PTC Therapeutics, Inc. today announced that the US Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to RG7916 for the treatment of patients with Spinal Muscular Atrophy (SMA).
RG7916 is part of PTC's joint development program in SMA with Roche and the SMA Foundation (SMAF). SMA is a rare genetic disorder that results in neuromuscular disability beginning in infancy and is the leading inherited cause of mortality in infants and young children.
RG7916 is an oral small molecule splicing modifier that directly targets the underlying molecular deficiency of SMA by modulating SMN2 splicing to increase expression of stable full-length SMN protein from the SMN2 gene.
RG7916 is currently under investigation in two clinical studies: SUNFISH, a trial in childhood onset (Type II/III) SMA patients, and FIREFISH, a trial in infant onset (Type I) patients.
"We are dedicated to the development of innovative treatments for patients, particularly in genetic disorders such as SMA," said Stuart W. Peltz, Ph.D., Chief Executive Officer of PTC Therapeutics.
"This orphan drug designation further supports the need for additional treatments for this devastating disease. As an oral small molecule with broad exposure, RG7916 has the potential to impact every aspect of this disease and provide hope to many patients."
In the US, orphan drug designation is granted by the FDA's Office of Orphan Products Development to promote the development of products that may offer therapeutic benefits for diseases with a prevalence of fewer than 200,000 individuals per year.
Orphan drug designation provides opportunities for grant funding towards clinical trial costs, tax advantages, FDA user-fee benefits, and seven years of market exclusivity in the United States, if granted FDA approval.
The SMA program was initially developed by PTC Therapeutics in partnership with the SMA Foundation in 2006 to accelerate the development of a treatment for SMA.
In November 2011, Roche gained an exclusive worldwide license to the PTC/SMA Foundation SMN2 alternative splicing program. The development of these compounds is being executed by Roche and overseen by a joint steering committee with members from PTC, Roche, and the SMA Foundation.