FDA Removes Warnings on Menopause Hormone Therapy
Summary:
The FDA is dropping long-standing “boxed” warnings on hormone replacement therapies, reversing decades of cautions about cancer and heart disease. The move follows a review of scientific evidence and expert input, emphasizing HRT’s benefits for menopausal symptoms, bone health, and possibly cognitive health.
Lilly Partners with MeiraGTx on Gene Therapy for Rare Blindness
Summary:
Eli Lilly has secured exclusive rights to AAV-AIPL4, an experimental gene therapy that restored vision in early trials for children born with Leber congenital amaurosis-4. The deal includes $75 million upfront, potential milestones of $400 million, and access to MeiraGTx’s gene therapy tools, as Lilly expands into ophthalmology.
Pfizer Wins $10B Bidding War for Obesity Drug Developer Metsera
Summary:
Pfizer will acquire Metsera for over $10 billion, topping Novo Nordisk’s bid and ending a high-profile battle for the obesity drug developer. The deal includes $65.60 per share upfront, with up to $20.65 per share in milestone-based payouts, and has the full backing of Metsera’s board. The acquisition positions Pfizer strongly in the competitive obesity drug market, giving it access to next-generation therapies that may require less frequent dosing than current treatments.
Neurocrine’s Experimental Depression Drug Fails Mid-Stage Trial
Summary:
Neurocrine Biosciences’ experimental depression drug NBI-’770 failed a mid-stage trial, showing no significant improvement over placebo in adults with major depressive disorder. Despite the setback, expectations for the drug were low, and the company plans to continue analyzing the data to determine next steps. Neurocrine’s other pipeline assets, including the more advanced osavampator, remain the focus for major depression treatment development.
FDA Proposes New Plausible Mechanism Pathway to Accelerate Custom Gene Editing Therapies
Summary:
The FDA has introduced a new “plausible mechanism pathway” aimed at speeding approvals for individualized gene-editing therapies by focusing on known biological mechanisms rather than large clinical trials. Inspired by the successful bespoke CRISPR treatment for “baby KJ,” the pathway would allow developers to seek approval based on demonstrated target engagement, biological plausibility, and early clinical improvement, particularly for rare diseases with clear molecular causes. While the framework could significantly expand access to personalized therapies, experts say detailed FDA guidance is still needed to clarify how broadly it will apply beyond ultra-rare conditions.
