Hemophilia B: UK Approves Gene Therapy that May Reduce Need for Factor IX Injections
What is Hemophilia B?
Hemophilia B is a rare, inherited genetic disorder that impairs the body’s ability to produce clotting factor IX. This factor is essential for blood to clot properly, so people with the condition experience severe bleeding even after minor injuries.
What is the New Gene Therapy for Hemophilia B?
A new gene therapy called Hemgenix (etranacogene dezaparvovec) from CSL Behring has been approved by the English drugs cost regulator for use in the country’s public health system. Hemgenix is given as a single infusion and has the potential to replace the need for regular factor IX injections in eligible patients.
How Effective is Hemgenix?
Hemgenix has shown promising results in clinical trials, with many trial patients staying off injections for years. One patient who endured serious, painful bleeds for decades said he feels “cured” after taking the therapy. However, it is not yet clear how long the therapy will be effective for and it has not worked for everyone. Some patients may still need to take regular factor IX injections.
What is the Innovative Drugs Fund?
The Innovative Drugs Fund is a £680m ($860m) scheme designed to bring “pioneering” drugs to patients quickly through the country’s public health system, the National Health Service (NHS). Hemgenix is the first gene therapy to be funded through this scheme. Eligible patients will get access to the drug for free under the NHS.
What is the Cost of Factor IX Injections?
A year’s worth of factor IX shots usually costs England’s public health system between £150,000 ($190,000) and £200,000 ($255,000) per patient. The cost of Hemgenix is not yet known, as the deal was brokered directly with CSL Behring.
What is the Eligibility for Hemgenix?
The therapy is available for people with moderately severe or severe hemophilia B, which is around 250 of the 2,000 people in England thought to have the condition, according to the NHS. The therapy is not suitable for everyone and eligibility will be determined by a healthcare professional.
Conclusion
The approval of Hemgenix is a major milestone for the hemophilia community and has the potential to transform the lives of eligible patients. While the long-term effectiveness of the therapy is not yet known, it offers hope for a reduction in the need for regular factor IX injections, which can significantly improve the quality of life for people with hemophilia B.
Originally Post From https://www.forbes.com/sites/katherinehignett/2024/06/27/uk-patients-to-get-33m-hemophilia-gene-therapy-for-free/
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