Duchenne muscular dystrophy (DMD) treatment

Search documents
FIGHT DMD Trial Results Selected for Late-Breaking Presentation at MDA Clinical & Scientific Conference
Prnewswireยท 2025-03-19 20:05
Core Insights - Cumberland Pharmaceuticals announced significant results from its Phase 2 FIGHT DMD clinical trial, demonstrating a 5.4% improvement in cardiac function for patients with Duchenne muscular dystrophy (DMD) [1][3][4] Group 1: Clinical Trial Results - The Phase 2 FIGHT DMD trial showed that high-dose ifetroban treatment resulted in a 3.3% improvement in left ventricular ejection fraction (LVEF) compared to placebo [3] - When compared to propensity-matched natural history controls, high-dose treatment provided a significant 5.4% overall improvement in LVEF, while control patients experienced a 3.6% decline [3] - These improvements in cardiac function could lead to enhanced quality of life and survival for DMD patients [3] Group 2: Drug Information - Ifetroban is an oral thromboxane receptor antagonist that has received Orphan Drug Designation and Rare Pediatric Disease Designation from the FDA for DMD-related heart disease [4][7][8] - If approved, ifetroban would be the first therapy specifically indicated for DMD-related heart disease [8] Group 3: Company and Industry Context - Cumberland Pharmaceuticals is focused on developing treatments for rare diseases, with a growing portfolio of patents related to ifetroban for DMD [5][11] - The company is preparing for an end-of-Phase-2 meeting with the FDA to discuss the next steps for ifetroban's development and commercialization [5] - Current therapies for DMD primarily focus on preserving muscle function, highlighting the urgent need for treatments addressing cardiac complications [4][6]