Regenxbio and Novartis down on data issue related to SMA gene therapy

REGENXBIO (RGNX -2.1%) slips on increased volume in response to a data integrity issue related to Novartis (NVS -3.4%) unit AveXis’ gene therapy Zolgensma (onasemnogene abeparvovec-xioi), approved by the FDA in May for pediatric patients with spinal muscular atrophy (SMA).

In a statement, the agency says AveXis notified it about a “data manipulation issue” impacting the accuracy of certain tests performed in animals, the results of which were included in the marketing application. The FDA is “carefully assessing” the situation and “remains confident” that the product should remain on the market, but adds that AveXis failed to inform it on the matter before the product was approved despite being aware beforehand.

AveXis in-licensed RGNX’s NAV AAV9 vector used in Zolgensma in March 2014.

SMA competitors Biogen (BIIB +1.3%) and Ionis Pharmaceuticals (IONS+3.1%) are up on the news.

https://seekingalpha.com/news/3487771-regenxbio-novartis-data-issue-related-sma-gene-therapy