Neuralstem announced that the US FDA has granted it Orphan Drug Designation for the treatment of Amyotrophic Lateral Sclerosis (ALS) with its human spinal cord derived neural stem cells (NSI-566RSC), currently in phase I study to evaluate the safety of the product and the surgical route of administration in a wide range of ALS patients.
“Congress passed the Orphan Drug Act because it recognised that adequate drugs for many rare diseases have not been developed,” said Richard Garr, president and CEO of Neuralstem. “The designation of our spinal cord stem cells as an orphan drug underscores the importance of developing effective treatments for patients with ALS.”
“In addition to providing a seven-year term of market exclusivity for our stem cells for ALS upon FDA approval, Orphan Drug Designation also positions Neuralstem to take advantage of certain financial and regulatory benefits, including government grants for conducting clinical trials, waiver of FDA user fees for the submission of a biologics license application for NSI-566RSC, and certain tax credits. It is an important step forward for the company,” Garr noted.
About the Orphan Drug Designation programme
According to the FDA, the Orphan Drug Designation programme is managed by FDA’s Office of Orphan Products Development (OOPD) and provides orphan status to drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the USA or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment drug.