NICE has published final draft guidance regarding the recommendation of routine NHS funding for onasemnogene abeparvovec.
The treatment is a potentially curative one-off gene therapy for treating a rare genetic condition, spinal muscular atrophy (SMA). It will be used specifically among babies aged up to 12 months, before they develop symptoms.
The drug – also known as Zolgensma – is produced by Novartis Gene Therapies. The recommendation has emerged after NICE reviewed the evidence to determine whether it should be available for routine NHS funding in England and Wales…
