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GAMIFANT emapalumab-lzsg 50 mg/10mL Injection — NDC 66658-0505-01 package photo
Label image from the product's FDA listing (DailyMed) — may show a different pack size or an older label revision.

GAMIFANT emapalumab-lzsg 50 mg/10mL Injection — NDC 66658-505-01 (Billing 66658-0505-01)

by Swedish Orphan Biovitrum AB (publ) · 1 VIAL, SINGLE-USE in 1 CARTON / 10 mL in 1 VIAL, SINGLE-USE

This is a package of GAMIFANT emapalumab-lzsg 50 mg/10mL Injection from Swedish Orphan Biovitrum AB (publ), marketed since May 2019 and currently FDA-listed. It is this product's only package size.

NDC 66658-0505-01
🏷️ FDA NDC (as labeled) 66658-505-01 billing pads the product segment with a zero
Rx only Brand On market Non-controlled ⇄ Compare with another NDC
🗂️ FDA directory synced Oct 1, 2026 · this listing last changed Jul 24, 2026 · sources: openFDA · FDA label (DailyMed) · FDA Orange & Purple Book · First Databank · CMS NADAC, ASP, Medicare & Medicaid · RxNorm
📋 All sources & update times →

This page is curated, coded, and maintained by

Compiled from public FDA, CMS, DailyMed, NADAC, and related drug data sources. Editorial policy

NDC database record

One package, one record: these facts belong to NDC 66658-505-01 alone.

Record
FDA NDC Directory package listing · Human prescription drug
Code segments
66658 labeler · 505 product · 01 package
Package marketed since
May 17, 2019
Sample package
No — commercial package
Listing certified through
Dec 31, 2027
Barcode (UPC-A, from the NDC)
3 6665850501 5
Medicaid fills, this package
15 prescriptions in the last four reported quarters
FDA record last changed
Jul 24, 2026

Identity & classification

Regulatory identifiers FDA, NLM and CMS codes for this package

FDA NDC (as labeled) 66658-505-01
Product NDC 66658-505
11-digit billing NDC 66658050501
NCPDP billing unit ML — per mL (volume)
RxCUI 2104611, 2104616, 2104621, 2104622, 2380771, 2380772, 2665325, 2665328,
UNII 3S252O2Z4X
Application # BLA761107
SPL Set ID a865e0ef-8685-4f69-8838-648c4f3bab47
Established class (EPC) Interferon gamma Blocker
Mechanism of action Interferon gamma Antagonists
DEA schedule Non-controlled
Marketing category BLA
Marketing status On market
FDA listing status Listed (active directory)
Marketing start 2019-05-17
Route INTRAVENOUS
Dosage form INJECTION
Substance EMAPALUMAB
Biologic (Purple Book) 351(a)

Drug-database identifiers Medi-Span GPI and First Databank GCN / HICL / AHFS classification

GPI-14 99405035402040
GPI class Gamifant
GCN Seq No 079316
GCN 45801
HICL code 045503
Ingredient (HICL) Emapalumab-Lzsg
HIC1 code Z
Therapeutic class — broad (HIC1) Body As A Whole
HIC2 code Z2
Therapeutic class — intermediate (HIC2) Antihistamines, Antiserotonins, Immunosuppressants
HIC3 code Z25
Therapeutic class — specific (HIC3) Immunosuppressant-Interferon Inhibitor, Mab
AHFS code 90:28.20.92
AHFS class Interferon Gamma Inhibitor Agents, Misc
FDB label name GAMIFANT 50 MG/10 ML VIAL
FDB brand name Gamifant
Legend status F — Federal legend — prescription drug or device
Quick answers
  • GSN (GCN sequence number): 079316
  • GCN: 45801
  • GPI-14 (Medi-Span): 99405035402040
  • HICL (First Databank): 045503
  • AHFS class code: 90:28.20.92
  • RxCUI (RxNorm): 2104611
Why two NDCs? The FDA registers this code as 66658-505-01 — a 5-3-2 layout, and that's what's printed on the package and shown on DailyMed. For insurance claims, every NDC is standardized to a uniform 11-digit 5-4-2 format by adding a zero to the product segment → 66658-0505-01. Same drug, same package — only the format differs.
Where does this data come from?
Identifiers from the FDA openFDA NDC Directory and Structured Product Labeling; RxCUI from RxNorm (NLM); GPI from Medi-Span; GCN / HIC / AHFS / legend from First Databank.

RxNorm drug class

This medicine belongs to the Interferon gamma Blocker class.

Pharmacologic class Interferon gamma Blocker
Drug family (ATC) Monoclonal antibodies
How it works Interferon gamma Antagonists
Where does this data come from?
Therapeutic classes from RxNorm RxClass (U.S. National Library of Medicine) — Established Pharmacologic Class (FDA), ATC drug family (WHO) and mechanism of action, matched by this product’s RxCUI.

Clinical

Label name GAMIFANT 50 MG/10 ML VIAL Ingredient Emapalumab-Lzsg
📗 Our plain-language guide HelloPharmacist
  • Gamifant is used to treat a rare but life-threatening condition called hemophagocytic lymphohistiocytosis — HLH for short — where the immune system goes into dangerous overdrive an...
  • What exactly is Gamifant being used for?
  • No, Gamifant is not a pill. It's given as an IV infusion — a slow drip directly into a vein — and it takes about one hour each time. It's always given in a clinical setting by a he...
  • How is this medicine given — do I take a pill?
📖 Read our full Emapalumab-lzsg Injection guide →
Where does this data come from?
Plain-language summary from MedlinePlus (U.S. National Library of Medicine); supplement & herbal interactions and nutrient depletion data from the Natural Medicines database; our full guide is HelloPharmacist editorial content.

Pricing

A drug doesn't have one price. Each row is a different public payment system, and none is what you'd pay at the counter — that depends on your insurance. The ⓘ on each row explains what it measures.

Price systemPer eachPer package
Retail pharmacies payNADAC · weekly Not in the retail survey — common for institutional, discontinued, or low-volume packs.
Medicaid paysCMS SDUD · 12 mo No recent Medicaid claims on file for this NDC — rare and low-volume NDCs are suppressed in the public data.
Medicare drug plans payPart D · quarterly No Part D plan price is available for this NDC in our data.
Medicare Part B allowsASP · J9210 $392.780 / J9210 unit —
Where does this data come from?
NADAC (National Average Drug Acquisition Cost) is the CMS weekly pharmacy-acquisition-cost survey — what pharmacies pay. ASP (Average Sales Price) is the CMS Medicare Part B drug-payment file, published quarterly. Medicaid pays is computed by us from CMS State Drug Utilization Data (total reimbursed ÷ units, trailing 12 months) — gross of rebates and inclusive of dispensing fees, so it reflects what Medicaid paid, not an acquisition cost. Medicare drug plans pay is the median negotiated point-of-sale unit cost across plans listing this NDC in the CMS quarterly Prescription Drug Plan pricing files, before rebates. The VA pays is the federal contract price (FSS, and the statutory Big 4 ceiling where listed) from the VA National Acquisition Center pharmaceutical price file. All are free public government data; each measures a different payer, so the figures are not directly comparable.

Billing & reimbursement

FDA NDC (as labeled)66658-505-01
11-digit billing NDC66658-0505-01
Format5-3-2 as registered → padded to 5-4-2 for billing (zero added to the product segment)
HCPCS J-codeJ9210
DescriptorINJECTION, EMAPALUMAB-LZSG, 1 MG
Billing units / pkg5 units
How the units are derivedThis package is 10 ML; the HCPCS unit is 1 MG, so one package = 5 billing units.
Medicare Part B spend (2026 (Q1))$2,031,768 · 24 claims · $84,657.02 per claim (all NDCs under J9210)
Crosswalk sourcePDAC NDC-HCPCS crosswalk (DME MAC / DMEPOS)
Where does this data come from?
The HCPCS J-code crosswalk comes from the CMS ASP NDC-HCPCS crosswalk and the DMEPDAC (DME MAC) NDC-HCPCS crosswalk — free public CMS data. Billing units are derived from the code’s descriptor and the package amount.

