The pharmaceutical industry is gearing up for an eventful end to 2025, with the FDA set to make crucial approval decisions on groundbreaking drugs across various indications. Some of the biggest players in pharma are eagerly awaiting upcoming PDUFA dates that could open the doors to new formulations of their flagship products.
Merck & Co. is anticipating a verdict on the subcutaneous version of its top-selling oncology drug Keytruda by Sept. 23, with plans to launch the new formulation by Oct. 1. Amidst looming generic competition, the introduction of a subcutaneous form could extend Keytruda’s market dominance while offering a more convenient administration method for patients and healthcare providers.
Novo Nordisk is also on the cusp of a game-changing decision as the FDA evaluates its application for an oral version of the GLP-1 semaglutide. After achieving significant success with semaglutide drugs like Wegovy and Ozempic, Novo Nordisk is now eyeing the oral route as the next frontier in diabetes and obesity treatment. The FDA is expected to announce its decision in the fourth quarter.
In addition to these established players, there are several new drugs on the horizon that could revolutionize the treatment landscape. Let’s take a closer look at some breakthrough therapies that are awaiting FDA approval.
Stealth Biotherapeutics’ Barth Syndrome Treatment
On Sept. 26, Stealth Biotherapeutics is anxiously awaiting the FDA’s decision on elamipretide, a first-of-its-kind mitochondrial targeted therapy for Barth syndrome. With a tiny patient population of about 150 individuals in the U.S., Barth syndrome can lead to muscle weakness, heart failure, and infections, often proving fatal. Stealth is seeking accelerated approval based on an intermediate endpoint of extensor muscle strength, with the potential to become the first approved treatment for this rare disease.
Biohaven’s Troriluzole for Spinocerebellar Ataxia
Biohaven’s troriluzole, originally intended for OCD, is now being repurposed for spinocerebellar ataxia (SCA), a rare and life-threatening disease with no approved treatments. Clinical trials have shown promising results in slowing disease progression by 50% to 70%, positioning troriluzole as a potential game-changer in the SCA space. The FDA is set to make a final decision in the fourth quarter, with analysts predicting a 40% chance of success.
Fortress Biotech and Sentynl Therapeutics’ Menkes Disease Treatment
Menkes disease, a fatal condition affecting up to 225 babies in the U.S. annually, could see a glimmer of hope with CUTX-101, a groundbreaking treatment developed by Fortress Biotech and Sentynl Therapeutics. Clinical trials have demonstrated a nearly 80% reduction in the risk of death, offering a ray of hope for patients and their families. The FDA is expected to announce its decision by Sept. 30, potentially ushering in a new era of treatment for this devastating disease.
As the pharmaceutical landscape continues to evolve, these upcoming FDA approvals have the potential to reshape the treatment paradigm and improve outcomes for patients facing challenging medical conditions. Stay tuned for the latest updates on these groundbreaking therapies as they navigate the regulatory pathway towards approval.
