Stem Cell Based Treatment for Spinal Muscular Atrophy Receives Orphan Drug Designation
News Dec 09, 2009
California Stem Cell, Inc. (CSC) and Families of Spinal Muscular Atrophy (FSMA) announced that the FDA has granted orphan drug designation to MOTORGRAFT™, a stem cell-derived motor neuron product, for the treatment of Spinal Muscular Atrophy (SMA).
Orphan drug designation, granted by the FDA Office of Orphan Products Development, provides several incentives to companies in the private sector developing novel drugs or biologics to treat diseases with relatively small market potential. These include seven years market exclusivity following FDA approval, clinical trial design assistance, reduced user fees and tax credits.
CSC developed MOTORGRAFT™ as a stem cell-derived motor neuron replacement product for the treatment of SMA. Pre-clinical GLP safety and efficacy studies, funded significantly by the Families of Spinal Muscular Atrophy and conducted by Professor Hans S. Keirstead of the University of California at Irvine, have demonstrated safety and functional benefit in several animal models.
SMA, the leading genetic killer of infants, causes the deterioration of the muscles that control crawling, walking, swallowing and breathing and affects approximately 1 in 6000 babies born. In the US approximately 7.5 million people (1 in 40) are carriers of the defective gene. There are no currently approved therapies for the treatment of SMA.
CSC recently completed a formal pre-IND meeting with the FDA to discuss the clinical and regulatory pathway for submission of an application to initiate human trials using this therapy for the treatment of SMA Type I. The company expects to file an IND to begin a Phase I safety study in 2010.
“We are extremely encouraged to receive orphan-drug designation for MOTORGRAFT™,” said Chris Airriess, CSC’s Chief Operating Officer, “this is a major milestone in realizing our commitment to find a treatment for SMA.”
“Families of SMA is pleased to see this promising therapy advancing towards clinical trials,” said Jill Jarecki, Research Director for FSMA, “Orphan Designation will provide CSC with all the opportunities afforded by Orphan Drug Act of 1983, which is intended to facilitate a close working relationship between regulatory agencies and companies with the aim of accelerating the drug development and approval processes for rare diseases.”