Home › NDC Lookup › Ingredients › Tofersen › 64406-0109-01
QALSODY tofersen 100 mg/15mL Injection — NDC 64406-0109-01 package photo
Label image from the product's FDA listing (DailyMed) — may show a different pack size or an older label revision.

QALSODY tofersen 100 mg/15mL Injection — NDC 64406-109-01 (Billing 64406-0109-01)

by Biogen Inc. · 1 VIAL, SINGLE-USE in 1 CARTON / 15 mL in 1 VIAL, SINGLE-USE

This is a package of QALSODY tofersen 100 mg/15mL Injection from Biogen Inc., marketed since Apr 2023 and currently FDA-listed. It is this product's only package size.

NDC 64406-0109-01
🏷️ FDA NDC (as labeled) 64406-109-01 billing pads the product segment with a zero
Rx only Brand On market Non-controlled ⇄ Compare with another NDC
🗂️ FDA directory synced Oct 8, 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 64406-109-01 alone.

Record
FDA NDC Directory package listing · Human prescription drug
Code segments
64406 labeler · 109 product · 01 package
Package marketed since
Apr 25, 2023
Sample package
No — commercial package
Listing certified through
Dec 31, 2026
Barcode (UPC)
0364406109010
FDA record last changed
Jul 24, 2026

Identity & classification

Regulatory identifiers FDA, NLM and CMS codes for this package

FDA NDC (as labeled) 64406-109-01
Product NDC 64406-109
11-digit billing NDC 64406010901
NCPDP billing unit ML — per mL (volume)
RxCUI 2634999, 2635005
UNII 2NU6F9601K
UPC 0364406109010
Application # NDA215887
SPL Set ID 81356b45-1cb7-4eef-88ea-e44cc18b47c5
Established class (EPC) Antisense Oligonucleotide
Chemical class Oligonucleotides, Antisense
DEA schedule Non-controlled
Marketing category NDA
Marketing status On market
FDA listing status Listed (active directory)
Marketing start 2023-04-25
Route INTRATHECAL
Dosage form INJECTION
Substance TOFERSEN

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

GPI-14 74504080002020
GCN Seq No 084682
GCN 54035
HICL code 048878
Ingredient (HICL) Tofersen
HIC1 code H
Therapeutic class — broad (HIC1) Nervous System (Except Autonomic)
HIC2 code H6
Therapeutic class — intermediate (HIC2) Drugs Acting Principally On The Midbrain
HIC3 code H6I
Therapeutic class — specific (HIC3) Amyotrophic Lateral Sclerosis Agents
AHFS code 28:44.00.00
AHFS class Amyotrophic Lateral Sclerosis(Als) Agent
FDB label name QALSODY 100 MG/15 ML VIAL
FDB brand name Qalsody
Legend status F — Federal legend — prescription drug or device
Quick answers
  • GSN (GCN sequence number): 084682
  • GCN: 54035
  • GPI-14 (Medi-Span): 74504080002020
  • HICL (First Databank): 048878
  • AHFS class code: 28:44.00.00
  • RxCUI (RxNorm): 2634999
Why two NDCs? The FDA registers this code as 64406-109-01 — 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 → 64406-0109-01. 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 Antisense Oligonucleotide class.

Pharmacologic class Antisense Oligonucleotide
Drug family (ATC) Other nervous system drugs
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 QALSODY 100 MG/15 ML VIAL Ingredient Tofersen
📖 What it is MedlinePlus · NLM

Tofersen injection is used to treat a certain type of amyotrophic lateral sclerosis (ALS, Lou Gehrig's disease; a condition in which the nerves that control muscle movement slowly die, causing the muscles to shrink and weaken) in adults with a certain genetic make-up. Tofersen injection is in a class of medications called antisense oligonucleotides. It works by decreasing the production of a harmful protein that builds up in the brain and spinal cord in ALS patients.

Read the full MedlinePlus article ↗

Supplement & herbal interactions

🌿 Drug–supplement interaction checker →
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 · J1304 $164.816 / J1304 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)64406-109-01
11-digit billing NDC64406-0109-01
Format5-3-2 as registered → padded to 5-4-2 for billing (zero added to the product segment)
HCPCS J-codeJ1304
DescriptorINJECTION, TOFERSEN, 1 MG
Billing units / pkg6.7 units
How the units are derivedThis package is 15 ML; the HCPCS unit is 1 MG, so one package = 6.7 billing units.
Medicare Part B spend (2026 (Q1))$2,718,644 · 172 claims · $15,806.07 per claim (all NDCs under J1304)
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
64406-0109-01 You're viewing this Main listing 1 VIAL, SINGLE-USE in 1 CARTON / 15 mL in 1 VIAL, SINGLE-USE 2023-04-25 — Active

Therapeutic equivalents

ProductLabelerPackNADAC/unitTEStatusPrice vs. this
Qalsody 100 mg/15mLthis 64406-0109-01 Biogen 1 vial — — FDA listed —
About this product: this is the brand-name version. We did not find an FDA-approved generic match for this exact strength, form and route.
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 & generic status

🏛️
2023
First FDA approval
Apr 2023
📍
2026
Currently FDA-listed
3 years listed
🛡️
2035
Latest patent/protection listed
not a guaranteed launch date
🔒No FDA-approved generic found

We did not find an FDA-approved generic match for this exact strength, form and route. Patent/protection dates below may affect future generic timing.

🛡️ Latest patent/protection date listed: FDA patent/protection data lists protections through Apr 2035. This may affect when a full generic version becomes widely available, but it is not a guaranteed launch date.
📅 FDA approved Apr 25, 2023 RLD RS ⏳ ~8.5 yr to latest listed protection

Why the date isn’t exact: Generic timing can change because patents may be challenged, settled, licensed, added, removed, or worked around with a narrower label — and FDA approval does not always mean a pharmacy can get the generic today.

