rarerelated tag: Definitions/ Other Names

FDA Lifts Partial Clinical Hold on Novartis’ SMA Drug Programme

The US Food and Drug Administration (FDA) has lifted the partial clinical trial hold on Novartis’ intrathecal (IT) OAV-101 (AVXS-101) programme for patients with spinal muscular atrophy (SMA).

The latest decision comes after the FDA analysed data from the company’s nonclinical toxicology study in non-human primates that addressed all detected issues, including questions related to dorsal root ganglia (DRG) injury after IT injection.

With the FDA approval and input, along with guidance from the European Medicines Agency (EMA), Novartis intends to commence the Phase III STEER trial to assess the clinical efficacy, safety and tolerability of a single IT dose of OAV-101 in SMA patients aged two to 18 years.