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.

Solution

Premier Consulting helped the sponsor obtain orphan drug designation for the product and negotiate the complex approval process. We identified the 505(b)(2) pathway as the best pathway for approval and prepared and then led interactions with the FDA’s Center for Drug Evaluation and Research and Office of Orphan Products Development to give the sponsor clarity on what the development plan should look like.

Our understanding of the FDA’s language and ability to convert meeting notes into actionable insights were essential to helping the sponsor move the program through development in several ways, including:

• Selection of the appropriate listed drug to include in the program’s clinical pharmacokinetic bridging study
• Design and interpretation of drug-drug interaction studies
• Pivoting the clinical protocol from a single-dose study to a placebo-controlled trial to accommodate shifts in the FDA’s thinking

Premier Consulting also helped the sponsor coordinate with the FDA to include a National Organization for Rare Disorders member and a patient advocate in key Type B and Type C meetings. The patient advocate, who also served as the president of the disorder’s patient organization, explained to the FDA how the product would improve quality of life for the patients she represented; this patient-centric approach to an unmet need was novel at the time of the product’s development.

Takeaway

Following a collaboration with Premier Consulting that spanned the length of the program, the sponsor obtained full FDA approval for the orphan product, and the quality of life for patients with the rare disease improved greatly.

Outcome

Sponsor obtained full FDA approval for the orphan product, and the quality of life for patients with the rare disease improved greatly.