Patents & exclusivity — FDA Orange Book
US 10968453 — method of use (U-3575)
US 10669546 — method of use (U-3575)
US 10385341 — drug substance
Exclusivity NCE
Exclusivity ODE-432
2023 2025 2027 2029 2031 2033 2035
Today
LOE
Substance patent Formulation patent Method-of-use patent Exclusivity Pediatric +6mo
🏛️FDA exclusivity
FDA-granted marketing protection. It’s separate from patents and may be shorter than patent protection.
🧪Product / substance patents
Patents covering the active ingredient, product, formulation, or related drug features.
🎯Method-of-use patents
Patents covering specific approved uses. These can sometimes be carved out with a “skinny label,” but not always.
🛈 What do these terms mean?
Patent
Legal protection listed in the Orange Book that may delay generic approval or launch. Issued by the U.S. Patent & Trademark Office.
Substance patent
Covers the active drug molecule itself — the hardest to design around. A generic generally can’t launch until it expires.
Formulation (product) patent
Covers a specific formulation or dosage form. A generic can sometimes work around it with a different formulation.
Method-of-use patent
A patent covering one specific approved use of the drug — not necessarily the whole molecule. A generic can sometimes launch with a “skinny label” that carves out the protected use and keeps the others.
Skinny label
A generic label that omits a still-patented use when the FDA allows it — letting a generic reach the market for the unprotected uses.
Exclusivity
FDA-granted marketing protection, separate from patents — e.g. 5-yr new chemical entity, 7-yr orphan drug, or a +6-month pediatric extension.
Paragraph IV
A generic applicant’s formal challenge to a listed patent. It can potentially lead to earlier generic entry, but often involves litigation or a settlement.
RLD / RS
Reference Listed Drug — the brand product the FDA uses as the reference for generic applications. Reference Standard — the product the FDA expects generics to compare against in bioequivalence testing.
TE / AB rating
FDA therapeutic-equivalence rating. An AB rating generally means the FDA considers a generic therapeutically equivalent to — and substitutable for — the brand.
LOE (loss of exclusivity)
The latest patent or exclusivity currently listed — the loss-of-exclusivity / latest-listed-protection date shown on this page. Paragraph-IV challenges and settlements can move the real date earlier; FDA approval and a manufacturer’s decision to market can move it later.

Built from the FDA Orange Book. The bars above are scaled to each protection’s expiry; the red LOE marker is the last one to lapse.

Listed patents (3)
PatentTypeUse codeExpires
US 10968453 ↗ Method of use U-3575 Apr 1, 2035
US 10669546 ↗ Method of use U-3575 Apr 1, 2035
US 10385341 ↗ Drug substance — Apr 1, 2035
FDA exclusivity
CodeWhat it grantsExpires
NCENew Chemical Entity (5-year)Apr 25, 2028
ODE-432Orphan Drug Exclusivity (7-year)Apr 25, 2030
Common questions
Is there a generic version of QALSODY 100 MG/15 ML VIAL?
No FDA-approved generic equivalent is currently listed in the FDA Orange Book for QALSODY 100 MG/15 ML VIAL. Based on the patents and exclusivity currently listed, the Orange Book estimate is that full-label generic entry may be delayed until Apr 2035 — an estimate, not a guaranteed launch date.
The FDA approved a generic — why can’t I get it at my pharmacy yet?
FDA approval and pharmacy availability are two different things. The FDA can approve a generic years before it actually reaches pharmacies, because the brand company may still hold patents or have a settlement that delays the launch. A manufacturer also has to choose to make and sell it, and have supply ready. So a drug can be “FDA-approved generic exists” and still be brand-only at the counter today.
Why do different websites show different generic release dates?
Generic availability is not based on one single date. Some sources use the first exclusivity expiration, some use the last product patent, and others use the latest method-of-use patent. Patent challenges, settlements, licenses, and label carve-outs can also change the real-world launch date. This page shows the underlying Orange Book dates so you can see why estimates may differ.
What does “FDA listed” mean?
It means the product appears in the FDA’s official NDC directory. That’s a good sign a product exists and is intended for the U.S. market, but on its own it does not confirm a pharmacy can fill it today. Where we have recent retail pricing data (NADAC) for a product, 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 Orange Book for a patent or exclusivity on the brand product. It can affect when a full generic version becomes widely available — but it is not a guaranteed generic launch date. Generics sometimes arrive earlier (through a settlement or patent challenge) or later (a manufacturer still has to make and sell one).
What does “current Orange Book estimate” mean?
It means we are using the latest patent and exclusivity dates currently listed in the FDA Orange Book. It is not a guaranteed launch date.
Can a generic come out before the last patent expires?
Sometimes. A generic company may challenge a patent, settle with the brand manufacturer, receive a license, or obtain approval with a narrower label that avoids a patented use. In other cases, the last listed protection may delay full-label generic competition.
Can a generic come out after the listed dates?
Yes. Even after patents or exclusivity expire, a generic still needs FDA approval and a manufacturer must choose to market it. Supply, litigation, business decisions, or regulatory issues can delay actual availability.
What is the difference between patents and exclusivity?
Patents are legal protections usually issued by the U.S. Patent and Trademark Office. FDA exclusivity is marketing protection granted by the FDA. They are separate, and either one can affect generic timing.
Why are there multiple patent dates?
One drug can have several patents covering different things: the active ingredient, a formulation, a manufacturing process, or a specific approved use. That is why a page may show several expiration dates instead of one simple generic date.
Built from FDA Orange Book patent and exclusivity data. Dates are refreshed from public FDA data when available; the marker is max(latest patent expiry, latest exclusivity expiry). Paragraph-IV settlements and first-filer 180-day exclusivity can shift the real date; a method-of-use patent may allow an earlier skinny-label generic for non-protected indications. Generic launch timing is an estimate, not a guarantee.
Where does this data come from?
Patents and exclusivity from the FDA Orange Book (small-molecule drugs), refreshed from public FDA data. Generic 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.

