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Spinraza Nusinersen 10 mg/mL Injection, Solution — NDC 64406-0037-01 package photo
Label image from the product's FDA listing (DailyMed) — may show a different pack size or an older label revision.

Spinraza Nusinersen 10 mg/mL Injection, Solution — NDC 64406-037-01 (Billing 64406-0037-01)

by Biogen Inc. · 1 VIAL, SINGLE-USE in 1 BOX / 5 mL in 1 VIAL, SINGLE-USE

This is a package of Spinraza Nusinersen 10 mg/mL Injection, Solution from Biogen Inc., marketed since Mar 2026 and currently FDA-listed. It is this product's only package size.

NDC 64406-0037-01
🏷️ FDA NDC (as labeled) 64406-037-01 billing pads the product segment with a zero
Rx only Brand On market Non-controlled ⇄ Compare with another NDC
🗂️ FDA directory synced Oct 1, 2026 · this listing last changed Jul 24, 2026 · sources: openFDA · FDA label (DailyMed) · FDA Orange & Purple Book · First Databank · CMS NADAC, ASP, Medicare & Medicaid · RxNorm
📋 All sources & update times →

This page is curated, coded, and maintained by

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

NDC database record

One package, one record: these facts belong to NDC 64406-037-01 alone.

Record
FDA NDC Directory package listing · Human prescription drug
Code segments
64406 labeler · 037 product · 01 package
Package marketed since
Mar 27, 2026
Sample package
No — commercial package
Listing certified through
Dec 31, 2027
Barcode (UPC-A, from the NDC)
3 6440603701 6
FDA record last changed
Jul 24, 2026

Identity & classification

Regulatory identifiers FDA, NLM and CMS codes for this package

FDA NDC (as labeled) 64406-037-01
Product NDC 64406-037
11-digit billing NDC 64406003701
NCPDP billing unit ML — per mL (volume)
RxCUI 1863560, 1863565, 2739995, 2739998, 2740001, 2740004
UNII 5Z9SP3X666
Application # NDA209531
SPL Set ID dd70cd5f-b0fc-4ba4-a5ea-89a34778bd94
Established class (EPC) Survival Motor Neuron-2-directed RNA Interaction; Antisense Oligonucleotide
Physiologic effect Increased Protein Synthesis
Chemical class Oligonucleotides, Antisense
DEA schedule Non-controlled
Marketing category NDA
Marketing status On market
FDA listing status Listed (active directory)
Marketing start 2026-03-27
Route INTRATHECAL
Dosage form INJECTION, SOLUTION
Substance NUSINERSEN

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

GPI-14 74701050002040
GCN Seq No 088818
GCN 58989
HICL code 044016
Ingredient (HICL) Nusinersen Sodium/Pf
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 Z1T
Therapeutic class — specific (HIC3) Genetic D/O Tx - Smn Protein Deficiency Treatment
AHFS code 92:18.00.00
AHFS class Antisense Oligonucleotides
FDB label name SPINRAZA 50 MG/5 ML VIAL
FDB brand name Spinraza
Legend status F — Federal legend — prescription drug or device
Quick answers
  • GSN (GCN sequence number): 088818
  • GCN: 58989
  • GPI-14 (Medi-Span): 74701050002040
  • HICL (First Databank): 044016
  • AHFS class code: 92:18.00.00
  • RxCUI (RxNorm): 1863560
Why two NDCs? The FDA registers this code as 64406-037-01 — 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 → 64406-0037-01. 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, Survival Motor Neuron-2-directed RNA Interaction
Drug family (ATC) Other drugs for disorders of the musculo-skeletal system
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 SPINRAZA 50 MG/5 ML VIAL Ingredient Nusinersen Sodium/Pf
📖 What it is MedlinePlus · NLM

Nusinersen injection is used for the treatment of spinal muscular atrophy (an inherited condition that reduces muscle strength and movement) in infants, children, and adults. Nusinersen injection is in a class of medications called antisense oligonucleotide inhibitors. It works by increasing the amount of a certain protein necessary for the muscles and nerves to work normally.

Read the full MedlinePlus article ↗
📗 Our plain-language guide HelloPharmacist
  • Spinraza treats spinal muscular atrophy (SMA) in children and adults. SMA is a genetic condition caused by too little SMN protein. Spinraza helps your body make more of it.
  • It is injected into the fluid around your spinal cord by a trained healthcare professional. You start with a few loading doses, then get maintenance doses about every 4 months. You...
  • Spinraza can lower platelets, affect clotting and put stress on the kidneys. Your team checks platelets, clotting and urine protein before each dose. This helps catch problems earl...
  • Common ones include fever, headache, vomiting, back pain, respiratory infections and constipation, depending on age and type of SMA. Call your care team about unusual bleeding or b...
📖 Read our full Nusinersen Injection guide →
Where does this data come from?
Plain-language summary from MedlinePlus (U.S. National Library of Medicine); supplement & herbal interactions and nutrient depletion data from the Natural Medicines database; our full guide is HelloPharmacist editorial content.

Pricing

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

Price systemPer 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 · J2326 No ASP payment limit on file for J2326 this quarter.
ℹ️
No price is published for this exact package yet. CMS surveys NADAC per package size, so a different pack of the same drug often has one.
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)64406-037-01
11-digit billing NDC64406-0037-01
Format5-3-2 as registered → padded to 5-4-2 for billing (zero added to the product segment)
HCPCS J-codeJ2326
DescriptorINJECTION, NUSINERSEN, 0.1 MG
Billing units / pkg100 units
How the units are derivedThis package is 5 ML; the HCPCS unit is 0.1 MG, so one package = 100 billing units.
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
64406-0037-01 You're viewing this Main listing 1 VIAL, SINGLE-USE in 1 BOX / 5 mL in 1 VIAL, SINGLE-USE 2026-03-27 — Active

Therapeutic equivalents

ProductLabelerPackNADAC/unitTEStatusPrice vs. this
Spinraza 10 mg/mLthis 64406-0037-01 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

