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Patient-Centric Approach and FDA Expertise Lead to Orphan Approval

Challenge

An international company developing an orphan drug product needed support navigating the US regulatory process from beginning formulation to NDA approval from a partner with experience in FDA interactions and a deep understanding of the Agency’s evolving thinking.

Background

The sponsor’s product used a novel delivery platform technology designed to reduce the treatment burden for people 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

Camargo supported the sponsor with obtaining orphan drug designation for the product and then navigating the complex approval process. We identified the 505(b)(2) pathway as the best pathway for product approval and prepared and led interactions with CDER and the Office of Orphan Products Development to help the sponsor gain clarity on what the development plan should look like.

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

  • Selection of the appropriate listed drug to include in the program’s clinical pharmacokinetic (PK) bridging study,
  • The design and interpretation of drug-drug interaction studies, and
  • Planning of the pivotal clinical protocol to accommodate the FDA’s changing thinking from a single-dose study to a placebo-controlled trial.

Camargo also assisted the sponsor in coordinating with the FDA to include a representative from the National Organization for Rare Disorders (NORD) 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 those she represented; this patient-centric approach to an unmet need was novel at the time of the product’s development.

Outcome

Following a collaboration with Camargo 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.

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