Alhemo recommended for European approval as first daily subcutaneous prophylactic treatment for haemophilia A or B patients with inhibitors
Novo Nordisk has announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a positive recommendation for Alhemo (concizumab) as the first once-daily subcutaneous prophylactic treatment for individuals aged 12 and older with haemophilia A or B who have developed inhibitors.
Haemophilia is a rare disorder that affects the body’s ability to form blood clots, which are essential for stopping bleeding. Traditional treatment involves replacing the missing clotting factors through intravenous infusions, but in some cases, the body creates inhibitors that prevent these therapies from working. These inhibitors limit treatment options, affecting up to 30% of people with severe haemophilia A and between 5-10% of those with severe haemophilia B.
Alhemo is an anti-tissue factor pathway inhibitor (TFPI) monoclonal antibody designed to block the TFPI protein, which interferes with the clotting process. By inhibiting TFPI, Alhemo promotes thrombin production, allowing blood to clot even in the absence of other essential clotting factors. If approved by the European Commission, Alhemo will offer patients a convenient, once-daily subcutaneous treatment option, even for those who have developed inhibitors to traditional therapies.
The CHMP’s positive opinion is based on results from the phase 3 explorer7 trial, which evaluated the efficacy and safety of Alhemo in patients with haemophilia A or B with inhibitors. If approved, the treatment will be available in a pre-mixed, prefilled pen for easy subcutaneous administration, aiming to reduce the burden of frequent intravenous infusions. Novo Nordisk expects the European Commission’s final decision within the next two months.
About Alhemo (concizumab)
Alhemo (concizumab) is a monoclonal antibody designed to inhibit the tissue factor pathway inhibitor (TFPI) protein, which disrupts the blood clotting process. By blocking TFPI, Alhemo helps the body produce thrombin, allowing blood to clot and preventing excessive bleeding. Alhemo has already been approved for use in Australia and Switzerland for patients aged 12 and older with haemophilia A or B with inhibitors. In Japan, it is approved for both inhibitor and non-inhibitor patients with haemophilia A or B and is used for routine prophylaxis to reduce the frequency of bleeding episodes.
About the explorer7 study
The explorer7 trial involved 133 male participants aged 12 or older, randomized into different treatment groups. Some participants received no prophylaxis for at least 24 weeks, while others received Alhemo prophylaxis for at least 32 weeks. The study's primary analysis compared the annualized bleeding rate (ABR) between these groups.
Results showed an 86% reduction in spontaneous and traumatic bleeding episodes for patients on Alhemo prophylaxis, with an average ABR of 1.7 compared to 11.8 for those not on prophylaxis. The median ABR for Alhemo was zero, while it was 9.8 for those without prophylaxis. The safety profile of Alhemo was in line with expectations.
About Haemophilia
Haemophilia is a rare genetic bleeding disorder that affects the body's ability to form blood clots. It impacts an estimated 1,125,000 people worldwide. Since haemophilia is an X-linked recessive disorder, it primarily affects males, with about 88% of diagnosed cases being male. The disorder is classified into two main types: haemophilia A, which is caused by a deficiency or defect in clotting Factor VIII (FVIII), and haemophilia B, which results from a deficiency or defect in clotting Factor IX (FIX). Both types lead to difficulty in stopping bleeding, making proper treatment critical for affected individuals.
AstraZeneca's Wainzua received CHMP positive opinion for treating adults with hereditary transthyretin-mediated amyloidosis (ATTRv) who have stage 1 or stage 2 polyneuropathy
The Committee for Medicinal Products for Human Use (CHMP) has given a positive recommendation for the marketing authorization of Wainzua, which is intended for adults with hereditary transthyretin-mediated amyloidosis (ATTRv) and stage 1 or 2 polyneuropathy.
Wainzua must be prescribed and monitored by a healthcare professional with expertise in treating patients with hereditary transthyretin-mediated amyloidosis (ATTRv).
Wainzua will be offered as a 45 mg solution for injection, delivered in pre-filled pens. Its active ingredient, eplontersen, is an antisense oligonucleotide inhibitor (ATC code: N07XX21) that specifically targets and binds to human transthyretin (TTR) mRNA, causing its breakdown. This action prevents the production of both mutant and normal TTR proteins, which effectively reduces amyloid fibril buildup and significantly slows the progression of ATTRv.
Novartis receives favorable CHMP opinion for Kisqali to lower recurrence risk in HR+/HER2- early breast cancer
If approved, Kisqali (ribociclib) combined with an aromatase inhibitor would become an adjuvant therapy option in Europe for patients with stage II or III HR+/HER2- early breast cancer (EBC) who are at high risk of recurrence, including those with node-negative disease.
This recommendation is based on findings from the Phase III NATALEE trial, where Kisqali, added to endocrine therapy (ET), significantly reduced the risk of recurrence by 25% compared to ET alone across a broad group of patients with HR+/HER2- early breast cancer.
Individuals with stage II or III HR+/HER2- EBC are at significant risk of cancer returning, often progressing to metastatic disease, which is incurable, despite receiving adjuvant ET, regardless of whether lymph nodes are involved.
In September, the FDA approved Kisqali for use in this setting. At the 2024 ESMO Congress, an updated analysis of the NATALEE trial revealed a deepening benefit in invasive disease-free survival (iDFS).
Novartis announced that the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion and recommended Kisqali for marketing authorization for use in adults with HR+/HER2- EBC at high risk of recurrence, including those without node involvement.
Breast cancer remains the most commonly diagnosed cancer across Europe. HR+/HER2- is the most frequent subtype, representing around 70% of all breast cancers, and more than 40% of these cases are diagnosed at stage II or III.
The CHMP’s positive recommendation is supported by robust data from the Phase III NATALEE trial. In this study, Kisqali combined with ET reduced the risk of cancer recurrence by 25.1% in stage II and III HR+/HER2- EBC patients compared to ET alone. The results demonstrated a meaningful and consistent iDFS benefit across key subgroups, with a hazard ratio (HR) of 0.749 and a P-value of 0.0006. The trial also confirmed that the 400 mg dose of Kisqali was well-tolerated, with mostly mild symptomatic side effects.
An updated analysis of NATALEE presented at the European Society for Medical Oncology (ESMO) Congress 2024 further strengthens the evidence of Kisqali’s ability to consistently reduce recurrence risk in a broad patient population. The updated data shows that the iDFS benefit continued to improve beyond the three-year Kisqali treatment period, including among patients with node-negative disease.
The NATALEE trial is an international Phase III, randomized, open-label study that is evaluating the efficacy and safety of Kisqali combined with ET as an adjuvant treatment versus ET alone in patients with stage II and III HR+/HER2- EBC. The trial is conducted in partnership with TRIO. The ET used in both arms of the study was a non-steroidal aromatase inhibitor (NSAI; either anastrozole or letrozole), with goserelin added where applicable.
The primary goal of the NATALEE trial is to assess iDFS based on the Standardized Definitions for Efficacy End Points (STEEP) criteria. A total of 5,101 patients from 20 countries participated in the trial.
About Kisqali (ribociclib)
Kisqali (ribociclib) is a selective inhibitor of cyclin-dependent kinases (CDK) 4 and 6, which are proteins that regulate the cell cycle. By blocking the action of these proteins, Kisqali helps slow the growth of cancer cells. When CDK4 and CDK6 are overactive, they can cause cancer cells to divide too rapidly. Targeting these proteins with greater precision may help prevent cancer cells from growing out of control.
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