During the second week of January 2025, the U.S. Food and Drug Administration (FDA) took several significant regulatory actions, reflecting an active period in pharmaceutical advancements, particularly for treatments addressing chronic and underserved conditions.
On January 8, COUR Pharmaceuticals achieved a key regulatory breakthrough when its investigational therapy CNP-104 received Orphan Drug Designation for the treatment of primary biliary cholangitis (PBC). This autoimmune liver disease currently lacks curative therapies, and CNP-104’s positive results from its Phase 2a clinical trial have reinforced its potential. The Orphan Drug Designation offers COUR several benefits, including market exclusivity upon approval, tax credits, and waiver of certain FDA fees—critical incentives for developing treatments for rare diseases.
Astellas Pharma announced on January 9 that the FDA had accepted its supplemental New Drug Application (sNDA) for avacincaptad pegol, commercially known as IZERVAY. This anti-complement C5 inhibitor is being evaluated for geographic atrophy secondary to age-related macular degeneration (AMD), a leading cause of irreversible vision loss. The acceptance of the sNDA sets a Prescription Drug User Fee Act (PDUFA) action date for February 26, 2025. If approved, this would broaden the therapeutic scope of IZERVAY, which is already approved for other retinal conditions.
On January 10, Bayer submitted an sNDA for finerenone (KERENDIA) to expand its indication to include heart failure patients with a left ventricular ejection fraction (LVEF) greater than or equal to 40%. This subgroup represents those with either preserved or mildly reduced ejection fraction, a population for whom treatment options remain limited. Finerenone, already approved for chronic kidney disease associated with type 2 diabetes, has shown promise in reducing cardiovascular risks in this extended patient cohort.
Perhaps the most groundbreaking development came on January 15, when Eli Lilly received full FDA approval for mirikizumab, marketed as Omvoh, for the treatment of moderate-to-severe Crohn’s disease. This marks the first biologic therapy approved for Crohn’s disease in over 15 years that includes two-year data from Phase 3 clinical trials. Omvoh targets the p19 subunit of interleukin-23 (IL-23), offering a novel mechanism of action that could set a new standard for long-term disease management in inflammatory bowel disease.
Collectively, these FDA actions highlight a week of substantial regulatory momentum. From orphan drug designations to approvals in immunology and cardiology, the agency’s decisions reflect an ongoing commitment to addressing complex and high-burden medical conditions through innovative therapies.
