In a week of significant developments for the biopharma industry, an FDA advisory committee issued pivotal recommendations on two high-profile blood disorder drugs, while PureTech Health announced a leadership change at its helm.
On July 26, 2024, the U.S. Food and Drug Administration’s advisory committee gathered to evaluate the risks and benefits of two therapies: Bristol Myers Squibb and Merck’s Reblozyl, and GSK’s Blenrep, according to BioPharma Dive. Both drugs are being considered for expanded uses in treating challenging blood cancers and disorders.
Reblozyl, already approved for certain anemia patients, underwent review for its use in treating anemia caused by myelodysplastic syndromes (MDS) and beta thalassemia. The panel overwhelmingly supported Reblozyl’s use in these broader patient groups, citing strong evidence for its efficacy in raising hemoglobin levels and reducing the need for blood transfusions. The committee concluded that the benefits of Reblozyl outweigh its risks, which include blood clots and other side effects, especially when weighed against the limited treatment alternatives for these patients.
In contrast, GSK’s Blenrep, a therapy for multiple myeloma, faced a more skeptical reception. The panel scrutinized data from a recent study that compared Blenrep to standard treatments. While Blenrep showed some promise, concerns lingered about its overall survival benefit and the risk of significant side effects, particularly eye toxicity. The committee voted against recommending Blenrep for wider use, reflecting a cautious stance about its safety and efficacy based on the current evidence.
These recommendations do not constitute final FDA decisions, but they heavily influence the agency’s approval process. For Bristol Myers Squibb and Merck, the positive outcome for Reblozyl could pave the way for increased adoption and an expanded patient base. For GSK, the setback on Blenrep means the company may need to conduct additional studies or refine its patient selection criteria to address the panel’s concerns.
In an unrelated but notable leadership shift, PureTech Health—an innovative biotech focused on developing medicines for brain and immune system disorders—announced that CEO Daphne Zohar will step down. Zohar, who co-founded the company and led it for over 15 years, will be succeeded by Dennis Ausiello, currently PureTech’s board chair and a prominent physician-scientist. The company credited Zohar with spearheading its growth from a startup to a publicly traded biotech with multiple assets in clinical development. The leadership change comes as PureTech seeks to advance its pipeline and forge new partnerships.