Packaging — all sizes for this product

Package NDCDescription Marketing startMarketing endStatus
66658-0505-01 You're viewing this Main listing 1 VIAL, SINGLE-USE in 1 CARTON / 10 mL in 1 VIAL, SINGLE-USE 2019-05-17 — Active

Therapeutic equivalents

ProductLabelerPackNADAC/unitTEStatusPrice vs. this
Gamifant 50 mg/10mLthis 66658-0505-01 Swedish 1 vial — — FDA listed —
About this product: this is a biologic. Biologics don't have small-molecule generics — competition comes from FDA-licensed biosimilars (shown above), not generics.
Where does this data come from?
Equivalents are other NDCs of the same ingredient, form and route from the openFDA NDC Directory, ranked least-expensive-first by NADAC. Therapeutic-equivalence (AB) ratings come from the FDA Orange Book; biologics use the FDA Purple Book for biosimilar & interchangeable status.

Availability & biosimilar status

🏛️
2018
First FDA approval
Nov 2018
📍
2026
Currently FDA-listed
8 years listed
🛡️
2030
Latest patent/protection listed
not a guaranteed launch date
🧬Biologic — competition comes from biosimilars

Biologics have no small-molecule generics; biosimilar competition is tracked in the FDA Purple Book.

🛡️ Latest patent/protection date listed: FDA patent/protection data lists protections through Nov 2030. This may affect when biosimilars become widely available, but it is not a guaranteed launch date.
📅 FDA approved Nov 20, 2018 ⏳ ~4.1 yr to latest listed protection

Why the date isn’t exact: Biosimilar timing can change because patents may be challenged, settled, licensed, added or removed, and litigation can move the real date earlier or later.

FDA Purple Book — biosimilars & interchangeables ⓘ
Reference product
Gamifantemapalumab-lzsg
🔒 No FDA-licensed biosimilars or interchangeable biosimilars are listed yet for this biologic. It currently has no biosimilar competition in the FDA Purple Book.
Source: FDA Purple Book (purplebooksearch.fda.gov), matched on the reference product’s active ingredient.
Patents & exclusivity — FDA Purple Book
Exclusivity RefProduct
2018 2020 2022 2024 2026 2028 2030
Today
LOE
Biologic patent Exclusivity
🏛️Reference-product exclusivity
A flat 12 years of FDA market protection from first licensure. No biosimilar can be licensed before it ends — regardless of patents.
🧪Listed biologic patents
Patents the reference maker lists covering the molecule, formulation, or manufacturing. A biosimilar generally can’t launch until these resolve.
🔁Interchangeability
An interchangeable biosimilar may be substituted at the pharmacy (state laws vary). The first one can earn its own exclusivity period.
🛈 What do these terms mean?
Biologic patent
A patent the reference product’s maker has publicly listed. A biosimilar generally can’t launch until these expire — unless they’re invalidated or resolved in a settlement.
Reference-product exclusivity
A flat 12 years of FDA market protection from the biologic’s first licensure (the BPCIA). No biosimilar can be licensed before it ends, regardless of patents.
Interchangeable exclusivity
The first interchangeable biosimilar can earn a period as the only interchangeable version (pharmacists can substitute it without the prescriber).
Earliest biosimilar (LOE)
The latest of all the dates above — the soonest a biosimilar can realistically reach the market. Litigation and settlements can move it earlier.

Biologics have no small-molecule “generics” — competition comes from FDA-licensed biosimilars, tracked in the FDA Purple Book.

FDA exclusivity
CodeWhat it grantsExpires
RefProductReference-product exclusivity (12-year, BPCIA) — no biosimilar can be licensed before this dateNov 20, 2030
Common questions
Is there a biosimilar for GAMIFANT 50 MG/10 ML VIAL?
No FDA-licensed biosimilar is currently listed for this biologic in the FDA Purple Book.
Why do different websites show different biosimilar dates?
Biosimilar availability isn’t based on one single date. Some sources use the reference-product exclusivity, some use the last listed patent, and patent litigation, settlements, and licenses can all change the real-world launch date. This page shows the underlying Purple Book dates so you can see why estimates differ.
Can a biosimilar launch before the last patent expires?
Sometimes. A biosimilar maker may settle with the reference manufacturer or receive a license to launch earlier. In other cases, the last listed protection delays competition.
What does “current Purple Book estimate” mean?
It means we’re using the latest patent and exclusivity dates currently listed in the FDA Purple Book. It is not a guaranteed launch date.
What does “FDA listed” mean?
It means the product appears in the FDA’s official directory. That’s a good sign a product exists for the U.S. market, but on its own it does not confirm a pharmacy can get it today. Where we have recent retail pricing data, we label it “Availability likely” instead.
What does a patent or protection date mean here?
It’s the latest date currently listed in the FDA Purple Book for a patent or exclusivity on the reference biologic. It can affect when a biosimilar becomes widely available — but it is not a guaranteed launch date. Settlements and licenses can move the real date earlier or later.
Built from the FDA Purple Book Patent List (patents the reference-product sponsor has publicly listed under the BPCIA) plus reference-product exclusivity. Biosimilars cannot launch until these clear; patent litigation and settlements can shift the real date. Biologics have no small-molecule generics — competition comes from FDA-licensed biosimilars, not the Orange Book.
Where does this data come from?
Patents and exclusivity from the FDA Purple Book (biologics), refreshed from public FDA data. Biosimilar launch timing is an estimate, not a guarantee.

Inactive Ingredients / Excipients

Inactive ingredients, also called excipients, are components of the drug product other than the active ingredient. They may include fillers, dyes, coatings, preservatives, flavors, or other formulation ingredients.

Loading inactive ingredients from the official FDA label in the background. No external source is being called by this page request.
Where does this data come from?
Source: official FDA Structured Product Labeling (SPL) via DailyMed and the openFDA label index. Structured IACT rows and label-wide narrative are kept separate; availability and product-level specificity depend on the submitted label.

Inactive ingredient FAQ

Are inactive ingredients the same for every manufacturer?
No. Inactive ingredients can differ by manufacturer, dosage form, strength, and package / product version.
Why might an inactive ingredient be missing?
Some SPLs do not provide a complete structured inactive-ingredient list, and older or unusual labels may only include the information in narrative text.
Can inactive ingredients matter?
Yes. They can matter for allergies, intolerances, dyes, gluten / lactose concerns, preservatives, and formulation differences — but confirm with a pharmacist or the manufacturer when it’s clinically important.

Manufacturer & labeler

LabelerSwedish Orphan Biovitrum AB (publ)
FDA applicationBLA761107 (BLA)
Labeler code66658
First marketedMay 2019
Product typeHuman Prescription Drug
Portfolio16 products on file

More NDCs from Swedish Orphan Biovitrum AB (publ) labeler code 66658

The labeler markets the product; the application holder owns the FDA approval. They’re often the same company but can differ (e.g. a repackager or an authorized generic). A mailing address / phone appears here when the manufacturer includes it in the product’s FDA label (not all do).
Where does this data come from?
Labeler, application holder and registered establishment from the FDA openFDA NDC Directory and Drugs@FDA; address/contact from the product’s FDA label.

Full prescribing information FDA SPL

The complete FDA label for this product — the official prescribing information, verbatim, section by section. Very long sections are excerpted here and marked; the full text is on DailyMed (linked in the sources below). Jump with a chip, search within the label, or expand everything.
🎯 Indications and Usage ⏱️ Dosage and Administration 💊 Dosage Forms and Strengths ⛔ Contraindications ⚠️ Warnings and Cautions 🤒 Adverse Reactions 🔄 Drug Interactions 👥 Use in Specific Populations 🤰 Pregnancy 🧒 Pediatric Use 🧓 Geriatric Use 🆘 Overdosage 🧬 Clinical Pharmacology 🧬 Mechanism of Action 📦 How Supplied / Storage and Handling 📋 Description 💬 Information for Patients 💬 Medication Guide 🧬 Pharmacokinetics 🧬 Pharmacodynamics 🔬 Clinical Studies 🧪 Nonclinical Toxicology 📄 Carcinogenesis, Mutagenesis, Impairment of Fertility 📄 Recent Major Changes 📄 Package Label / Principal Display Panel
🎯 Indications and Usage 138 words ▾

1 INDICATIONS AND USAGE GAMIFANT is indicated for the treatment of: adult and pediatric patients with primary hemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent or progressive disease or intolerance with conventional HLH therapy. adult and pediatric patients with HLH/macrophage activation syndrome (MAS) in known or suspected Still’s disease, including systemic Juvenile Idiopathic Arthritis (sJIA), with an inadequate response or intolerance to glucocorticoids, or with recurrent MAS. GAMIFANT is an interferon gamma (IFNγ) neutralizing antibody indicated for the treatment of: adult and pediatric patients with primary hemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent or progressive disease or intolerance with conventional HLH therapy.

