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ELELYSO Taliglucerase alfa 200 U/5mL Injection, Powder, Lyophilized, For Solution — NDC 00069-0106-01 package photo

ELELYSO Taliglucerase alfa 200 U/5mL Injection, Powder, Lyophilized, For Solution

by Pfizer Laboratories Div Pfizer Inc · 1 VIAL, GLASS in 1 CARTON (0069-0106-01) / 5 mL in 1 VIAL, GLASS
NDC 00069-0106-01
🏷️ FDA NDC (as labeled) 0069-0106-01 billing pads the labeler segment with a zero
Rx only Brand On market Non-controlled
🗂️ Data synced 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

🆔 Identity & classification

FDA NDC (as labeled) 0069-0106-01
Product NDC 0069-0106
11-digit billing NDC 00069010601
NCPDP billing unit EA — each (per item)
RxCUI 1292879, 1292881
UNII 0R4NLX88O4
Application # BLA022458
SPL Set ID fa3cbd5d-677c-4b19-9032-d9182cb69a83
Established class (EPC) Hydrolytic Lysosomal Glucocerebroside-specific Enzyme
Chemical class Glucosylceramidase
DEA schedule Non-controlled
Marketing category BLA
Marketing status On market
FDA listing status Listed (active directory)
Marketing start 2012-05-01
Route INTRAVENOUS
Dosage form INJECTION, POWDER, LYOPHILIZED, FOR SOLUTION
Substance TALIGLUCERASE ALFA
GPI-14 82700080102120
GPI class Elelyso
GCN Seq No 069127
GCN 32078
HICL code 038937
Ingredient (HICL) Taliglucerase Alfa
HIC1 code Z
Therapeutic class — broad (HIC1) Body As A Whole
HIC2 code Z1
Therapeutic class — intermediate (HIC2) Positive Effects On Wide-Spread Tissue
HIC3 code Z1I
Therapeutic class — specific (HIC3) Metabolic Disease Enzyme Replacement, Gaucher's Dx
AHFS code 44:00.00.00
AHFS class Enzymes
FDB label name ELELYSO 200 UNITS VIAL
FDB brand name Elelyso
Legend status F — Federal legend — prescription drug or device
Biologic (Purple Book) 351(a)
Why two NDCs? The FDA registers this code as 0069-0106-01 — a 4-4-2 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 labeler segment → 00069-0106-01. 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 Hydrolytic Lysosomal Glucocerebroside-specific Enzyme class.

Pharmacologic class Hydrolytic Lysosomal Glucocerebroside-specific Enzyme
Drug family (ATC) Enzymes
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.

🏭 Manufacturer & labeler

LabelerPfizer Laboratories Div Pfizer Inc
FDA applicationBLA022458 (BLA)
Labeler code00069
First marketedMay 2012
Product typeHuman Prescription Drug
Portfolio242 products on file
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.

🩺 Clinical

Label name ELELYSO 200 UNITS VIAL Ingredient Taliglucerase Alfa

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.

🧪 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.

  • 206.7 mg / 5 mL UNII 3OWL53L36A
    A natural sugar alcohol derived from seaweed or synthesized in the lab. It's used as a filler to add bulk, a sweetener in sugar-free formulas, and a disintegrant to help tablets break apart in the stomach.
  • 0.56 mg / 5 mL UNII 6OZP39ZG8H
    Polysorbate 80 is a synthetic emulsifier derived from sorbitol and oleic acid. It helps mix oil and water-based ingredients together in medications and improves how the product disperses in the body.
  • 30.4 mg / 5 mL UNII 1Q73Q2JULR
    Sodium citrate is a salt derived from citric acid. It works as a buffer to maintain the medicine's pH level and may also help improve taste or act as a preservative in the formulation.

3 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.

💲 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 mLPer 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 · J3060 $42.675 / J3060 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)0069-0106-01
11-digit billing NDC00069-0106-01
Format4-4-2 as registered → padded to 5-4-2 for billing (zero added to the labeler segment)
HCPCS J-codeJ3060
DescriptorInjection, taliglucerase alfa, 10 units
Billing units / pkg20 units
Crosswalk sourceCMS ASP NDC-HCPCS Crosswalk
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.

