Spinal muscular atrophy
After bringing Zolgensma to market in 2019 as the first gene therapy for spinal muscular atrophy, Novartis is back with an intrathecal formulation intended for older patients.
Biohaven in recent months has reported a clinical stumble in spinal muscular atrophy, alongside a Phase I readout for its protein degrader candidate that investors found underwhelming.
Evrysdi is the first, and so far only, noninvasive disease-modifying treatment for spinal muscular atrophy.
In a good-news-bad-news week for Biogen, the company will cut an undisclosed number of employees, just as a higher dose of its Ionis-partnered therapy Spinraza for spinal muscular atrophy will be considered by the FDA and EMA.
While taldefgrobep alfa failed to show improved motor function in spinal muscular atrophy, treated patients saw a marked reduction in body fat. Biohaven plans to launch a Phase II trial in obesity by the end of the year.
With Monday’s data from SAPPHIRE, Scholar Rock is building toward regulatory submissions for apitegromab in spinal muscular atrophy in the first quarter of 2025.
The Swiss pharma is looking to leverage Voyager Therapeutics’ capsid technology for gene therapies aimed at treating Huntington’s disease and spinal muscular atrophy.
The deal with PTC Therapeutics increases Royalty Pharma’s stake to 13% of total royalties garnered by Evrysdi, the blockbuster drug for spinal muscular atrophy licensed and marketed by Roche.
New data from Scholar Rock’s Phase II TOPAZ trial showed its investigational antibody apitegromab improves quality of life in types 2 and 3 spinal muscular atrophy patients.
Novartis’ gene therapy Zolgensma is again under a cloud of doubt after Nature Biotechnology retracted its 2010 article noting “issues” regarding data cited in a key figure in the report.
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