Pfizer: There’s need for innovation in access to hemophilia treatments

Pfizer, a global pharmaceutical and biotechnology company, has re-emphasised its commitment to patients living with hemophilia across the globe.

The global company made its commitment known on Thursday during a media roundtable to commemorate the World Hemophilia Day.

Hemophilia, a rare genetic bleeding disorder that causes the blood to take a long time to clot because of a deficiency in one of several blood clotting factors, is almost exclusively found in males.

During the roundtable, the company reiterated that there is need for innovation in access to hemophilia treatments.

According to Pfizer, its goal is to make sure patients living with hemophilia are seen, heard, and never forgotten as the company continues to work to find breakthrough solutions and therapeutic options to change the lives of patients.

Kodjo Soroh, medical director, Sub-Saharan Africa, Pfizer, said: “Pfizer’s efforts at advancing treatment for hemophilia and bridge gaps in access to care resonates with this year’s theme of World Hemophilia Day (WHD) Access For All: Prevention of Bleeds.

“Pfizer’s commitment to equity and continued investment in hemophilia is evident in its more than 30 years of experience in developing therapies for hematological disorders as it has a deep understanding of the significant challenges that people living with hemophilia continually face. Therefore, we will continue to amplify and celebrate the work of the global hemophilia community as we create awareness on the need for innovation in access to hemophilia treatments.”

Akanmu Sulaimon, professor of hematology and blood transfusion at the University of Lagos, said people with hemophilia are at risk for excessive and recurrent bleeding from modest injuries, which have the potential to be life threatening.

“People with severe hemophilia often bleed spontaneously into their muscles or joints, or rarely into other critical closed spaces such as the intracranial space, where bleeding can be fatal,” Sulaimon said.

“According to the World Federation of Hemophilia, an international not-for-profit organisation, more than 38,000 people worldwide were living with hemophilia B in 2021. WFH works closely in partnership with hemophilia treatment centers (HTCs) in 29 African countries to share knowledge and build global awareness through information exchange, education, and training.

“WFH also provides ∼24 million units of CFCs per year to patients in sub-Saharan Africa through a humanitarian aid program. Pfizer is a visionary partner of the World Federation of Hemophilia for WHD.”

Contributing to the discussion, Vivian Painstil, senior lecturer at the department of child health, Kwame Nkrumah University of Science and Technology (KNUST), Kumasi, Ghana, said the burden people living with hemophilia B face is significant, with many receiving routine infusions or injections which can interfere with their ability to take part in day-to-day activities that many take for granted.

“The BENEGENE-2 data demonstrate the promise of this gene therapy candidate as a potential one-time option for people living with hemophilia B as a means of reducing the clinical and treatment burden over the long term,” said Adam Cuker, director, Penn Comprehensive and Hemophilia Thrombosis Programme.