🔁 Therapeutic equivalents

ProductLabelerPackNADAC/unitTEStatusPrice vs. this
Elelyso 200 U/5mLthis 00069-0106-01 Pfizer 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

🏛️
2012
First FDA approval
May 2012
📍
2026
Currently FDA-listed
14 years listed
🔓
·
Biosimilar pathway open
listed protections lapsed 2024
🧬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: The latest listed FDA patent/protection lapsed May 2024 — those protections no longer apply, though a biosimilar still needs FDA licensure and a manufacturer to market it.
📅 FDA approved May 1, 2012

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
Elelysotaliglucerase alfa
🔒 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
2012 2014 2016 2018 2020 2022 2024
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 dateMay 1, 2024
Common questions
Is there a biosimilar for ELELYSO 200 UNITS 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.

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

Medicare Part D (outpatient prescription) spending for Elelyso — 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 Elelyso. 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
$761.3K
Claims incl. refills
15
Beneficiaries
—
Spend / beneficiary
—
Spend / claim
$50,753.81
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.

📦 Packaging — all sizes for this product

Package NDCDescription Marketing startStatus
00069-0106-01 You're viewing this 1 VIAL, GLASS in 1 CARTON (0069-0106-01) / 5 mL in 1 VIAL, GLASS 2012-05-01 Active

🧭 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 FDA registers it as 0069-0106-01, which is what is printed on the packaging and shown on DailyMed. Insurance claims use a fixed 11-digit 5-4-2 format, so the short segment is padded with a leading zero: 00069-0106-01, written without dashes as 00069010601. The Identity section at the top of this page lists every form of this code.
What do the three segments of this NDC mean?
In 00069-0106-01, the first segment (00069) is the labeler code FDA assigned to Pfizer Laboratories Div Pfizer Inc; the middle segment (0106) identifies this specific product — its ingredient, strength, and dosage form; and the last segment (01) identifies this exact package size and type. Together they name one specific package of one specific product.
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 Pfizer Laboratories Div Pfizer Inc. Listing status can change — the directory data on this page refreshes weekly.
Who lists this product with the FDA?
Pfizer Laboratories Div Pfizer 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 J3060 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.

📄 Full prescribing information FDA SPL

The complete FDA label for this product — the official prescribing information, verbatim, section by section. Jump with a chip, search within the label, or expand everything.
🚨 Boxed Warning 🎯 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
🚨 Boxed Warning 182 words ▾

WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS Patients treated with enzyme replacement therapies have experienced life‑threatening hypersensitivity reactions, including anaphylaxis. Anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy. Initiate ELELYSO in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment.

If a severe hypersensitivity reaction (e.g., anaphylaxis) occurs, discontinue ELELYSO and immediately initiate appropriate medical treatment, including use of epinephrine. Inform patients of the symptoms of life‑threatening hypersensitivity reactions, including anaphylaxis and to seek immediate medical care should symptoms occur [see Warnings and Precautions (5.1) ] . WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS See full prescribing information for complete boxed warning. • Anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy.

( 5.1 ) • Initiate ELELYSO in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment. ( 5.1 ) • If a severe hypersensitivity reaction (e.g., anaphylaxis) occurs, discontinue ELELYSO and immediately initiate appropriate medical treatment, including use of epinephrine. ( 5.1 )

🎯 Indications and Usage 56 words ▾

1 INDICATIONS AND USAGE ELELYSO is indicated for the treatment of patients 4 years of age and older with a confirmed diagnosis of Type 1 Gaucher disease. ELELYSO is a hydrolytic lysosomal glucocerebroside-specific enzyme indicated for the treatment of patients 4 years and older with a confirmed diagnosis of Type 1 Gaucher disease ( 1 ).

⏱️ Dosage and Administration ~3 min read ▾

2 DOSAGE AND ADMINISTRATION Recommendations Prior to ELELYSO Treatment ( 2.1 ): • Administration of ELELYSO should be supervised by a healthcare provider knowledgeable in the management of hypersensitivity reactions including anaphylaxis. Recommended Dosage in Patients 4 Years and Older ( 2.2 ): • Treatment-naïve : 60 units/kg administered every other week as a 60- to 120-minute intravenous infusion. • Patients switching from imiglucerase : Initiate ELELYSO intravenous treatment (60- to 120-minute infusion) with the same units/kg imiglucerase dosage and subsequently administer ELELYSO every other week.

