According to a news release from April 2021, a new partnership is working to improve the commercialization of rare disease drugs for patients in China. Together, rare disease therapeutics company Citrine Medicine (“Citrine”) and pharmaceutical company Sinopharm Group (“Sinopharm”) will benefit patients and improve treatment access.
Rare Disease Awareness
In the United States, a rare disease is defined as one affecting 200,000 individuals or less. Alternately, in the European Union (EU), it is considered one which affects less than 1 in every 2,000 people. So what is a rare disease in China? Unfortunately, knowledge of rare conditions in China is still growing. An estimated 20 million people in China live with a rare disease. Yet patient advocacy and drug accessibility is still an issue. According to an article published in the Orphanet Journal of Rare Diseases:
Most patient organizations do not have official registration, employees, written standard operation protocol, or reliable financial resources, [so] no organizations have been able to sponsor academic research, unlike their counterparts in Western countries. As to challenges, 71.4% of interviewees listed lack of rare disease awareness among the general public, while 67.9% selected lack of financial resources.
While there are patient advocacy groups in China, such as those for amyotrophic lateral sclerosis (ALS), there is still a desperate need for additional drug commercialization and increased access for patients.
The New Partnership
Currently, Citrine is considered one of China’s leading biopharmaceutical companies. Through this partnership with Sinopharm, Citrine will develop a supply chain and distribution channels, allowing for increased drug distribution to patients in need. Considering Sinopharm’s reach, this is extremely important. Altogether, the partnership will increase drug access in 31 Chinese provinces.
One drug to look out for within Citrine’s drug development platform is Wakix (pitolisant). This investigational therapy, which is orally administered, is designed to treat patients with narcolepsy and obstructive sleep apnea. Currently, Citrine has already begun the Investigational New Drug (IND) application process. Further, Citrine will soon commercialize Alkindi in China. The therapy is designed for pediatric patients with congenital adrenal hyperplasia (CAH), a group of inherited genetic disorders which impact the adrenal glands.
