The narcolepsy drug Wakix can now be prescribed to patients in China with urgent unmet medical needs
BOSTON, Mass., and Boao, Hainan, China, May 07, 2021 – Citrine Medicine, a China-based rare disease therapeutics company, today announced that the narcolepsy therapy Wakix
® (pitolisant) has been approved by the Health Commission and Medical Products Administration of Hainan Province for its Named Patient Program (NPP). Through this program, patients can be prescribed Wakix
® in the Boao Lecheng International Medical Tourism Pilot Zone in Hainan Province.
Under the NPP, patients in Boao Pilot Zone with urgent clinical needs can make individual requests, via a special approval procedure, for access to drugs approved in the United States (U.S.), Europe, and other parts of the world but not yet approved by the Chinese National Medical Products Administration (NMPA). Patients may be able to take Wakix
® outside of Boao for personal use if approved in advance by the Hainan NMPA.
“We are very excited to be able to offer Wakix
® to patients with narcolepsy in China via the Named Patient Program only a few months after we obtained the China rights to this therapy,” said Melissa Bradford-Klug, President and Chief Business Officer of Citrine Medicine, Inc. “This is a remarkable milestone, because Wakix
® now becomes one of the very few rare disease drugs accessible to Chinese patients via the NPP, significantly expanding access to eligible patients. Our mission at Citrine is to build a rare disease ecosystem in China, and one of the ways we are doing this is by bringing much-needed, clinically-validated rare disease drugs to patients with high unmet medical needs. We look forward to continuing to work with our partners and with China’s regulatory agencies to find expedited pathways to the treatment of rare disease.”
Through a partnership with Bioprojet, Citrine has exclusive rights for the commercialization and development of Wakix
® in China. The partnership deal was announced in Q4, 2020. Speaking of the partnership, Chong Xu, Ph.D., Principal at F-Prime Capital and Citrine Medicine Board Member, said, “F-Prime has long been committed to investing in companies making a difference in the lives of individuals with rare diseases and their families, and we are pleased to see Citrine make Wakix
® available to Chinese patients.”
Wakix
® is a selective histamine H3-receptor antagonist/inverse agonist that enhances the activity of histaminergic neurons and increases the release of the brain chemical histamine to increase a patient's wakefulness and alertness. It is approved by the European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA) to treat adults suffering from narcolepsy, a serious, chronic disorder characterized by excessive daytime sleepiness (EDS), with or without cataplexy.
About Citrine Medicine
Citrine Medicine is dedicated to improving the lives of patients with rare and intractable diseases by making diagnosis and essential treatments available and accessible to those who need them in China. Our mission is to build the first rare disease ecosystem in China, and in doing so, enable people with rare diseases to live more normal lives. In addition to developing and marketing rare disease drugs, Citrine aims to establish a patient-centric platform which educates people on rare diseases, trains doctors on diagnosis and treatment, and helps doctors develop a full disease management protocol. Citrine’s lead product candidate, Wakix
® (pitolisant), is an investigational oral drug in development for the treatment of narcolepsy and obstructive sleep apnea in China. Citrine has initiated an Investigational New Drug (IND) submission for pitolisant in the treatment of narcolepsy and completed the IND submission in the first quarter of 2021. Citrine also recently announced two strategic partnerships that will allow the company exclusive rights to develop, register, and commercialize Alkindi
® for pediatric congenital adrenal hyperplasia (CAH) patients and Efmody
® for adolescent and adult CAH patients—both hydrocortisone treatments—in Greater China. Citrine is headquartered in Shanghai and has other offices in Beijing, China and Cambridge, Mass.
For more information, visit
www.citrinemed.com
About Pitolisant
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 EU and the US for the treatment of narcolepsy. The drug pitolisant is distributed under the trade name Wakix
® in the European Union and the United States for the treatment of narcolepsy in adult patients with and without cataplexy.
About Narcolepsy
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 in of hypocretin-1 levels, while Type 2 is not associated with cataplexy.
Important Safety Information
In clinical trials conducted in patients with narcolepsy, the most frequent adverse drug reactions (ADRs) reported with pitolisant were insomnia (8.4%), headache (7.7%), nausea (4.8%), anxiety (2.1%), irritability (1.8%), dizziness (1.4%), depression (1.3%), tremor (1.2%), sleep disorders (1.1%), fatigue (1.1%), vomiting (1.0%), vertigo (1.0%), dyspepsia (1.0%), weight increase (0.9%), and upper abdominal pain (0.9%). Pitolisant is contraindicated in patients with severe hepatic impairment (Child-Pugh C) and in women breastfeeding their children.
Citrine Medicine Media Contacts:
Melissa Bradford-Klug
mbk@citrinemed.com
781-999-2665