The majority of the large pharmas have now reported third quarter earnings—spoiler alert: Eli Lilly made more money than anyone expected. Plus, the FDA dropped another initiative aimed at accelerating rare disease treatments; HHS greenlights the compounding of six unapproved peptides; and uniQure’s embattled gene therapy for Huntington’s disease has finally been accepted for review.
Big Pharma earnings continued over the past week, with no fewer than a dozen companies reporting third quarter results, including AstraZeneca, Biogen, GSK, Bristol Myers Squibb, Merck, Sanofi, AbbVie and Regeneron.
Pfizer reported flat revenue for the quarter but still beat expectations, pleasing investors. Eli Lilly, meanwhile, made more money with its tirzepatide franchise than Pfizer did across its entire portfolio. Lilly’s sales were so good, in fact, that analysts questioned why the pharma wasn’t better at predicting its own growth.
On the other hand, Alnylam suffered through a tough earnings call, even as the company reported a 59% revenue increase for its transthyretin franchise. CEO Yvonne Greenstreet admitted that the franchise has not been performing as Alnylam had hoped.
On the regulatory front, the FDA issued new draft guidance for the regulation of gene-edited therapies. The “plug and play” strategy would allow one regulatory application to cover gene-editing therapies addressing various disease-causing mutations in a single gene. Analysts flagged Beam Therapeutics and Prime Medicine as two potential beneficiaries of this approach.
Another bit of regulatory news this week was not so well received by industry. A new interim policy from the Department of Health and Human Services (HHS) essentially allows compounding pharmacies to produce six unapproved peptides. As Robert Popovian, a pharmacist and chief science policy officer at the Global Healthy Living Foundation, wrote in an opinion piece for BioSpace, the policy could not only put Americans in harm’s way but also threaten the overall industry by weakening incentives for rigorous drug development.
And uniQure has finally had an application for its embattled gene therapy for Huntington’s disease accepted for review by the FDA, with an action date of May 1, 2027. May Day is a European festival that marks the beginning of summer. Will it also be the beginning of a disease-modifying treatment for Huntington’s?
