Asklepion Pharmaceuticals, LLC announces receipt of Orphan Drug Designation from U.S. FDA

Asklepion Pharmaceuticals, LLC announces receipt of Orphan Drug Designation from the U.S. FDA for the prevention of clinical sequelae of acute lung injury induced by cardiopulmonary bypass in pediatric patients.

 

On January 9, 2017, Asklepion Pharmaceuticals, LLC announced that the U.S. Food and Drug Administration (FDA) has granted orphan-drug designation for L-citrulline, which is being evaluated for the prevention of clinical sequelae of acute lung injury induced by cardiopulmonary bypass in pediatric patients.

Asklepion Pharmaceuticals currently has a Phase 3 trial underway to determine whether L-citrulline is effective and safe in the prevention of clinical sequelae of acute lung Injury in pediatric subjects undergoing surgery for congenital heart defects. More information can be found at www.clinicaltrials.gov, under the identifier NCT02891837.

The FDA’s Office of Orphan Products Development grants orphan status to drugs and biologic products which treat, diagnose or prevent rare diseases or disorders which affect less than 200,000 people in the United States. The designation offers certain benefits and incentives to the drug developer.

 

About Asklepion Pharmaceuticals, LLC

Asklepion Pharmaceuticals is a Baltimore-based, biopharmaceutical company, focused on the discovery, development, and commercialization of pharmaceutical products to help patients who have little to no therapeutic options, due to the rarity of their conditions. In 2015 the Company obtained FDA approval for an oral treatment indicated for bile acid synthesis defects. For more information please visit: www.asklepionpharm.com