Dosage adjustments can be based on achievement and maintenance of each patient’s therapeutic goals. Preparation and Administration ( 2.3, 2.4, 2.5 ): • Reconstitute, dilute and administer under the supervision of a healthcare professional. • See Full Prescribing Information for complete instructions.

2.1Recommendations Prior to ELELYSO Treatment Administration of ELELYSO should be supervised by a healthcare provider knowledgeable in the management of hypersensitivity reactions including anaphylaxis [see Warnings and Precautions (5.1) ] . Initiate ELELYSO in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment [see Warnings and Precautions (5.1) ] . To reduce the risk of hypersensitivity reactions, consider pretreatment with antihistamines and/or corticosteroids [see Warnings and Precautions (5.1) ] .

2.2Recommended Dosage in Patients 4 Years and Older Treatment-Naïve Patients 4 Years of Age and Older The recommended dosage of ELELYSO is 60 units/kg (based on actual body weight) administered every other week as a 60- to 120-minute intravenous infusion. Patients 4 Years of Age and Older Switching from Imiglucerase If it is acceptable to switch from a stable imiglucerase dosage to ELELYSO, initiate ELELYSO intravenous treatment (60- to 120-minute infusion) with the same units/kg imiglucerase dosage and subsequently administer ELELYSO every other week.

Dosage adjustments can be made based on achievement and maintenance of each patient's therapeutic goals.

2.3Preparation Instructions ELELYSO should be reconstituted, diluted, and administered under the supervision of a healthcare professional. Prepare ELELYSO according to the following steps using aseptic technique: a. Determine the number of vials to be reconstituted based on the patient's weight in kg and the recommended dose [see Dosage and Administration (2.2) ] .

Round the number of vials up to the next whole number. b. Remove the required number of vials from the refrigerator. Do not leave these vials at room temperature longer than 24 hours prior to reconstitution.

Do not heat or microwave these vials. c. Reconstitute each vial of ELELYSO with 5.1 mL of Sterile Water for Injection, USP to yield a reconstituted product with a concentration of 40 units/mL and an extractable volume of 5 mL. (1) Upon reconstitution, mix vials gently.

DO NOT SHAKE. (2) Prior to further dilution, visually inspect the reconstituted solution in the vials for particulate matter and discoloration. The solution should be clear and colorless.

Discard if particulate matter is present or the solution is discolored. d. Withdraw the calculated dose of drug from the appropriate number of vials and dilute with 0.9% Sodium Chloride Injection, USP, to a final volume of 100 to 200 mL. Discard any unused reconstituted solution.

(1) For pediatric patients 4 years of age and older, use a final volume of 100 to 120 mL. (2) For adult patients, may use a final volume of 130 to 150 mL. However, if the volume of reconstituted product alone is equal to or greater than 130 to 150 mL, then the final volume should not exceed 200 mL. e.

Mix the diluted solution gently. DO NOT SHAKE. Since this is a protein solution, slight flocculation (described as translucent fibers) occurs occasionally after dilution. f.

Discard any unused diluted soluti…

💊 Dosage Forms and Strengths 35 words ▾

3 DOSAGE FORMS AND STRENGTHS For injection: 200 units white to off-white lyophilized powder in a single-dose vial for reconstitution. For injection: 200 units lyophilized powder in a single-dose vial for reconstitution ( 3 )

⛔ Contraindications 7 words ▾

4 CONTRAINDICATIONS None. None ( 4 )

⚠️ Warnings and Cautions ~2 min read ▾

5 WARNINGS AND PRECAUTIONS See boxed warning ( 5.1 )

5.1Hypersensitivity Reactions Including Anaphylaxis Life‑threatening h ypersensitivity reactions, including anaphylaxis, have occurred in patients treated with enzyme replacement therapies, including ELELYSO . In clinical trials, (patients were not routinely pretreated with antihistamines and/or corticosteroids prior to ELELYSO infusions during the clinical trials): • 2 of 72 (3%) ELELYSO-treated patients experienced signs and symptoms consistent with anaphylaxis including urticaria, hypotension, flushing, wheezing, chest tightness, nausea, vomiting, and dizziness.

