Research & Development
US FDA approves PTC Therapeutics' Evrysdi
10 August 2020 -

The United States Food and Drug Administration (FDA) has approved United States-based PTC Therapeutics Inc's. (NASDAQ: PTCT) Evrysdi (risdiplam), the first at-home, orally administered treatment for spinal muscular atrophy in adults and children two months and older, it was reported on Friday.

The product indicated clinical improvement in motor function and received developmental milestones across two trials in patients aged two months and above and across all levels of disease severity, including types one, two, and three spinal muscular atrophy.

Evrysdi was studied in a clinical trial program in spinal muscular atrophy with over 450 patients and subjects. It will be offered in the United States within two weeks for direct delivery to patients' homes via Accredo Health Group Inc, an Express Scripts specialty pharmacy.