(Yicai) Sept. 10 -- Dalnacogene Ponparvovec, a gene therapy for adults with moderate to severe hemophilia B that holds the title of the most expensive drug in China with a price tag of CNY2.8 million (USD417,000), has welcomed its first patient over a year after launching, following its developer Belief BioMed reducing patients' out-of-pocket costs to around CNY1 million.

The first Dalnacogene Ponparvovec prescription was recently issued in northern Tianjin, Belief BioMed told Yicai yesterday. This marks a significant milestone in the commercialization of the Shanghai-based company's independently developed gene therapy innovations, officially bringing them to clinical practice, founder, Chairman, and Chief Scientist Xiao Xiao added.

Dalnacogene Ponparvovec was approved in April last year, making it China's first gene therapy for hemophilia. However, its high cost delayed adoption despite its significant therapeutic advantages, leading Belief BioMed to reduce the price in June. With certain provinces and municipalities including the drug in insurance reimbursement programs, the out-of-pocket costs for patients dropped to about CNY1 million.

Hemophilia B is a rare genetic disorder caused by a deficiency or functional defect of clotting factor IX. Patients with the condition have abnormal blood coagulation, which makes them prone to bleeding. In severe cases, internal bleeding, particularly in the brain, can pose a life-threatening risk.

Clinical treatment of Hemophilia B has long relied primarily on factor replacement therapy, requiring patients to undergo lifelong injections of clotting factor replacement products that significantly affect their daily lives. However, Dalnacogene Ponparvovec involves a single injection that delivers the clotting factor IX gene into the patient's body, helping them to restore the ability to produce clotting factors independently and potentially providing a one-time cure.

Because the price of Dalnacogene Ponparvovec remains high even after the cut, Belief BioMed is collaborating with various stakeholders to establish a special medical insurance system. For example, the drug has been included in inclusive commercial health insurance plans in Beijing and Shanghai, gradually reducing patients' out-of-pocket burden.

In addition, Belief BioMed has applied to include Dalnacogene Ponparvovec in the National Healthcare Security Administration's new list of innovative drugs for commercial health insurance. Relevant negotiations concluded yesterday, but no results have been announced yet.

Editors: Dou Shicong, Martin Kadiev