(Yicai) April 11 -- China's top drugs regulator has given the green light to a gene therapy developed by Belief BioMed's unit Xinzhi Medicine Technology for treating moderate-to-severe hemophilia B in adult patients, becoming the first such treatment approved in the country.

China's National Medical Products Administration approved BBM-H901, whose generic name is dalnacogene ponparvovec injection, to provide a new treatment option for adults with factor IX deficiency, the regulator announced on its website yesterday.

Belief BioMed inked an exclusive deal with Takeda China in October 2023, granting the Chinese arm of Japan's Takeda Pharmaceutical the commercialization rights to BBM-H901 in the Chinese mainland, Hong Kong, and Macau.

"BBM-H901, based on a recombinant adeno-associated virus vector, can deliver the optimized human coagulation FIX gene into liver cells of patients," Belief BioMed and Takeda China jointly announced. "Then, coagulation FIX is continuously expressed and secreted into the bloodstream using the host cell gene transcription system, thereby promoting coagulation."

BBM-H901's price will be revealed closer to its commercial launch, which will likely be around the middle of this year, Takeda China told Yicai.

Many gene therapies remain expensive worldwide due to technical complexity, high development costs, and manufacturing challenges. Treatments can range from CNY1 million to over CNY 10 million (USD136,700 to USD1.4 million) per patient, hindering their broader commercial adoption.

Hemophilia B is a rare X chromosome-linked recessive hereditary disorder that causes recurrent spontaneous bleeding in joints, muscles, and soft tissues. Prolonged bleeding can lead to joint deformities, muscle atrophy, or even death. China has around 3,800 hemophilia B patients with limited treatment options.

Gene therapy, or introducing exogenous normal genes into target cells to correct or compensate for diseases caused by defects and abnormal genes, has emerged as a promising direction for treating rare diseases.

Editor: Martin Kadiev