🏛️
2016
First FDA approval
Dec 2016
📍
2026
Currently FDA-listed
10 years listed
🛡️
2036
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 Mar 2036. This may affect when a full generic version becomes widely available, but it is not a guaranteed launch date.
📅 FDA approved Dec 23, 2016 RLD RS ⏳ ~9.4 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 9717750 — method of use (U-1942)
US 9717750 — method of use (U-2094)
US 9717750 — method of use (U-2093)
US 10436802 — method of use (U-2094)
US 10436802 — method of use (U-1943)
US 10436802 — method of use (U-1941)
US 10436802 — method of use (U-2093)
US 10436802 — method of use (U-1944)
US 10436802 — method of use (U-1942)
US 9717750 — method of use (U-1943)
US 9926559 — method of use (U-1943)
US 12013403 — method of use (U-1941)
US 12013403 — method of use (U-1942)
US 12013403 — method of use (U-1943)
US 12013403 — method of use (U-1944)
US 12013403 — method of use (U-2093)
US 12013403 — method of use (U-2094)
US 8980853 — method of use (U-1941)
US 9717750 — method of use (U-1943)
US 9717750 — method of use (U-2093)
US 9717750 — method of use (U-2094)
US 9717750 — method of use (U-1942)
US 9717750 — method of use (U-1943)
US 9717750 — method of use (U-2093)
US 9717750 — method of use (U-2094)
US 9717750 — method of use (U-1942)
US 8980853 — method of use (U-1941)
US 8980853 — method of use (U-1941)
US 8361977 — drug substance
US 8361977 — drug substance
US 7838657 — drug substance
US 7838657 — drug substance
US 7838657 — drug substance
US 8361977 — drug substance
Exclusivity NS
Exclusivity NS
2016 2018 2020 2022 2024 2026 2028 2030 2032 2034 2036
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 (34)
PatentTypeUse codeExpires
US 9717750 ↗ Method of use U-1942 Jun 17, 2030
US 9717750 ↗ Method of use U-2094 Jun 17, 2030
US 9717750 ↗ Method of use U-2093 Jun 17, 2030
US 10436802 ↗ Method of use U-2094 Sep 11, 2035
US 10436802 ↗ Method of use U-1943 Sep 11, 2035
US 10436802 ↗ Method of use U-1941 Sep 11, 2035
US 10436802 ↗ Method of use U-2093 Sep 11, 2035
US 10436802 ↗ Method of use U-1944 Sep 11, 2035
US 10436802 ↗ Method of use U-1942 Sep 11, 2035
US 9717750 ↗ Method of use U-1943 Jun 17, 2030
US 9926559 ↗ Method of use U-1943 Jan 9, 2034
US 12013403 ↗ Method of use U-1941 Mar 4, 2036
US 12013403 ↗ Method of use U-1942 Mar 4, 2036
US 12013403 ↗ Method of use U-1943 Mar 4, 2036
US 12013403 ↗ Method of use U-1944 Mar 4, 2036
US 12013403 ↗ Method of use U-2093 Mar 4, 2036
US 12013403 ↗ Method of use U-2094 Mar 4, 2036
US 8980853 ↗ Method of use U-1941 Nov 24, 2030
US 9717750 ↗ Method of use U-1943 Jun 17, 2030
US 9717750 ↗ Method of use U-2093 Jun 17, 2030
US 9717750 ↗ Method of use U-2094 Jun 17, 2030
US 9717750 ↗ Method of use U-1942 Jun 17, 2030
US 9717750 ↗ Method of use U-1943 Jun 17, 2030
US 9717750 ↗ Method of use U-2093 Jun 17, 2030
US 9717750 ↗ Method of use U-2094 Jun 17, 2030
US 9717750 ↗ Method of use U-1942 Jun 17, 2030
US 8980853 ↗ Method of use U-1941 Nov 24, 2030
US 8980853 ↗ Method of use U-1941 Nov 24, 2030
US 8361977 ↗ Drug substance — Dec 23, 2030
US 8361977 ↗ Drug substance — Dec 23, 2030
US 7838657 ↗ Drug substance — Jul 11, 2027
US 7838657 ↗ Drug substance — Jul 11, 2027
US 7838657 ↗ Drug substance — Jul 11, 2027
US 8361977 ↗ Drug substance — Dec 23, 2030
FDA exclusivity
CodeWhat it grantsExpires
NSNew StrengthMar 27, 2029
NSNew StrengthMar 27, 2029
Common questions
Is there a generic version of SPINRAZA 50 MG/5 ML VIAL?
No FDA-approved generic equivalent is currently listed in the FDA Orange Book for SPINRAZA 50 MG/5 ML VIAL. Based on the patents and exclusivity currently listed, the Orange Book estimate is that full-label generic entry may be delayed until Mar 2036 — 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.

🧪 Avoiding an ingredient? See Nusinersen Injection inactive ingredients by manufacturer: every current product's list side by side, so you can ask your pharmacy for the version that does not list it.

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

  • 0.21 mg / 1 mL 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 QTT17582CB
    A strong acid used to adjust and maintain the proper pH level in liquid medicines, ensuring stability and preventing breakdown of active ingredients.
  • 0.16 mg / 1 mL 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.
  • 0.22 mg / 1 mL 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.
  • 8.11 mg / 1 mL 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 55X04QC32I
    A strong alkaline chemical used to adjust and maintain the pH balance of liquid medicines. It helps keep the medicine stable and ensures it stays effective during storage.
  • 0.10 mg / 1 mL UNII 22ADO53M6F
    A mineral salt derived from phosphoric acid, used as a buffer to maintain the pH balance of the medication and help stabilize the active ingredients.
  • 0.05 mg / 1 mL UNII 5QWK665956
    A salt derived from phosphoric acid and sodium, used primarily as a buffer to control the acidity or pH of a medication, helping keep it stable during storage and use.
  • 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.

