Partners to bring PNH treatment Fabhalta to low-resource countries
Max Foundation, Novartis target 53 countries across globe
Patients with paroxysmal nocturnal hemoglobinuria (PNH) in 53 countries with limited access to life-saving treatments will be able to receive treatment with Fabhalta (iptacopan).
The Max Foundation, a global nonprofit organization, and Novartis are expanding their collaboration to address the unmet needs of patients across Sub-Saharan Africa, as well as regions in Latin America, the Caribbean, Southeast Asia, and Asia-Pacific.
The initiative will first focus on identifying healthcare providers who are ready to treat PNH patients and creating pathways for treatment access.
“We are thrilled to expand our reach to include people living with PNH in low-resource countries without access to an innovative treatment for this life-threatening condition,” Pat Garcia-Gonzalez, CEO of The Max Foundation, said in a foundation press release. “This collaboration reaffirms the deeply shared value of health equity for both organizations by making the latest treatment approved for the disease available at no cost, fulfilling our mission to accelerate health equity and enabling people to live with dignity and hope.”
PNH treatment raises hemoglobin levels
In PNH, the abnormal activation of the complement system, a group of immune proteins, results in the breakdown of red blood cells (hemolysis). This leads to a drop in the levels of hemoglobin, the protein responsible for oxygen transport in red blood cells, causing anemia and other related symptoms.
Fabhalta, an oral medication approved for adults with PNH in the U.S., works by targeting factor B, a protein involved in the earlier steps of complement activation process. By inhibiting factor B, Fabhalta helps prevent hemolysis occurring both inside and outside blood vessels.
Data from clinical trials have shown that Fabhalta was able to increase hemoglobin levels in PNH patients who had and had not been previously treated with complement inhibitors targeting another complement protein, C5.
In addition to improving access to medications like Fabhalta, the foundation aims to collaborate with medical professionals to improve health systems. Efforts include providing education and training, identifying potential partners in low- and middle-income countries, building capacity at medical institutions, and fostering dialogue and partnerships to broaden treatment accessibility.
The Max Foundation and Novartis have been collaborating to improve access to medications since 2001, starting with cancer therapies and now expanding to treatments for several rare diseases. Novartis is part of Max’s Humanitarian Partnership for Access to Critical Treatments, a network of professional, nonprofit, and commercial organizations that work together to improve access to treatment, care, and support for people living with serious diseases around the world.
“For more than 20 years, Novartis has successfully partnered with The Max Foundation to advance health equity by providing access to our innovative oncology medicines. Together, we have reached more than 100,000 patients by now, delivering sustainable and profound impact on communities around the globe,” said Lutz Hegemann, the company’s president of global health. “Novartis is proud to further expand our strong partnership, thereby accelerating early access for PNH patients in need.”
About the Author