( 1 ) adult and pediatric patients with HLH/macrophage activation syndrome (MAS) in known or suspected Still’s disease, including systemic Juvenile Idiopathic Arthritis (sJIA), with an inadequate response or intolerance to glucocorticoids, or with recurrent MAS. ( 1 )

⏱️ Dosage and Administration ~3 min read ▾

2 DOSAGE AND ADMINISTRATION For intravenous infusion only: Primary HLH recommended starting dosage: 1 mg/kg as an intravenous infusion over 1 hour twice per week. ( 2.1 ) HLH/MAS in Still’s disease recommended dosage schedule: 6 mg/kg, followed by 3 mg/kg every 3 days for 5 doses, then 3 mg/kg twice per week. Given as an intravenous infusion over 1 hour. ( 2.2 ) For both indications, the dose may be titrated up to a maximum of 10 mg/kg. ( 2.5 , 2.6 )

2.1Recommended Dosage for Primary Hemophagocytic Lymphohistiocytosis The recommended starting dose of GAMIFANT is 1 mg/kg given as a central or peripheral intravenous infusion over 1 hour twice per week (every three to four days). Doses subsequent to the initial dose may be increased based on clinical and laboratory criteria [see Dosage and Administration ( 2.5 )] . Administer GAMIFANT until hematopoietic stem cell transplantation (HSCT) is performed or unacceptable toxicity.

Discontinue GAMIFANT when a patient no longer requires therapy for the treatment of HLH.

2.2Recommended Dosage for Hemophagocytic Lymphohistiocytosis/Macrophage Activation Syndrome GAMIFANT is administered as a central or peripheral intravenous infusion over 1 hour according to the dosage schedule in Table 1. Doses may be increased based on clinical and laboratory criteria [see Dosage and Administration ( 2.6 )] . Discontinue GAMIFANT when a patient no longer requires therapy for the treatment of HLH/MAS.

Table 1. GAMIFANT Dosage in Patients with HLH/MAS Treatment Day GAMIFANT Dosage Day 1 6 mg/kg Days 4 to 16 3 mg/kg every 3 days for 5 doses From Day 19 onward 3 mg/kg twice per week (i.e., every 3 to 4 days)

2.3Monitoring to Assess Safety Before Initiating GAMIFANT Treatment Conduct testing for latent tuberculosis infections using the purified protein derivative (PPD) or IFNγ release assay and evaluate patients for tuberculosis risk factors prior to initiating GAMIFANT. Administer tuberculosis prophylaxis to patients at risk for tuberculosis, or known to have a positive PPD test result, or positive IFNγ release assay. During GAMIFANT Treatment Monitor for herpes zoster infection, adenovirus, EBV and CMV as clinically indicated.

2.4Prophylaxis and Concomitant Medication Information Prophylaxis Consider prophylaxis for herpes zoster, Pneumocystis jirovecii , and for fungal infections prior to GAMIFANT administration. Concomitant Medications For primary HLH patients who are not receiving baseline dexamethasone treatment, begin dexamethasone at a daily dose of at least 5 mg/m 2 to 10 mg/m 2 the day before GAMIFANT treatment begins. For patients who were receiving baseline dexamethasone, they may continue their regular dose provided the dose is at least 5 mg/m2.

Dexamethasone can be tapered according to the judgment of the treating physician [see Clinical Studies ( 14 )] .

2.5Dose Modification Based on Response for Primary Hemophagocytic Lymphohistiocytosis The GAMIFANT dose may be titrated up if disease response is unsatisfactory (see Table 2) [see Clinical Pharmacology ( 12.3 )] . After the patient's clinical condition is stabilized, decrease the dose to the previous level to maintain clinical response. Table 2: Dose Titration Criteria Treatment Day GAMIFANT Dose Criteria for Dose Increase Day 1 Starting Dose of 1 mg/kg N/A From Day 4 onwards Increase to 3 mg/kg Unsatisfactory improvement in clinical condition, as assessed by a healthcare provider AND at least one of the following: Fever - persistence or recurrence Platelet count If baseline < 50,000/mm 3 and no improvement to > 50,000/mm 3 If baseline > 50,000/mm 3 and less than 30% improvement If baseline > 100,000/mm 3 and decrease to < 100,000/mm 3 Neutrophil count If baseline < 500/mm 3 and no improvement to > 500/mm 3 If baseline > 500/mm 3 to 1,000/mm 3 and decrease to < 500/mm 3 If baseline 1,000/mm 3 to 1,500/mm 3 and decrease to < 1,000/mm 3 Ferritin (ng/mL) If baseline ≥3,000 ng/mL and < 20% decrease If baseline < 3,000 ng/mL and any increase to… [Excerpted — this section continues on DailyMed.]

💊 Dosage Forms and Strengths 190 words ▾

3 DOSAGE FORMS AND STRENGTHS GAMIFANT is a clear to slightly opalescent, colorless to slightly yellow preservative-free solution available as: Injection: • 10 mg/2 mL (5 mg/mL) in a single-dose vial • 50 mg/10 mL (5 mg/mL) in a single-dose vial • 100 mg/20 mL (5 mg/mL) in a single-dose vial • 50 mg/2 mL (25 mg/mL) in a single-dose vial • 100 mg/4 mL (25 mg/mL) in a single-dose vial • 250 mg/10 mL (25 mg/mL) in a single-dose vial • 500 mg/20 mL (25 mg/mL) in a single-dose vial Injection: • 10 mg/2 mL (5 mg/mL) solution in a single-dose vial ( 3 ) • 50 mg/10 mL (5 mg/mL) solution in a single-dose vial ( 3 ) • 100 mg/20 mL (5 mg/mL) solution in a single-dose vial ( 3 ) • 50 mg/2 mL (25 mg/mL) solution in a single-dose vial ( 3 ) • 100 mg/4 mL (25 mg/mL) solution in a single-dose vial ( 3 ) • 250 mg/10 mL (25 mg/mL) solution in a single-dose vial ( 3 ) • 500 mg/20 mL (25 mg/mL) solution in a single-dose vial ( 3 )

⛔ Contraindications 7 words ▾

4 CONTRAINDICATIONS None. None. ( 4 )

⚠️ Warnings and Cautions ~3 min read ▾

5 WARNINGS AND PRECAUTIONS Infections: Monitor patients for signs and symptoms and treat promptly. Test for latent tuberculosis. Consider administering prophylactic treatment against herpes zoster, Pneumocystis jirovecii and fungal infections.

( 5.1 ) Live Vaccines: Do not administer live or live attenuated vaccines to patients receiving GAMIFANT. ( 5.2 ) Infusion-Related Reactions: Monitor patients for infusion-related reactions. Interrupt infusion for infusion reactions and institute appropriate medical management.

( 5.3 )

5.1Infections GAMIFANT may increase the risk of fatal and serious infections to include specific pathogens favored by IFNγ neutralization, including mycobacteria, herpes zoster virus, and Histoplasma capsulatum . Do not administer GAMIFANT in patients with infections caused by these pathogens until appropriate treatment has been initiated. In patients with primary HLH receiving GAMIFANT in clinical trials, serious infections such as sepsis, pneumonia, bacteremia, disseminated histoplasmosis, necrotizing fasciitis, viral infections, and perforated appendicitis were observed in 32% of patients.

The reported infections were viral (41%), bacterial (35%), fungal (9%), and the pathogen was not identified in 15% of cases. In patients with HLH/MAS in Still’s disease receiving GAMIFANT in clinical trials, serious infections such as pneumonia, cytomegalovirus infection, cytomegalovirus infection reactivation, and sepsis were observed in 13% of patients. The reported infections were viral (44%), bacterial (13%), fungal (3%) and the pathogen was not identified in (13%) of patients.

In the GAMIFANT clinical trials, additional infections were reported in patients treated with GAMIFANT including herpes zoster (2.4%) and mycobacterial infections (2.4%). Evaluate patients for tuberculosis risk factors and test for latent infection (PPD testing, PCR, or IFNγ release assay) prior to initiating GAMIFANT. Administer tuberculosis prophylaxis to patients at risk for tuberculosis or known to have a positive purified protein derivative (PPD) test result [see Dosage and Administration ( 2.2 )] .

