Patient-Centric Approach And FDA Expertise Lead To Orphan Approval

Background

An international company developing an orphan drug product needed a partner experienced in FDA interactions to help it navigate the U.S. regulatory process from beginning formulation to NDA approval. The sponsor’s product used a novel delivery platform designed to reduce the treatment burden on patients with a rare chronic pituitary gland disorder. The standard of care at the time required either multiple daily subcutaneous injections or painful monthly injections that did not manage symptoms well for the entire treatment period. The sponsor’s oral product had the potential to substantially increase quality of life for patients.