9 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 INC
FDA applicationNDA209531 (NDA)
Labeler code64406
First marketedMar 2026
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 📄 Recent Major Changes 📄 Package Label / Principal Display Panel
🎯 Indications and Usage 46 words ▾

1 INDICATIONS AND USAGE SPINRAZA is indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients. SPINRAZA is a survival motor neuron-2 (SMN2)-directed antisense oligonucleotide indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients ( 1 )

⏱️ Dosage and Administration ~3 min read ▾

2 DOSAGE AND ADMINISTRATION SPINRAZA is administered intrathecally ( 2.1 ) Recommended Dosage ( 2.1 ) The recommended dosage is one of two options: Low Dose Regimen: Administer one 12 mg loading dose every 14 days for three doses; then a fourth 12 mg loading dose 30 days after the third dose; then administer a 12 mg maintenance dose once every 4 months thereafter. High Dose Regimen: Administer one 50 mg loading dose followed by a second 50 mg loading dose 14 days later; then administer a 28 mg maintenance dose once every 4 months thereafter.

Important Preparation and Administration Instructions ( 2.3 ) Allow to warm to room temperature prior to administration Administer within 4 hours of removal from vial Prior to administration, remove 5 mL of cerebrospinal fluid Administer as intrathecal bolus injection over 1 to 3 minutes Laboratory Testing and Monitoring to Assess Safety ( 2.4 ) At baseline and prior to each dose, obtain a platelet count, coagulation laboratory testing, and quantitative spot urine protein testing

2.1Recommended Dosage SPINRAZA is administered intrathecally by, or under the direction of, healthcare professionals experienced in performing lumbar punctures. Two dosing regimen options for SPINRAZA, which consist of loading followed by maintenance dosages, are presented in Table 1 . Table 1: Recommended Dosage for SPINRAZA Loading Dosages Maintenance Dosage Low Dose Regimen (Low dose with four loading doses) Administer a total of four loading doses as follows: one 12 mg dose every 14 days for three doses, then a fourth 12 mg dose 30 days after the third dose.

Administer 12 mg once every 4 months starting 4 months after the last loading dose. High Dose Regimen (High dose with two loading doses) Administer a total of two loading doses as follows: one 50 mg dose followed by a second 50 mg dose 14 days later. Administer 28 mg once every 4 months starting 4 months after the last loading dose.

2.2Missed Doses Missed Dose of Low Dose Regimen Missed Loading Dose If a 12 mg loading dose (any of the 4 loading doses) is missed, administer the missed loading dose as soon as possible; adjust the date for the subsequent doses to maintain the recommended interval between doses. Missed Maintenance Dose Less than 8 months from last maintenance dose Administer the missed 12 mg maintenance dose as soon as possible; then administer the next maintenance dose per the originally scheduled date, as long as these two doses are administered at least 14 days apart.

At least 8 months but less than 16 months from last maintenance dose Administer the missed 12 mg maintenance dose as soon as possible, followed by one additional dose 14 days later, and then administer the next maintenance dose 4 months thereafter. At least 16 months but less than 40 months from last maintenance dose Administer the missed 12 mg maintenance dose as soon as possible, followed by two additional doses 14 days apart, and then administer the next maintenance dose 4 months thereafter. At least 40 months from last dose Restart Low Dose Regimen with 12 mg loading dosesas described in Recommended Dosage.

Missed Dose of High Dose Regimen Missed Second 50 mg Loading Dose Administer the missed 50 mg loading dose as soon as possible; then administer 28 mg maintenance doses every 4 months thereafter. Missed 28 mg Maintenance Dose Less than 8 months from last maintenance dose Administer the missed 28 mg maintenance dose as soon as possible; administer the next 28 mg maintenance dose per the originally scheduled date, as long as these two doses are administered at least 14 days apart; then administer 28 mg every 4 months thereafter.

At least 8 months to less than 40 months from last maintenance dose Administer a 50 mg loading dose as soon as possible; then administer 28 mg maintenance doses every 4 months thereafter. At least 40 months from last maintenance dose Restart High Dose Regimen with two 50 mg loading doses as described in Recommended Dosage.

2.3Important Preparati… [Excerpted — this section continues on DailyMed.]

💊 Dosage Forms and Strengths 75 words ▾

3 DOSAGE FORMS AND STRENGTHS Injection: nusinersen is a clear and colorless solution supplied in single-dose vials in the following strengths: 12 mg/5 mL (2.4 mg/mL) 28 mg/5 mL (5.6 mg/mL) 50 mg/5 mL (10 mg/mL) Injection: 12 mg/5 mL (2.4 mg/mL) in a single-dose vial ( 3 ) Injection: 28 mg/5 mL (5.6 mg/mL) in a single-dose vial ( 3 ) Injection: 50 mg/5 mL (10 mg/mL) in a single-dose vial ( 3 )

⛔ Contraindications 4 words ▾

4 CONTRAINDICATIONS None. None.

⚠️ Warnings and Cautions ~1 min read ▾

5 WARNINGS AND PRECAUTIONS Thrombocytopenia and Coagulation Abnormalities: Increased risk for bleeding complications; testing required at baseline and before each dose and as clinically needed ( 5.1 , 2.3 ) Renal Toxicity: Quantitative spot urine protein testing required at baseline and prior to each dose ( 5.2 , 2.3 )

5.1Thrombocytopenia and Coagulation Abnormalities Coagulation abnormalities and thrombocytopenia, including acute severe thrombocytopenia, have been observed after administration of some antisense oligonucleotides. In the sham-controlled studies for patients with infantile-onset (Study 1) and later-onset (Study 2) SMA who received Low Dose Regimen [see Clinical Studies ( 14.1 , 14.2 )] , 24 of 146 (16%) SPINRAZA-treated patients with high, normal, or unknown platelet count at baseline developed a platelet level below the lower limit of normal, compared to 10 of 72 (14%) sham-controlled patients.

In Study 2, two SPINRAZA-treated patients developed platelet counts less than 50,000 cells per microliter, with a lowest level of 10,000 cells per microliter recorded on study day 28. In patients who received High Dose Regimen, decreases in platelet counts were also observed. Because of the risk of thrombocytopenia and coagulation abnormalities from SPINRAZA, patients may be at increased risk of bleeding complications.

