Sarepta's Major DMD Approval, Alnylam's Clinical Success, and ADA Key Points

Last week saw significant developments in the pharmaceutical world, most notably the expanded approval of Sarepta's Duchenne muscular dystrophy gene therapy, Elevidys. The Director of the FDA's Center for Biologics Evaluation and Research (CBER), Peter Marks, made the controversial decision to fully approve Elevidys and expand its label despite the therapy missing the primary endpoint in a Phase III confirmatory study, contradicting the recommendations of FDA staff and review teams.

This major approval has drawn attention to other significant FDA decisions anticipated this year. One of the most closely watched is the potential approval of Eli Lilly’s anti-amyloid antibody, donanemab. If approved, donanemab would directly compete with Biogen and Eisai’s Leqembi, another anti-amyloid antibody that modifies the disease.

Looking ahead, Verona Pharma is expecting an FDA decision on ensifentrine by Wednesday. If approved, it would introduce a novel mechanism for treating chronic obstructive pulmonary disease (COPD), a first in over a decade. In August, the FDA is set to make a decision on Lykos' MDMA-assisted therapy for PTSD, despite an advisory committee voting against it earlier this month.

In a related development, Alnylam achieved a significant milestone with a Phase III success in treating transthyretin amyloidosis with cardiomyopathy (ATTR-CM). According to Alnylam’s Chief Medical Officer Pushkal Garg, the data from the trial were "overwhelmingly positive," showing that Amuvttra considerably reduced the risk of death and recurrent cardiovascular events in patients suffering from ATTR-CM.

Meanwhile, the biopharma conference season is in full swing, with the American Diabetes Association’s annual conference taking place this past weekend. Eli Lilly presented promising results for its blockbuster drug Zepbound, which demonstrated significant improvements in disease severity for patients with obesity and sleep apnea. Additionally, Altimmune reported a Phase II success with its drug pemvidutide. This treatment proved effective in aiding weight loss while preserving lean muscle mass in patients.

These developments mark significant strides in the pharmaceutical industry, with potential new therapies offering hope for conditions that have seen limited advancements in recent years. From gene therapies and anti-amyloid antibodies to novel COPD treatments and promising weight loss drugs, the upcoming FDA decisions could reshape the treatment landscape for multiple diseases.