💡 Tap an ingredient (hover on desktop) to see what it is and why it’s used.

  • UNII M4I0D6VV5M
    Calcium chloride is a salt compound that acts as a firming agent and source of calcium ions in medications. It's used in formulations to help maintain tablet structure, improve texture, or serve as a buffering agent.
  • UNII 02F3473H9O
    A mineral salt used in medications as a source of magnesium. It helps bind ingredients together, improve texture, and maintain the product's stability during storage.
  • UNII 660YQ98I10
    Potassium chloride is a mineral salt used in medicines as a buffer and to help maintain the proper pH balance and stability of the formulation during storage and use.
  • UNII 451W47IQ8X
    Sodium chloride is common table salt. It's used in medicines as a buffer to maintain proper pH, as a filler to add bulk, or to adjust the osmotic balance in liquid formulations.
  • UNII GR686LBA74
    Sodium phosphate, dibasic is a salt derived from phosphoric acid. It acts as a buffer to help maintain the medicine's pH balance and may serve as a binder or filler in tablets and capsules.
  • UNII 3980JIH2SW
    A salt form of phosphoric acid that acts as a buffer and pH adjuster in medicines. It helps keep the product at the correct acidity level for stability and effectiveness.
  • UNII 059QF0KO0R
    Water is a liquid solvent that dissolves and mixes ingredients together in liquid medicines, syrups, and injections. It helps distribute the active drug evenly throughout the product.

7 inactive ingredients listed in the exact product block matched to this NDC.

Where does this data come from?
Data sourced from official FDA Structured Product Labeling (SPL) via DailyMed — ingredient classCode="IACT" elements from the exact product block matched by this NDC. Label-section narrative from DailyMed / the openFDA label index is shown separately when available.

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

LabelerBiogen Inc.
Application holderBIOGEN MA INC
FDA applicationNDA215887 (NDA)
Labeler code64406
First marketedApr 2023
Product typeHuman Prescription Drug
Portfolio47 products on file

More NDCs from Biogen Inc. labeler code 64406

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 👥 Use in Specific Populations 🤰 Pregnancy 🧒 Pediatric Use 🧓 Geriatric Use 🧬 Clinical Pharmacology 🧬 Mechanism of Action 📦 How Supplied / Storage and Handling 📦 Storage and Handling 📋 Description 💬 Information for Patients 🧬 Pharmacokinetics 🧬 Pharmacodynamics 🔬 Clinical Studies 🧪 Nonclinical Toxicology 📄 Carcinogenesis, Mutagenesis, Impairment of Fertility 📄 Package Label / Principal Display Panel
🎯 Indications and Usage 144 words ▾

1 INDICATIONS AND USAGE QALSODY is indicated for the treatment of amyotrophic lateral sclerosis (ALS) in adults who have a mutation in the superoxide dismutase 1 ( SOD1 ) gene. This indication is approved under accelerated approval based on reduction in plasma neurofilament light chain (NfL) observed in patients treated with QALSODY [see Clinical Studies ( 14 )] . Continued approval for this indication may be contingent upon verification of clinical benefit in confirmatory trial(s).

QALSODY is an antisense oligonucleotide indicated for the treatment of amyotrophic lateral sclerosis (ALS) in adults who have a mutation in the superoxide dismutase 1 ( SOD1 ) gene. This indication is approved under accelerated approval based on reduction in plasma neurofilament light chain observed in patients treated with QALSODY. Continued approval for this indication may be contingent upon verification of clinical benefit in confirmatory trial(s).

( 1 )

⏱️ Dosage and Administration ~2 min read ▾

2 DOSAGE AND ADMINISTRATION QALSODY is administered intrathecally ( 2.1 ) Dosing Information ( 2.1 ) Recommended dose: 100 milligrams (15 mL) per administration Initiate QALSODY treatment with 3 loading doses administered at 14day intervals. A maintenance dose should be administered once every 28 days thereafter. Preparation and Administration Instructions ( 2.2 ) Allow to warm to room temperature prior to administration Administer within 4 hours of removal from vial Prior to administration, remove approximately 10 mL of cerebrospinal fluid Administer as an intrathecal bolus injection over 1 to 3 minutes 2.1.

Dosing Information Recommended Dosage Administer QALSODY intrathecally using a lumbar puncture by, or under the direction of, healthcare professionals experienced in performing lumbar punctures. The recommended dosage is 100 mg (15 mL) of QALSODY per administration. Initiate QALSODY treatment with three (3) loading doses administered at 14-day intervals.

Administer a maintenance dose every 28 days thereafter. Missed Dose If the second loading dose is missed, administer QALSODY as soon as possible, and administer the third loading dose 14 days later. If the third loading dose or a maintenance dose is missed, administer QALSODY as soon as possible, and administer the next dose 28 days later.

2.2. Preparation and Administration Instructions Use aseptic technique when preparing and administering QALSODY intrathecally. Prepare and administer QALSODY according to the following steps: Preparation Vial preparation instructions Allow refrigerated QALSODY vial to warm to room temperature (25°C/77°F) prior to administration without using external heat sources [see Storage and Handling ( 16.2 )] .

Inspect the solution in the QALSODY vial prior to administration. Do not administer if particles are observed or the liquid in the vial is not clear and colorless to slightly yellow. Do not shake the QALSODY vial.

Procedural preparation instructions If indicated by the clinical condition of the patient, consider sedation. If indicated by the clinical condition of the patient, consider imaging to guide intrathecal administration of QALSODY. Prior to removing the vial's cap on the aluminum overseal, confirm readiness of the patient.

An unopened QALSODY vial can be returned to the refrigerator [see Storage and Handling ( 16.2 )] . Evaluate patients prior to and after intrathecal injection for the presence of potential conditions related to lumbar puncture, to avoid serious procedural complications. Administration Prior to administration, remove approximately 10 mL of cerebrospinal spinal fluid (CSF) using a lumbar puncture needle.

