AveXis, a Novartis company, announced today that the European Commission (EC) have granted conditional approval for Zolgensma for the treatment of patients with 5q SMA, activating a ‘Day One’ access programme.
The conditional approval includes treatment for babies and young children up to 21kg with 5q SMA with a bi-allelic mutation in the SMN1 gene and a clinical diagnosis of SMA type 1, as well as patients with 5q SMA with a bi-allelic mutation in the SMN1 gene and up to three copies of the SMN2 gene. This is with accordance with the approved dosing guidance.
The ‘Day One’ access programme helps with the cost of treatment. This will start while to company negotiates actual cost with the national health departments across the EU. The program will help with keeping the pricing consistent in the EU as there are different medicine payment systems including :
– deferred payments and installment options
In the UK pricing will require a review by NICE and an agreement by the NHS, though special access programs have been put in place like the RESTORE registry. In addition the company will provide training for hospitals.