Perform a platelet count and coagulation laboratory testing at baseline and prior to each administration of SPINRAZA and as clinically needed.

5.2Renal Toxicity Renal toxicity, including potentially fatal glomerulonephritis, has been observed after administration of some antisense oligonucleotides. SPINRAZA is present in and excreted by the kidney [see Clinical Pharmacology ( 12.3 )] . In Study 1 and Study 2, 71 of 123 (58%) of SPINRAZA-treated patients had elevated urine protein, compared to 22 of 65 (34%) sham-controlled patients.

Conduct quantitative spot urine protein testing (preferably using a first morning urine specimen) at baseline and prior to each dose of SPINRAZA. For urinary protein concentration greater than 0.2 g/L, consider repeat testing and further evaluation.

🤒 Adverse Reactions ~3 min read ▾

6 ADVERSE REACTIONS The following serious adverse reactions are described in detail in other sections of the labeling: Thrombocytopenia and Coagulation Abnormalities [see Warnings and Precautions ( 5.1 )] Renal Toxicity [see Warnings and Precautions ( 5.2 )] The most common adverse reactions that occurred in at least 20% of SPINRAZA-treated patients who received Low Dose Regimen and occurred at least 5% more frequently than in control patients were: lower respiratory infection and constipation in patients with infantile-onset SMA ( 6.1 ) pyrexia, headache, vomiting, and back pain in patients with later-onset SMA ( 6.1 ) The most common adverse reactions in at least 10% of SPINRAZA-treated patients who received High Dose Regimen and occurred at least 5% more frequently than in historic matched sham-control were: pneumonia, COVID-19, pneumonia aspiration, and malnutrition in patients with infantile-onset SMA ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Biogen at 1-844-477-4672 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 SPINRAZA cannot be directly compared to rates in clinical trials of other drugs and may not reflect the rates observed in practice. SPINRAZA Low Dose Regimen (12 mg loading doses/12 mg maintenance doses) In clinical studies, 385 patients (47% male, 68% Caucasian, and 12% Asian) were treated with SPINRAZA Low Dose Regimen [see Dosage and Administration ( 2.1 )] , including 353 exposed for at least 6 months, 314 exposed for at least 1 year, and 256 exposed for at least 5 years.

Clinical Trial in Infantile-Onset SMA (Study 1) In Study 1, baseline disease characteristics were largely similar in the SPINRAZA-treated patients and sham-control patients except that SPINRAZA-treated patients at baseline had a higher percentage compared to sham-control patients of paradoxical breathing (89% vs 66%), pneumonia or respiratory symptoms (35% vs 22%), swallowing or feeding difficulties (51% vs 29%), and requirement for respiratory support (26% vs 15%). The most common adverse reactions that occurred in at least 20% of SPINRAZA-treated patients and occurred at least 5% more frequently than in control patients were lower respiratory infection and constipation.

Serious adverse reactions of atelectasis were more frequent in SPINRAZA-treated patients (18%) than in control patients (10%). Because patients in Study 1 were infants, adverse reactions that are verbally reported could not be assessed in this study. Table 2.

Adverse Reactions that Occurred in at Least 5% of SPINRAZA Patients and Occurred at Least 5% More Frequently or At Least 2 Times as Frequently Than in Control Patients with Infantile-Onset SMA (Study 1) 1 Low Dose Regimen [see Dosage and Administration ( 2.1 )] 2 Includes adenovirus infection, bronchiolitis, bronchitis, bronchitis viral, corona virus infection, Influenza, lower respiratory tract infection, lower respiratory tract infection viral, lung infection, parainfluenzae virus infection, pneumonia, pneumonia bacterial, pneumonia influenzal, pneumonia moraxella, pneumonia parainfluenzae viral, pneumonia pneumococcal, pneumonia pseudomonal, pneumonia respiratory syncytial viral, pneumonia viral, and respiratory syncytial virus bronchiolitis.

Adverse Reactions SPINRAZA 12 mg 1 N = 80 % Sham-Procedure Control N = 41 % Lower respiratory infection 2 55 37 Constipation 35 22 Teething 18 7 Urinary tract infection 9 0 Upper respiratory tract congestion 8 2 Ear infection 6 2 Flatulence 5 2 Decreased weight 5 2 In an open-label clinical study in infants with symptomatic SMA, severe hyponatremia was reported in a patient treated with SPINRAZA requiring salt supplementation for 14 months. Cases of rash were reported in patients treated with SPINRAZA. One patient, 8 months after starting SPINRAZA treatment, developed painless red macular lesions on the f… [Excerpted — this section continues on DailyMed.]

👥 Use in Specific Populations ~2 min read ▾

8 USE IN SPECIFIC POPULATIONS Pregnancy: Based on animal data, may cause fetal harm ( 8.1 )

8.1Pregnancy Risk Summary There are no adequate data on the developmental risk associated with the use of SPINRAZA in pregnant women. When nusinersen was administered by subcutaneous injection to mice throughout pregnancy and lactation, developmental toxicity (long-term neurobehavioral impairment) was observed at all doses tested (see Data ) . 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 When nusinersen (0, 3, 10, or 25 mg/kg) was administered subcutaneously to male and female mice every other day prior to and during mating and continuing in females throughout organogenesis, no adverse effects on embryofetal development were observed. Subcutaneous administration of nusinersen (0, 6, 12.6, or 25 mg/kg) to pregnant rabbits every other day throughout organogenesis produced no evidence of embryofetal developmental toxicity.

When nusinersen (1.4, 5.8, or 17.2 mg/kg) was administered to pregnant female mice by subcutaneous injection every other day throughout organogenesis and continuing once every six days throughout the lactation period, adverse neurobehavioral effects (alterations in locomotor activity, learning and memory deficits) were observed when offspring were tested after weaning or as adults. A no-effect level for neurobehavioral impairment was not established.

