China-based Hong Kong WinHealth Pharma Group Ltd has announced receiving market approval from the National Medical Products Administration (NMPA) for its glycerol phenylbutyrate, a drug used as a long-term treatment for patients with urea cycle disorders (UCDs). This approval specifically targets patients who cannot achieve disease control through protein intake limitation and/or amino acid supplementation.
Understanding Urea Cycle Disorders (UCDs)
UCDs are a group of rare genetic metabolic disorders characterized by the reduction or lack of enzymes or transporters required in the urea cycle, leading to the obstruction of ammonia urea synthesis and excessive ammonia accumulation in the body, known as hyperammonemia. Clinical symptoms include neurological and digestive system issues caused by acute and chronic elevated blood ammonia levels, which can result in high mortality and disability rates, recurrent symptoms, and the need for long-term management. Acute hyperammonemia may lead to acute encephalopathy symptoms such as nausea, vomiting, and disorders of consciousness, while long-term hyperammonemia can cause growth and development delays and cognitive impairment.
Benefits of Glycerol Phenylbutyrate for UCD Patients
The oral preparation of glycerol phenylbutyrate can maintain normal blood ammonia levels, reduce the incidence of high ammonia crisis, improve cognitive function and growth and development levels in patients, and enhance the patient’s medication experience with its colorless and odorless taste. This significantly improves patient compliance and quality of life.
WinHealth’s Licensing Deal and Regional Rights
WinHealth struck a licensing deal with Immedica Pharma AB in December 2020, obtaining exclusive commercialization rights to the drug in Greater China, South Korea, Singapore, Vietnam, Indonesia, Malaysia, Philippines, and Thailand. This deal expands the reach of glycerol phenylbutyrate and provides patients in these regions with a new treatment option for managing UCDs.-Fineline Info & Tech