Consider prophylaxis for herpes zoster, Pneumocystis jirovecii ,and fungal infection to mitigate the risk to patients while receiving GAMIFANT [see Dosage and Administration ( 2.3 )] .Employ surveillance testing during treatment with GAMIFANT. Closely monitor patients receiving GAMIFANT for signs or symptoms of infection, promptly initiate a complete diagnostic workup appropriate for an immunocompromised patient, and initiate appropriate antimicrobial therapy.

5.2Increased Risk of Infection with Use of Live Vaccines Do not administer live or live attenuated vaccines to patients receiving GAMIFANT and for at least 4 weeks after the last dose of GAMIFANT. The safety of immunization with live vaccines during or following GAMIFANT therapy has not been studied.

5.3Infusion-Related Reactions Infusion-related reactions in patients with primary HLH, including drug eruption, pyrexia, rash, erythema, and hyperhidrosis, were reported with GAMIFANT treatment in 27% of patients. In one-third of these patients, the infusion-related reaction occurred during the first infusion. Infusion-related reactions in patients with HLH/MAS in Still’s disease, including pyrexia, headache, paresthesia, bone pain, pruritic rash, and peripheral coldness, were reported with GAMIFANT treatment in 13% of patients.

Infusion-related reactions were reported as mild in 8% of patients and as moderate in 5% of patients. Infusion-related reactions were defined as any event reported to have occurred within 24 hours after the start of infusion and assessed as related to GAMIFANT. In the GAMIFANT clinical trials, additional infusion-related reactions that were reported in less than 3% of patients treated with GAMIFANT included: malaise, sinus tachycardia, feeling hot, and acute respiratory failure.

Monitor patients for infusion-related reactions which can be severe. Interrupt the… [Excerpted — this section continues on DailyMed.]

🤒 Adverse Reactions ~3 min read ▾

6 ADVERSE REACTIONS The following adverse reactions are described elsewhere in the labeling: Infections [see Warnings and Precautions ( 5.1 )] Increased Risk of Infection with Use of Live Vaccines [see Warnings and Precautions ( 5.2 )] Infusion-Related Reactions [see Warnings and Precautions ( 5.3 )] In patients with primary HLH, the most common adverse reactions (≥ 20%) were: infections, hypertension, infusion-related reactions, and pyrexia. ( 6.1 ) In patients with HLH/MAS in Still’s disease, the most common adverse reactions (≥20%) were: viral infections, including cytomegalovirus infection or reactivation, and rash.

( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact 1-866-773-5274 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

6.1Clinical Trial Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice. Primary HLH The safety data described in this section reflect exposure to GAMIFANT in which 34 patients with untreated primary HLH and previously treated patients with primary HLH ( NCT01818492 ) received GAMIFANT at a starting dose of 1 mg/kg every 3 days with dose increases up to 10 mg/kg [see Dosage and Administration ( 2.1 ) and Clinical Studies ( 14 )] .

The median duration of treatment with GAMIFANT was 59 days (range: 4 to 245 days) and the median cumulative dose was 25 mg/kg (range: 4 to 254 mg/kg). The median age of study population was 1 year (range: 0.1 to 13 years), 53% were female, and 65% were Caucasian. Serious adverse reactions were reported in 53% of patients.

The most common serious adverse reactions (≥3%) included infections, gastrointestinal hemorrhage, and multiple organ dysfunction. Fatal adverse reactions occurred in two (6%) of patients and included septic shock and gastrointestinal hemorrhage. Disseminated histoplasmosis led to drug discontinuation in one patient.

The most commonly reported adverse reactions (≥20%) were infections, hypertension, infusion-related reactions, and pyrexia. Adverse reactions reported in ≥10% of patients during treatment with GAMIFANT are presented in Table 4. Table 4 : Adverse Reactions Reported in 10% of Patients with Primary HLH Adverse Reactions GAMIFANT (%) (N = 34) Infections a 56 Hypertension b 41 Infusion-related reactions c 27 Pyrexia 24 Hypokalemia 15 Constipation 15 Rash 12 Abdominal pain 12 Cytomegalovirus infection 12 Diarrhea 12 Lymphocytosis 12 Cough 12 Irritability 12 Tachycardia 12 Tachypnea 12 a Includes viral, bacterial, fungal, and infections in which no pathogen was identified b Includes secondary hypertension c Includes events of drug eruption, pyrexia, rash, erythema, and hyperhidrosis Additional selected adverse reactions (all grades) that were reported in less than 10% of patients treated with GAMIFANT included: vomiting, acute kidney injury, asthenia, bradycardia, dyspnea, gastro-intestinal hemorrhage, epistaxis, and peripheral edema.

HLH/MAS The safety of GAMIFANT was evaluated in two open-label clinical studies in patients with HLH/MAS in Stills disease, including sJIA [see Clinical Studies ( 14.2 )] . The pooled safety data from these two studies included 39 patients who received an initial dose of 6 mg/kg followed by 3 mg/kg every 3 days until Day 16, and then twice weekly thereafter. The median duration of treatment with GAMIFANT was 29 days (range: 7 to 220 days) and the median cumulative dose was 33 mg/kg (range: 12 to 175 mg/kg).

Serious adverse reactions were reported in 12 patients (31%), with the most common serious adverse reaction being pneumonia (5%). Fatal adverse reactions occurred in two patients (5%) and included multiple organ dysfunction and circulatory shock. Pneumonia led to drug discontinuation in one patient (3%).

The most common adverse reactions (≥20%) were viral infections, including cytomegalovirus i… [Excerpted — this section continues on DailyMed.]

🔄 Drug Interactions 75 words ▾

7 DRUG INTERACTIONS

7.1Effect of GAMIFANT on Cytochrome P450 Substrates The formation of CYP450 enzymes may be suppressed by increased levels of cytokines (such as IFNγ) during chronic inflammation. By neutralizing IFNγ, use of GAMIFANT may normalize CYP450 activities which may reduce the efficacy of drugs that are CYP450 substrates due to increased metabolism. Upon initiation or discontinuation of concomitant GAMIFANT, monitor for reduced efficacy and adjust dosage of CYP450 substrate drugs as appropriate.

👥 Use in Specific Populations ~2 min read ▾

8 USE IN SPECIFIC POPULATIONS

8.1Pregnancy Risk Summary There are no available data on GAMIFANT use in pregnant women to inform a drug-associated risk of adverse developmental outcomes. In an animal reproduction study, a murine surrogate anti-mouse IFNγ antibody administered to pregnant mice throughout gestation crossed the placental barrier, and no fetal harm was observed (see Data ) . The estimated background risk of major birth defects and miscarriage for the indicated population is unknown.

All pregnancies have a background risk of birth defect, loss, or other adverse outcomes. In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2 to 4% and 15 to 20%, respectively. Data Animal Data In a mouse embryo-fetal development study, a murine surrogate anti-mouse IFNγ antibody was administered every 3-4 days throughout organogenesis and late gestation at doses of 0, 30, 75 or 150 mg/kg/occasion.

The surrogate antibody was detected in the plasma of all treated pregnant mice and their corresponding fetuses. No maternal toxicity occurred and there was no evidence of teratogenicity or effects on embryo-fetal survival or growth.

8.2Lactation Risk Summary There is no information regarding the presence of emapalumab-lzsg in human milk, the effects on the breastfed child, or the effects on milk production. Published data suggest that only limited amounts of therapeutic antibodies are found in breast milk and they do not enter the neonatal and infant circulations in substantial amounts. The developmental and health benefits of breastfeeding should be considered along with the mother's clinical need for GAMIFANT and any potential adverse effects on the breastfed child from GAMIFANT or from the underlying maternal condition.

8.4Pediatric Use Safety and effectiveness of GAMIFANT have been established in pediatric patients with primary HLH that is reactivated or refractory to conventional therapies and with HLH/MAS in Still’s Disease (including sJIA) with an inadequate response to glucocorticoids [see Clinical Studies ( 14 )]. Use of GAMIFANT is supported by 3 single-arm trials which included 57 pediatric patients: 27 with primary HLH and 30 with HLH/MAS in Still’s disease. These studies included pediatric patients in the following age groups: 5 patients newborn to 6 months, 13 patients 6 months to 2 years, 27 patients from 2 years to <12 years, and 12 patients from 12 years to <17 years.

