The U.S. Food and Drug Administration is set to deliver several significant decisions on new drugs in the second quarter, with a particularly busy schedule in April, according to a March 30 report from BioSpace.
These upcoming FDA verdicts could affect treatments for rare diseases, obesity, melanoma, Alzheimer’s disease agitation, and hypertension. The agency's rulings are closely watched by patients and industry stakeholders because they can impact treatment options and shape the competitive landscape for major pharmaceutical companies.
Among the most anticipated decisions is for Biogen’s high-dose Spinraza formulation for spinal muscular atrophy (SMA). After a previous rejection due to manufacturing issues rather than data concerns, Biogen resubmitted its application supported by updated Phase 2/3 study results showing improved motor skills compared to control groups. Shortly after publication of this story, Biogen announced that the FDA approved high-dose Spinraza. BMO Capital Markets said in a note that this approval “could reinvigorate Biogen’s SMA franchise.”
Eli Lilly is also awaiting an FDA decision on orforglipron, an oral weight loss pill intended to compete with Novo Nordisk’s oral Wegovy. Orforglipron has shown greater weight loss and glycemic control than oral semaglutide in recent studies. Lilly has prepared for rapid launch if approved and has stockpiled $1.5 billion worth of orforglipron while increasing production capacity since February 2024.
Replimune seeks approval for RP1, an oncolytic immunotherapy for advanced melanoma after previously being rejected due to study design concerns. The company resubmitted its data package with additional analyses; leadership changes at the FDA may influence this decision.
Other notable pending approvals include Axsome Therapeutics’ AXS-05 for agitation associated with Alzheimer’s disease—a condition affecting around seven million Americans—and AstraZeneca’s baxdrostat as a potential first-in-class aldosterone synthase inhibitor targeting resistant hypertension.
Biogen and Eisai are also seeking expanded use of their subcutaneous Leqembi Iqlik injection for Alzheimer’s therapy initiation following earlier maintenance approval. If cleared by regulators, it would allow home administration during both induction and maintenance phases.
These regulatory outcomes have implications not only for patient care but also signal trends in how novel therapies are evaluated amid evolving agency leadership.