Prior to administration, remove the plastic cap and attach a needle to the syringe, for the purpose of withdrawing QALSODY from the vial. Insert the needle into the vial through the center of the overseal and withdraw the required dose of 15 mL (equivalent to 100 mg) from the vial. Do not dilute QALSODY.

External filters are not required. Administer QALSODY using a lumbar puncture needle as an intrathecal bolus injection over 1 to 3 minutes. QALSODY contains no preservatives.

Once drawn into the syringe, the solution should be administered immediately (within 4 hours of removal from the vial) at room temperature; otherwise, it must be discarded. Any unused contents of the single-dose vial should be discarded.

💊 Dosage Forms and Strengths 38 words ▾

3 DOSAGE FORMS AND STRENGTHS Injection: 100 mg/15 mL (6.7 mg/mL) as a clear and colorless to slightly yellow solution in a single-dose vial. Injection: 100 mg/15 mL (6.7 mg/mL) solution in a single-dose vial ( 3 )

⛔ Contraindications 7 words ▾

4 CONTRAINDICATIONS None. None ( 4 )

⚠️ Warnings and Cautions ~2 min read ▾

5 WARNINGS AND PRECAUTIONS Myelitis and/ or Radiculitis: Serious events of myelitis and radiculitis have been reported. Monitor for symptoms; diagnostic workup and treatment should be initiated according to the standard of care. ( 5.1 ) Papilledema and Elevated Intracranial Pressure: Serious events of papilledema and elevated intracranial pressure have been reported.

Monitor for symptoms; diagnostic workup and treatment should be initiated according to standard of care. ( 5.2 ) Aseptic Meningitis: Serious events of aseptic meningitis have been reported. Monitor for symptoms; diagnostic workup and treatment should be initiated according to standard of care.

( 5.3 ) 5.1. Myelitis and/or Radiculitis Serious adverse reactions of myelitis and radiculitis have been reported in patients treated with QALSODY. Six patients treated with QALSODY experienced myelitis or radiculitis in the clinical studies.

Two patients discontinued treatment with QALSODY and required symptomatic management with full resolution of symptoms. In the remaining 4 patients, symptoms resolved without discontinuation of QALSODY. If symptoms consistent with myelitis or radiculitis develop, diagnostic workup and treatment should be initiated according to the standard of care.

Management may require interruption or discontinuation of QALSODY. 5.2. Papilledema and Elevated Intracranial Pressure Serious adverse reactions of papilledema and elevated intracranial pressure have been reported in patients treated with QALSODY.

Four patients developed elevated intracranial pressure and/or papilledema. All patients received treatment with standard of care with resolution of symptoms, and no events led to discontinuation of QALSODY. If symptoms consistent with papilledema or elevated intracranial pressure develop, diagnostic workup and treatment should be initiated according to the standard of care.

5.3. Aseptic Meningitis Serious adverse reactions of aseptic meningitis (also called chemical meningitis or drug-induced aseptic meningitis) have been reported in patients treated with QALSODY. One patient experienced a serious adverse reaction of chemical meningitis, which led to discontinuation of QALSODY.

One patient experienced a serious adverse reaction of aseptic meningitis, which did not lead to discontinuation of QALSODY. In addition, nonserious adverse drug reactions of CSF white blood cell increased, and CSF protein increased have also been reported with QALSODY [see Adverse Reactions ( 6.1 )]. If symptoms consistent with aseptic meningitis develop, diagnostic workup and treatment should be initiated according to the standard of care.

🤒 Adverse Reactions ~2 min read ▾

6 ADVERSE REACTIONS The following clinically significant adverse reactions are discussed elsewhere in the labeling: Myelitis and/or Radiculitis [see Warnings and Precautions ( 5.1 )] Papilledema and Elevated Intracranial Pressure [see Warnings and Precautions ( 5.2 )] Aseptic Meningitis [see Warnings and Precautions ( 5.3 )] The most common adverse reactions ( ≥ 10% of patients treated with QALSODY and greater than placebo) were pain, fatigue, arthralgia, cerebrospinal fluid white blood cell increased, and myalgia. (Section 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Biogen at 1-877-725-7639 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

6.1. Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of QALSODY cannot be directly compared to rates in clinical trials of other drugs and may not reflect the rates observed in practice. The safety of QALSODY 100 mg was evaluated in 147 patients with SOD1-ALS.

The median patient exposure was 119.4 weeks (range from 4 to 212 weeks). QALSODY was evaluated in the placebo-controlled Study 1 and in the open label extension Study 2. In Study 1 Part C, approximately 43% were female; 57% were male; 64% were White and 8% were Asian.

The mean age at entry in Study 1 Part C was 49.8 years (range from 23 to 78 years). The most common adverse reactions (≥ 10% of patients treated with QALSODY and greater than placebo) were pain, fatigue, arthralgia, CSF white blood cell increased, and myalgia. Table 1 shows the common adverse reactions that occurred in at least 5% of patients treated with QALSODY and at a 5% or higher frequency than placebo.

Table 1: Adverse Drug Reactions That Occurred in At Least 5% of Patients Treated with QALSODY and at >5% Higher Frequency Than Placebo ‡‡ Pain includes preferred terms of pain, back pain, and pain in extremity. * CSF white blood cell increased includes preferred terms of CSF white blood cell increased and pleocytosis. Adverse Reaction Study 1 Part C QALSODY 100 mg (n = 72) % Placebo (n = 36) % Pain ‡‡ 42 22 Fatigue 17 6 Arthralgia 14 6 CSF white blood cell increased * 14 0 Myalgia 14 6 CSF protein increased 8 3 Musculoskeletal stiffness 6 0 Neuralgia 6 0 Less Common Adverse Reactions Serious adverse reactions of myelitis and radiculitis; papilledema and elevated intracranial pressure; and aseptic meningitis have occurred in patients treated with QALSODY [see Warnings and Precautions ( 5.1 , 5.2 , 5.3 )] .

