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Neuromuscular Drug Gets Fast-Track, Orphan Approval

Drug vials (Hey Paul/Flickr)Repligen Corp. in Waltham, Massachusetts says its drug candidate for spinal muscular atrophy received fast-track and orphan drug status from U.S. and European regulators. The U.S. Food and Drug Administration (FDA) granted fast-track status to Repligen’s RG3039, while the European Medicines Agency gave the drug its orphan medicinal product designation.

Spinal muscular atrophy (SMA) is an inherited disease that affects mainly infants, with about four out of every 100,000 people having the condition. Infants with SMA type I, the most severe form of the disease, are born with very little muscle tone, weak muscles, and feeding and breathing problems. Often, weakness is first noted in the shoulder muscles and proximal leg muscles. Weakness gets worse over time and eventually becomes severe.

SMA occurs when a defect in the survival motor neuron (SMN1) gene results in low levels of SMN protein. Patients lacking a functional SMN1 gene survive only because humans carry a  second gene called SMN2 which produces low levels of SMN protein.

RG3039, taken orally, inhibits an RNA processing enzyme that targets SMN2 and has been shown to increase production of SMN protein in cells derived from patients. RG3039 has also been shown to improve mobility and lifespan in preclinical animal models of SMA.

With FDA’s fast-track designation, early and frequent communication between FDA and a drug company is encouraged throughout the entire drug development and review process.  The frequency of communication encourages questions and issues to be resolved quickly, which can lead to earlier drug approval. 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.

Repligen has received approval from the FDA to initiate a phase 1 clinical trial of RG3039, a double-blind study to test the body’s reaction to the drug and safety of escalating doses of RG3039 in up to 40 healthy volunteers. Preclinical research was funded by the organization Families of Spinal Muscular Atrophy, which licensed its technology to Repligen in 2009.

Read more: Blood Pressure Drug Helps Regenerate Muscle, Prevent Atrophy

Photo: Hey Paul/Flickr

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