EC approval and activation of "Day One" access program for Zolgensma in SMA

AveXis today announced the European Commission (EC) granted conditional approval for Zolgensma (onasemnogene abeparvovec) 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 for patients with 5q SMA with a bi-allelic mutation in the SMN1  gene and up to three copies of the SMN2 gene.

The approval for this one-time gene therapy covers babies and young children with SMA up to 21kg according to the approved dosing guidance, noted AveXis, which Novartis (NOVN: VX) acquired in 2018 for $8.7 billion. The overall cost of care burden of the disease in Europe is estimated to be between 2.5 million and 4 million euros ($4.3million) in the first 10 years of life.

Zolgensma was approved by the US Food and Drug Administration in May 2019, and dubbed the ‘most expensive drug ever’ with a US wholesale acquisition cost set by the company of $2.125 million paid over five years. The drug is expected to generate global sales of $2.5 billion by 2025 and lead the spinal muscular atrophy (SMA) market, according to data and analytics company GlobalData. In March this year, the drug picked up approval Japan, where it has been given a lower price tag of around $1.55 million. In the first quarter of this year, sales were just $170 million.