8.2Lactation Risk Summary There are no data on the presence of nusinersen in human milk, the effects on the breastfed infant, or the effects of the drug on milk production. Nusinersen was detected in the milk of lactating mice when administered by subcutaneous injection. The developmental and health benefits of breastfeeding should be considered along with the mother's clinical need for SPINRAZA and any potential adverse effects on the breastfed infant from SPINRAZA or from the underlying maternal condition.

8.4Pediatric Use The safety and effectiveness of SPINRAZA in pediatric patients from newborn to 17 years have been established [see Clinical Studies ( 14.1 )]. Juvenile Animal Toxicity Data In intrathecal toxicity studies in juvenile monkeys, administration of nusinersen (0, 0.3, 1, or 3 mg/dose for 14 weeks and 0, 0.3, 1, or 4 mg/dose for 53 weeks) resulted in brain histopathology (neuronal vacuolation and necrosis/cellular debris in the hippocampus) at the mid and high doses and acute, transient deficits in lower spinal reflexes at the high dose in each study.

In addition, possible neurobehavioral deficits were observed on a learning and memory test at the high dose in the 53-week monkey study. In a combined 6 and 13 week toxicity study in juvenile monkeys, intrathecal administration of nusinersen at higher doses (0, 5, 10, or 15 mg/dose) resulted in additional acute, transient effects, including limited use of limbs at the mid and high dose and uncoordinated movement at the high dose. The no-effect dose for neurohistopathology in monkeys (0.3 mg/dose) is approximately equivalent to and lower than the recommended clinical maintenance doses of 12 and 28 mg, respectively, when calculated on annual dose basis and corrected for species differences in CSF volume.

8.5Geriatric Use Clinical studies of SPINRAZA did not include sufficient numbers of subjects aged 65 and over to determine whether they respond differently from younger subjects.

🤰 Pregnancy ~1 min read ▾

8.1Pregnancy Risk Summary There are no adequate data on the developmental risk associated with the use of SPINRAZA in pregnant women. When nusinersen was administered by subcutaneous injection to mice throughout pregnancy and lactation, developmental toxicity (long-term neurobehavioral impairment) was observed at all doses tested (see Data ) . 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 When nusinersen (0, 3, 10, or 25 mg/kg) was administered subcutaneously to male and female mice every other day prior to and during mating and continuing in females throughout organogenesis, no adverse effects on embryofetal development were observed. Subcutaneous administration of nusinersen (0, 6, 12.6, or 25 mg/kg) to pregnant rabbits every other day throughout organogenesis produced no evidence of embryofetal developmental toxicity.

When nusinersen (1.4, 5.8, or 17.2 mg/kg) was administered to pregnant female mice by subcutaneous injection every other day throughout organogenesis and continuing once every six days throughout the lactation period, adverse neurobehavioral effects (alterations in locomotor activity, learning and memory deficits) were observed when offspring were tested after weaning or as adults. A no-effect level for neurobehavioral impairment was not established.

🧒 Pediatric Use 204 words ▾

8.4Pediatric Use The safety and effectiveness of SPINRAZA in pediatric patients from newborn to 17 years have been established [see Clinical Studies ( 14.1 )]. Juvenile Animal Toxicity Data In intrathecal toxicity studies in juvenile monkeys, administration of nusinersen (0, 0.3, 1, or 3 mg/dose for 14 weeks and 0, 0.3, 1, or 4 mg/dose for 53 weeks) resulted in brain histopathology (neuronal vacuolation and necrosis/cellular debris in the hippocampus) at the mid and high doses and acute, transient deficits in lower spinal reflexes at the high dose in each study.

In addition, possible neurobehavioral deficits were observed on a learning and memory test at the high dose in the 53-week monkey study. In a combined 6 and 13 week toxicity study in juvenile monkeys, intrathecal administration of nusinersen at higher doses (0, 5, 10, or 15 mg/dose) resulted in additional acute, transient effects, including limited use of limbs at the mid and high dose and uncoordinated movement at the high dose. The no-effect dose for neurohistopathology in monkeys (0.3 mg/dose) is approximately equivalent to and lower than the recommended clinical maintenance doses of 12 and 28 mg, respectively, when calculated on annual dose basis and corrected for species differences in CSF volume.

🧓 Geriatric Use 27 words ▾

8.5Geriatric Use Clinical studies of SPINRAZA did not include sufficient numbers of subjects aged 65 and over to determine whether they respond differently from younger subjects.

🧬 Clinical Pharmacology ~3 min read ▾

12 CLINICAL PHARMACOLOGY

12.1Mechanism of Action SPINRAZA is an antisense oligonucleotide (ASO) designed to treat SMA caused by mutations in chromosome 5q that lead to SMN protein deficiency. Using in vitro assays and studies in transgenic animal models of SMA, SPINRAZA was shown to increase exon 7 inclusion in SMN2 messenger ribonucleic acid (mRNA) transcripts and production of full-length SMN protein.

12.2Pharmacodynamics Autopsy samples from patients (n=3) had higher levels of SMN2 messenger ribonucleic acid (mRNA) containing exon 7 in the thoracic spinal cord compared to untreated SMA infants. Cardiac Electrophysiology Across the sham-controlled studies in 247 patients with spinal muscular atrophy who received either SPINRAZA Low Dose Regimen or sham-control, QTcF values > 500 ms and change from baseline values > 60 ms were observed in 4 (2.4%) patients receiving SPINRAZA. Compared to the sham-control, there was no increase in the incidence of cardiac adverse reactions associated with delayed ventricular repolarization in patients treated with SPINRAZA.

12.3Pharmacokinetics Absorption Intrathecal injection of SPINRAZA into the cerebrospinal fluid (CSF) allows nusinersen to be distributed from the CSF to the target central nervous system (CNS) tissues. Following intrathecal administration, trough plasma concentrations of nusinersen were relatively low, compared to the trough CSF concentration. Median plasma Tmax values ranged from 1.7 to 6.0 hours.

