PITTSBURGH, Dec. 23, 2024 /PRNewswire/ -- PANTHERx Rare, a leader in rare disease product patient access and support services in the United States, is pleased to announce that it was selected by Neurocrine Biosciences, Inc. for the distribution of CRENESSITY (crinecerfont), an oral selective corticotropin-releasing factor type 1 (CRF1) receptor antagonist indicated to improve androgen control and enable a reduced glucocorticoid dose in patients 4 years of age and older with classic congenital adrenal hyperplasia (CAH). CRENESSITY is the first non-steroidal treatment approved for classic CAH and a first-in-class therapy that provides a novel approach to treatment for this rare disorder.

CAH is a rare, autosomal recessive genetic disorder that results from deficiency of one of the enzymes needed for adrenal hormone production. The most common cause of CAH is 21-hydroxylase (21-OHD) deficiency, which accounts for approximately 95% of cases.2 21-OHD deficiency prevents the body from producing cortisol, which is essential to respond to stress and illness. In about 75% of cases, aldosterone production is also affected, which impairs the body's ability to regulate salt and water levels, and thereby blood pressure.2,3 In response to low levels of cortisol and aldosterone, the hypothalamus-pituitary-adrenal (HPA) axis is activated, leading to overproduction of androgens.2 Excess androgen production can cause a variety of symptoms, including anxiety, acne, hirsutism, atypical genital development in affected females, fertility issues, early-onset puberty, and rapid growth followed by premature completion resulting in short stature.3 These symptoms can significantly impact both the physical and mental health of those affected with CAH.

"We are proud to partner with Neurocrine Biosciences for the distribution of CRENESSITY. This collaboration underscores PANTHERx's dedication to providing personalized care and seamless access to transformative therapies for patients with rare and complex conditions," said Rob Snyder, Executive Chair, PANTHERx Rare Pharmacy.

About PANTHERx Rare
PANTHERx Rare is a dual-accredited specialty pharmacy focused on rare and orphan disease, distributing several orphan products and providing access and support services to those needing them most. PANTHERx transforms lives by delivering medicine breakthroughs, clinical excellence, and access solutions to people living with rare and devastating conditions. While each rare condition affects few people, together all rare diseases impact an estimated 25 to 30 million Americans.3 Currently over 7,000 rare diseases have been identified and more than 90% of rare diseases are still without an FDA-approved treatment. 3 Changes in federal policy and advances in science have led to a surge in FDA orphan drug approvals, providing tremendous hope to the rare disease community.

PANTHERx is a dual-accredited specialty pharmacy, holding distinctions in rare disease including the Accreditation Commission for Health Care (ACHC) Distinction in Orphan Drugs and Utilization Review Accreditation Commission (URAC) Rare Disease Center of Excellence. As a pharmacy focused on patient satisfaction, PANTHERx is now a six-time winner of the prestigious MMIT Patient Choice Award, including the 2023 honor. PANTHERx is headquartered in Pittsburgh, Pennsylvania and is licensed in all 50 states and US territories.

1. CRENESSITY (crinecerfont) [package insert]. Neurocrine Biosciences, Inc. 2024.
2. Congenital Adrenal Hyperplasia (CAH). Neurocrine Biosciences, Inc. Accessed October 21, 2024.
3. Congenital Adrenal Hyperplasia. NORD (National Organization for Rare Disorders). Published June 8, 2023. Accessed October 21, 2024.

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SOURCE PANTHERx Rare Pharmacy