FDA Q4 2025 Decisions: Potential Breakthroughs in Rare Diseases and Oncology

As 2025 enters its final quarter, the pharmaceutical industry anticipates a series of pivotal FDA decisions that could reshape treatment landscapes across multiple therapeutic areas. From rare genetic disorders to oncology, these rulings have the potential to introduce novel therapies and expand indications for existing treatments.

Rare Disease Innovations on the Horizon

Two notable FDA decisions in Q4 focus on rare diseases, potentially offering new hope for patients with limited treatment options. Arrowhead Pharmaceuticals' plozasiran, an RNA interference therapeutic for familial chylomicronemia syndrome (FCS), awaits a verdict by November 18. FCS, affecting only 1 to 2 people per million, leads to dangerously high triglyceride levels and various complications. Plozasiran demonstrated an impressive 80% reduction in triglyceride levels in its Phase III PALISADE trial.

Ascendis Pharma's TransCon CNP (navepegritide) for achondroplasia in children faces a decision by November 30. This rare genetic disorder, characterized by dwarfism and other complications, affects approximately 250,000 people worldwide. The Phase II ACcomplisH study showed significant improvements in annualized growth velocity and other key benefits for patients treated with TransCon CNP.

Oncology Advancements and Comebacks

In the oncology space, Bristol Myers Squibb's CAR T therapy Breyanzi could become the first of its class approved for marginal zone lymphoma (MZL) by December 5. The Phase II TRANSCEND FL study reported a 95.5% overall response rate in MZL patients, with 62.1% achieving a complete response.

GSK's Blenrep seeks a comeback as a second-line treatment for relapsed/refractory multiple myeloma, with a decision expected by October 23. Despite strong efficacy data from the DREAMM-7 and DREAMM-8 trials, concerns about ocular toxicity led an FDA advisory committee to vote 7-1 against approval in July. The extended review period concludes this month, determining whether Blenrep will return to the market.

Cardiovascular and Ophthalmology Updates

Cytokinetics' aficamten for obstructive hypertrophic cardiomyopathy (oHCM) faces an FDA decision by December 26. The Phase III MAPLE-HCM study demonstrated significant improvements in exercise capacity compared to the standard-of-care metoprolol. If approved, aficamten will compete with BMS's Camzyos in the oHCM market.

Regeneron awaits two verdicts for high-dose Eylea in the fourth quarter, including an expansion into macular edema after retinal vein occlusion and a monthly dosing regimen across all approved indications. The review was extended due to manufacturing issues at a third-party facility, but Regeneron remains optimistic about a favorable outcome.

These upcoming FDA decisions represent potential paradigm shifts in multiple therapeutic areas, offering new treatment options for patients and shaping the competitive landscape in the pharmaceutical industry as we approach 2026.