Mean plasma Cmax and AUC values increased approximately dose-proportionally up to a dose of 12 mg. Distribution Autopsy data from patients (n=3) showed that SPINRAZA administered intrathecally was distributed within the CNS and peripheral tissues, such as skeletal muscle, liver, and kidney. Elimination Metabolism Nusinersen is metabolized via exonuclease (3'- and 5')-mediated hydrolysis and is not a substrate for, or inhibitor or inducer of CYP450 enzymes.

Excretion The mean terminal elimination half-life is estimated to be 135 to 177 days in CSF, and 63 to 87 days in plasma. The primary route of elimination is likely by urinary excretion for nusinersen and its chain-shortened metabolites. At 24 hours, only 0.5% of the administered dose was recovered in the urine.

12.6Immunogenicity The observed incidence of anti-drug antibodies is highly dependent on the sensitivity and specificity of the assay. Differences in assay methods preclude meaningful comparisons of the incidence of anti-drug antibodies in the studies described below with the incidence of anti-drug antibodies in other studies, including those of SPINRAZA or of other nusinersen products. The immunogenic response to nusinersen in patients who received SPINRAZA Low Dose Regimen was evaluated in 367 patients with post-baseline plasma samples for anti-drug antibodies (ADAs).

Thirty eight patients (10%) developed treatment-emergent ADAs, of which 16 were transient and 22 were considered to be persistent. Persistent was defined as having one positive test followed by another one more than 100 days after the first positive test. In addition, “persistent” is also defined as having one or more positive samples and no sample more than 100 days after the first positive sample.

Transient was defined as having one or more positive results and not confirmed to be persistent. The immunogenic response to nusinersen in patients who received SPINRAZA High Dose Regimen was evaluated in 117 patients with post-baseline plasma samples for ADAs. Eleven patients (9%) developed treatment-emergent ADAs, of which 5 were transient and 6 were persistent.

There are insufficient data to evaluate an effect of ADAs on clinical response, adverse events, or the pharmacokinetic profile of nusinersen.

🧬 Mechanism of Action 59 words ▾

12.1Mechanism of Action SPINRAZA is an antisense oligonucleotide (ASO) designed to treat SMA caused by mutations in chromosome 5q that lead to SMN protein deficiency. Using in vitro assays and studies in transgenic animal models of SMA, SPINRAZA was shown to increase exon 7 inclusion in SMN2 messenger ribonucleic acid (mRNA) transcripts and production of full-length SMN protein.

📦 How Supplied / Storage and Handling ~1 min read ▾

16 HOW SUPPLIED/STORAGE AND HANDLING

16.1How Supplied SPINRAZA injection is a sterile, clear and colorless, preservative-free solution in single-dose glass vials supplied as one vial per carton in the following strengths: 12 mg/5 mL (2.4 mg/mL) (NDC 64406-058-01) 28 mg/5 mL (5.6 mg/mL) (NDC 64406-036-01) 50 mg/5 mL (10 mg/mL) (NDC 64406-037-01)

16.2Storage and Handling Store in a refrigerator between 2°C to 8°C (36°F to 46°F) in the original carton to protect from light. Do not freeze. SPINRAZA should be protected from light and kept in the original carton until time of use.

If no refrigeration is available, SPINRAZA may be stored in its original carton, protected from light at or below 30 o C (86 o F) for up to 14 days. Prior to administration, unopened vials of SPINRAZA can be removed from and returned to the refrigerator, if necessary. If removed from the original carton, the total combined time out of refrigeration should not exceed 30 hours at a temperature that does not exceed 25 o C (77 o F).

16.1How Supplied SPINRAZA injection is a sterile, clear and colorless, preservative-free solution in single-dose glass vials supplied as one vial per carton in the following strengths: 12 mg/5 mL (2.4 mg/mL) (NDC 64406-058-01) 28 mg/5 mL (5.6 mg/mL) (NDC 64406-036-01) 50 mg/5 mL (10 mg/mL) (NDC 64406-037-01)

📦 Storage and Handling 121 words ▾

16.2Storage and Handling Store in a refrigerator between 2°C to 8°C (36°F to 46°F) in the original carton to protect from light. Do not freeze. SPINRAZA should be protected from light and kept in the original carton until time of use.

If no refrigeration is available, SPINRAZA may be stored in its original carton, protected from light at or below 30 o C (86 o F) for up to 14 days. Prior to administration, unopened vials of SPINRAZA can be removed from and returned to the refrigerator, if necessary. If removed from the original carton, the total combined time out of refrigeration should not exceed 30 hours at a temperature that does not exceed 25 o C (77 o F).

📋 Description ~2 min read ▾

11 DESCRIPTION Nusinersen is a modified antisense oligonucleotide, where the 2'-hydroxy groups of the ribofuranosyl rings are replaced with 2'-O-2-methoxyethyl groups and the phosphate linkages are replaced with phosphorothioate linkages. Nusinersen binds to a specific sequence in the intron downstream of exon 7 of the SMN2 transcript. The structural formula is: SPINRAZA is supplied as a sterile, preservative-free, colorless solution for intrathecal use in a single-dose glass vial in the following strengths: 12 mg/5 mL (2.4 mg/mL) 28 mg/5 mL (5.6 mg/mL) 50 mg/5 mL (10 mg/mL) Each 1 mL solution of the 12 mg/5 mL strength contains 2.4 mg of nusinersen (equivalent to 2.53 mg of nusinersen sodium salt).

Each 1 mL also contains calcium chloride dihydrate (0.21 mg) USP, magnesium chloride hexahydrate (0.16 mg) USP, potassium chloride (0.22 mg) USP, sodium chloride (8.77 mg) USP, sodium phosphate dibasic anhydrous (0.10 mg) USP, sodium phosphate monobasic dihydrate (0.05 mg) USP, and Water for Injection USP. Each 1 mL solution of the 28 mg/5 mL strength contains 5.6 mg of nusinersen (equivalent to 5.90 mg of nusinersen sodium salt). Each 1 mL also contains calcium chloride dihydrate (0.21 mg) USP, magnesium chloride hexahydrate (0.16 mg) USP, potassium chloride (0.22 mg) USP, sodium chloride (8.39 mg) USP, sodium phosphate dibasic anhydrous (0.10 mg) USP, sodium phosphate monobasic dihydrate (0.05 mg) USP, and Water for Injection USP.