In the long-term extension study, nonserious adverse reactions of pyrexia have occurred with repeat administration of QALSODY.

👥 Use in Specific Populations ~2 min read ▾

8 USE IN SPECIFIC POPULATIONS 8.1. Pregnancy Risk Summary There are no adequate data on developmental risks associated with the use of QALSODY in pregnant women to evaluate for a drug-associated risk of major birth defects, miscarriage, or other adverse maternal or fetal 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.

The background risk of major birth defects and miscarriage for the indicated population is unknown. Data Animal Data Subcutaneous administration of tofersen (0, 3, 10, 30 mg/kg) every other day to pregnant mice during the period of organogenesis resulted in no adverse effects on embryofetal development. Plasma exposure at the highest dose tested (30 mg/kg) was approximately 4 times that in humans at the recommended human dose (RHD) of 100 mg.

Subcutaneous administration of tofersen (0, 3, 10, 30 mg/kg) every other day to pregnant rabbits during the period of organogenesis resulted in no adverse effects on embryofetal development. Plasma exposure at the highest dose tested (30 mg/kg) was approximately 20 times that in humans at the RHD. Subcutaneous administration of tofersen (0, 3, 10, or 30 mg/kg) every other day to male and female mice prior to and during mating and continuing in females throughout organogenesis resulted in no adverse effects on pre- or postnatal development.

Plasma exposures at the highest dose tested (30 mg/kg) were approximately 4 times that in humans at the RHD. 8.2. Lactation Risk Summary There are no data on the presence of tofersen or its metabolites in human milk, the effects on the breastfed infant, or the effects on milk production.

Tofersen was detected in the milk of lactating mice following subcutaneous administration. The developmental and health benefits of breastfeeding should be considered along with the mother's clinical need for QALSODY and any potential adverse effects on the breastfed infant from QALSODY or from the underlying maternal condition. 8.4.

Pediatric Use Safety and effectiveness in pediatric patients have not been established. 8.5. Geriatric Use A total of 13.5% (22/162) patients were 65 years of age and older and 1.2% (2/162) patients were 75 years of age and older at initiation of treatment in clinical studies for ALS in patients who have a mutation in the superoxide dismutase 1 ( SOD1 ) gene [see Clinical Studies ( 14 )] .

No overall differences in safety or effectiveness were observed between these patients and younger patients, but a greater sensitivity of some older individuals cannot be ruled out. There is no evidence for special dosage considerations based on age when QALSODY is administered.

🤰 Pregnancy ~1 min read ▾

8.1. Pregnancy Risk Summary There are no adequate data on developmental risks associated with the use of QALSODY in pregnant women to evaluate for a drug-associated risk of major birth defects, miscarriage, or other adverse maternal or fetal 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.

The background risk of major birth defects and miscarriage for the indicated population is unknown. Data Animal Data Subcutaneous administration of tofersen (0, 3, 10, 30 mg/kg) every other day to pregnant mice during the period of organogenesis resulted in no adverse effects on embryofetal development. Plasma exposure at the highest dose tested (30 mg/kg) was approximately 4 times that in humans at the recommended human dose (RHD) of 100 mg.

Subcutaneous administration of tofersen (0, 3, 10, 30 mg/kg) every other day to pregnant rabbits during the period of organogenesis resulted in no adverse effects on embryofetal development. Plasma exposure at the highest dose tested (30 mg/kg) was approximately 20 times that in humans at the RHD. Subcutaneous administration of tofersen (0, 3, 10, or 30 mg/kg) every other day to male and female mice prior to and during mating and continuing in females throughout organogenesis resulted in no adverse effects on pre- or postnatal development.

Plasma exposures at the highest dose tested (30 mg/kg) were approximately 4 times that in humans at the RHD.

🧒 Pediatric Use 13 words ▾

8.4. Pediatric Use Safety and effectiveness in pediatric patients have not been established.

🧓 Geriatric Use 100 words ▾

8.5. Geriatric Use A total of 13.5% (22/162) patients were 65 years of age and older and 1.2% (2/162) patients were 75 years of age and older at initiation of treatment in clinical studies for ALS in patients who have a mutation in the superoxide dismutase 1 ( SOD1 ) gene [see Clinical Studies ( 14 )] . No overall differences in safety or effectiveness were observed between these patients and younger patients, but a greater sensitivity of some older individuals cannot be ruled out.

There is no evidence for special dosage considerations based on age when QALSODY is administered.

🧬 Clinical Pharmacology ~3 min read ▾

12 CLINICAL PHARMACOLOGY 12.1. Mechanism of Action Tofersen is an antisense oligonucleotide that causes degradation of SOD1 mRNA through binding to SOD1 mRNA, which results in a reduction of SOD1 protein synthesis. 12.2.

Pharmacodynamics Effect of Tofersen on Total CSF SOD1 Protein Total CSF SOD1, an indirect measure of target engagement, was evaluated in Study 1 Part C in SOD1-ALS patients [see Clinical Studies ( 14 )] . At Week 28 in Study 1 Part C, a reduction in total CSF SOD1 protein of 35% (geometric mean ratio to baseline) in the tofersen-treated group versus a 2% decrease from baseline in the corresponding placebo subjects in the ITT population was observed (difference in geometric mean ratios for tofersen to placebo: 34%; nominal p<0.0001).

Effect of Tofersen on Neurofilament Proteins Plasma NfL, a blood-based biomarker of axonal injury and neurodegeneration, was evaluated in Study 1 Part C in SOD1-ALS patients [see Clinical Studies ( 14 )] . At Week 28 in Study 1 Part C, mean plasma NfL was reduced 55% (geometric mean ratio to baseline) in the QALSODY-treated subjects, compared to a 12% increase with placebo in ITT population (difference in geometric mean ratios for QALSODY to placebo: 60%; nominal p<0.0001). Plasma NfL declined until approximately Day 113, after which the reductions were sustained.

