The one-time gene therapy BBM-H901 (dalnacogene ponparvovec) has been officially approved in the Macao region of China for the treatment of adults with moderate to severe hemophilia B, thus expanding access to the medication beyond mainland China.
Developed by Belief Biomed (BBM), BBM-H901 became China’s first and only approved hemophilia B gene therapy when it was cleared by regulators last year for use in patients in the Asian nation. Macao, although part of China, has its own regulatory framework for drug approvals.
“As the first gene therapy drug for hemophilia B in both mainland China and Macao … we are delighted to leverage Macao’s position as an international gateway to extend the reach of this innovative achievement to the Macao region and even broader markets,” Xiao Xiao, BBM’s cofounder, chairman, and chief science officer, said in a company press release announcing the approval.
“This approval reflects the Macao Special Administrative Region Government’s forward-looking support for cutting-edge medical technologies and provides patients in the region with a breakthrough treatment option that keeps pace with international standards,” Xiao added.
BBM manufactures BBM-H901, while Takeda China is responsible for marketing it in mainland China, Hong Kong, and Macao.
“Looking ahead, BBM will continue to collaborate with Takeda China to accelerate the accessibility of innovative therapies, and hope this approval is an opportunity to deepen our integration with the international market and enable China’s advanced gene therapy to benefit more patients worldwide,” Xiao said.
Hemophilia B is caused by mutations in the F9 gene that result in low levels of clotting factor IX (FIX), a protein needed for blood to clot. As a result, patients often experience excessive and prolonged bleeding. Standard treatment involves clotting factor replacement therapy, in which patients receive regular infusions of FIX to restore the blood’s ability to clot and prevent bleeding. While such treatment is usually successful, it is lifelong.
Now-approved gene therapy shown to reduced joint bleeds in trial
BBM-H901 is a gene therapy administered as a single infusion into the bloodstream. It uses a modified, harmless virus to deliver a highly functional copy of the F9 gene to liver cells, allowing them to produce a working version of the FIX protein, which is then released into the bloodstream, enabling more normal blood clotting.
“BBM-H901 is a gene therapy designed to enable sustained and stable expression of … [FIX] in the body through a single administration, potentially freeing patients from the physical, psychological, and economic burdens associated with lifelong frequent injections required by conventional therapies,” Xiao said.
Results from a small Phase 1 clinical trial (NCT04135300) involving 10 men with moderate to severe hemophilia B showed that the gene therapy increased FIX activity over about a year, while also reducing bleeding rates and the need for FIX replacement therapy. Sustained increases in FIX activity were observed for most participants after five years, according to BBM.
BBM-H901 is a gene therapy designed to enable sustained and stable expression of … [clotting factor IX (FIX)] in the body through a single administration, potentially freeing patients from the physical, psychological, and economic burdens associated with lifelong frequent injections required by conventional therapies.
A Phase 3 clinical study (NCT05203679) then assessed the therapy’s safety and efficacy in 26 people with moderate to severe hemophilia B.
A year after dosing, the mean annualized bleed rate was 0.6 bleeds per year, reaching the threshold for statistical superiority over the historically observed five bleeds per year in Chinese hemophilia B patients receiving standard preventive therapies.
The frequency of joint bleeds was also significantly reduced, and the average number of FIX infusions decreased from 58.2 to 2.9 per year after gene therapy. Long-term follow-up for trial participants is ongoing.
BBM-H901 has also demonstrated good safety in clinical studies, with no serious side effects reported. The most common treatment-related side effects in the Phase 3 study were elevated liver enzymes.
