Spinal muscular atrophy
President Donald Trump continues to warn of tariffs on the pharmaceutical industry; Susan Monarez replaces Dave Weldon as CDC director nominee; Novo Nordisk joins the triple-G race; Alnylam wins approval for Amvuttra in ATTR-CM; and Cassava Sciences ends development of simufilam in Alzheimer’s.
Since 2016, the FDA has approved three disease-modifying treatments for spinal muscular atrophy, with several companies—including Novartis, Scholar Rock and Biogen—progressing novel candidates through clinical trials.
This is the third indication for Fabhalta after Novartis won FDA approval of the small molecule in paroxysmal nocturnal hemoglobinuria and primary immunoglobulin A nephropathy.
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.
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