tMMJ-002, a cannabis investigational product for the treatment of Huntington’s disease, has been granted Orphan Drug designation by the U.S. Food and Drug Administration, announced its developer MMJ International Holdings.
This new status is expected to expedite the clinical development, regulatory review, and potential marketing approval of the therapy, which is designed to alleviate many of the debilitating effects of Huntington’s.
“We are working diligently to address the significant unmet medical need in people suffering from this deadly disease and this designation furthers our mission to develop impactful cannabinoid-derived medicines to improve clinical outcomes for patients with Huntington’s disease,” Elio Mariani, PhD, director of research and development at MMJ, said in a press release.
MMJ-002 is a cannabinoid-based product designed to provide patients with the medical benefits of cannabis, but without the psychoactive side effects that are commonly associated with the cannabis plant and the health dangers associated with smoking.
The designation follows MMJ’s request to the FDA to grant Investigational New Drug status to MMJ-002 and allows the company to initiate clinical studies. If approved, MMJ will become the first company to advance a product based on natural botanical cannabis extracts to use in human controlled clinical trials.
MMJ expects this approach will enable the company to gather new evidence to demonstrate the therapeutic activity of MMJ-002 and support its commercial development as a treatment for Huntington’s.
“MMJ’s scheduled Phase 2 clinical trial for this program will demonstrate the ability of this therapy to slow or stop the deleterious effects of Huntington’s chorea,” Duane Boise, president and CEO of MMJ, said.
The company filed a request with the U.S. Patent and Trademark Office in December 2017 for its part-cannabinoid therapy to treat patients with cannabinoid-responsive diseases, which includes Huntington’s disease and multiple sclerosis. In addition, MMJ is the first company to obtain a license from Health Canada to grow medical marijuana in Canada, and extract cannabinoids from the cannabis plant for use in FDA-approved clinical studies.
“MMJ has established a leading position in the development of plant-derived cannabinoid therapeutics through its proven drug discovery and development processes, intellectual property portfolio and regulatory and manufacturing expertise,” Boise said.
The benefits of Orphan Drug status include seven-year marketing exclusivity, tax credits for clinical trials, federal grants, and FDA guidance.