Shots: The NDA submission is based on FIREFISH study assessing Risdiplam in infants with symptomatic SMA Type 1 & SUNFISH study in children and young adults with SMA Type 2 or 3 FIRESISH study results: improvement in survival and motor milestones in infants. SUNFISH study… Read More »Chugai Reports NDA Submission of Risdiplam to the MHLW as the First Oral Drug for SMA in Japan
spinal muscular atrophy
FDA’s Ok to Roche’s Oral SMA Therapy; Roche’s Etrolizumab Mixed Results in Ulcerative Colitis; Secura receives rights to Copiktra from Verastem
Roche Set to Compete With Biogen and Novartis in the SMA Market With its First Oral Therapy The US FDA has recommended the use of Evrysdi (risdiplam), a candidate of Genentech, a Roche company, for spinal muscular atrophy (SMA), an inherited neuromuscular disease resulting in… Read More »FDA’s Ok to Roche’s Oral SMA Therapy; Roche’s Etrolizumab Mixed Results in Ulcerative Colitis; Secura receives rights to Copiktra from Verastem
Analysts noted that Evrysdi would be priced significantly lower than Biogen’s Spinraza or Novartis’ Zolgensma. While this will provide a market advantage, it would also yield lower per-patient revenues.
The company plans to enroll 60 patients who have received its drug, Spinraza, following suboptimal response to Novartis’ Zolgensma, which was approved last year. The trial, RESPOND, is anticipated to start enrolling in the first quarter of 2021, pending regulatory approval.