Hemlibra Now Available to Patients in Developing Countries via Aid Program

Patricia Inácio, PhD avatar

by Patricia Inácio, PhD |

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SIG-001 orphan drug

Hemlibra (emicizumab), the sole approved prophylactic treatment for hemophilia A, is now available to patients in developing countries via the World Federation of Hemophilia (WFH) Humanitarian Aid Program after Roche and its subsidiaries Chugai and Genentech joined the program.

The donation will provide access to Hemlibra for a period of five years for over 1,000 people with hemophilia A, including those with hemophilia A with factor VIII inhibitors and children without factor VIII inhibitors.

The companies will provide Hemlibra and donate funds to the WFH Program’s integrated care development training so that the prophylatic is made available along with trained medical experts and appropriate local infrastructures.

“Thanks to Roche’s donation, significantly more people with hemophilia A will be able to receive prophylaxis through the WFH Humanitarian Aid Program. Importantly, the donation will also provide a treatment option for people with hemophilia A with factor VIII inhibitors who previously had very limited or no treatment,” Alain Weill, WFH President, said in a press release.

Across the globe, approximately 70 percent of people living with hemophilia have limited access to prophylactic therapy. In developing countries, their resources are limited to emergency situations and acute bleeds.

This leads to a significant detrimental impact on patients’ quality of life and life expectancy. Moreover, lack of access to treatment often means that children with severe hemophilia fail to reach adulthood.

The WFH is an international not-for-profit organization composed of patient organizations in 140 countries. Its Aid Program is a landmark initiative focused on facilitating access to care and treatment for those living with bleeding disorders in developing countries. Currently, around 1,500 people with hemophilia A have access to prophylactic treatment via the WFH Humanitarian Aid Program.

“Increasing access to prophylactic treatments can make a profound difference in countries where hemophilia A remains underdiagnosed and untreated,” Weill said.

“We are proud to join the WFH Humanitarian Aid Program, a landmark initiative leading the effort to change the lack of access to care and treatment for people with inherited bleeding disorders in developing countries,” said Bill Anderson, CEO of Roche Pharmaceuticals. “Our partnership with the WFH reflects our commitment to the hemophilia community and to supporting rapid, broad and sustainable access to Hemlibra for all people with hemophilia A around the world who may benefit from this important treatment option.”

Hemlibra is an antibody therapy designed to combine factors IX and X of the blood clotting series and promote clotting. Restoring the missing function of factor VIII by an alternate method makes Hemlibra beneficial for the treatment of hemophilia A patients.

Initially created by Japan-based Chugai Pharmaceuticals, the therapy is now co-developed by Chugai, along with Roche and its subsidiary, Genentech.

“Chugai intends to contribute to tackling social issues through the creation of innovative medical products, and develop along with the society,” said Tatsuro Kosaka, Chugai’s President & CEO.

“We are very pleased that Hemlibra, our innovative drug, can play a great role in the global treatment for hemophilia A through the program by WFH. We will continuously engage in global health by creating shared values with the society through our business activities,” Kosaka said.