These reactions occurred during ELELYSO infusion. • 21 of 72 (29%) ELELYSO-treated patients experienced hypersensitivity reactions, including the 2 ELELYSO‑treated patients who experienced signs and symptoms consistent with anaphylaxis. Signs and symptoms of these hypersensitivity reactions included pruritus, angioedema, flushing, erythema, rash, nausea, vomiting, cough, chest tightness, and throat irritation. These reactions occurred during ELELYSO infusion and up to 3 hours after the start of infusion [see Adverse Reactions (6.1) ] .

Anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy. Administration of ELELYSO should be supervised by a healthcare provider knowledgeable in the management of hypersensitivity reactions including anaphylaxis. Initiate ELELYSO in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment.

Observe patients closely during and after the infusion. ELELYSO-treated patients who developed anti-taliglucerase alfa antibodies (referred to as anti-drug antibodies (ADA)) generally had a greater frequency of hypersensitivity reactions compared to those who did not develop ADA [see Adverse Reactions (6.1) ] . Closely monitor for hypersensitivity reactions in patients who develop ADA.

Management of hypersensitivity reactions should be based on the severity of the reaction and includes slowing or temporary interruption of the infusion and/or administration of antihistamines, antipyretics, and/or corticosteroids for mild reactions. To reduce the risk of hypersensitivity reactions, consider pretreatment with antihistamines and/or corticosteroids. If a severe hypersensitivity reaction (e.g., anaphylaxis) occurs, discontinue ELELYSO and immediately initiate appropriate medical treatment, including use of epinephrine.

Inform patients of the symptoms of life‑threatening hypersensitivity reactions, including anaphylaxis and to seek immediate medical care should symptoms occur. Consider the risks and benefits of re-administering ELELYSO in patients who have experienced a severe hypersensitivity reaction associated with ELELYSO. Caution should be exercised upon rechallenge [see Adverse Reactions (6.2) ].

🤒 Adverse Reactions ~3 min read ▾

6 ADVERSE REACTIONS The most common adverse reactions are: • Treatment-Naïve Adults (≥5%): headache, arthralgia, fatigue, nausea, dizziness, abdominal pain, pruritus, flushing, vomiting, urticaria ( 6.1 ). • Patients who Switched from Imiglucerase, after 9 Months on Treatment (≥10%): arthralgia, headache, pain in extremity ( 6.1 ). To report SUSPECTED ADVERSE REACTIONS, contact Pfizer Inc. at 1-800-438-1985 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch .

6.1Clinical Trials 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. Adverse Reactions from Clinical Trials of ELELYSO as Initial Therapy • Clinical Trial in Adult Patients The safety of ELELYSO at dosages of either 30 units/kg (n=16) (50% of the recommended dosage) [see Dosage and Administration (2.1) ] or 60 units/kg (n=16) administered intravenously every other week was assessed in 32 adult treatment-naïve patients (aged 19 to 74 years) with Type 1 Gaucher disease in a 9-month double-blind, randomized clinical trial (Trial 1) [see Clinical Studies (14) ] .

Table 1 presents the adverse reactions that occurred in these ELELYSO-treated patients. Table 1: Adverse Reactions in ≥5% of Treatment-Naïve Adult Patients Treated with ELELYSO Preferred Term Treatment-Naïve Adults (N=32) n (%) Headache 6 (19) Arthralgia 4 (13) Fatigue 3 (9) Nausea 3 (9) Dizziness 3 (9) Abdominal pain 2 (6) Pruritus 2 (6) Flushing 2 (6) Vomiting 2 (6) Urticaria 2 (6) • Clinical Trial in Pediatric Patients 16 Years of Age and Younger The safety of ELELYSO at dosages of either 30 units/kg (n=4) (50% of the recommended dosage) [see Dosage and Administration (2.1) ] or 60 units/kg (n=5) administered intravenously every other week was assessed in 9 pediatric treatment-naïve patients (aged 2 to 13 years) with Type 1 Gaucher disease in a 12-month randomized clinical trial (Trial 2) [see Clinical Studies (14) ] .

