China Grants Citrine Clinical Trial Waiver for Narcolepsy Therapy Wakix

Rare Daily Staff

China’s Citrine Medicine said it has received a clinical trial waiver from the National Medical Products Administration in China for Wakix, a drug approved in the European Union and the United States for the treatment of narcolepsy in adult patients with and without cataplexy.

Narcolepsy is a rare, chronic, debilitating neurological disorder characterized by excessive daytime sleepiness, cataplexy, a sudden loss of muscle control triggered by emotions, and hallucinations among other symptoms. Two types of narcolepsy are currently recognized according to the ICSD3 diagnostic criteria, Type 1 and 2. Type 1 is associated with cataplexy and a reduction of hypocretin-1 levels, while Type 2 is not associated with cataplexy.

Wakix (Pitolisant) is a selective histamine H3-receptor antagonist/inverse agonist which enhances the activity of histaminergic neurons. The drug was approved by the EMA in 2016 for the treatment of narcolepsy in adults with or without cataplexy and is marketed in major countries in Europe, and was approved by the FDA in 2018. Cataplexy is characterized by sudden and uncontrolled muscle weakness or paralysis. Pitolisant has an orphan designation in the European Union and the United States for the treatment of narcolepsy. 

The clinical trial waiver enables Citrine to submit a New Drug Application using data from the U.S. and Europe.

Separately, the company said that its holding company the RareStone Group has signed a five-year strategic partnership with the Chinese Alliance for Rare Diseases (CARD), a non-profit organization promoting the development of rare disease therapies and treatment access in China. Together, the company said these efforts reflect the creation of the first rare disease ecosystem in China.

As part of the strategic partnership, Citrine will work closely with the Chinese Alliance for Rare Diseases to promote advances in rare disease prevention, diagnosis, and treatment. The partnership will include rare disease research collaborations, the establishment of a patient registry, rare disease education projects and collaborations, the establishment of rare disease expert panels, and the launch of rare disease-focused conferences in China.

“Together, the clinical trial waiver for Wakix and our new partnership with CARD represent a significant step forward in our efforts to build Citrine into a leading rare disease platform company in China,” said Melissa Bradford-Klug, Citrine’s chief business officer and U.S. president. “The clinical trial waiver will vastly increase the speed at which we can bring Wakix to patients with narcolepsy and provides important validation of our business model and our platform.

Photo: Melissa Bradford-Klug, Citrine’s chief business officer and U.S. president