Orsini Partners with PTC Therapeutics to Enhance Care for PKU Patients with SEPHIENCE

Partnership Announcement

Orsini, a prominent name in rare disease pharmacy solutions, has been officially announced as a specialty pharmacy partner for PTC Therapeutics, focusing on the recently FDA-approved therapy, SEPHIENCE™ (sepiapterin). This collaboration is a significant advancement in the treatment options available for patients suffering from phenylketonuria (PKU), a rare genetic disorder characterized by the inability to metabolize phenylalanine (Phe).

Understanding PKU and SEPHIENCE

PKU is a metabolic disorder that can result in serious health complications if not managed effectively. The accumulation of Phe in the body can lead to symptoms such as severe brain damage and intellectual disabilities. SEPHIENCE is a promising oral therapy that specifically targets hyperphenylalaninemia (HPA) in adults and pediatric patients aged one month and older who are diagnosed with sepiapterin-responsive PKU.

Clinical trials have shown that SEPHIENCE significantly reduces blood Phe levels while allowing patients to safely increase their protein intake. This represents a notable improvement in the management of PKU, providing patients with more dietary flexibility and enhancing their quality of life.

Orsini's Commitment to Patient Care

Since its inception in 1987, Orsini has been dedicated to offering compassionate care specifically tailored for those with rare diseases. Their collaboration with PTC Therapeutics dates back to 2020, driven by a shared commitment to delivering outstanding service and making innovative treatments accessible to the rare disease community. Brandon Tom, CEO of Orsini, expressed enthusiasm over this new partnership, emphasizing the importance of SEPHIENCE in making vital treatment options available to PKU patients.

Tom stated, "Since we first began partnering with PTC in 2020, we've shared deep commitment to offering exceptional service and care and to facilitating access to innovative treatments to the rare disease community. SEPHIENCE gives us an exciting opportunity to continue that collaboration as we help the PKU community access this important new treatment as quickly as possible."

This partnership highlights Orsini's ongoing mission to ensure that no patient is left behind. With services that include pharmacy distribution, clinical management, and home infusion services, Orsini aims to simplify the process for patients seeking advanced treatments for their conditions.

Nationally Recognized Achievements

Orsini's reputation in the industry is reinforced by their accreditations, such as the Accreditation Commission for Health Care (ACHC), URAC’s Rare Disease Pharmacy Center of Excellence Designation, and recognition from The Joint Commission and NABP. These accolades demonstrate Orsini's commitment to the highest standard of care in the pharmacy industry.

By adding SEPHIENCE to its growing Endocrinology Center of Excellence, Orsini enhances its portfolio of rare disease management solutions. This underscores their commitment to improving patient outcomes through innovative therapies.

In conclusion, the partnership between Orsini and PTC Therapeutics signifies a considerable step forward in addressing the challenges faced by PKU patients. With SEPHIENCE now available through Orsini's services, hope and improved health prospects are on the horizon for those affected by this challenging disorder. For further information on their services or SEPHIENCE, please visit Orsini's website.