Nusinersen
Orphan Drug Cold Chain RequiredUnregistered in India
Contents
Description
Nusinersen is an antisense oligonucleotide drug used to treat spinal muscular atrophy (SMA). It works by modifying the splicing of the SMN2 gene to produce more functional SMN protein.
Indications & Therapeutic Use
Spinal Muscular Atrophy (SMA) types 1, 2, and 3
Linked Diseases:
Global Availability (3 countries)
| Country | Access Route | Regulatory Pathway | Lead Time | Status |
|---|---|---|---|---|
| Named Patient | — | 5 days | Available via NPP | |
| EAMS | UK Early Access to Medicines Scheme (EAMS) | 10 days | Available via EAMS | |
| Named Patient | India Named Patient Import (Form 12B) | 7 days | Available via NPP |
Access Programs
Named Patient ProgramNamed Patient Program
Framework: Country-specific regulatory approvals
Typical timeline: 21 days
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Available Through
This drug is available for procurement through the following fulfilment partner.
Fulfilment Partner
Nusinersen
| Generic Name | Nusinersen |
| Brands | 1 brand available |
| Active Ingredient | Nusinersen sodium |
| Drug Class | Spinal Muscular Atrophy (SMA) types 1 |
| Manufacturer | Biogen |
| Dosage Forms | Intrathecal injection 12mg/5mL |
| Medical Code | RxNorm:1860484 |
| Orphan Status | Yes — Orphan Drug |
| Cold Chain | Required |
| Lead Time | 7 days |
| Reg. Status | Unregistered in India |
| Countries | 3 countries |
Authority Signals
Last Verified3/11/2026
Data ConfidenceHigh (AI + Pharmacist)
Source Citations4 Validated Nodes