China-based Hong Kong WinHealth Pharma Group Ltd announced that its Swiss partner Immedica Pharma AB’s Ravicti (glycerol phenylbutyrate) has received orphan drug designation (ODD) status in Taiwan. This rare disease drug, which is awaiting regulatory decisions in China with priority review status, can now be used as a long-term adjuvant therapy for patients with urea cycle disorder (UCD) that cannot be controlled by limiting protein intake and/or simply supplementing amino acids.
Understanding Urea Cycle Disorder
Urea cycle disorder (UCD) is a rare genetic metabolic disease primarily characterized by hyperammonemia in the neonatal period or later. Clinical manifestations include changes in the level of consciousness similar to encephalitis or drug poisoning, acute encephalopathy, epilepsy, lethargy, and mental disorders. Hyperammonemia can lead to severe neurocognitive impairment, coma, or even death. Currently, no effective therapeutic drugs for UCD have been approved for marketing in China.
Licensing Deal and Regulatory Progress
WinHealth struck a licensing deal with Immedica in December 2020, securing exclusive commercialization rights to Ravicti in Greater China, South Korea, Singapore, Vietnam, Indonesia, Malaysia, the Philippines, and Thailand. The drug, already approved for marketing in Europe and the US, was included in the Center for Drug Evaluation’s (CDE) 44th batch of generic preparation reference list drugs in August of last year.
Future Outlook
With the ODD status in Taiwan, WinHealth is poised to advance the development and commercialization of Ravicti for the treatment of UCD. The company aims to leverage the drug’s regulatory approvals and market presence to address the unmet needs of patients with this rare disease.-Fineline Info & Tech