Each 1 mL solution of the 50 mg/5 mL strength contains 10 mg of nusinersen (equivalent to 10.54 mg of nusinersen sodium salt). Each 1 mL also contains calcium chloride dihydrate (0.21 mg) USP, magnesium chloride hexahydrate (0.16 mg) USP, potassium chloride (0.22 mg) USP, sodium chloride (8.11 mg) USP, sodium phosphate dibasic anhydrous (0.10 mg) USP, sodium phosphate monobasic dihydrate (0.05 mg) USP, and Water for Injection USP. For all strengths the product may contain hydrochloric acid or sodium hydroxide to adjust pH.

The pH is ~7.2. The molecular formula of SPINRAZA is C 234 H 323 N 61 O 128 P 17 S 17 Na 17 and the molecular weight is 7501.0 daltons. Structural Formula

💬 Information for Patients 131 words ▾

17 PATIENT COUNSELING INFORMATION Thrombocytopenia and Coagulation Abnormalities Inform patients and caregivers that SPINRAZA could increase the risk of bleeding. Inform patients and caregivers of the importance of obtaining blood laboratory testing at baseline and prior to each dose to monitor for signs of increased potential for bleeding. Instruct patients and caregivers to seek medical attention if unexpected bleeding occurs [see Warnings and Precautions ( 5.1 )] .

Renal Toxicity Inform patients and caregivers that SPINRAZA could cause renal toxicity. Inform patients and caregivers of the importance of obtaining urine testing at baseline and prior to each dose to monitor for signs of potential renal toxicity [see Warnings and Precautions ( 5.2 )] . 60110-01 Manufactured for: Biogen Cambridge, MA 02142 SPINRAZA is a registered trademark of Biogen. © Biogen 2016-2026

🧬 Pharmacokinetics 174 words ▾

12.3Pharmacokinetics Absorption Intrathecal injection of SPINRAZA into the cerebrospinal fluid (CSF) allows nusinersen to be distributed from the CSF to the target central nervous system (CNS) tissues. Following intrathecal administration, trough plasma concentrations of nusinersen were relatively low, compared to the trough CSF concentration. Median plasma Tmax values ranged from 1.7 to 6.0 hours.

Mean plasma Cmax and AUC values increased approximately dose-proportionally up to a dose of 12 mg. Distribution Autopsy data from patients (n=3) showed that SPINRAZA administered intrathecally was distributed within the CNS and peripheral tissues, such as skeletal muscle, liver, and kidney. Elimination Metabolism Nusinersen is metabolized via exonuclease (3'- and 5')-mediated hydrolysis and is not a substrate for, or inhibitor or inducer of CYP450 enzymes.

Excretion The mean terminal elimination half-life is estimated to be 135 to 177 days in CSF, and 63 to 87 days in plasma. The primary route of elimination is likely by urinary excretion for nusinersen and its chain-shortened metabolites. At 24 hours, only 0.5% of the administered dose was recovered in the urine.

🧬 Pharmacodynamics 97 words ▾

12.2Pharmacodynamics Autopsy samples from patients (n=3) had higher levels of SMN2 messenger ribonucleic acid (mRNA) containing exon 7 in the thoracic spinal cord compared to untreated SMA infants. Cardiac Electrophysiology Across the sham-controlled studies in 247 patients with spinal muscular atrophy who received either SPINRAZA Low Dose Regimen or sham-control, QTcF values > 500 ms and change from baseline values > 60 ms were observed in 4 (2.4%) patients receiving SPINRAZA. Compared to the sham-control, there was no increase in the incidence of cardiac adverse reactions associated with delayed ventricular repolarization in patients treated with SPINRAZA.

🔬 Clinical Studies ~3 min read ▾

14 CLINICAL STUDIES The efficacy of SPINRAZA Low Dose Regimen was demonstrated in two double-blind, sham-procedure controlled clinical trials in patients with symptomatic infantile-onset and later-onset SMA (Study 1 and Study 2) and was supported by open-label clinical trials conducted in patients with presymptomatic (Study 3) and symptomatic SMA [see Dosage and Administration ( 2.1 )] . The efficacy of SPINRAZA High Dose Regimen was demonstrated in a double-blind, randomized, external-controlled trial in patients with symptomatic infantile-onset and later-onset SMA (Study 4).

The overall findings from these trials support the effectiveness of SPINRAZA administered with either the Low Dose Regimen or the High Dose Regimen across the range of ages and severities in patients with SMA, and support the early initiation of treatment with SPINRAZA.

14.1Infantile-Onset SMA Study 1 (NCT02193074) was a multicenter, randomized, double-blind, sham-procedure controlled study in 121 symptomatic infants ≤ 7 months of age at the time of first dose, diagnosed with SMA (symptom onset before 6 months of age). Patients were randomized 2:1 to receive either SPINRAZA Low Dose Regimen or sham injection as a series of loading doses administered intrathecally followed by maintenance doses administered every 4 months. Patients in this study were deemed most likely to develop Type 1 SMA.

A planned interim efficacy analysis was conducted based on patients who died, withdrew, or completed at least 183 days of treatment. Of the 82 patients included in the interim analysis (52 patients in the SPINRAZA-treated group and 30 in the sham-control group), 44% were male, 87% were Caucasian, 2% were Black, and 4% were Asian. Age at first treatment ranged from 30 to 262 days (median 181).

Length of treatment ranged from 6 to 442 days (median 261 days). Baseline demographics were balanced between the SPINRAZA and control groups with the exception of age at first treatment (median age 175 vs. 206 days, respectively).

The SPINRAZA and control groups were balanced with respect to gestational age, birth weight, disease duration, and SMN2 copy number. Median disease duration was 14 weeks. There was some imbalance in age at symptom onset with 88% of subjects in the SPINRAZA group and 77% in the control group experiencing symptoms within the first 12 weeks of life.

