Risdiplam
Orphan DrugUnregistered in India
Contents
Description
Risdiplam is a small molecule SMN2 pre-mRNA splicing modifier used for the treatment of spinal muscular atrophy.
Indications & Therapeutic Use
Spinal Muscular Atrophy (SMA) types 1, 2, and 3
Linked Diseases:
Global Availability (1 countries)
| Country | Access Route | Regulatory Pathway | Lead Time | Status |
|---|---|---|---|---|
| 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
Buy / Source Risdiplam — GNH India
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Available Through
This drug is available for procurement through the following fulfilment partner.
Fulfilment Partner
Risdiplam
| Generic Name | Risdiplam |
| Brands | 1 brand available |
| Active Ingredient | Risdiplam |
| Drug Class | Spinal Muscular Atrophy (SMA) types 1 |
| Manufacturer | Genentech / Roche |
| Dosage Forms | Oral solution 0.75mg/mL |
| Medical Code | RxNorm:2413676 |
| Orphan Status | Yes — Orphan Drug |
| Cold Chain | Not Required |
| Lead Time | 7 days |
| Reg. Status | Unregistered in India |
| Countries | 1 countries |
Authority Signals
Last Verified3/11/2026
Data ConfidenceHigh (AI + Pharmacist)
Source Citations2 Validated Nodes