The reductions in phosphorylated neurofilament heavy chain (pNfH) were similar compared to reductions in NfL, as were reductions in CSF compared to plasma. Cardiac Electrophysiology At the maximum approved recommended dosing regimen, QALSODY does not prolong the QTc interval to any clinically relevant extent. 12.3.

Pharmacokinetics Absorption Intrathecal administration of QALSODY into the CSF allows tofersen to be distributed from the CSF to central nervous system tissues. The maximum CSF trough concentration occurred at the third dose, which was the last dose of the loading period. There was little to no accumulation for CSF tofersen with monthly dosing after the loading phase.

Tofersen is transferred from CSF into the systemic circulation, with median time to maximum concentration (T max ) plasma values ranging from 2 to 6 hours. There was no accumulation in plasma tofersen exposure following monthly maintenance dosing. Distribution Autopsy tissue from patients treated with tofersen (n=3) showed that tofersen administered intrathecally was distributed within the central nervous system tissues.

Elimination Metabolism Tofersen is expected to be metabolized through exonuclease (3'- and 5')-mediated hydrolysis and is not a substrate for, or inhibitor or inducer of CYP450 enzymes. Excretion The primary route of elimination has not been characterized. The effective half-life in CSF is estimated to be 4 weeks.

Drug Interaction Studies No clinical drug interaction studies have been performed. In vitro, QALSODY is not a substrate or inhibitor/inducer of major CYP enzymes or a substrate or inhibitor of major transporters. Specific Populations Effect of sex, race, age, and body weight on tofersen exposure in plasma was not clinically significant.

The effect of these factors on tofersen exposure in CSF is unknown. Patients with Renal or Hepatic Impairment No clinical studies have been conducted to evaluate the pharmacokinetics of tofersen in patients with renal or hepatic impairment. Tofersen is not expected to undergo metabolism by hepatic enzymes.

12.6. Immunogenicity As with all therapeutic oligonucleotides, there is a potential for immunogenicity. Immunogenicity assay results are highly dependent on the sensitivity and specificity of the assay and may be influenced by several factors such as: assay methodology, sample handling, timing of sample collection, concomitant medications, and underlying disease.

For these reasons, comparison of the incidence of antibodies to tofersen with the incidence of antibodies to other products may be misleading. The immunogenic response to QALSODY was evaluated in 166 patients with post-baseline… [Excerpted — this section continues on DailyMed.]

🧬 Mechanism of Action 29 words ▾

12.1. Mechanism of Action Tofersen is an antisense oligonucleotide that causes degradation of SOD1 mRNA through binding to SOD1 mRNA, which results in a reduction of SOD1 protein synthesis.

📦 How Supplied / Storage and Handling 167 words ▾

16 HOW SUPPLIED/STORAGE AND HANDLING 16.1. How Supplied QALSODY injection is a sterile, clear and colorless to slightly yellow solution supplied as 100 mg/15 mL (6.7 mg/mL) solution in a single-dose glass vial free of preservatives. The NDC is 64406-109-01.

16.2. Storage and Handling Store refrigerated between 2°C to 8°C (36°F to 46°F) in the original carton to protect from light. Do not freeze.

If no refrigeration is available, QALSODY may be stored in its original carton, protected from light at or below 30°C (86°F) for up to 14 days. If removed from the original carton, unopened vials of QALSODY can be removed from and returned to the refrigerator, if necessary, for not more than 6 hours per day at or below 30°C (86°F) for a maximum of 6 days (36 hours).

16.1. How Supplied QALSODY injection is a sterile, clear and colorless to slightly yellow solution supplied as 100 mg/15 mL (6.7 mg/mL) solution in a single-dose glass vial free of preservatives. The NDC is 64406-109-01.

📦 Storage and Handling 92 words ▾

16.2. Storage and Handling Store refrigerated between 2°C to 8°C (36°F to 46°F) in the original carton to protect from light. Do not freeze.

If no refrigeration is available, QALSODY may be stored in its original carton, protected from light at or below 30°C (86°F) for up to 14 days. If removed from the original carton, unopened vials of QALSODY can be removed from and returned to the refrigerator, if necessary, for not more than 6 hours per day at or below 30°C (86°F) for a maximum of 6 days (36 hours).

📋 Description 216 words ▾

11 DESCRIPTION Tofersen, an antisense oligonucleotide, is a 20-base residue (20-mer) 5-10-5 MOE gapmer mixed backbone oligonucleotide. Of the nineteen internucleotide linkages, fifteen are 3′-O to 5′-O phosphorothioate diesters, and four are 3′-O to 5′-O phosphate diesters. Ten of the twenty sugar residues are 2-deoxy-D-ribose and the remainder are 2′-O-(2-methoxyethyl)-D-ribose (MOE).

The residues are arranged so that there are five MOE nucleosides at the 5′ and 3′-ends of the molecule flanking a gap of ten 2′-deoxynucleosides. The cytosine and uridine bases are methylated at the 5-position. The structural formula is: Figure 1: Structural Formula for Tofersen The molecular formula is C 230 H 317 N 72 O 123 P 19 S 15 and the molecular weight is 7127.86 atomic mass units (amu).

QALSODY is supplied as a sterile, preservative-free, clear, and colorless to slightly yellow solution in a Type I glass vial to be administered by intrathecal administration. Each vial of drug product contains a single dose of 100 mg tofersen at a concentration of 6.7 mg/mL in a formulation containing 0.21 mg/mL calcium chloride dihydrate, 0.11 mg/mL dibasic sodium phosphate, 0.16 mg/mL magnesium chloride hexahydrate, 0.03 mg/mL monobasic sodium phosphate, 0.22 mg/mL potassium chloride, 8.77 mg/mL sodium chloride, and water for injection.

