FDA
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.
FEATURED STORIES
Drug developers increasingly have the option of seeking regulatory approval and commercializing a medicine themselves, rather than accepting an early exit offer. Doing so requires a worldwide strategy.
Disc Medicine’s bitopertin—rejected by the FDA in February—significantly reduced levels of a key disease biomarker and significantly increased the time patients with erythropoietic protoporphyria were able to spend in the sun and artificial light.
Leveraging recently proposed regulatory flexibilities, the gene-editing sector is working to cut R&D costs and unlock indications that were previously economically unviable, according to a new playbook published by the Alliance for Regenerative Medicine.
Subscribe to ClinicaSpace
Clinical trial results, research news, the latest in cancer and cell and gene therapy, in your inbox every Monday
THE LATEST
The FDA has outlined a process for allowing a single application to cover multiple variants of a gene therapy. At the same time, the Centers for Medicare & Medicaid Services has revealed plans to expand the Cell and Gene Therapy Access Model to increase patient access to these products.
After cardiac safety signals led to a full clinical hold on Aardvark Therapeutics’ studies of ARD-101, the biotech conducted an additional safety study, in which no cardiac events were detected. Aardvark plans to resume testing the candidate next year.
Troriluzole, in development for spinocerebellar ataxia, was rejected nearly a year ago when the FDA took issue with the design of Biohaven’s registrational study. The agency has now agreed to review a new application and render a decision in the first quarter of 2027.
The approval of Hibsago has created a new market for a handful of biopharmas, including Gilead, Arrowhead, and Vir, to fill in.
The recurrence of violations at multiple sites led the FDA to conclude that Baxter management must improve its oversight and control of manufacturing.
Hibsago’s approval—a functional cure for certain patients with a hepatitis B infection—further validates Ionis Pharmaceuticals’ antisense platform, which has already generated two other new drug approvals this year.
Inconsistent reporting at a Pennsylvania site affected microbiological analyses of components and drug products that analytical testing service provider Eurofins ran for customers.
Following several recent initiatives, the FDA’s newest rare disease framework sets out to standardize the agency’s approach to scientific evidence and bring rare disease therapies to patients faster.
After one patient death prompted an FDA hold this spring, Newron Pharmaceuticals has now shared that a total of four fatalities have been recorded among participants receiving its schizophrenia candidate. A fifth death was recorded among those taking the placebo.
Despite the regulatory delay, Novo is still expecting a 2027 launch for its hemophilia A drug denecimig.