FDA Gears Up for Busy August with Key Decisions on Novel Therapies

The U.S. Food and Drug Administration (FDA) is poised for a bustling August, with at least eight target action dates lined up for potentially groundbreaking therapies. These decisions span a wide range of indications, from rare genetic disorders to chronic lung conditions, and could usher in new treatment paradigms for patients with limited options.

Insmed's Brensocatib: A Potential First for Bronchiectasis

New Jersey-based Insmed is awaiting the FDA's verdict on brensocatib, an oral DPP1 inhibitor for the treatment of bronchiectasis, due by August 12. If approved, brensocatib would become the first drug specifically indicated for this chronic lung disease and introduce DPP1 inhibition as a novel therapeutic mechanism.

The application is supported by the Phase III ASPEN study, which demonstrated that 10 mg of brensocatib reduced the annualized pulmonary exacerbation rate by 21.1% compared to placebo. The drug also showed improvements in lung function and other secondary outcomes over 52 weeks.

Pioneering Treatments for Rare Diseases

Several companies are vying for approvals in rare disease indications this month. Tonix Pharmaceuticals' TNX-102 SL, a non-opioid therapy for fibromyalgia, could receive approval by August 15. If successful, it would be the first new treatment for this condition in over 15 years.

PTC Therapeutics is expecting a decision on vatiquinone for Friedreich's ataxia by August 19, despite mixed results from its Phase III MOVE-FA trial. The company is leveraging additional long-term studies to support its application.

Ionis Pharmaceuticals' donidalorsen, an RNA-targeting therapy for hereditary angioedema (HAE) prevention, faces an August 21 deadline. Phase III data showed an 81% reduction in HAE attacks compared to placebo over 25 weeks.

Potential Breakthroughs in Ophthalmology and Respiratory Care

Outlook Therapeutics is anticipating an August 27 decision for ONS-5010, an eye drop formulation of bevacizumab for wet age-related macular degeneration. The company aims to market the product as Lytenava if approved.

On the same day, Precigen expects a verdict on PRGN-2012, a gene therapy for recurrent respiratory papillomatosis (RRP). If successful, it would be the first approved treatment for this rare and potentially fatal neoplastic lung disease.

Sanofi and Biogen/Eisai Round Out the Month

Sanofi's rilzabrutinib, a BTK inhibitor for immune thrombocytopenia, has an August 29 target date. Phase III results showed promising platelet responses and reduced need for rescue therapy.

Capping off the month, Biogen and Eisai await an August 31 decision on their subcutaneous maintenance formulation of Leqembi for Alzheimer's disease. This new delivery method could potentially reduce treatment burden for patients and healthcare providers.

As the pharmaceutical industry holds its breath for these crucial decisions, August promises to be a pivotal month for innovative therapies and the patients who may benefit from them.