The pH of QALSODY is approximately 7.2 (range 6.7 to 7.7). Figure 1

💬 Information for Patients 140 words ▾

17 PATIENT COUNSELING INFORMATION Myelitis and/or Radiculitis Inform patients and caregivers that QALSODY could cause myelitis and radiculitis. Instruct patients and caregivers to contact their healthcare provider if symptoms consistent with these adverse reactions develop [see Warnings and Precautions ( 5.1 )] . Papilledema and Elevated Intracranial Pressure Inform patients and caregivers that QALSODY could cause papilledema and elevated intracranial pressure.

Instruct patients and caregivers to contact their healthcare provider if symptoms consistent with these adverse reactions develop [see Warnings and Precautions ( 5.2 )] . Aseptic Meningitis Inform patients and caregivers that QALSODY could cause aseptic meningitis. Instruct patients and caregivers to contact their healthcare provider if symptoms consistent with meningitis develop [see Warnings and Precautions ( 5.3 )].

57294-01 Manufactured by: Biogen MA Inc. Cambridge, MA 02142 QALSODY is a trademark of Biogen MA Inc. © Biogen Inc.

🧬 Pharmacokinetics ~1 min read ▾

12.3. Pharmacokinetics Absorption Intrathecal administration of QALSODY into the CSF allows tofersen to be distributed from the CSF to central nervous system tissues. The maximum CSF trough concentration occurred at the third dose, which was the last dose of the loading period.

There was little to no accumulation for CSF tofersen with monthly dosing after the loading phase. Tofersen is transferred from CSF into the systemic circulation, with median time to maximum concentration (T max ) plasma values ranging from 2 to 6 hours. There was no accumulation in plasma tofersen exposure following monthly maintenance dosing.

Distribution Autopsy tissue from patients treated with tofersen (n=3) showed that tofersen administered intrathecally was distributed within the central nervous system tissues. Elimination Metabolism Tofersen is expected to be metabolized through exonuclease (3'- and 5')-mediated hydrolysis and is not a substrate for, or inhibitor or inducer of CYP450 enzymes. Excretion The primary route of elimination has not been characterized.

The effective half-life in CSF is estimated to be 4 weeks. Drug Interaction Studies No clinical drug interaction studies have been performed. In vitro, QALSODY is not a substrate or inhibitor/inducer of major CYP enzymes or a substrate or inhibitor of major transporters.

Specific Populations Effect of sex, race, age, and body weight on tofersen exposure in plasma was not clinically significant. The effect of these factors on tofersen exposure in CSF is unknown. Patients with Renal or Hepatic Impairment No clinical studies have been conducted to evaluate the pharmacokinetics of tofersen in patients with renal or hepatic impairment.

Tofersen is not expected to undergo metabolism by hepatic enzymes.

🧬 Pharmacodynamics ~1 min read ▾

12.2. Pharmacodynamics Effect of Tofersen on Total CSF SOD1 Protein Total CSF SOD1, an indirect measure of target engagement, was evaluated in Study 1 Part C in SOD1-ALS patients [see Clinical Studies ( 14 )] . At Week 28 in Study 1 Part C, a reduction in total CSF SOD1 protein of 35% (geometric mean ratio to baseline) in the tofersen-treated group versus a 2% decrease from baseline in the corresponding placebo subjects in the ITT population was observed (difference in geometric mean ratios for tofersen to placebo: 34%; nominal p<0.0001).

Effect of Tofersen on Neurofilament Proteins Plasma NfL, a blood-based biomarker of axonal injury and neurodegeneration, was evaluated in Study 1 Part C in SOD1-ALS patients [see Clinical Studies ( 14 )] . At Week 28 in Study 1 Part C, mean plasma NfL was reduced 55% (geometric mean ratio to baseline) in the QALSODY-treated subjects, compared to a 12% increase with placebo in ITT population (difference in geometric mean ratios for QALSODY to placebo: 60%; nominal p<0.0001). Plasma NfL declined until approximately Day 113, after which the reductions were sustained.

The reductions in phosphorylated neurofilament heavy chain (pNfH) were similar compared to reductions in NfL, as were reductions in CSF compared to plasma. Cardiac Electrophysiology At the maximum approved recommended dosing regimen, QALSODY does not prolong the QTc interval to any clinically relevant extent.

🔬 Clinical Studies ~3 min read ▾

14 CLINICAL STUDIES The efficacy of QALSODY was assessed in a 28-week randomized, double-blind, placebocontrolled clinical study in patients 23 to 78 years of age with weakness attributable to ALS and a SOD1 mutation confirmed by a central laboratory (Study 1 Part C, NCT02623699). One hundred eight (108) patients were randomized 2:1 to receive treatment with either QALSODY 100 mg (n = 72) or placebo (n = 36) for 24 weeks (3 loading doses followed by 5 maintenance doses). Concomitant riluzole and/or edaravone use was permitted for patients.

The prespecified primary analysis population (n = 60, modified intent to treat [mITT]) had a slow vital capacity (SVC) ≥ 65% of predicted value and met prognostic enrichment criteria for rapid disease progression, defined based on their pre-randomization ALS Functional Rating Scale–Revised (ALSFRS-R) decline slope and SOD1 mutation type. The non-mITT population (n = 48) had a slow vital capacity (SVC) ≥ 50% of predicted value and did not meet the enrichment criteria for rapid disease progression. Baseline disease characteristics in the overall intent-to-treat (ITT) population (combined mITT and non-mITT population) were generally similar in patients treated with QALSODY and patients who received placebo, with slightly shorter time from symptom onset and higher plasma NfL at baseline in the QALSODY group.

At baseline, 62% of patients were taking riluzole, and 8% of patients were taking edaravone. Mean baseline ALSFRS-R score was 36.9 (5.9) in the QALSODY treatment group and 37.3 (5.8) in the placebo group. Median time from symptom onset was 11.4 months in the QALSODY treatment group and 14.6 months in the placebo group.

