Neurocrine Biosciences Collaborates with PANTHERx
PANTHERx Rare, a leader in rare disease accessibility and support, has been chosen by Neurocrine Biosciences, Inc. to distribute CRENESSITY™ (crinecerfont). This oral medication serves as a selective corticotropin-releasing factor type 1 (CRF1) receptor antagonist, aiding in managing androgen levels and reducing reliance on glucocorticoids in patients aged four and older diagnosed with classic congenital adrenal hyperplasia (CAH). Notably, CRENESSITY is the first non-steroidal treatment approved for classic CAH and represents a pioneering therapy aimed at addressing this rare condition.
Understanding Congenital Adrenal Hyperplasia
CAH is a genetic disorder characterized by a deficiency in specific enzymes crucial for adrenal hormone production. Most commonly, it results from a deficiency in 21-hydroxylase (21-OHD), responsible for about 95% of CAH cases. When 21-OHD is lacking, the body struggles to produce cortisol, a hormone vital in managing stress and illness. Additionally, around 75% of patients may experience impaired aldosterone production, which can disrupt the body's ability to balance salt and water levels and maintain healthy blood pressure.3
The low levels of these hormones activate the hypothalamus-pituitary-adrenal (HPA) axis, leading to an overproduction of androgens, which can manifest in various symptoms. Individuals with CAH may experience anxiety, acne, hirsutism, unusual genital development in females, fertility struggles, early puberty, and accelerated growth which may result in short stature once growth plates close prematurely. The combination of these symptoms can severely impact both the physical and psychological well-being of patients.
The Significance of the Partnership
Rob Snyder, Executive Chair of PANTHERx Rare Pharmacy, expressed enthusiasm about the collaboration, stating, "We are proud to partner with Neurocrine Biosciences for the distribution of CRENESSITY. This partnership highlights our commitment to providing personalized care and ensuring access to innovative therapies for patients living with rare and complex conditions." The collaboration aims to enhance patient care and accessibility to treatments that can significantly improve the quality of life for those affected.
PANTHERx Rare: A Leader in Specialty Pharmacy
PANTHERx Rare operates as a dual-accredited specialty pharmacy dedicated to supporting those affected by rare and orphan diseases. The pharmacy not only distributes a variety of orphan products but also provides essential access and support services to patients. PANTHERx is committed to transforming the lives of individuals living with rare and devastating conditions by delivering innovative therapies, offering clinical excellence, and ensuring practical access solutions.
Despite the rarity of each condition, collectively, all rare diseases impact approximately 25 to 30 million individuals across the nation. Currently, there are over 7,000 rare diseases classified, with more than 90% lacking an FDA-approved treatment. Recent advancements in science and shifts in federal policy have resulted in a notable increase in FDA approvals for orphan drugs, offering significant hope and improved treatment options for the rare disease community.
Accreditations and Recognition
PANTHERx holds esteemed distinctions in rare diseases, including the Accreditation Commission for Health Care (ACHC) Distinction in Orphan Drugs and Utilization Review Accreditation Commission (URAC) Rare Disease Center of Excellence. The pharmacy has consistently focused on patient satisfaction, earning the prestigious MMIT Patient Choice Award six times, including the most recent accolade in 2023. Headquartered in Pittsburgh, Pennsylvania, PANTHERx is licensed to operate in all 50 states and U.S. territories.
Frequently Asked Questions
1. What is CRENESSITY and its purpose?
CRENESSITY is an oral medication designed to act as a selective CRF1 receptor antagonist, helping to improve androgen control in patients with classic congenital adrenal hyperplasia (CAH).
2. Who can benefit from CRENESSITY?
This medication is indicated for patients aged four years and older with classic congenital adrenal hyperplasia, aiming to help manage symptoms and reduce glucocorticoid dosages.
3. What is PANTHERx Rare known for?
PANTHERx Rare specializes in providing access and support for patients with rare and orphan diseases, ensuring availability of various orphan drugs.
4. What impact does CAH have on patients?
Patients with CAH may experience a range of symptoms affecting physical and mental health, including anxiety, fertility issues, and unusual physical developments.
5. How does PANTHERx ensure patient satisfaction?
PANTHERx prioritizes patient care and satisfaction, demonstrated by multiple awards and recognition for its excellence in service and support within the specialty pharmacy field.
