Neurocrine’s Crinecerfont Aims to Break 70-Year Drought In Congenital Adrenal Hyperplasia Drug Development

The FDA accepts New Drug Applications and grants Priority Review for crinecerfont, potentially the first new treatment for congenital adrenal hyperplasia (CAH) in 70 years, with a PDUFA action date set for late December 2024.

Key points:

  • Crinecerfont, a highly selective CRF1 antagonist, could be the first-in-class therapy for CAH, addressing a significant unmet medical need.
  • The drug has received Orphan Drug and Breakthrough Therapy designations, expediting its development and potential market exclusivity.
  • Phase 3 CAHtalyst study results showed compelling efficacy and excellent safety profile in both pediatric and adult patients with CAH.
Neurocrine Biosciences
Image Source: Neurocrine Biosciences.

In a groundbreaking development for patients with congenital adrenal hyperplasia (CAH), the U.S. Food and Drug Administration (FDA) has accepted two New Drug Applications (NDA) with Priority Review designations for Neurocrine Biosciences‘ (NASDAQ: NBIX) crinecerfont in the treatment of children, adolescents and adults with classic CAH.

Monday, Neurocrine Biosciences announced that the FDA set Prescription Drug User Fee Act (PDUFA) target action dates of December 29 and 30, 2024, for the capsule and oral solution formulations of crinecerfont, respectively.

Dr. Eiry W. Roberts, Chief Medical Officer at Neurocrine Biosciences, expressed enthusiasm about the FDA’s decision: “Receipt of a Priority Review reflects the FDA’s agreement that CAH is a serious condition and there is an urgent need for patients to have access to new treatments.

Crinecerfont’s compelling efficacy results and excellent safety profile support our filing.”

Crinecerfont, a highly selective CRF1 antagonist, represents a novel approach to treating CAH, a disorder that affects the production of essential adrenal hormones.

Current treatments rely on glucocorticoids, which, while life-saving, often lead to significant side effects when used at high doses over extended periods.

The drug’s journey through the regulatory process has been marked by several milestones.

It received Orphan Drug designation in March 2019, granting potential seven-year market exclusivity upon approval.

In December 2023, the FDA awarded crinecerfont Breakthrough Therapy designation, acknowledging its potential to offer substantial improvement over existing therapies.

These designations were based on strong results from the CAHtalyst Phase 3 studies in both pediatric and adult patients.

The findings, published in The New England Journal of Medicine in June, demonstrated crinecerfont’s efficacy and safety in managing CAH.

For the estimated 1 in 10,000 to 1 in 15,000 people born with CAH in the United States, crinecerfont could represent a significant leap forward in treatment options.

The disorder, caused by an enzyme deficiency that alters adrenal hormone production, can lead to severe health complications if left untreated.

While specific market projections for crinecerfont are not available, its potential as the first new CAH treatment in 70 years, coupled with Orphan Drug status, suggests significant commercial potential for Neurocrine Biosciences in this rare disease space.

If approved, crinecerfont could transform the treatment landscape for CAH, offering hope for improved management of this challenging condition with potentially fewer long-term complications.

The FDA’s decision to not currently plan an advisory committee meeting for these applications further underscores the agency’s confidence in the data presented.

 

Image Source: BioMediaHub.

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