8.5Geriatric Use Clinical studies of GAMIFANT did not include sufficient numbers of subjects aged 65 and over to determine whether they respond differently from younger subjects. Other reported clinical experience has not identified differences in responses between the elderly and younger patients.

🤰 Pregnancy 182 words ▾

8.1Pregnancy Risk Summary There are no available data on GAMIFANT use in pregnant women to inform a drug-associated risk of adverse developmental outcomes. In an animal reproduction study, a murine surrogate anti-mouse IFNγ antibody administered to pregnant mice throughout gestation crossed the placental barrier, and no fetal harm was observed (see Data ) . The estimated background risk of major birth defects and miscarriage for the indicated population is unknown.

All pregnancies have a background risk of birth defect, loss, or other adverse outcomes. In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2 to 4% and 15 to 20%, respectively. Data Animal Data In a mouse embryo-fetal development study, a murine surrogate anti-mouse IFNγ antibody was administered every 3-4 days throughout organogenesis and late gestation at doses of 0, 30, 75 or 150 mg/kg/occasion.

The surrogate antibody was detected in the plasma of all treated pregnant mice and their corresponding fetuses. No maternal toxicity occurred and there was no evidence of teratogenicity or effects on embryo-fetal survival or growth.

🧒 Pediatric Use 110 words ▾

8.4Pediatric Use Safety and effectiveness of GAMIFANT have been established in pediatric patients with primary HLH that is reactivated or refractory to conventional therapies and with HLH/MAS in Still’s Disease (including sJIA) with an inadequate response to glucocorticoids [see Clinical Studies ( 14 )]. Use of GAMIFANT is supported by 3 single-arm trials which included 57 pediatric patients: 27 with primary HLH and 30 with HLH/MAS in Still’s disease. These studies included pediatric patients in the following age groups: 5 patients newborn to 6 months, 13 patients 6 months to 2 years, 27 patients from 2 years to <12 years, and 12 patients from 12 years to <17 years.

🧓 Geriatric Use 43 words ▾

8.5Geriatric Use Clinical studies of GAMIFANT did not include sufficient numbers of subjects aged 65 and over to determine whether they respond differently from younger subjects. Other reported clinical experience has not identified differences in responses between the elderly and younger patients.

🆘 Overdosage 18 words ▾

10 OVERDOSAGE Consider contacting the Poison Help line (1-800-222-1222) or a medical toxicologist for additional overdose management recommendations.

🧬 Clinical Pharmacology ~3 min read ▾

12 CLINICAL PHARMACOLOGY

12.1Mechanism of Action Emapalumab-lzsg is a monoclonal antibody that binds to and neutralizes interferon gamma (IFNγ). Nonclinical data suggest that IFNγ plays a pivotal role in the pathogenesis of HLH by being hypersecreted.

12.2Pharmacodynamics IFNγ Inhibition Emapalumab-lzsg reduces the plasma concentrations of CXCL9, a chemokine induced by IFNγ. Cardiac Electrophysiology At a dose of 3 mg/kg GAMIFANT does not prolong the QT interval to any clinically relevant extent.

12.3Pharmacokinetics The pharmacokinetics of emapalumab-lzsg were evaluated in healthy adult subjects and in patients with HLH. Following a 1 mg/kg emapalumab-lzsg dose, median steady state peak concentration was 44 mcg/mL, which was 2.9 times higher than after the first dose. The median steady state trough concentration was 25 mcg/mL, which was 4.3 times higher than after the first dose.

Emapalumab-lzsg AUC increases slightly more than proportionally between 1 and 3 mg/kg doses, and less than proportionally at 3, 6, and 10 mg/kg doses. Emapalumab-lzsg exhibits target-mediated-like clearance dependent on IFNγ production, which can vary between and within patients as a function of time and can affect the recommended dosage [see Dosage and Administration ( 2.2 )] . Emapalumab-lzsg steady state is achieved by the 7th infusion when the IFNγ production is moderate.

At high IFNγ production, steady-state is reached earlier due to a shorter half-life. Distribution The central and peripheral volumes of distribution in a subject with body weight of 70 kg are 2.8 and

4.4L, respectively. Elimination Emapalumab-lzsg elimination half-life is approximately 22 days in healthy subjects and ranged from 2.5 to 18.9 days in HLH patients. In patients with HLH, the elimination half-life of emapalumab-lzsg was significantly influenced by the amount of IFNγ within the patient, demonstrating a target mediated clearance-like mechanism.

The elimination half-life of emapalumab-lzsg is reduced as the concentration of IFNγ increases. Emapalumab-lzsg clearance is approximately 0.007 L/h in healthy subjects. Metabolism The metabolic pathway of emapalumab-lzsg has not been characterized.

Like other protein therapeutics, GAMIFANT is expected to be degraded into small peptides and amino acids via catabolic pathways. Specific Populations Body weight (3 to 80 kg) was a significant covariate of emapalumab-lzsg pharmacokinetics, supporting body weight-based dosing. No clinically significant differences in the pharmacokinetics of emapalumab-lzsg were observed based on age (0.02 to 64 year), sex (53% Females), race (71.4% Caucasian, 12.2% Asian and 8.2% Black), renal impairment including dialysis, or hepatic impairment (mild, moderate, and severe).

Drug Interaction Studies No drug-drug interaction studies have been conducted with GAMIFANT.

12.4Immunogenicity As with all therapeutic proteins, there is potential for immunogenicity. The detection of antibody formation is highly dependent on the sensitivity and specificity of the assay. Additionally, the observed incidence of antibody (including neutralizing antibody) positivity in an assay may be influenced by several factors, including assay methodology, sample handling, timing of sample collection, concomitant medications, and underlying disease.

For these reasons, comparison of the incidence of antibodies in the studies described below with the incidence of antibodies in other studies or to other emapalumab products may be misleading. The immunogenicity of emapalumab-lzsg has been evaluated using an electrochemiluminescence-based immunoassay (ECLIA). Treatment-emergent anti-drug antibodies (ADAs) were detected in 1/33 (3%) of patients in the primary HLH clinical trial.

The ADAs in this patient were found to have neutralizing ability. Treatment-emergent ADAs were detected in 5/35 patients (14%) in the two HLH/MAS clinical trials. None of the ADAs in these 5 patients were found to have neutralizing ability.

No evidence of… [Excerpted — this section continues on DailyMed.]

🧬 Mechanism of Action 34 words ▾

12.1Mechanism of Action Emapalumab-lzsg is a monoclonal antibody that binds to and neutralizes interferon gamma (IFNγ). Nonclinical data suggest that IFNγ plays a pivotal role in the pathogenesis of HLH by being hypersecreted.

📦 How Supplied / Storage and Handling 139 words ▾

16 HOW SUPPLIED/STORAGE AND HANDLING GAMIFANT (emapalumab-lzsg) injection is a sterile, clear to slightly opalescent, colorless to slightly yellow solution supplied in the following packaging configuration: NDC 66658-501-01 – containing one 10 mg/2 mL (5 mg/mL) single-dose vial NDC 66658-505-01 – containing one 50 mg/10 mL (5 mg/mL) single-dose vial NDC 66658-510-01 – containing one 100 mg/20 mL (5 mg/mL) single-dose vial NDC 66658-522-01 – containing one 50 mg/2 mL (25 mg/mL) single-dose vial NDC 66658-523-01 – containing one 100 mg/4 mL (25 mg/mL) single-dose vial NDC 66658-524-01 – containing one 250 mg/10 mL (25 mg/mL) single-dose vial NDC 66658-525-01 – containing one 500 mg/20 mL (25 mg/mL) single-dose vial Store GAMIFANT in a refrigerator at 2ºC to 8ºC (36ºF to 46ºF) in original carton to protect from light.

Do not freeze or shake. This product contains no preservative.

📋 Description 189 words ▾

11 DESCRIPTION Emapalumab-lzsg is an interferon gamma (IFNγ) neutralizing antibody. Emapalumab-lzsg is produced in Chinese Hamster Ovary cells by recombinant DNA technology. Emapalumab-lzsg is an IgG1 immunoglobulin with a molecular weight of approximately 148 kDa.

