Swiss drugmaker Roche said 59% of infants with the severest form of spinal muscular atrophy could sit for at least five seconds after getting the company’s medicine Evrysdi for two years, according to trial data, up from 33% after a year of treatment.
Novartis’ bid to expand the company’s $2.1 million-per-patient gene therapy Zolgensma to more spinal muscular atrophy patients faces a possible delay after U.S. regulators requested another study in older children getting the drug via a spinal infusion.
Roche said two-thirds of spinal muscular atrophy (SMA) patients taking the Swiss drugmaker’s newly approved medicine Evrysdi have previously received rival treatments Zolgensma from Novartis or Biogen’s Spinraza.
A look at U.S. Food and Drug Administration PDUFA dates for end-of-August and early-September 2020.
The U.S. Food and Drug Administration approved an oral treatment from Roche Holding AG and PTC Therapeutics Inc. for spinal muscular atrophy in adults and children two months and above.
One of medicine’s costliest treatment areas could become even more pricey as Biogen seeks to layer the company’s $750,000 spinal muscular atrophy drug on top of the $2.1 million-per-patient Novartis gene therapy Zolgensma.
Biogen announced new data from the NURTURE trial of pre-symptomatic patients with spinal muscular atrophy (SMA).
Roche company Genentech presented one-year data from FIREFISH Part 2, a pivotal trial of risdiplam in infants one to seven months old with symptomatic Type 1 spinal muscular atrophy (SMA).
Biogen reported full-year 2019 financial results, citing total revenues of $14.378 billion, an increase of 7% from the previous year.
Roche company Genentech announced positive top-line results from the pivotal Part 2 of the FIREFISH study looking at risdiplam in infants aged 1-7 months with Type 1 spinal muscular atrophy (SMA).
