After a year in which the U.S. Food and Drug Administration (FDA) approved 56 new therapeutics for cancer, neurological, rare diseases and other conditions, the agency is preparing for six significant regulatory decisions in the first quarter of 2026. Four of these are continuations from the previous quarter.
Jefferies analysts noted in an investor update that FDA approval and rejection rates remained within historical ranges. The Center for Drug Evaluation and Research (CDER) granted 46 approvals, slightly down from 49 in 2024, while the Center for Biologics Evaluation and Research (CBER) issued 10 approvals, consistent with prior years. In total, there were 19 rejections in 2025—15 from CDER and four from CBER—with Jefferies stating that CBER’s number was higher than usual.
Delays in regulatory deadlines have become more common. According to RBC Capital Markets’ report on global biotech trends for 2026, the FDA met its review deadlines about three-quarters of the time during the latter half of 2025.
One upcoming decision involves Travere Therapeutics’ Filspari as a treatment for focal segmental glomerulosclerosis (FSGS), a rare kidney disease affecting about seven people per million. After initially failing to show superiority over Sanofi’s Avapro in improving kidney function during a Phase III trial, Travere resubmitted its application with additional data showing Filspari reduced proteinuria compared to Avapro. If approved, it would be the first drug specifically indicated for FSGS in the United States.
REGENXBIO is awaiting an FDA verdict on RGX-121 gene therapy for Hunter syndrome after submitting further information that led to a delayed decision date now set for February 8, 2026. The therapy aims to deliver a functioning copy of a key gene into patients’ central nervous systems and has shown promising results: an 86% median decrease in disease biomarkers and most patients able to avoid enzyme replacement therapy at last assessment. “If approved, RGX-121 would be the first one-time therapy that directly addresses the underlying genetic cause of Hunter syndrome,” REGENXBIO said.
Ascendis Pharma also faces an extended review period for TransCon CNP (navepegritide), targeting achondroplasia—the most common form of dwarfism caused by mutations affecting bone growth pathways. Data published showed improved annualized growth velocity compared with placebo recipients. If cleared by regulators, TransCon CNP will compete with BioMarin’s Voxzogo, which became available in late 2021.
Rocket Pharmaceuticals expects an FDA decision by March on Kresladi as a treatment option for leukocyte adhesion deficiency-I (LAD-I), a rare immune disorder primarily affecting children globally. After addressing manufacturing concerns raised during its initial submission last year—and following several organizational setbacks—Rocket hopes this approval will mark progress after recent clinical holds and strategic withdrawals involving other pipeline products.
Rhythm Pharmaceuticals seeks expanded use of Imcivree to treat acquired hypothalamic obesity—a severe weight gain condition linked to brain injury—following positive Phase III data indicating significant reductions in body mass index over one year compared with placebo controls. Imcivree was previously approved for certain genetically confirmed obesity disorders; Rhythm now awaits word on broader indications.
Finally, Aldeyra Therapeutics’ reproxalap remains under review as a potential dry eye disease treatment after two prior rejections due to insufficient evidence of efficacy on key symptoms. The latest application includes new trial data showing improvement but not reaching statistical significance; thus, regulators extended their evaluation period by three months pending further analysis.
As these decisions approach throughout early 2026, they reflect ongoing challenges around clinical evidence requirements and timely regulatory reviews amid evolving standards across therapeutic areas.