GAMIFANT (emapalumab-lzsg) injection for intravenous use is a sterile, preservative-free, clear to slightly opalescent, colorless to slightly yellow solution provided in single-dose vials that require dilution prior to intravenous infusion. GAMIFANT 5 mg/mL (2 mL, 10 mL and 20 mL) Each vial contains 10 mg/2 mL, 50 mg/10 mL, or 100 mg/20 mL emapalumab-lzsg at a concentration of 5 mg/mL. Each mL also contains the following inactive ingredients: L-Histidine (1.55 mg), L-Histidine monohydrochloride, monohydrate (3.14 mg), Polysorbate 80 (0.05 mg), sodium chloride (7.30 mg), and Water for Injection, USP.

GAMIFANT 25 mg/mL (2 mL, 4 mL, 10 mL and 20 mL) Each vial contains 50 mg/2 mL, 100 mg/4 mL, 250 mg/10 mL or 500 mg/20 mL emapalumab- lzsg at a concentration of 25 mg/mL. Each mL also contains the following inactive ingredients: L-Histidine (1.55 mg), L-Histidine monohydrochloride, monohydrate (3.14 mg), Polysorbate 80 (0.05 mg), sodium chloride (7.30 mg), and Water for Injection, USP.

💬 Information for Patients 146 words ▾

17 PATIENT COUNSELING INFORMATION Advise the patient to read the FDA-approved patient labeling ( Medication Guide ). Infections Inform patients and their caregivers of the risk of developing infections during treatment with GAMIFANT, and to report any symptoms of infection [see Warnings and Precautions ( 5.1 )] . Vaccinations Advise patients and their caregivers that the patient should not receive live or live attenuated vaccines during GAMIFANT treatment [see Warnings and Precautions ( 5.2 )] .

Infusion-Related Reactions Advise patients and their caregivers of the potential for developing infusion-related reactions during treatment with GAMIFANT [see Warnings and Precautions ( 5.3 )] . Manufactured by: Swedish Orphan Biovitrum AB (publ) Stockholm, Sweden U.S. License Number 1859 Distributed by: Sobi Inc.

77 Fourth Avenue, 3 rd Floor Waltham, MA 02451-7559 Manufactured at: Patheon Italia S.p.A 2° Trav. SX Via Morolense, 5 03013-Ferentino Italy Product of the United Kingdom

💬 Medication Guide ~3 min read ▾

MEDICATION GUIDE GAMIFANT ® (gam i fant) (emapalumab-lzsg) injection, for intravenous use What is the most important information I should know about GAMIFANT? GAMIFANT can cause serious side effects including: Infections. GAMIFANT is a medicine that affects your immune system and may lower the ability of your immune system to fight infections.

GAMIFANT may increase your risk of serious infections that can lead to death. These infections include tuberculosis (TB), histoplasmosis, pneumonia, cytomegalovirus infection or reactivation of infection, sepsis, herpes zoster infection (shingles) and other infections caused by viruses, fungi or bacteria that can spread throughout the body. Your healthcare provider will: test you for TB before you start treatment with GAMIFANT. treat you with a medicine for TB if you are at risk for TB or if you have a known positive TB test.

Infections are common in people treated with GAMIFANT. Before starting GAMIFANT, tell your healthcare provider if you: had TB in the past, or if you or a member of your family have been in recent close contact with someone with TB. have ever had a positive TB skin test (PPD). currently have or have had history of infections, including histoplasmosis, cytomegalovirus, or herpes zoster (shingles). are being treated for an active infection. have symptoms of an infection, such as fever, sweat and chills, cough, breathing problems, blood in mucus (phlegm), warm, red, or painful skin or sores on your body.

Your healthcare provider may give you medicine to help prevent certain infections before you receive GAMIFANT. After starting GAMIFANT , tell your healthcare provider if: new symptoms of an infection appear. symptoms of an infection that you already had when starting GAMIFANT worsen. Your healthcare provider will monitor you closely for signs and symptoms of infections during treatment with GAMIFANT.

See " What are the possible side effects of GAMIFANT? " for more information about side effects. What is GAMIFANT? GAMIFANT is a prescription medicine used for the treatment of hemophagocytic lymphohistiocytosis (HLH) in adults and children with: primary HLH whose disease has come back or progressed, or other medicines have not worked well enough or cannot be tolerated.

HLH/macrophage activation syndrome (MAS) in known or suspected Still's disease, including systemic Juvenile Idiopathic Arthritis (sJIA), when glucocorticoids have not worked well enough or cannot be tolerated, or with MAS that has come back. Before you receive GAMIFANT, tell your healthcare provider about all of your medical conditions including if you: have an infection (see What is the most important information I should know about GAMIFANT? ) have received or are scheduled to receive an immunization (vaccine). You should not receive "live or live attenuated" vaccines during your treatment with GAMIFANT and for at least 4 weeks after the last dose of GAMIFANT. are pregnant or plan to become pregnant.

It is not known if GAMIFANT can harm your unborn baby. are breastfeeding or plan to breastfeed. It is not known if GAMIFANT passes into your breast milk. Talk to your healthcare provider about the best way to feed your baby during treatment with GAMIFANT.

Tell your healthcare provider about all the medicines you take , including prescription and over-the-counter medicines, vitamins, and herbal supplements. How will I receive GAMIFANT? You will receive GAMIFANT through a vein by intravenous (IV) infusion over 1 hour.

Your healthcare provider will monitor you during the infusion for side effects. For primary HLH: o GAMIFANT is given 2 times a week (every 3 to 4 days). o GAMIFANT is used with another prescription medicine called dexamethasone. You can ask your healthcare provider for information about dexamethasone.

For HLH/MAS in Still's disease, after your first dose, GAMIFANT is given every 3 days for 5 doses, then 2 times a week (every 3 to 4 days). Your healthcare provider will do blood tests during your tre… [Excerpted — this section continues on DailyMed.]

🧬 Pharmacokinetics ~2 min read ▾

12.3Pharmacokinetics The pharmacokinetics of emapalumab-lzsg were evaluated in healthy adult subjects and in patients with HLH. Following a 1 mg/kg emapalumab-lzsg dose, median steady state peak concentration was 44 mcg/mL, which was 2.9 times higher than after the first dose. The median steady state trough concentration was 25 mcg/mL, which was 4.3 times higher than after the first dose.

Emapalumab-lzsg AUC increases slightly more than proportionally between 1 and 3 mg/kg doses, and less than proportionally at 3, 6, and 10 mg/kg doses. Emapalumab-lzsg exhibits target-mediated-like clearance dependent on IFNγ production, which can vary between and within patients as a function of time and can affect the recommended dosage [see Dosage and Administration ( 2.2 )] . Emapalumab-lzsg steady state is achieved by the 7th infusion when the IFNγ production is moderate.

At high IFNγ production, steady-state is reached earlier due to a shorter half-life. Distribution The central and peripheral volumes of distribution in a subject with body weight of 70 kg are 2.8 and

4.4L, respectively. Elimination Emapalumab-lzsg elimination half-life is approximately 22 days in healthy subjects and ranged from 2.5 to 18.9 days in HLH patients. In patients with HLH, the elimination half-life of emapalumab-lzsg was significantly influenced by the amount of IFNγ within the patient, demonstrating a target mediated clearance-like mechanism.

The elimination half-life of emapalumab-lzsg is reduced as the concentration of IFNγ increases. Emapalumab-lzsg clearance is approximately 0.007 L/h in healthy subjects. Metabolism The metabolic pathway of emapalumab-lzsg has not been characterized.

Like other protein therapeutics, GAMIFANT is expected to be degraded into small peptides and amino acids via catabolic pathways. Specific Populations Body weight (3 to 80 kg) was a significant covariate of emapalumab-lzsg pharmacokinetics, supporting body weight-based dosing. No clinically significant differences in the pharmacokinetics of emapalumab-lzsg were observed based on age (0.02 to 64 year), sex (53% Females), race (71.4% Caucasian, 12.2% Asian and 8.2% Black), renal impairment including dialysis, or hepatic impairment (mild, moderate, and severe).

Drug Interaction Studies No drug-drug interaction studies have been conducted with GAMIFANT.

