Novartis will launch a new phase three study to expand the use of Zolgensma – the world’s most expensive one-time therapy at $2.1 million per patient treatment – after the U.S. regulator lifted its restrictions.
Roche presented results from several Phase II and III trials at the 7th Congress of the European Academy of Neurology (EAN) Annual Meeting held virtually June 19-22.
Swiss drugmakers Roche and Novartis each won European approval for drugs on March 30 in a sign that the rival Basel-based companies are increasingly competing for the same patients.
Novartis is closing a manufacturing facility in Longmont, Colorado acquired by company subsidiary AveXis during 2019 that has provided additional production capacity for Zolgensma, a gene therapy treatment for spinal muscular atrophy.
BioSpace reviewed the top 10 novel drug approvals of 2020, loosely based on projected earnings in the upcoming years.
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.