Repurposing an EU Therapeutic for Hemoglobinopathies

National Center For Advancing Translational Sciences

Investigators

Linked publications, trials & patents

Abstract

The TRND team completed pharmacology and efficacy studies to recapitulate key data originally generated by the collaborator. The team received recommendations from the Food and Drug Administration (FDA) of studies required to support filing of an Investigational New Drug (IND) application via pre-IND interactions. Pharmacokinetic, bioanalytical, ADME (absorption, distribution, metabolism, excretion) and IND-enabling toxicology studies were completed, as were formulation and manufacturing of the drug product. These data and materials enabled the collaborator to file regulatory applications, which were cleared by both the US FDA and Health Canada, to begin clinical trials for beta-thalassemia intermedia. The ongoing trials have expanded, as planned, to enroll SCD patients. The TRND team has provided supplementary clinical pharmacology support to the Phase 1b/2a trial related to bioanalytical methods. The TRND team has continued to conduct stability studies of the Good Manufacturing Practice (GMP)-compliant drug product, and is engaged in ongoing regulatory activities between Phoenicia and the FDA and Health Canada.