🧬 Pharmacodynamics 36 words ▾

12.2Pharmacodynamics IFNγ Inhibition Emapalumab-lzsg reduces the plasma concentrations of CXCL9, a chemokine induced by IFNγ. Cardiac Electrophysiology At a dose of 3 mg/kg GAMIFANT does not prolong the QT interval to any clinically relevant extent.

🔬 Clinical Studies ~3 min read ▾

14 CLINICAL STUDIES

14.1Primary Hemophagocytic Lymphohistiocytosis The efficacy of GAMIFANT was evaluated in a multicenter, open-label, single-arm trial NI-0501-04 ( NCT01818492 ) in 27 pediatric patients with suspected or confirmed primary Hemophagocytic lymphohistiocytosis (HLH) with either refractory, recurrent, or progressive disease during conventional HLH therapy or who were intolerant of conventional HLH therapy. Patients were required to fulfill the following criteria for enrollment: primary HLH based on a molecular diagnosis or family history consistent with primary HLH or five out of the 8 criteria fulfilled: fever, splenomegaly, cytopenias affecting 2 of 3 lineages in the peripheral blood (hemoglobin < 9, platelets < 100 x 10 9 /L, neutrophils < 1 x 10 9 /L), hypertriglyceridemia (fasting triglycerides > 3 mmol/L or ≥265 mg/dL) and/or hypofibrinogenemia (≤1.5 g/L), hemophagocytosis in bone marrow, spleen, or lymph nodes with no evidence of malignancy, low or absent NK-cell activity, ferritin ≥500 mcg/L, soluble CD25 ≥2400 U/mL.

Patients had to have evidence of active disease as assessed by the treating physician. Patients had to fulfill one of the following criteria as assessed by the treating physician: having not responded or not achieved a satisfactory response or not maintained a satisfactory response to conventional HLH therapy, or intolerance to conventional HLH treatments. Patients with active infections caused by specific pathogens favored by IFNγ neutralization were excluded from the trial (e.g., mycobacteria and Histoplasma c apsulatum ).

Patients received prophylaxis for herpes zoster, Pneumocystis jirovecii , and fungal infections. Twenty-seven patients enrolled and received treatment in the study and twenty patients (74%) completed the study. Seven patients (26%) were prematurely withdrawn.

Twenty-two patients (81%) enrolled onto the open-label extension study which monitored patients for up to 1 year after HSCT or after the last GAMIFANT infusion (NI-0501-05; NCT02069899 ). The study treatment duration was up to 8 weeks after which patients could continue treatment on the extension study. All patients received an initial starting dose of GAMIFANT of 1 mg/kg every 3 days.

Subsequent doses could be increased to a maximum of 10 mg/kg based on clinical and laboratory parameters interpreted as unsatisfactory response. Forty-four percent of patients remained at a dose of 1 mg/kg, 30% of patients increased to 3-4 mg/kg and 26% of patients increased to 6-10 mg/kg. The median time to dose increase was 27 days (range: 3-31 days) with 22% of patients requiring a dose increase in the first week of treatment.

All patients received dexamethasone as background HLH treatment with doses between 5 to 10 mg/m 2 /day. Cyclosporine A was continued if administered prior to screening. Patients receiving methotrexate and glucocorticoids administered intrathecally at baseline could continue these treatments.

In Study NI-0501-04, the median patient age was 1 year (0.2 to 13). Fifty-nine percent of the patients were female, 63% were Caucasian, 11% were Asian, and 11% were Black. A genetic mutation known to cause HLH was present in 82% of patients.

The most frequent causative mutations were FHL3-UNC13D (MUNC 13-4) (26%), FHL2-PRF1 (19%), and Griscelli Syndrome type 2 (19%). The HLH mutations in the population enrolled are described in Table 6 Table 6: HLH Mutations in Patients with Primary HLH with Prior Therapy GAMIFANT (N=27) HLH Genetic Confirmation 22 (82) FHL3-UNC13D 7 (26) FHL2-PRF1 5 (19) Griscelli Syndrome type 2 (RAB27A) 5 (19) FHL5-STXBP2 (UNC18B) 2 (7.4) FHL4-STX11 1 (3.7) X-linked Lymphoproliferative Disorder 1 1 (3.7) X-linked Lymphoproliferative Disorder 2 1 (3.7) All patients received previous HLH treatments.

Patients received a median of 3 prior agents before enrollment into the trial. Prior regimens included combinations of the following agents: dexamethasone, etoposide, cyclosporine A, and anti-thymocyte globul… [Excerpted — this section continues on DailyMed.]

🧪 Nonclinical Toxicology 55 words ▾

13 NONCLINICAL TOXICOLOGY

13.1Carcinogenesis, Mutagenesis, Impairment of Fertility No carcinogenicity or genotoxicity studies have been conducted with emapalumab-lzsg. No studies have been conducted to evaluate the effects of emapalumab-lzsg on fertility; however, no adverse effects on male or female reproductive organs were observed in the 8- or 13-week repeat-dose toxicity studies in cynomolgus monkeys.

📄 Carcinogenesis, Mutagenesis, Impairment of Fertility 52 words ▾

13.1Carcinogenesis, Mutagenesis, Impairment of Fertility No carcinogenicity or genotoxicity studies have been conducted with emapalumab-lzsg. No studies have been conducted to evaluate the effects of emapalumab-lzsg on fertility; however, no adverse effects on male or female reproductive organs were observed in the 8- or 13-week repeat-dose toxicity studies in cynomolgus monkeys.

📄 Recent Major Changes 28 words ▾

RECENT MAJOR CHANGES Indications and Usage ( 1 ) 6/2025 Dosage and Administration ( 2.2 , 2.3 ) 6/2025 Warnings and Precautions ( 5.1 , 5.3 ) 03/2026

📄 Package Label / Principal Display Panel ~1 min read ▾

Principal Display Panel - 10 mg/2 mL Carton Label NDC 66658-501-01 Gamifant ® (emapalumab-lzsg) Injection 10 mg/2 mL (5 mg/mL) For intravenous infusion only. Requires dilution prior to administration. Single-dose vial. Discard unused portion. Rx only Dispense the enclosed Medication Guide to each patient. image description

Principal Display Panel - 50 mg/10 mL Carton Label NDC 66658-505-01 Gamifant ® (emapalumab-lzsg) Injection 50 mg/10 mL (5 mg/mL) For intravenous infusion only. Requires dilution prior to administration. Single-dose vial. Discard unused portion. Rx only Dispense the enclosed Medication Guide to each patient. image description

Principal Display Panel - 100 mg/20 mL Carton Label NDC 66658-510-01 Gamifant ® (emapalumab-lzsg) Injection 100 mg/20 mL (5 mg/mL) For intravenous infusion only. Requires dilution prior to administration. Single-dose vial. Discard unused portion. Rx only Dispense the enclosed Medication Guide to each patient. image description

Principal Display Panel – 50 mg/2 mL Carton Label NDC 66658-522-01 Gamifant ® (emapalumab-lzsg) Injection 50 mg/2 mL (25 mg/mL) For intravenous infusion only. Requires dilution prior to administration. Single-dose vial. Discard unused portion. Rx only Dispense the enclosed Medication Guide to each patient. image description

Principal Display Panel – 100 mg/4 mL Carton Label NDC 66658-523-01 Gamifant ® (emapalumab-lzsg) Injection 100 mg/4 mL (25 mg/mL) For intravenous infusion only. Requires dilution prior to administration. Single-dose vial. Discard unused portion. Rx only Dispense the enclosed Medication Guide to each patient. image description

Principal Display Panel – 250 mg/10 mL Carton Label NDC 66658-524-01 Gamifant ® (emapalumab-lzsg) Injection 250 mg/10 mL (25 mg/mL) For intravenous infusion only. Requires dilution prior to administration. Single-dose vial. Discard unused portion. Rx only Dispense the enclosed Medication Guide to each patient. image description

Principal Display Panel – 500 mg/20 mL Carton Label NDC 66658-525-01 Gamifant ® (emapalumab-lzsg) Injection 500 mg/20 mL (25 mg/mL) For intravenous infusion only. Requires dilution prior to administration. Single-dose vial. Discard unused portion. Rx only Dispense the enclosed Medication Guide to each patient. image description

Source: FDA Structured Product Labeling, mirrored from DailyMed / openFDA. Prefer the government’s original formatting? View this label on DailyMed ↗