The primary efficacy analysis was the change from baseline to Week 28 in the ALSFRS-R total score in the mITT population, analyzed using the joint rank test to account for mortality in conjunction with multiple imputation (MI) to account for missing data for withdrawals other than death. Patients treated with QALSODY experienced less decline from baseline in the ALSFRS-R compared to placebo, but the results were not statistically significant (QALSODY-placebo adjusted mean difference [95% CI]: 1.2 [-3.2, 5.5]). Other clinical secondary outcomes also did not reach statistical significance.

Secondary endpoints of change from baseline at Week 28 in plasma NfL and CSF SOD1 protein were nominally statistically significant (see Table 2 ). NfL reduction was consistently observed for all subgroups based on sex, disease duration since symptom onset, site of onset, and riluzole/edaravone use. Table 2: Biomarker Results of QALSODY in Study 1 Part C at Week 28 Note 1: N is the number of patients with baseline value.

Note 2: MI was used for missing data. Model included treatment, use of riluzole or edaravone, relevant baseline score and postbaseline values (natural log transformed data). Separate models for mITT and nonmITT were used and combined for ITT analyses.

Note 3: Adjusted geometric mean ratios to baseline, treatment differences in adjusted geometric mean ratios to baseline and corresponding 95% CIs and nominal p-values were obtained from the ANCOVA model for change from baseline including treatment as a fixed effect and adjusting for the following covariates: baseline disease duration since symptom onset, relevant baseline score, and use of riluzole or edaravone. The analysis was based on natural log transformed data. Biomarker Endpoints QALSODY Placebo Plasma NfL ITT population N=72 N=36 Adjusted geometric mean ratio to baseline 0.45

1.12QALSODY to placebo difference in geometric mean ratio (95% CI) 0.40 (0.33, 0.49) Nominal p-value (ANCOVA+MI) <0.0001 mITT population N=39 N=21 Adjusted geometric mean ratio to baseline 0.40

1.20QALSODY to placebo difference in geometric mean ratio (95% CI) 0.33 (0.25, 0.45) Nominal p-value (ANCOVA+MI) <0.0001 CSF SOD1 Protein ITT population N=72 N=36 Adjusted geometric mean ratio to baseline 0.65

0.98QALSODY to placebo difference in geometric m… [Excerpted — this section continues on DailyMed.]

🧪 Nonclinical Toxicology 162 words ▾

13 NONCLINICAL TOXICOLOGY 13.1. Carcinogenesis, Mutagenesis, Impairment of Fertility Carcinogenesis Studies to assess the carcinogenic potential of tofersen have not been conducted. Mutagenesis Tofersen was negative in in vitro (bacterial reverse mutation and mammalian cell chromosomal aberration) and in vivo (mouse micronucleus) assays.

Impairment of Fertility In a study to assess effects on fertility and reproductive function, tofersen (0, 3, 10, 30 mg/kg) was administered every other day to male and female mice prior to and during mating and continuing in females to gestation day (GD) 7. Adverse effects on male reproductive organs (seminiferous tubular degeneration, seminiferous tubule dilatation, spermatid retention, apoptosis of epithelial cells, increased cellular debris in the testes, and hypospermia in the epididymis) were observed at the highest dose tested; however, there were no adverse effects on functional endpoints.

Plasma exposure at the no-effect dose (10 mg/kg) for adverse effects on male reproductive organs was approximately 2 times that in humans at the recommended human dose of 100 mg.

📄 Carcinogenesis, Mutagenesis, Impairment of Fertility 159 words ▾

13.1. Carcinogenesis, Mutagenesis, Impairment of Fertility Carcinogenesis Studies to assess the carcinogenic potential of tofersen have not been conducted. Mutagenesis Tofersen was negative in in vitro (bacterial reverse mutation and mammalian cell chromosomal aberration) and in vivo (mouse micronucleus) assays.

Impairment of Fertility In a study to assess effects on fertility and reproductive function, tofersen (0, 3, 10, 30 mg/kg) was administered every other day to male and female mice prior to and during mating and continuing in females to gestation day (GD) 7. Adverse effects on male reproductive organs (seminiferous tubular degeneration, seminiferous tubule dilatation, spermatid retention, apoptosis of epithelial cells, increased cellular debris in the testes, and hypospermia in the epididymis) were observed at the highest dose tested; however, there were no adverse effects on functional endpoints.

Plasma exposure at the no-effect dose (10 mg/kg) for adverse effects on male reproductive organs was approximately 2 times that in humans at the recommended human dose of 100 mg.

📄 Package Label / Principal Display Panel 66 words ▾

Principal Display Panel - Carton Label 1 vial NDC: 64406- 109 -01 QALSODY ™ (tofersen) Injection 100 mg/15 mL (6.7 mg/mL) Sterile solution for Intrathecal Injection Only Rx only Biogen ® Principal Display Panel - Carton Label

Principal Display Panel - Vial Label QALSODY ™ (tofersen) Injection 100 mg/15 mL (6.7 mg/mL) Sterile solution for Intrathecal Injection Only Rx only Principal Display Panel - Vial Label

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

Medicare Part D spend CMS · PART D · 2026 (Q1)

Medicare Part D (outpatient prescription) spending for Qalsody — 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 Qalsody. 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.
Period
Total Part D spend
$177.8K
Claims incl. refills
13
Beneficiaries
—
Spend / beneficiary
—
Spend / claim
$13,680.09
Trend by period
💵 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.

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) ✓ 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) — Not published for this NDC CMS reports utilization only for NDCs with Medicaid claims above its privacy threshold.
“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 Biogen Inc.. Listing status can change — the directory data on this page refreshes weekly.
Who lists this product with the FDA?
Biogen Inc. 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 J1304 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.
← 64406-0058-01 · Biogen 64406-0120-10 · Biogen →
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.