Magdalena Kegel,  —

Magdalena is a writer with a passion for bridging the gap between the people performing research, and those who want or need to understand it. She writes about medical science and drug discovery. She holds an MS in Pharmaceutical Bioscience and a PhD — spanning the fields of psychiatry, immunology, and neuropharmacology — from Karolinska Institutet in Sweden.

Articles by Magdalena Kegel

After 2-Year Hiatus, Novartis Resumes Branaplam Clinical Trial in SMA Type 1 Infants

Novartis will resume clinical development of branaplam to treat spinal muscular atrophy after a two-year pause brought on by safety concerns, the Swiss company announced in a letter published by Cure SMA. The ongoing clinical trial will start enrolling patients as soon as the research team obtains approvals by health authorities and the ethics committee. The study recruits infants younger than six months with type 1 SMA. All will receive branaplam, the name Novartis chose for its compound when reopening clinical investigations. While the study originally only included four European study centers — in Belgium, Germany, Denmark and Italy — Novartis said it plans to add more sites including some in the United States, following a green light from the U.S. Food and Drug Administration (FDA). The branaplam clinical trial halted new enrollments in May 2016 after simultaneous animal studies linked branaplam to unexpected nerve damage and other injuries. Infants already enrolled continued treatment and have been closely monitored. Novartis has worked with outside experts to understand the meaning of the animal findings. The fact that enrollment has resumed signals that Novartis — which has not commented on its relevance — no longer perceives the animal data as an immediate patient hazard. Besides resuming recruitment, the company has modified its trial design after considering feedback from the babies’ parents and study investigators. For instance, infants can now receive their weekly dose of branaplam orally, instead of via a feeding tube, which was the only option when the trial started. The company also added nerve tests to the trial as an additional safety procedure. As clinical development of branaplam continues, Novartis is also working with regulators to expand drug testing to include patients with other types of SMA.