The designation offers several benefits for Organovo, but more importantly, brings a potentially useful solution for alpha-1 antitrypsin deficiency to market.
Organovo Holdings Ltd., the company that designs and creates 3D human tissues for drug discovery and clinical trials, has recently been granted orphan drug designation by the US Food and Drug Administration (FDA). Orphan drug designation is a program by the FDA which lends orphan status to drugs in development giving them a seal of approval. In other words, these drugs are deemed safe and effective to use.
Organovo has received the designation for its 3D bioprinted liver therapeutic tissue for treatment of alpha-1 antitrypsin deficiency (A1AT).
A1At is a rare, inherited condition that can cause lung and liver issues. An estimated 100,000 people are affected in the US. The disease is marked by lack of enzyme inhibitor alpha-1 antitrypsin, making patients more vulnerable to smoke and dust inhalation.
Current therapeutic measures are exceeding costs of $250,000 for drug treatment alone. This renders Organovo’s latest treatment an even more important option for patients.
Native human liver tissue versus ExVive 3D Bioprinted Human Liver Tissue (Image: Organovo)
FDA Approval to Guarantee Market Exclusivity
Taylor J. Crouch, CEO, Organovo, explained:
“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 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 program has a long-standing reputation to offer incentives to develop drug and biologics therapies for rare diseases – those classified as affecting fewer than 200,000 people in the US.
Through the latest qualification, Organovo is now able to seek assistance through FDA interactions and protocol assistance as well as tax credits for clinical research costs.
As part of the designation, certain fees are also being waived. Market exclusivity for the tissue is guaranteed for seven years as part of the FDA approval.
Organovo’s 3D printed tissues such as the ExVive Human Liver and Kidney tissues are being used for drug profiling and compound screening tests of disease models as well as toxicological studies. In addition, Organovo is expanding its preclinical programs to develop NovoTissues liver tissues for therapeutic use.