The US Food and Drug Administration has officially approved Novo Nordisk’s Alhemo® (concizumab-mtci) injection as a once-daily prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 years of age and older who are suffering from hemophilia A or B with inhibitors,
In case you weren’t aware, Hemophilia is a rare bleeding disorder that affects approximately 800,000 people worldwide and 32,000 people in the US alone. Caused by mutation in a gene that provides instructions for making the clotting factor proteins needed to form a blood clot, the disease is known for impairing the body’s ability to make blood clots.
More on the given condition would reveal how an estimated 30% of patients living with severe hemophilia A, and 5-10% of those with severe hemophilia B, develop inhibitors, which makes treatment in some cased significantly more challenging. Now, while treatments have improved the lives of many living with hemophilia, those with hemophilia B with inhibitors continue to experience a disease and treatment burden because of limited prophylactic treatment options to prevent bleeding.
“The development of inhibitors remains the most serious treatment-related complication for people living with hemophilia. For patients with inhibitors, especially in hemophilia B, their hemophilia may remain poorly controlled and pose a life-threatening risk,” said Amy Shapiro, CEO and co-medical director at the Indiana Hemophilia & Thrombosis Center. “The approval of Alhemo® – a first-of-its-kind, prophylaxis, subcutaneous injection pen for adults and children 12 years and older with hemophilia A and B with inhibitors – provides a much-needed alternative to the current standard of care in hemophilia B with inhibitors, while offering patients with hemophilia A with inhibitors more treatment options.”
In response to the problem statement here, Novo Nordisk trialed its Alhemo injection during a comprehensive study, which compared the number of treated spontaneous and traumatic bleeding episodes, as measured by annual bleeding rate (ABR). More on that would reveal how the estimated mean ABR was 1.7 for patients on Alhemo® prophylaxis, compared to 11.8 for patients with no prophylaxis. All in all, the median ABR was zero for treated spontaneous and traumatic bleeds when compared to 9.8 ABR in patients with no prophylaxis.
As for the results, they showed an estimated 86% reduction of ABR in patients randomized to receive Alhemo® prophylaxis (ABR ratio of 0.14, 95% confidence interval [CI], 0.07 to 0.29, p-value <0.001). Beyond that, as a supportive secondary efficacy endpoint, 64% of the patients randomized to receive Alhemo® prophylaxis treatment were found to have experienced zero treated spontaneous and traumatic bleeds during the first 24 weeks of treatment vs. 11% with no prophylaxis.
If we dig into possible adverse reactions of this injection, they were observed in ≥5% of patients.. These side effects included site reactions (18%) and urticaria (6%). Serious adverse reactions were renal infarct and hypersensitivity reaction.
Taking an even deeper view of Novo Nordisk’s explorer7 study, it established the efficacy and safety of Alhemo® for adults and pediatric patients who were 12 years of age and older living with hemophilia A or B with inhibitors. To do so, the exercise had 52 males randomly assigned in a 1:2 ratio to receive no prophylaxis (arm 1, n=19), or Alhemo® prophylaxis (arm 2, n=33), whereas 81 males were non-randomly assigned to receive Alhemo® prophylaxis (arms 3 and 4).
Among other things, we ought to ought to mention how the initial loading dose of Alhemo® was 1 mg per kilogram of body weight, followed by 0.2 mg per kilogram daily,
“The approval of Alhemo® signifies a remarkable achievement in prophylactic hemophilia treatment for individuals with inhibitors aged 12 years and older who, in some cases, currently have few options,” said Anna Windle, SVP Clinical Development, Novo Nordisk. “As the first treatment of its kind for this population, Alhemo® represents a significant step in helping to address the unmet needs of patients with hemophilia with inhibitors, highlighting Novo Nordisk’s commitment to patient-centric innovations in rare diseases.”
