This release is available in French.
In addition to their suffering, rare disease patients often have to face the harsh reality that few pharmaceutical companies will ever be able to offer new treatments for their condition because the costs of new treatments will never be recovered from such a small market. But there are ways they can be helped. The U.S. Food and Drug Administration's "Orphan Drug Designation" offers a wide range of benefits that help organizations developing treatments for diseases and conditions affecting fewer than 200,000 patients in the United States. It was recently granted to McGill University for research conducted at the MUHC into the use of the drug fenretinide for the treatment of pulmonary infections caused by Pseudomonas aeruginosa in patients with Cystic Fibrosis (CF).
CF is a chronic, hereditary disease affecting the respiratory and digestive systems of approximately 70,000 people worldwide. Pseudomonas aeruginosa infects more than 80 per cent of CF patients and contributes to the decline in pulmonary function - the ability of the body to pump blood between the heart and the lungs. The infection is a major cause of sickness and death and is difficult to treat since it is resistant to many drugs.
"We strongly believe that fenretinide has the potential to improve morbidity and prognosis in CF patients at different levels," said Dr. Danuta Radzioch, a professor of Experimental Medicine at the MUHC and McGill's Faculty of Medicine. "Our research has demonstrated that fenretinide reduced lung inflammation as well as the frequency and the severity of pulmonary infections." Fenretinide is a drug that was originally investigated for potential use in the treatment of cancer but has not been commercially available.
Orphan Drug Designation provides opportunities for grant funding toward clinical-trial costs, tax advantages, FDA user-fee benefits, and seven years of market exclusivity in the U.S. following drug approval by the FDA. "We felt extremely proud when McGill received the FDA's Orphan Drug status for fenretinide. This is a very important step in our development and commercialization objectives for this truly innovative approach for the management of pulmonary infections in CF patients" said Dr. Mounia Azzi, Investment Manager at MSBi Valorisation, a partner organization.
"This Orphan Drug Designation is a critical, value-added step towards the development of fenretidine treatment for the benefit of CF patients" said Dr. Costas Karatzas, Director of Business Development at the Research Institute of the MUHC. Dr. Michèle Beaulieu at McGill's Office of Sponsored Research stated that the next step will be a clinical trial to assess safety, tolerability and efficacy of fenretinide in CF patients, to be undertaken in collaboration with the Montreal Chest Institute.