Galapagos NV has received Investigational New Drug (IND) clearance from the FDA to expand its ATALANTA-1 phase 1/2 clinical trial (NCT06561425) to the United States. The trial, which is already underway in Europe, is designed to evaluate the safety and efficacy of Galapagos' novel treatment for hemophilia A.
The decision to bring the ATALANTA-1 trial to the US follows promising initial findings. According to CGTLive, among more than 100 patients with hemophilia A, no FVIII-specific responses showed associations with the assessed safety or efficacy parameters.
ATALANTA-1 Trial Design
The ATALANTA-1 trial is a phase 1/2 study designed to assess the safety, tolerability, and preliminary efficacy of Galapagos' investigational therapy in patients with hemophilia A. The trial includes multiple sites across Europe and will now expand to include sites in the United States. Specific details regarding dosing regimens, administration routes, and primary/secondary endpoints were not disclosed in the provided source material.
Hemophilia A Treatment Landscape
Hemophilia A is a genetic bleeding disorder caused by a deficiency in factor VIII (FVIII), a protein essential for blood clotting. Current treatments for hemophilia A include replacement therapy with FVIII concentrates and bypassing agents. Gene therapy represents a potentially curative approach by delivering a functional copy of the FVIII gene to patients. Mark Walters, MD, a professor at the University of California, has discussed gene therapy’s ongoing transformation of the treatment landscape for such diseases.
Other Gene Therapy Advancements
In related news, the NMPA in China made a decision with reference to results from the phase 2 CARTIFAN-1 clinical trial (NCT03758417), which took place at multiple sites in China, highlighting the global progress in cell and gene therapies.
