California Stem Cell, Inc. (CSC) and Families of Spinal Muscular Atrophy
(FSMA) announced today 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
“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
“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
California Stem Cell, Inc.