FDA Approves Alhemo: A Breakthrough In Hemophilia Treatment
In a significant advancement for hemophilia care, the U.S. Food and Drug Administration (FDA) has approved Alhemo® (concizumab-mtci) as a once-daily prophylactic treatment for adults and children aged 12 years and older with hemophilia A or B who have developed inhibitors to factor VIII or IX.
This approval introduces the first subcutaneous prophylactic therapy available in a prefilled pen for this patient population, offering a more convenient alternative to traditional intravenous infusions.
Addressing A Critical Need
Hemophilia A and B are genetic bleeding disorders caused by deficiencies in clotting factors VIII and IX, respectively.
Some patients develop inhibitors—antibodies that neutralize the effectiveness of replacement therapies—making bleeding episodes more challenging to control.
Alhemo® targets this issue by inhibiting the tissue factor pathway inhibitor (TFPI), thereby enhancing thrombin generation and improving blood clotting.
Alhemo Clinical Efficacy And Safety
The FDA’s approval is based on data from the explorer7 Phase 3 clinical trial, which demonstrated that Alhemo® significantly reduced the annualized bleeding rate in patients with hemophilia A or B with inhibitors.
Participants receiving Alhemo® experienced a median annual bleed rate of 0, compared to 9.8 in those not on prophylaxis, representing an 86% reduction.
Dr. Amy Shapiro, CEO and co-medical director at the Indiana Hemophilia & Thrombosis Center, commented, “The approval of Alhemo® provides a much-needed alternative to the current standard of care, offering patients with inhibitors more treatment options and the opportunity to personalize their care.”
Innovative Delivery Method
Alhemo concizumab-mtci is administered via a prefilled, premixed pen designed for subcutaneous injection, eliminating the need for intravenous access.
The pen delivers doses of 60 mg, 150 mg, or 300 mg through a 32-gauge, 4 mm needle, enhancing patient comfort and adherence.
This user-friendly design is particularly beneficial for patients managing chronic conditions.
Global Regulatory Approvals
Beyond the United States, Alhemo® has received regulatory approvals in several countries.
Health Canada approved the therapy in March 2023, followed by the Therapeutic Goods Administration in Australia in July 2023.
The European Medicines Agency granted approval in December 2024, further expanding access to this innovative treatment.
Implications For The Hemophilia Community
The introduction of Alhemo® marks a pivotal moment in hemophilia care, particularly for patients with inhibitors who have limited treatment options.
Its efficacy, coupled with a convenient administration method, has the potential to improve quality of life and reduce the burden of disease management.
As the hemophilia community continues to seek advancements in treatment, Alhemo® represents a promising step forward in addressing unmet needs and enhancing patient outcomes.
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