Core Insights - Cumberland Pharmaceuticals Inc. has received Orphan Drug Designation and Rare Pediatric Disease Designation from the FDA for Ifetroban, aimed at treating cardiomyopathy associated with Duchenne muscular dystrophy (DMD) [1][2] - The company is conducting the FIGHT DMD™ trial, a Phase II study to evaluate the pharmacokinetics, safety, and efficacy of Ifetroban in DMD patients, with results expected later this year [1][2] Regulatory Designations - The Rare Pediatric Disease Designation is intended to incentivize drug development for life-threatening diseases affecting fewer than 200,000 children in the U.S., providing potential benefits such as a priority review voucher for other products [2] - Orphan Drug Designation offers multiple incentives, including exemption from user fees, tax credits for clinical trials, and potential market exclusivity for seven years post-approval [3] Disease Overview - Duchenne muscular dystrophy is a rare genetic disorder affecting approximately 1 in every 3,300 male births globally, leading to severe damage to skeletal and cardiac muscles, with cardiomyopathy being a primary cause of mortality [4] - Ifetroban is being evaluated specifically for its effects on heart disease associated with DMD, an area that has seen limited therapeutic focus [4] Drug Profile - Ifetroban is characterized as a potent and selective thromboxane-prostanoid receptor antagonist, showing promise in preclinical models by preventing cardiac fibrosis and dysfunction, and improving mortality rates [5] Additional Research - Cumberland is also exploring Ifetroban for other conditions, including systemic sclerosis and pulmonary fibrosis, with ongoing trials assessing its safety and efficacy in idiopathic pulmonary fibrosis [7] Company Background - Cumberland Pharmaceuticals focuses on delivering high-quality prescription brands for hospital acute care, gastroenterology, and rheumatology, with a portfolio that includes several FDA-approved products [8][9]
CUMBERLAND PHARMACEUTICALS RECEIVES FDA ORPHAN DRUG AND RARE PEDIATRIC DISEASE DESIGNATIONS FOR NEW TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY