2 Information about onasemnogene abeparvovec

Marketing authorisation indication


Onasemnogene abeparvovec (Zolgensma, Novartis Gene Therapies) is indicated for 'the treatment of patients with 5q spinal muscular atrophy (SMA) with a bi-allelic mutation in the SMN1 gene and a clinical diagnosis of SMA Type 1, or patients with 5q SMA with a bi-allelic mutation in the SMN1 gene and up to 3 copies of the SMN2 gene'.

Dosage in the marketing authorisation



The price for onasemnogene abeparvovec is £1,795,000 for a one-time dose (excluding VAT; company submission).


The company has a commercial arrangement. This makes onasemnogene abeparvovec available to the NHS with a discount. The size of the discount is commercial in confidence. It is the company's responsibility to let relevant NHS organisations know details of the discount.

  • National Institute for Health and Care Excellence (NICE)