The most common adverse reaction (≥10%) was vomiting, which occurred in 4 of 9 patients. Two patients developed hypersensitivity reactions; 1 patient experienced severe vomiting and gastrointestinal inflammation, and 1 experienced mild throat irritation and chest discomfort. Both patients responded to treatment with antihistamines and continued ELELYSO treatment.

Adverse Reactions in a Clinical Trial in Patients Who Switched from Imiglucerase to ELELYSO The safety of ELELYSO was assessed in 31 patients (26 adult and 5 pediatric patients), ages 6 to 66 years old, with Type 1 Gaucher disease who had previously been receiving imiglucerase treatment for a minimum of 2 years (Trial 3). ELELYSO was administered intravenously every other week for 9 months at the same number of units as each patient's previous imiglucerase dose. Table 2 presents the adverse reactions in these ELELYSO-treated patients.

Table 2: Adverse Reactions in ≥10% of ELELYSO-Treated Patients Who Switched from Imiglucerase to ELELYSO (after 9 months on treatment) Preferred Term Adult and Pediatric Patients Switched from Imiglucerase (N=31) n (%) Arthralgia 4 (13) Headache 4 (13) Pain in extremity 3 (10) Immunogenicity: Anti-Drug Antibody-Associated Adverse Reactions Trials 1, 2, and 3 evaluated ELELYSO enzyme replacement therapy (ERT)-naïve and ERT-experienced adult and pediatric patients with Gaucher disease [see Clinical Studies (14.1, 14.2) ] .

In patients with Type 1 Gaucher disease, hypersensitivity reactions occurred in 36% (9/25) of ELELYSO-treated patients who developed ADA during the treatment period and in 15% (6/41) of ELELYSO-treated patients who did not develop ADA during the treatment period [see Warnings and Precautions (5.1) and Clinical Pharmacology (12.6) ] . Of the 9 ELELYSO-treated patients who tested positive for ADA and who developed hypersensitivity reactions, 2 patients had anaphylaxis and 1 a…

👥 Use in Specific Populations ~3 min read ▾

8 USE IN SPECIFIC POPULATIONS

8.1Pregnancy Risk Summary Available data, including data from a product registry and pharmacovigilance reports, have not identified a drug-associated risk of major birth defects, miscarriage, or other adverse maternal or fetal outcomes in pregnant women exposed to ELELYSO. Additionally, there are risks associated with untreated symptomatic Type I Gaucher disease in pregnant women ( see Clinical Considerations ). In animal reproduction studies when pregnant rats and rabbits were administered taliglucerase alfa at intravenous doses up to 5 times the recommended human dose (RHD), there was no evidence of embryo-fetal toxicity ( see Data ).

The background risk of major birth defects and miscarriage for the indicated population is unknown. 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. Clinical Considerations Disease-Associated Maternal and/or Embryo/Fetal Risk Women with Type 1 Gaucher disease have an increased risk of spontaneous abortion if disease symptoms are not treated and controlled pre-conception and during a pregnancy.

Pregnancy may exacerbate existing Type 1 Gaucher disease symptoms or result in new disease manifestations. Type 1 Gaucher disease manifestations may lead to adverse pregnancy outcomes, including hepatosplenomegaly which can interfere with the normal growth of a fetus and thrombocytopenia which can lead to increased bleeding and possible postpartum hemorrhage requiring transfusion. Data Animal Data Reproduction studies have been performed with taliglucerase alfa administered during the period of organogenesis in rats and rabbits.

In rats, intravenous doses up to 55 mg/kg/day (about 5 times the RHD of 60 units/kg based on the body surface area) did not cause any adverse effects on embryo-fetal development. In rabbits, intravenous doses up to 27.8 mg/kg/day (about 5 times the RHD of 60 units/kg based on the body surface area) did not show any embryo-fetal toxicity.

8.2Lactation Risk Summary There are no data on the presence of taliglucerase alfa in human milk or the effects of taliglucerase alfa on milk production. Lactation data from a product registry have not identified adverse reactions in infants exposed to taliglucerase alfa during breastfeeding. The developmental and health benefits of breastfeeding should be considered along with the mother’s clinical need for ELELYSO and any potential adverse effects on the breastfed child from ELELYSO or from the underlying maternal condition.