The primary endpoint assessed at the time of interim analysis was the proportion of responders: patients with an improvement in motor milestones according to Section 2 of the Hammersmith Infant Neurologic Exam (HINE). This endpoint evaluates seven different areas of motor milestone development, with a maximum score between 2-4 points for each, depending on the milestone, and a total maximum score of 26. A treatment responder was defined as any patient with at least a 2-point increase (or maximal score of 4) in ability to kick (consistent with improvement by at least 2 milestones), or at least a 1-point increase in the motor milestones of head control, rolling, sitting, crawling, standing or walking (consistent with improvement by at least 1 milestone).

To be classified as a responder, patients needed to exhibit improvement in more categories of motor milestones than worsening. Of the 82 patients who were eligible for the interim analysis, a statistically significantly greater percentage of patients achieved the definition of a motor milestone responder in the SPINRAZA group (40%) compared to the sham-control group (0%). Results from the final analysis were consistent with those from the interim analysis ( Table 5 ).

Fifty-one percent of patients in the SPINRAZA group achieved the definition of a motor milestone responder compared to 0% of patients in the sham-control group. Figure 1 is a descriptive display of the distribution of net change from baseline in the total motor milestone score for Section 2 of the HINE for patients in the final efficacy set who did not die or withdraw from the study. The primary end… [Excerpted — this section continues on DailyMed.]

🧪 Nonclinical Toxicology 118 words ▾

13 NONCLINICAL TOXICOLOGY

13.1Carcinogenesis, Mutagenesis, Impairment of Fertility Carcinogenesis Administration of nusinersen (0, 5, 15, or 50 mg/kg) to male and female mice by subcutaneous injection, once every two weeks for 2 years, resulted in an increase in the incidence of vascular tumors (combined hemangioma and hemangiosarcoma) at the highest dose tested. Mutagenesis Nusinersen demonstrated no evidence of genotoxicity in in vitro (Ames and chromosomal aberration in CHO cells) and in vivo (mouse micronucleus) assays. Impairment of Fertility When nusinersen (0, 3, 10, or 25 mg/kg) was administered by subcutaneous injection to mice every other day prior to and during mating and continuing in females throughout organogenesis, no adverse effects on male or female fertility were observed.

📄 Carcinogenesis, Mutagenesis, Impairment of Fertility 115 words ▾

13.1Carcinogenesis, Mutagenesis, Impairment of Fertility Carcinogenesis Administration of nusinersen (0, 5, 15, or 50 mg/kg) to male and female mice by subcutaneous injection, once every two weeks for 2 years, resulted in an increase in the incidence of vascular tumors (combined hemangioma and hemangiosarcoma) at the highest dose tested. Mutagenesis Nusinersen demonstrated no evidence of genotoxicity in in vitro (Ames and chromosomal aberration in CHO cells) and in vivo (mouse micronucleus) assays. Impairment of Fertility When nusinersen (0, 3, 10, or 25 mg/kg) was administered by subcutaneous injection to mice every other day prior to and during mating and continuing in females throughout organogenesis, no adverse effects on male or female fertility were observed.

📄 Recent Major Changes 20 words ▾

Dosage and Administration ( 2.1 , 2.2 , 2.3 , 2.5 ) 03/2026 Warnings and Precautions ( 5.1 ) 03/2026

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

Principal Display Panel - Spinraza 12 mg/5 mL Carton Label NDC 64406- 058 -01 Spinraza ® (nusinersen) Injection 12 mg/5 mL (2.4 mg/mL) Sterile solution for Intrathecal Use Only Rx Only Biogen ® Principal Display Panel - Spinraza 12 mg/5 mL Carton Label

Principal Display Panel - Spinraza 12 mg/5 mL Vial Label NDC 64406- 058 -01 Spinraza® (nusinersen) injection 12 mg/5 mL (2.4 mg/mL) For Intrathecal Use Only Manufactured For: Biogen Inc. Principal Display Panel - Spinraza 12 mg/5 mL Vial Label

Principal Display Panel - Spinraza 28 mg/5 mL Carton Label NDC 64406- 0 36 -01 Spinraza ® (nusinersen) Injection 28 mg/5 mL (5.6 mg/mL) Sterile solution for Intrathecal Use Only Rx Only Biogen ® Principal Display Panel - Spinraza 28 mg/5 mL Carton Label

Principal Display Panel - Spinraza 28 mg/5 mL Vial Label NDC 64406- 0 36 -01 Spinraza ® (nusinersen) injection 28 mg/5 mL (5.6 mg/mL) For Intrathecal Use Only Manufactured For: Biogen Inc. Principal Display Panel - Spinraza 28 mg/5 mL Vial Label

Principal Display Panel - Spinraza 50 mg/5 mL Carton Label NDC 64406- 0 37 -01 Spinraza ® (nusinersen) Injection 50 mg/5 mL (10 mg/mL) Sterile solution for Intrathecal Use Only Rx Only Biogen ® Principal Display Panel - Spinraza 50 mg/5 mL Carton Label

Principal Display Panel - Spinraza 50 mg/5 mL Vial Label NDC 64406- 0 37 -01 Spinraza ® (nusinersen) injection 50 mg/5 mL (10 mg/mL) For Intrathecal Use Only Manufactured For: Biogen Inc. Principal Display Panel - Spinraza 50 mg/5 mL 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 · 2025 (Q1-Q4)

Medicare Part D (outpatient prescription) spending for Spinraza — 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 Spinraza. That’s a different level of aggregation than the Medicaid card above, which is specific to this exact 11-digit NDC (pack size included), so the two aren’t directly comparable.
Total Part D spend
$4.55M
Claims incl. refills
31
Beneficiaries
11
Spend / beneficiary
$413,638.52
Spend / claim
$146,774.96
💵 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

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NDC identity (package / product / labeler codes) ✓ Available
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NADAC pharmacy acquisition price (CMS) — Not published for this NDC CMS publishes NADAC only for NDCs reported in its retail-pharmacy survey.
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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 J2326 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.
← 64406-0036-01 · Biogen 64406-0058-01 · 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.