Repligen announced yesterday that it has been granted Fast Track and orphan drug status by the US FDA and the European Medicines Agency respectively for its drug RG3039, a potential treatment for the rare disease Spinal Muscular Atrophy.
Fast Track status in the U.S. means the FDA will make faster decisions about Repligen’s marketing application. Orphan drug status in the EU comes with up to 10 years of marketing exclusivity if approved. Both incentives are used to encourage treatments for rare diseases.
“Receipt of Fast Track designation and a positive opinion for European orphan medicinal product designation for RG3039 demonstrates the FDA and EMA commitment to the study and development of treatments for rare and serious diseases,” stated Walter C. Herlihy, President and Chief Executive Officer of Repligen Corporation. “This regulatory support adds momentum to our efforts to develop a novel treatment for patients with Spinal Muscular Atrophy.”
Repligen has received approval from the FDA to initiate a Phase 1 clinical trial of RG3039.
The prevalence of SMA in the U.S. and Europe is approximately 20,000 patients and there is currently no treatment or cure for the disease.
For more information on orphan drugs check out the World Orphan Drug Congress USA.