Medicaid utilization & spend

📍 This exact package only: Medicaid data is reported per full 11-digit NDC — labeler, product and pack size — so every number here is for this package alone, not the drug overall. Other pack sizes report separately.
💊 Pharmacy benefit only: These are Medicaid outpatient pharmacy claims, billed by NDC. They exclude the medical benefit — clinic- or hospital-administered drugs billed under HCPCS J-codes — so drugs used mostly that way (e.g. Avastin, Lucentis, Keytruda) can look low or missing here. That’s expected, not an error.
📅 Q1 2025 – Q4 2025 · 4 quarters of data
ⓘ The newest quarter is usually incomplete when first published; states restate recent quarters in later CMS releases, so the latest figures typically revise upward. State coverage-policy changes can also shift quarter-to-quarter totals.
Prescriptions last 4 qtrs
15
Units reimbursed last 4 qtrs
168
Latest quarter Q4 2025
0Rx
Fee-for-service vs managed care ⓘ
100% MCO
Fee-for-service · 0 Rx Managed care · 15 Rx
State Medicaid map
Alaska: no data reported AK Maine: no data reported ME Washington: no data reported WA Idaho: no data reported ID Montana: no data reported MT North Dakota: no data reported ND Minnesota: no data reported MN Wisconsin: no data reported WI Michigan: no data reported MI New York: no data reported NY Vermont: no data reported VT New Hampshire: no data reported NH Oregon: no data reported OR Nevada: no data reported NV Wyoming: no data reported WY South Dakota: no data reported SD Iowa: no data reported IA Illinois: no data reported IL Indiana: no data reported IN Ohio: no data reported OH Pennsylvania: no data reported PA New Jersey: no data reported NJ Massachusetts: no data reported MA California: no data reported CA Utah: no data reported UT Colorado: no data reported CO Nebraska: 168 units · 8.5 per 100k residents NE Missouri: no data reported MO Kentucky: no data reported KY West Virginia: no data reported WV Virginia: no data reported VA Maryland: no data reported MD Connecticut: no data reported CT Rhode Island: no data reported RI Arizona: no data reported AZ New Mexico: no data reported NM Kansas: no data reported KS Arkansas: no data reported AR Tennessee: no data reported TN North Carolina: no data reported NC South Carolina: no data reported SC Delaware: no data reported DE Oklahoma: no data reported OK Louisiana: no data reported LA Mississippi: no data reported MS Alabama: no data reported AL Georgia: no data reported GA D.C.: no data reported DC Hawaii: no data reported HI Texas: no data reported TX Florida: no data reported FL
Units reimbursed · per 100k residents
8.58.5
gray = no data reported ⓘ
Colors are per 100,000 residents, so big states don’t automatically dominate. Tap or hover a state for its actual totals.
Tap or hover a state
…for its Medicaid breakdown
🏆 Top states by units · per 100k residents
1 Nebraska 8.5 /100k
National units — by quarter
💵 About the dollar figures: “reimbursed” is what Medicaid paid pharmacies before confidential manufacturer rebates, so the program’s real net cost is lower than these numbers. Fee-for-service and managed-care claims are combined unless split above. Source: CMS State Drug Utilization Data; per-100k rates use 2023 Census population estimates.

Medicare Part D spend CMS · PART D · 2025 (Q1-Q4)

Medicare Part D (outpatient prescription) spending for Gamifant — the program that covers self-administered drugs. 1 manufacturer.
⚠️ Drug-level data: CMS publishes Part D spending by drug, not by NDC — these figures combine every manufacturer, strength and package size sold under the name Gamifant. That’s a different level of aggregation than the Medicaid card above, which is specific to this exact 11-digit NDC (pack size included), so the two aren’t directly comparable.
Total Part D spend
$4.61M
Claims incl. refills
67
Beneficiaries
—
Spend / beneficiary
—
Spend / claim
$68,746.50
💵 About the dollar figures: spending is what Part D plans paid before confidential manufacturer rebates, so the program’s real net cost is lower. A blank patient count means fewer than 11 people — CMS hides counts that small to protect privacy. Source: CMS Medicare Quarterly Part D Spending by Drug (data.cms.gov), updated quarterly.

Reported adverse events (FAERS)

Read carefully: FAERS reports are voluntary and unverified. Counts are not incidence, do not establish causation, are subject to reporting bias, and cannot be used to compare one drug to another. Shown for signal context only. Reports for GAMIFANT (this brand).

Top reported reactions

Haemophagocytic Lymphohistiocytosis58
Condition Aggravated47
Death26
Septic Shock19
Epstein-barr Virus Infection14
Adenovirus Infection13
Hypertension12

Age at onset

Infant5
Child7
Adolescent4
Adult14
Elderly2

Reporter sex

0 reports
Male · 51%
Female · 49%

Serious outcomes

Hospitalization55
Life-threatening25
Reports over time (by year) — tap or hover for the count & year
2019 2021 2023 2026 62 0
Most recent year is provisional (FAERS lags ~3 months).
Where does this data come from?
Adverse-event reports from the FDA Adverse Event Reporting System (FAERS) via openFDA. FAERS reports are voluntary and unverified — counts are not incidence and don’t establish causation.

About this NDC listing & data coverage

Finished prescription product
What data is (and isn’t) available for this NDC — tap to expand
NDC identity (package / product / labeler codes) ✓ Available
Labeler ✓ Available
Product & package description ✓ Available
Marketing category & status ✓ Available
Active ingredient / dosage form / route ✓ Available
FDA label (SPL via DailyMed) ✓ Available
Package photos ✓ Available
Inactive ingredients (structured) — Not published for this NDC The labeler did not submit a structured excipient list, or no SPL is available.
NADAC pharmacy acquisition price (CMS) — Not published for this NDC CMS publishes NADAC only for NDCs reported in its retail-pharmacy survey.
Orange Book / therapeutic-equivalence data ✓ Available
HCPCS J-code billing crosswalk ✓ Available
Medicaid utilization (CMS SDUD) ✓ Available
“Not published” reflects what the public FDA / CMS / NLM sources provide for this exact package code — it is a property of the data feeds, not a judgment about the product.

Questions about this listing

Why is there no price listed?
The pricing shown on our NDC pages comes from CMS NADAC, a voluntary survey of retail community pharmacy invoices. CMS does not publish a NADAC for every NDC — packages outside the retail survey (institutional and hospital products, bulk packages, discontinued items, and many OTC items) may never receive one. A missing price reflects the survey's scope, not this product's actual cost, and does not mean the product is free or unavailable.
Is the NDC printed on the package the same as the 11-digit billing NDC?
Yes, they identify this exact package in different formats. The form printed on the packaging and shown on DailyMed is the one the FDA registered. Insurance claims use a fixed 11-digit 5-4-2 format, so the short segment is padded with a leading zero and the dashes are dropped. The Identity section at the top of this page lists each form of this code.
Is this package still being marketed?
Yes, per the latest FDA NDC Directory data on this page: this package is listed as actively marketed, with no marketing end date reported by Swedish Orphan Biovitrum AB (publ). Listing status can change — the directory data on this page refreshes weekly.
Who lists this product with the FDA?
Swedish Orphan Biovitrum AB (publ) is the labeler of record for this NDC — the company under whose FDA-assigned code the package is listed. The labeler may be the manufacturer itself or a distributor marketing the product under its own code.
Do I need a prescription for this product?
This NDC is listed with FDA as a prescription product, so it is dispensed under a prescriber's order. Your pharmacist can tell you whether any over-the-counter forms of the same medication exist.
Does this product have a billing J-code?
Yes — this NDC cross-references HCPCS code J9210 for medical-claim billing (typically used when a product is administered in a clinical setting rather than dispensed at a retail pharmacy). See the Billing section on this page.
This page identifies an FDA-listed package (the NDC) and reports public regulatory and pricing data about the listing. It is reference information, not a medical recommendation — talk to your pharmacist or prescriber about your own medication.
Where does this data come from?
Listing facts (marketing category, packager status, marketing dates) from the FDA openFDA NDC Directory; label availability from DailyMed; pricing coverage from CMS NADAC; equivalence scope from the FDA Orange Book.
← 66658-0501-01 · Swedish 66658-0510-01 · Swedish →
For educational and professional reference only — not medical advice. Pricing reflects published NADAC and CMS ASP (free public data) and may differ from your acquisition cost; always verify before billing or dispensing.