DMD New Drug Project ifetroban receives FDA grant for orphan drug product development program

Based on preclinical findings, the FDA has approved Cumberland's application to investigate ifetroban in patients 7 years and older with DMDIn addition, the FDA has allocated more than $1 million through the Orphan Drug Funding Program to support Cumberland in a Phase II Phase II DMD clinical studyIt is worth noting that this is the first DMD clinical program approved to be funded through the FDA Orphan Product Development ProgramDMD is a rare and fatal genetic neuromuscular disease characterized by the gradual loss of muscle, leading to the degeneration of skeletal, heart and lung musclesThis deterioration can lead to loss of movement and wheelchair dependenceIt is estimated that one in every 3,500-5,000 male children suffer from the disease, making it the most common muscle disease in childhoodCardiomyopathy is now the leading cause of death in patients with DMDThere is currently no universal and effective treatment for DMD-related cardiomyopathy, and there are significantly unmet medical needs in this fieldifetroban molecular structure (photo: ebiochemicals.com) ifetroban is a selective, powerful, thrombotosis-procycline receptor (TPr) antagonistPreclinical studies of this molecule have shown that blocking TPr with ifetroban can improve heart survival and increase heart output in multiple animal modelsThese encouraging findings led Cumberland to develop a clinical project to evaluate the efficacy of ifetroban's treatment of DMD cardiomyopathyThis new grant for orphan drugs will support a Phase II multi-center study to assess the safety and effectiveness of ifetroban's treatment of DMD cardiomyopathy and to improve the quality of life of Children and men with DMD"Our mission is to develop innovative drugs that meet the needs of our medical care, and this new project is very much aligned with our company's strategy," said A.JKazimi, CEO ofCumberlandSince ifetroban may be the only drug to address heart failure associated with this deadly disease, we are grateful for the FDA's funding to fund the development of new treatments for these critically ill patientsOriginal Origin: Cumberland Pharmaceuticals Announces FDA OrphanS FDA Grant Grant Grant New Phase II Clinical Program