Today, European Medicines Agency (EMA) recommended a conditional approval of the breakthrough gene therapy drug Zolgensma® for treating spinal muscular atrophy across the European Union.
The Agency wrote in today’s press release:
Zolgensma 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.
Unlike in the case of the US and Japanese labels, the European regulator has not proposed restrictions based on patient’s age.
While the current press release does not specify it, the drug’s safety profile will certainly limit it to children within a specific body weight bracket. We are awaiting details.
A conditional approval usually indicates that the data was not strong or complete enough for EMA to recommend full approval, and the manufacturer will have been requested to supply additional data in the next year or two. However, the drug can be legally prescribed for the time being, with the agency reserving a right to revise the decision depending on data.
The final approval by the European Commission is usually concluded within 67 days from EMA recommendation and will also apply to the UK.
NICE has already started an appraisal process for Zolgensma, unfortunately only in type 1 children. TreatSMA is part of the process.
Important note: as the second bullet point in the EMA criteria above does not mention clinical diagnosis, it is unclear whether this applies to symptomatic or only to presymptomatic children. We are in the process of clarifying it; hopefully, the final label document will offer more clarity.