Today, Corbus Pharmaceuticals announced that the European Commission has approved orphan designation for lenabasum in the treatment of dermatomyositis in the European Union (EU).
Earlier this year, lenabasum was granted Orphan Drug Designation in the U.S. by the FDA. This designation allows for incentives for continued testing required for FDA approval.
Similar to the U.S., Orphan Designation is granted by the European Commission to drugs that are intended for the treatment, prevention or diagnosis of life-threatening or chronically debilitating rare diseases where no satisfactory method of diagnosis, prevention or treatment of the condition concerned is authorized.
Lenabasum is a synthetic, oral, non-psychoactive cannabinoid receptor type 2 (CB2) agonist that has shown significant promise for treating dermatomyositis in earlier clinical trials. The drug works by resolving inflammation and fibrosis and halting the production of additional inflammation.
One of the values of this treatment is that it does not suppress the immune system like prednisone and other medications typically used to treat myositis, and studies so far have shown it has few side effects.
Phase 2 studies in diffuse cutaneous systemic sclerosis (scleroderma), dermatomyositis, and cystic fibrosis suggest that the drug may have clinical benefit. Additional clinical studies are being conducted and/or planned to confirm these preliminary results and support applications for regulatory approval in both the US and in Europe.