November 3, 2023
Medicines Development for Global Health Receives United States FDA Orphan Drug Designation for Dovramilast to Treat Leprosy Type 2 Reaction
Medicines Development for Global Health announced today that the United States Food and Drug Administration (FDA) has granted Orphan Drug Designation to dovramilast for the treatment of leprosy type 2 reaction. This designation represents a significant milestone in our commitment to addressing the unmet treatment needs of patients suffering from this debilitating condition.
Leprosy is a neglected disease caused by infection with Mycobacterium leprae or M. lepromatosis. Medicines Development for Global Health is developing dovramilast for the treatment of leprosy type 2 reaction (also known as erythema nodosum leprosum), an immune-mediated reactional state affecting people with multibacillary leprosy. Commonly characterised by the presence of inflamed and painful skin nodules, leprosy type 2 reaction is actually a multisystem disorder and it can be sufficiently serious to require prolonged hospitalization. Patients with leprosy type 2 reaction are, in general, chronically ill and fatigued, feverish, in pain and suffering from insomnia, and can also experience painful inflammation in multiple systems or organs. Consequences of recurrent leprosy type 2 reaction may be serious and long lasting, in the worst cases leading to permanent nerve damage and deformities. The current management of leprosy type 2 reaction requires prolonged treatment with anti-inflammatory and immunosuppressive drugs such as prednisolone and/or thalidomide, both of which can add to the existing disease burden, highlighting the need for the development of new treatments, such as dovramilast.
Orphan Drug Designation is granted by the FDA to medicines that demonstrate promise for the treatment of rare diseases affecting fewer than 200,000 people in the United States. Dovramilast’s designation provides several benefits to Medicines Development for Global Health, including seven years of market exclusivity upon FDA approval, tax credits for clinical development expenses, and assistance with clinical trial design. Most importantly, it underscores the FDA's recognition of dovramilast’s potential to address the significant unmet medical need in leprosy type 2 reaction.
"We are extremely pleased to receive Orphan Drug Designation from the FDA for dovramilast to treat leprosy," said Dr Danielle Smith, Global Head of Regulatory Affairs at Medicines Development for Global Health. "This designation reflects our commitment to improving the lives of patients living with neglected diseases. We are dedicated to advancing the global development of dovramilast for patients with leprosy type 2 reaction, and look forward to working closely with the FDA to bring this potentially life-changing therapy to those in need."