Novartis is seeking to repurpose the company’s investigational oral spinal muscular atrophy (SMA) medicine branaplam to treat Huntington’s disease, as the Swiss drugmaker plans a clinical trial after winning U.S. orphan drug designation.
Novartis said new interim data from an ongoing phase clinical trial for the company’s $2.1 million-per-patient gene therapy Zolgensma showed spinal muscular atrophy patients experienced significant therapeutic benefit.
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).