8.4Pediatric Use The safety and effectiveness of ELELYSO for the treatment of pediatric patients 4 years of age and older with a confirmed diagnosis of Type 1 Gaucher disease has been established. The use of ELELYSO for this indication is supported by evidence of effectiveness from adequate and well-controlled trials of ELELYSO in adults, with additional pharmacodynamic data from 5 pediatric patients and pharmacokinetic data from 9 pediatric patients who participated in clinical trials [see Clinical Studies (14.1 , 14.2) and Clinical Pharmacology (12.3) ].

Data from 14 pediatric patients were included in the safety evaluation [see Adverse Reactions (6.1) ]. The safety and effectiveness of ELELYSO has not been established in patients less than 4 years of age. Pediatric patients experienced a higher frequency of vomiting during ELELYSO treatment (4 of 9 treatment-naïve patients) than adult patients, and this may be a symptom of hypersensitivity reaction.

The frequencies of other adverse reactions were similar between pediatric and adult patients treated with ELELYSO [see Adverse Reactions (6.1) ] .

8.5Geriatric Use During clinical trials, 8 patients aged 65 or older were treated with ELELYSO. Clinical trials of ELELYSO did not include sufficient numbers of patients aged 65 and over to determine whether they respond differently from younger patients.

🤰 Pregnancy ~1 min read ▾

8.1Pregnancy Risk Summary Available data, including data from a product registry and pharmacovigilance reports, have not identified a drug-associated risk of major birth defects, miscarriage, or other adverse maternal or fetal outcomes in pregnant women exposed to ELELYSO. Additionally, there are risks associated with untreated symptomatic Type I Gaucher disease in pregnant women ( see Clinical Considerations ). In animal reproduction studies when pregnant rats and rabbits were administered taliglucerase alfa at intravenous doses up to 5 times the recommended human dose (RHD), there was no evidence of embryo-fetal toxicity ( see Data ).

The background risk of major birth defects and miscarriage for the indicated population is unknown. 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. Clinical Considerations Disease-Associated Maternal and/or Embryo/Fetal Risk Women with Type 1 Gaucher disease have an increased risk of spontaneous abortion if disease symptoms are not treated and controlled pre-conception and during a pregnancy.

Pregnancy may exacerbate existing Type 1 Gaucher disease symptoms or result in new disease manifestations. Type 1 Gaucher disease manifestations may lead to adverse pregnancy outcomes, including hepatosplenomegaly which can interfere with the normal growth of a fetus and thrombocytopenia which can lead to increased bleeding and possible postpartum hemorrhage requiring transfusion. Data Animal Data Reproduction studies have been performed with taliglucerase alfa administered during the period of organogenesis in rats and rabbits.

In rats, intravenous doses up to 55 mg/kg/day (about 5 times the RHD of 60 units/kg based on the body surface area) did not cause any adverse effects on embryo-fetal development. In rabbits, intravenous doses up to 27.8 mg/kg/day (about 5 times the RHD of 60 units/kg based on the body surface area) did not show any embryo-fetal toxicity.

🧒 Pediatric Use 170 words ▾

8.4Pediatric Use The safety and effectiveness of ELELYSO for the treatment of pediatric patients 4 years of age and older with a confirmed diagnosis of Type 1 Gaucher disease has been established. The use of ELELYSO for this indication is supported by evidence of effectiveness from adequate and well-controlled trials of ELELYSO in adults, with additional pharmacodynamic data from 5 pediatric patients and pharmacokinetic data from 9 pediatric patients who participated in clinical trials [see Clinical Studies (14.1 , 14.2) and Clinical Pharmacology (12.3) ].

Data from 14 pediatric patients were included in the safety evaluation [see Adverse Reactions (6.1) ]. The safety and effectiveness of ELELYSO has not been established in patients less than 4 years of age. Pediatric patients experienced a higher frequency of vomiting during ELELYSO treatment (4 of 9 treatment-naïve patients) than adult patients, and this may be a symptom of hypersensitivity reaction.

The frequencies of other adverse reactions were similar between pediatric and adult patients treated with ELELYSO [see Adverse Reactions (6.1) ] .

🧓 Geriatric Use 40 words ▾

8.5Geriatric Use During clinical trials, 8 patients aged 65 or older were treated with ELELYSO. Clinical trials of ELELYSO did not include sufficient numbers of patients aged 65 and over to determine whether they respond differently from younger patients.

