The U.S. Food and Drug Administration (FDA) granted orphan drug designation for Organovo’s treatment of alpha-1 antitrypsin deficiency (“A1AT”) with its 3D bioprinted liver therapeutic tissue.
“We are extremely pleased to receive orphan designation for our NovoTissues®treatment of A1AT,” said Taylor J. Crouch, CEO, Organovo. “The FDA’s rapid action recognizes the importance of developing regenerative medicine therapeutic applications, and mirrors our own urgency in addressing this devastating disease. With tens of thousands of patients being treated for inborn errors of metabolism (“IEMs”) in the U.S., and an annual cost per patient that exceeds $250,000 for drug therapy alone, these patient populations are in desperate need of new treatment options.”
Crouch concluded, “This is a critical milestone that supports our ongoing development of 3D bioprinted tissues for therapeutic use. We remain on track for filing an Investigational New Drug (“IND”) application with the FDA in calendar-year 2020, as we continue to conduct safety and dosing investigations in small animal disease models and move to defining and scoping IND enabling studies.”
The FDA Orphan Drug designation program provides incentives to sponsors that are developing therapies for rare diseases which affect fewer than 200,000 people in the United States. Organovo is now qualified to receive significant benefits throughout its orphan drug development program including more frequent FDA interactions, protocol assistance, and tax credits for clinical research costs. The designation also includes a waiver of certain fees and a seven-year term of market exclusivity upon FDA approval of the orphan drug and can provide for a more streamlined and cost-effective path through to commercialization.