The U.S. Food and Drug Administration has granted orphan drug designation for San Diego-based Organovo’s 3D bioprinted tissue treatment of a protein deficiency disease.
The designation paves the way for more frequent FDA interactions, tax credits for clinical research costs and the potential for seven years of marketing exclusivity after the drug is approved.
Organovo designs and creates functional, three-dimensional human tissues for use in drug discovery, clinical development and therapeutic applications. The company’s NovoTissues is intended to treat alpha-1 antitrypsin deficiency, a condition in which the body does not make enough of a protein that protects the lungs and liver from damage.
The condition can lead to, among other things, liver disease.
“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 US, 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,” CEO Taylor Crouch said in a news release.
He added the company remains on track for filing an investigational new drug application with the FDA in calendar-year 2020.