🧬 Clinical Pharmacology ~3 min read ▾

12 CLINICAL PHARMACOLOGY

12.1Mechanism of Action Gaucher disease is an autosomal recessive disorder caused by mutations in the human glucocerebrosidase gene, which results in a reduced activity of the lysosomal enzyme glucocerebrosidase. Glucocerebrosidase catalyzes the conversion of the sphingolipid glucocerebroside into glucose and ceramide. The enzymatic deficiency results in accumulation of substrate glucocerebroside primarily in the lysosomal compartment of macrophages, giving rise to foam cells or "Gaucher cells," which accumulate in the liver, spleen and bone marrow.

ELELYSO, an enzyme replacement therapy, is a recombinant analog of human lysosomal glucocerebrosidase that catalyzes the hydrolysis of glucocerebroside to glucose and ceramide, reducing the amount of accumulated glucocerebroside. ELELYSO uptake into cellular lysosomes is mediated by binding of ELELYSO mannose oligosaccharide chains to specific mannose receptors on the cell surface leading to internalization and subsequent transport to the lysosomes.

12.3Pharmacokinetics Pharmacokinetics of taliglucerase alfa were evaluated in 38 patients (29 adult and 9 pediatric patients) who received intravenous infusions of ELELYSO 30 units/kg (50% of the recommended dosage) or 60 units/kg (the recommended dosage) [see Dosage and Administration (2.1) ] every other week. The pharmacokinetic parameters in adult and pediatric patients are summarized in Table 3. In adult Type 1 Gaucher disease patients treated with ELELYSO 30 units/kg (50% of the recommended dosage) or 60 units/kg (the recommended dosage) [see Dosage and Administration (2.1) ] (N=29) every other week as initial therapy, pharmacokinetics were determined with the first dose and at Week 38 of treatment.

The pharmacokinetics of taliglucerase alfa appeared to be nonlinear with a greater than dose-proportional increase in exposure at the doses studied. No significant accumulation or change in taliglucerase alfa pharmacokinetics over time from Weeks 1 to 38 was observed with repeated dosages of 30 units/kg (50% of the recommended dosage) or 60 units/kg (the recommended dosage) [see Dosage and Administration (2.1) ] every other week. Based on the limited data, there were no significant pharmacokinetic differences between male and female patients in this study.

The pharmacokinetics of taliglucerase alfa were evaluated in 9 pediatric patients 4 to 17 years of age with Type 1 Gaucher disease who were treated with ELELYSO for 10 to 27 months. Six of the 9 patients were treatment-naïve, and 3 patients were switched from imiglucerase. In both the 30 units/kg (50% of the recommended dosage) and 60 units/kg (the recommended dosage) [see Dosage and Administration (2.1) ] dose groups, clearance values in pediatric patients were similar to those in adult patients.

AUC values in pediatric patients were lower than AUC values in adult patients, due to weight-based dosing of taliglucerase alfa and lower body weights in pediatric patients. Table 3: Taliglucerase Alfa Pharmacokinetic Parameters after Repeated Dosing in Adult and Pediatric Patients with Type 1 Gaucher Disease Pediatric Patients (N=9) Median (Range) Adult Patients at Week 38 (N=29) Median (Range) 30 units/kg 30 units/kg is 50% of the recommended dose. n=5 60 units/kg n=4 30 units/kg n=14 60 units/kg n=15 Age (years) 15 (10, 17) 11 (4, 16) 35 (19, 74) 33 (19, 58) Weight (kg) 44.3 (22.8, 71.0) 28.6 (16.5, 50.4) 72.5 (51.5, 99.5) 73.5 (58.5, 87.0) n=14 AUC 0–∞ (ng*h/mL) Values were derived from concentration data expressed in ng/mL 1416 (535, 1969) 2984 (1606, 4273) 2007 (1007, 10092) 6459 (2548, 21020) T 1/2 (min) 37.1 (22.5, 56.8) 32.5 (18.0, 42.9) 18.9 (9.20, 57.9) 28.7 (11.3, 104) CL (L/h) 30.5 (17.4, 37.8) 15.8 (11.7, 24.9) 30.5 (6.79, 68.0) 18.5 (6.20, 37.9) V ss (L) 14.9 (10.1, 35.6) 8.80 (3.75, 21.4) 11.7 (2.3, 22.7) 10.7 (1.4, 18.5)

