2 Information about nusinersen and risdiplam

Marketing authorisation indications

2.1

Nusinersen (Spinraza, Biogen) is indicated for 'the treatment of 5q Spinal Muscular Atrophy'.

2.2

Risdiplam (Evrysdi, Roche) is indicated for 'the treatment of 5q spinal muscular atrophy (SMA) in patients with a clinical diagnosis of SMA Type 1, Type 2 or Type 3 or with one to 4 SMN2 copies'.

Dosage in the marketing authorisation

Price

2.5

The list price of nusinersen is £75,000 for 1 vial of 12 mg/5 ml solution for injection, £78,000 for 1 vial of 28 mg/5 ml solution, and £139,286 for 1 vial of 50 mg/5 ml of solution (excluding VAT; company submission). The company has a commercial arrangement (commercial access agreement). This makes nusinersen available to the NHS with a discount. The size of the discount is commercial in confidence.

2.6

The list price of risdiplam is £7,900 per 60 mg/80 ml oral solution (excluding VAT; BNF online accessed May 2026) and £18,433.00 for a pack of 5 mg tablets (28 tablets per pack; excluding VAT; company submission). The company has a commercial arrangement (simple discount patient access scheme). This makes risdiplam available to the NHS with a discount. The size of the discount is commercial in confidence.

Sustainability