SHANGHAI and BOSTON – Citrine Medicine, a China-based rare disease therapeutics company, today announced that it has received a clinical trial waiver from the National Medical Products Administration (NMPA) in China for Wakix® (pitolisant), a narcolepsy drug approved in the European Union and the United States for the treatment of narcolepsy in adult patients with and without cataplexy. The company also announced, during a ceremony that took place during Sleep Awareness Week, 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, these efforts support Citrine’s efforts to build the first rare disease ecosystem in China. The clinical trial waiver enables Citrine to accelerate its efforts to bring Wakix to patients in need. Wakix 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. The clinical trial waiver enables Citrine to submit a New Drug Application (NDA) using data from the U.S. and Europe.
As part of the strategic partnership, Citrine will work closely with CARD 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. We founded Citrine to build a rare disease ecosystem in China and bring much-needed drugs to patients affected by rare and orphan diseases. We thank our founding investors, F-Prime Capital, Vivo, and Eight Roads, for their continued support of our business model and platform, and look forward to working with the NMPA, as well as with physicians, patients, and families affected by narcolepsy, to bring Wakix and other important rare and orphan disease therapies to the Chinese market.”
“We are incredibly impressed by the speed that Citrine is moving towards bringing Wakix to patients,” said Dandan Dong, Ph.D., a managing director at Vivo Capital and member of Citrine’s Board of Investors. “After closing their Series A financing round late last year, the Citrine team is making incredible progress in their efforts to build a rare disease ecosystem to benefit patients and families in China.”
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 expects to complete the IND submission in the first quarter of 2021. Citrine also recently announced a strategic partnership that will allow the company exclusive rights to develop, register, and commercialize Alkindi®—the first clinically validated pediatric treatment for congenital adrenal hyperplasia—in the Greater China market. Citrine is a subsidiary of RareStone Group, and is headquartered in Shanghai and has other offices in Beijing, China and Cambridge, Mass.
For more information, visit www.citrinemed.com
CARD is a national, non-profit, cooperative exchange platform for rare disease therapy development, supported by Peking Union Medical College Hospital, PhiRDA, the Chinese Medical Association, the Chinese Hospital Association and the Chinese Research Hospital Association, as well as multiple universities, scientific research institutes and enterprises.
About Eight Roads
Eight Roads is a global proprietary investment firm backed by Fidelity. The flexible nature of its capital means Eight Roads can back and build innovative and ambitious businesses looking to disrupt or lead their chosen markets. Eight Roads has over 20 years of venture capital experience in China and has supported over 100 companies in the country’s healthcare and technology sectors. In the past 10 years, the Eight Roads ecosystem has invested almost $6 billion USD globally.
About Vivo Capital
Founded in 1996, Vivo Capital is a global investment firm focused on healthcare. Vivo has approximately $4.3 billion in assets under management, invested in over 250 public and private companies worldwide. Vivo provides a multi-fund investment platform, covering growth equity, private equity including buyout, venture capital, and public equity. The firm is headquartered in Palo Alto, California, with additional offices in Asia.
About F-Prime Capital
F-Prime Capital is a global venture capital firm investing in healthcare and technology. For the past 50 years, our independent venture capital group has had the privilege of backing great entrepreneurs building groundbreaking companies. With over two billion dollars under management and a global portfolio of more than 200 companies, we champion those dedicated to creating positive change in the world. F-Prime is headquartered in Cambridge, MA, with offices in London, UK and San Francisco, CA.
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 tradename WAKIX® 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 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.
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