Repligen Corporation announced that the US 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 I 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 programme 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 US Orphan Drug Designation for RG3039, providing important regulatory and marketing incentives for the programme.

The work led by Families of SMA was the very first drug development programme ever done for SMA.

SMA is an inherited neurodegenerative disease in which a defect in the SMN1 (“Survival Motor Neuron”) gene results in low levels of the protein SMN and leads to progressive damage to motor neurons, loss of muscle function and, in many patients, early death. Patients lacking a functional SMN1 gene survive only because humans carry a second gene called SMN2 which produces low levels of SMN protein. RG3039, an orally bioavailable compound, is an inhibitor of an RNA processing enzyme which targets SMN2 and has been shown to increase production of SMN protein in cells derived from patients. In addition, RG3039 has been shown to improve mobility and lifespan in preclinical animal models of SMA. RG3039 is a new chemical entity, which is the subject of worldwide composition of matter patent applications which, if allowed, will remain in force until 2028 prior to any patent term extensions. 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.

Families of SMA is a nonprofit 501(c)3 tax exempt organization with 30 Chapters throughout the United States and over 70,000 members and supporters. Families of SMA funds and directs the leading SMA research programs. The successful results and progress from basic research to drug discovery programs to clinical trials provide real hope for families and patients. Families of SMA is dedicated to creating a treatment and cure by: funding and advancing a comprehensive research program; supporting SMA families through networking, information and services; improving care for all SMA patients; educating healthcare professionals and the public about SMA; enlisting government support for SMA; embracing all touched by SMA in a caring community. FSMA’s vision is a world where Spinal Muscular Atrophy is treatable and curable.

The Muscular Dystrophy Association (MDA) is the leading nonprofit health agency dedicated to curing muscular dystrophy, ALS, SMA and related diseases by funding worldwide research.

Repligen Corporation is a biopharmaceutical company focused on building an integrated company by developing and marketing innovative drugs that deliver the benefits of protein therapies in the fields of neurology and gastroenterology.