Motorgraft receives orphan drug designation

California Stem Cell (CSC) and Families of Spinal Muscular Atrophy 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.
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 is a privately held company developing proprietary therapeutic products to treat several neurodegenerative diseases, as well as innovative stem cell based human cell products to facilitate research and conventional drug discovery. Its lead therapeutic candidate, Motorgraft, is 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 Keirstead of the University of California at Irvine, have demonstrated safety and functional benefit in several animal models.
CSC recently completed (last October) 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".

California Stem Cell also announced that it has completed a Series B financing round with private investors. The financing will enable the company to support the advancement of its therapeutic products to human clinical trials, as well as the generation and distribution of its tools to aid in stem cell research and drug discovery.
“The ability to raise money in this difficult economic environment is a testament to the significant progress we’ve made the last year in advancing our clinical development programs and developing innovative new research tools. Backed by excellent science and a strong management team, CSC has the momentum, vision, technology and now capital to advance its cell-based therapies and bring its innovative products to the market”, stated Chris Airriess, CSC’s Chief Operating Officer.

(sources: CSC and FSMA websites)