FDA Approvals and Rejections Shape Pharmaceutical Landscape in July

The U.S. Food and Drug Administration (FDA) made several significant decisions in July, issuing approvals for new drugs and expanded indications while also rejecting some high-profile applications. These rulings have far-reaching implications for patients, pharmaceutical companies, and the broader healthcare industry.

New Drug Approvals Offer Hope for Multiple Conditions

The FDA greenlit several new treatments this month, addressing various medical needs. Regeneron's Lynozyfic (linvoseltamab) received accelerated approval for relapsed or refractory multiple myeloma, demonstrating a 70% objective response rate in clinical trials. The bispecific antibody, targeting both BCMA and CD3 proteins, offers a convenient dosing schedule that could potentially set it apart from competitors.

KalVista's Ekterly (sebetralstat) became the first oral on-demand therapy for acute hereditary angioedema (HAE) attacks. Despite reported internal disagreements at the FDA, the drug's approval was supported by robust clinical data showing rapid symptom relief compared to placebo.

PTC Therapeutics' Sephience (sepiapterin) secured approval for phenylketonuria (PKU) in patients as young as one month old. The drug demonstrated significant reductions in phenylalanine levels, with nearly 85% of patients achieving levels below treatment guideline thresholds.

Label Expansions Broaden Treatment Options

Several established drugs received expanded indications, enhancing their utility in clinical practice. Eli Lilly's Alzheimer's therapy Kisunla now has an FDA-approved modified dosing regimen, which may reduce the risk of amyloid-related imaging abnormalities. Moderna's COVID-19 vaccine Spikevax gained full approval for high-risk children aged 6 months to 11 years, building on its previous emergency use authorization.

Bayer's Kerendia secured a new indication for reducing cardiovascular risks in certain heart failure patients, while GSK's shingles vaccine Shingrix received approval for a prefilled syringe formulation, simplifying administration for healthcare providers.

Setbacks for Cell and Gene Therapies

The month also saw notable rejections in the cell and gene therapy space. Capricor Therapeutics faced a significant setback when the FDA declined to approve deramiocel, a cell therapy for cardiomyopathy in Duchenne muscular dystrophy patients. The regulator cited insufficient evidence of effectiveness and manufacturing concerns, leading to a sharp decline in Capricor's stock price.

Ultragenyx encountered a similar fate with its gene therapy UX111 for Sanfilippo syndrome type A. While the FDA acknowledged the robustness of the neurodevelopmental outcome data, manufacturing issues not directly related to product quality led to the rejection. The company expects to address these concerns and resubmit its application in the coming months.

These decisions highlight the ongoing challenges in bringing advanced therapies to market, even as the FDA continues to evaluate and approve novel treatments across various therapeutic areas.