New Zolgensma 'inflection point' is here as Novartis snags EU nod for SMA gene therapy

As the number of U.S. spinal muscular atrophy (SMA) patients Zolgensma treats each quarter stabilizes, Novartis is counting on a set of “inflection points” for future growth. Now, it has one.

After a manufacturing-related delay, Novartis has won conditional approval in the EU for the one-time gene therapy to treat patients with a clinical diagnosis of SMA type 1 and others with up to three copies of the SMN2 backup gene, the company said Monday.