Repligen Corporation (NASDAQ: RGEN) announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for RG3039, a potential treatment for Spinal Muscular Atrophy (SMA). Fast Track is a process designed to facilitate the development and expedite the review of drugs that treat serious diseases and fill an unmet medical need. Once a drug receives Fast Track designation, frequent communication between the FDA and the sponsor is encouraged throughout the development and review process. In addition, RG3039 has received a positive opinion for orphan medicinal product designation from the European Medicines Agency. European orphan medicinal product designation aims to encourage the development of drugs involved in the diagnosis, prevention or treatment of a life-threatening or chronically debilitating condition that affects no more than five in 10,000 persons in the European Union.
"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 first clinical trial of a novel drug specifically designed to treat SMA and the first treatment approach which seeks to increase levels of the deficient protein SMN. This is a double-blind study to evaluate the pharmacokinetic and safety profile of escalating doses of RG3039 in up to 40 healthy volunteers. Repligen's ongoing research efforts are funded in part with grants from the Muscular Dystrophy Association.
This program was licensed in 2009 from Families of Spinal Muscular Atrophy. Families of SMA fully funded and directed the preclinical development work with an investment of more than $13 million prior to licensing RG3039 to Repligen. Families of SMA previously secured U.S. Orphan Drug Designation for RG3039, providing important regulatory and marketing incentives for the program. The work led by Families of SMA was the very first drug development program ever done for SMA.