The US Food and Drug Administration (FDA) maintained its position as the leading drug regulatory authority in 2024, approving 50 novel medicines containing new active substances, narrowly surpassing the European Medicines Agency's (EMA) 46 approvals. This represents a slight decrease from the FDA's 55 approvals in 2023, while the EMA showed improvement from its previous year's count of 39 authorizations.
Focus on Rare Diseases and Therapeutic Areas
The FDA's commitment to addressing rare diseases was evident, with more than half of its novel approvals targeting orphan conditions affecting fewer than 200,000 Americans. The agency also maintained its global leadership in expediting access to new treatments, with 68% of these novel drugs receiving first approval in the United States.
For the EMA, hematology and hemostaseology emerged as significant focus areas, with ten out of twelve approved medicines in this category containing new active ingredients. Oncology continued to dominate overall approvals for both agencies, maintaining a trend observed in previous years.
Notable Joint Approvals
Several groundbreaking treatments received authorization from both regulatory bodies in 2024. Pfizer's Hympavzi (marstacimab-hncq) marked a significant advancement in hemophilia treatment, becoming the first approved anti-tissue factor pathway inhibitor for adults and adolescents with severe hemophilia A or B without inhibitors.
In hepatology, both agencies greenlit two important treatments for primary biliary cholangitis: Ipsen's Iqirvo (elafibranor) and Gilead's Livdelzi (seladelpar). These approvals expanded the therapeutic options for patients with this chronic liver condition.
A notable innovation in anaphylaxis treatment came with ARS Pharmaceuticals' needle-free adrenaline nasal spray, marketed as Neffy in the US and Eurneffy in Europe. This breakthrough provides a more convenient alternative to traditional auto-injectors like EpiPen.
Divergent Approvals in Neurology
Significant differences between the agencies emerged in neurological treatments. The FDA approved Eli Lilly's Kisunla (donanemab) for Alzheimer's disease in July 2024, while the EMA approved Eisai and Biogen's Leqembi (lecanemab) later in the year, more than twelve months after its US authorization.
A historic approval came in September 2024 when the FDA authorized Bristol Myers Squibb's Cobenfy (xanomeline and trospium chloride), the first new schizophrenia drug in over three decades. While not yet submitted for EMA review, the treatment is expected to achieve blockbuster status by 2028.
EMA-Exclusive Approvals
The EMA independently approved several important medications in 2024, including Egetis Therapeutics' Emcitate (tiratricol) for Allan-Herndon-Dudley syndrome and Pfizer's Emblaveo (aztreonam-avibactam) for antibiotic-resistant bacterial infections, demonstrating the agency's distinct approach to addressing unmet medical needs in the European market.
