Europe Poised to Reject Sarepta's Elevidys as FDA Scrutinizes SSRI Risks and Pharma Giants Navigate Regulatory Hurdles

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European Regulators Lean Against Approval of Sarepta's Duchenne Therapy

In a potentially significant setback for Sarepta Therapeutics, European medicines regulators are reportedly preparing to recommend against approval of Elevidys, the company's gene therapy for Duchenne muscular dystrophy. This development comes just two years after the U.S. Food and Drug Administration (FDA) controversially approved the treatment despite mixed clinical trial data.

The Scientific Divide

Sources familiar with the European Medicines Agency's (EMA) review suggest the committee found insufficient evidence that Elevidys meaningfully improves motor function in Duchenne patients. The therapy's conditional approval in the U.S. was based primarily on its ability to increase dystrophin protein levels - a biomarker whose clinical relevance remains debated among neuromuscular experts.

Commercial Implications

A European rejection would deal a blow to Sarepta's partnership with Roche, which paid $1.15 billion upfront for ex-U.S. commercial rights. Analysts estimate the European Duchenne market could represent 20-30% of the therapy's potential global sales.

FDA Convenes Expert Panel to Examine SSRI Safety Concerns

Meanwhile, the FDA has announced plans to convene its Psychopharmacologic Drugs Advisory Committee to review emerging data about potential neurological risks associated with selective serotonin reuptake inhibitors (SSRIs), the most commonly prescribed class of antidepressants.

Focus on Long-Term Effects

The September meeting will specifically examine post-marketing reports suggesting possible links between prolonged SSRI use and subtle cognitive changes or movement disorders. While SSRIs remain a cornerstone of depression treatment, some neurologists have raised concerns about their chronic effects on neuroplasticity.

Balancing Act for Regulators

"This isn't about questioning SSRIs' established benefits," explained Dr. Aaron Kesselheim, a Harvard medical professor and former FDA advisor. "It's about ensuring we fully understand the risk-benefit profile for patients who may take these medications for decades."

Other Notable Developments in Global Pharma

The pharmaceutical industry saw several other significant developments this week:

GSK's Blenrep Makes Surprising Comeback

GSK received unexpected positive data for its multiple myeloma drug Blenrep, which the company withdrew from the U.S. market in 2022 after failing a confirmatory trial. New analyses suggest particular efficacy in specific genetic subgroups, potentially paving the way for a targeted relaunch.

Alzheimer's Pipeline Progress

Eli Lilly provided updates on its Kisunla (donanemab) launch, reporting stronger-than-expected early demand for the Alzheimer's treatment. Meanwhile, Novartis and Bavarian Nordic announced a collaboration to develop vaccines that might prevent neurodegenerative conditions.

As the pharmaceutical industry navigates these complex regulatory and scientific challenges, stakeholders emphasize the need for transparent risk communication and evidence-based decision making at all levels of drug development and approval.

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