12.6Immunogenicity The observed incidence of ADA (including neutralizing antibodies [Nab]) is highly dependent on the sensitivit…

🧬 Mechanism of Action 135 words ▾

12.1Mechanism of Action Gaucher disease is an autosomal recessive disorder caused by mutations in the human glucocerebrosidase gene, which results in a reduced activity of the lysosomal enzyme glucocerebrosidase. Glucocerebrosidase catalyzes the conversion of the sphingolipid glucocerebroside into glucose and ceramide. The enzymatic deficiency results in accumulation of substrate glucocerebroside primarily in the lysosomal compartment of macrophages, giving rise to foam cells or "Gaucher cells," which accumulate in the liver, spleen and bone marrow.

ELELYSO, an enzyme replacement therapy, is a recombinant analog of human lysosomal glucocerebrosidase that catalyzes the hydrolysis of glucocerebroside to glucose and ceramide, reducing the amount of accumulated glucocerebroside. ELELYSO uptake into cellular lysosomes is mediated by binding of ELELYSO mannose oligosaccharide chains to specific mannose receptors on the cell surface leading to internalization and subsequent transport to the lysosomes.

📦 How Supplied / Storage and Handling 66 words ▾

16 HOW SUPPLIED/STORAGE AND HANDLING ELELYSO (taliglucerase alfa) for injection is supplied as a sterile, preservative-free, white to off-white lyophilized powder in a single-dose vial. Each vial of ELELYSO contains 200 units of taliglucerase alfa. Each carton contains one vial (NDC 0069-0106-01). Refrigerate ELELYSO at 2 °C to 8 °C (36 °F to 46 °F) in the original carton to protect from light. Do not freeze.

📦 Storage and Handling 24 words ▾

Refrigerate ELELYSO at 2 °C to 8 °C (36 °F to 46 °F) in the original carton to protect from light. Do not freeze.

📋 Description 214 words ▾

11 DESCRIPTION Taliglucerase alfa is a hydrolytic lysosomal glucocerebroside-specific enzyme produced by recombinant DNA technology using plant cell culture (carrot). Taliglucerase alfa is a monomeric glycoprotein enzyme containing 4 N-linked glycosylation sites (kDa=60.8). Taliglucerase alfa differs from native human glucocerebrosidase by 2 amino acids at the N terminal and up to 7 amino acids at the C terminal.

Taliglucerase alfa is a glycosylated protein with oligosaccharide chains at the glycosylation sites having terminal mannose sugars. These mannose-terminated oligosaccharide chains of taliglucerase alfa are specifically recognized by endocytic carbohydrate receptors on macrophages, the cells that accumulate lipid in Gaucher disease. A unit is the amount of enzyme that catalyzes the hydrolysis of 1 micromole of the synthetic substrate para-nitrophenyl-β-D-glucopyranoside ( p NP-Glc) per minute at 37 °C.

ELELYSO (taliglucerase alfa) for injection is supplied as a sterile, preservative-free, lyophilized powder for reconstitution and dilution prior to intravenous infusion. Each single-dose vial contains 200 units of taliglucerase alfa and D-mannitol (206.7 mg), polysorbate 80 (0.56 mg), and sodium citrate (30.4 mg). Citric acid may be added to adjust the pH at the time of manufacture.

After reconstitution with 5.1 mL Sterile Water for Injection, USP, taliglucerase alfa concentration is 40 units/mL [see Dosage and Administration (2) ] . Reconstituted solutions have a pH of approximately 6.0.

💬 Information for Patients 91 words ▾

17 PATIENT COUNSELING INFORMATION Hypersensitivity Reactions Including Anaphylaxis Advise patients and caregivers that life-threatening hypersensitivity reactions, including anaphylaxis may occur with ELELYSO treatment. Advise patients and caregivers that anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy. Inform patients and caregivers of the symptoms of life‑threatening hypersensitivity reactions, including anaphylaxis and to seek immediate medical care should symptoms occur [see Warnings and Precautions (5.1) ] .

This product’s labeling may have been updated. For the most recent prescribing information, please visit www.pfizer.com .

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