Spinal muscular atrophy
The regulatory action marks the second rejection for a spinal muscular atrophy therapy this week after Scholar Rock’s apitegromab was issued a complete response letter on Tuesday, similarly on manufacturing grounds.
In its rejection letter, the FDA flagged problems at a third-party fill-finish site owned by Novo Nordisk. Issues at this site have previously been investigated by the regulator.
After a season of regulatory upheaval, obesity and rare genetic diseases will likely remain major themes for biopharma in 2025, according to Jefferies.
In combination with Eli Lilly’s tirzepatide, marketed as Zepbound for obesity, Scholar Rock’s monolonal antibody helped patients lose the same amount of weight as patients on tirzepatide alone while preserving more muscle mass.
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.
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