Cumberland Pharmaceuticals Secures Orphan Drug and Rare Pediatric Disease Designations for Ifetroban

Cumberland Pharmaceuticals Secures Orphan Drug and Rare Pediatric Disease Designations for Ifetroban

By, Admin 8 November 2024

Overview

Cumberland Pharmaceuticals has announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation and Rare Pediatric Disease Designation to Ifetroban for the treatment of cardiomyopathy associated with Duchenne muscular dystrophy (DMD).

The Duchenne Muscular Dystrophy

  • DMD is a rare and fatal genetic disorder that affects approximately 1 in 3,300 male births worldwide.
  • It causes damage to skeletal and cardiac muscle, with cardiomyopathy being a leading cause of death. 
  • Ifetroban is being investigated as a potential treatment for the heart disease associated with DMD.

About Ifetroban

  • Ifetroban is a potent and selective thromboxane-prostanoid receptor (TPr) antagonist. 
  • It has shown promise in preclinical models of muscular dystrophy by preventing cardiac fibrosis and dysfunction and improving survival.

Initiation of FIGHT DMD™ Trial

The company is currently completing the FIGHT DMD™ trial, a Phase II study investigating the safety and efficacy of Ifetroban in DMD patients. Results are expected later this year.

The Disease

  • Duchenne muscular dystrophy is a severe genetic disorder primarily affecting young boys. 
  • The FDA's designations for Ifetroban highlight the urgent need for effective treatments for this condition and will accelerate its development.

The Rare Pediatric Disease Designation

  • The FDA grants Rare Pediatric Disease Designation to encourage the development of treatments for life-threatening diseases affecting fewer than 200,000 children in the US. 
  • Companies that receive approval for a drug with this designation may be eligible for a priority review voucher.

Orphan Drug Designation 

  • Orphan Drug Designation is awarded by the FDA to support the development of new therapies for rare diseases or conditions affecting fewer than 200,000 people in the US. 
  • This designation offers various incentives, including fee exemptions, tax credits, and potential market exclusivity.

About Cumberland Pharmaceuticals

  • Cumberland Pharmaceuticals acquired the ifetroban program in collaboration with Vanderbilt University and Cumberland Emerging Technologies. 
  • The company is also evaluating ifetroban for systemic sclerosis and pulmonary fibrosis in the FIGHTING FIBROSIS Trial.

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