Zevra Therapeutics Selects Orsini as Specialty Pharmacy for OLPRUVA® (sodium phenylbutyrate)

Orsini Specialty Pharmacy and Zevra Therapeutics have announced a partnership involving the distribution of OLPRUVA® (sodium phenylbutyrate) for oral suspension. This pharmaceutical product is used in conjunction with specific therapies, including dietary modifications, for the long-term treatment of certain urea cycle disorders (UCDs) in both adults and children. Urea cycle disorders are a group of rare genetic conditions that hinder the body's ability to eliminate excess ammonia, leading to potential severe consequences such as brain damage, neurocognitive impairments, coma, and even death if not properly managed.

Joshua Schafer, Chief Commercial Officer at Zevra, emphasized the challenges faced by UCD patients, including developmental delays and cognitive impairments, and reiterated the company's commitment to ensuring that these patients have access to OLPRUVA. Brandon Tom, CEO of Orsini, highlighted the company's dedication to serving rare disease communities and expressed enthusiasm about partnering with Zevra to provide a crucial treatment option for UCD patients.

OLPRUVA® was approved in December 2022 for the management of UCDs involving deficiencies in carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS). It is important to note that OLPRUVA® is not indicated for the treatment of acute hyperammonemia, a rapid increase in blood ammonia levels that can be life-threatening and requires immediate medical attention. Instead, OLPRUVA® functions as a nitrogen scavenger, aiding in the removal of excess ammonia from the bloodstream.

The safety profile of OLPRUVA® includes potential side effects such as neurotoxicity, characterized by symptoms like sleepiness, tiredness, lightheadedness, vomiting, nausea, headache, and confusion. Other possible side effects include low potassium levels (hypokalemia) and conditions related to swelling (edema). Patients are advised to inform their doctors about all medications they are taking, especially corticosteroids, valproic acid, haloperidol, and probenecid, as these can elevate ammonia levels or cause serious side effects. Common side effects reported with OLPRUVA® include irregular menstrual periods, decreased appetite, body odor, taste aversion, and the avoidance of foods previously consumed before illness.

Orsini Specialty Pharmacy, established in 1987, is a leading provider of care for patients with rare diseases and gene therapies. The company collaborates with biopharma innovators, healthcare providers, and payors to ensure patients and their families have access to groundbreaking treatments for rare conditions. Orsini’s services encompass pharmacy distribution, patient services, clinical management, and home infusion services, all designed to simplify patient access to advanced therapies. The company prides itself on a high-touch care model delivered by experienced therapy care teams, ensuring no patient is left behind.

Zevra Therapeutics focuses on developing transformative therapies for rare diseases. Using a unique, data-driven approach to drug development and commercialization, Zevra aims to overcome complex challenges and make new treatments available to the rare disease community. The company also offers expanded access programs for its therapies, which are subject to regulatory guidelines in each jurisdiction and the discretion of treating physicians.

This partnership between Orsini and Zevra represents a significant step forward in providing much-needed treatment options to patients suffering from urea cycle disorders, thereby improving their quality of life and long-term health outcomes.