CHMP Meeting Highlights April 2024 New
This month, medicinal products for the following indications have received a positive opinion:
- Haemophilia A
- Colorectal cancer
- Hypertension
- Over Active Bladder (OAB) syndrome
- Breast cancer
New medicines recommended for approval:
Altuvoct (efanesoctocog alfa): has received a positive opinion forthe treatment and prophylaxis of bleeding in patients with haemophilia A (congenital factor VIII deficiency).
Congenital haemophilia is an X-linked recessive disease caused by mutations in the genes coding for the coagulation factor VIII (haemophilia A) or IX (haemophilia B). It is characterised by the inability to form blood clots, which results in a higher risk of bruising, internal bleeding and bleeding inside the joints. The disease can be classified as mild, moderate or severe, depending on the endogenous plasma activity levels of clotting factors. Patients who are treated with anti-haemophilic factors to replace the deficient coagulation factor can develop anti-factor VIII or XI alloantibodies (inhibitors), which neutralise the activity of the administered replacement factors.
Efanesoctocog alfa is a long-acting version of recombinant factor VIII (FVIII) for the control and prevention of bleeding episodes in individuals with haemophilia A. It is a genetically engineered fusion protein of FVIII, von-Willebrand factor (VWF) and a peptide which prevents the fusion protein from being degraded, resulting in a longer half-life. For more information please consult the product for Altuvoct on the EMA website.
Fruzaqla (fruquintinib): has received a positive opinion for the monotherapy treatment of adult patients with metastatic colorectal cancer (mCRC) who have been previously treated with available standard therapies, including fluoropyrimidine-, oxaliplatin-, and irinotecan-based chemotherapies, anti-VEGF agents, and anti-EGFR agents, and who have progressed on or are intolerant to treatment with either trifluridine-tipiracil or regorafenib.
Colorectal cancer (CRC) is a common and lethal disease. CRC can be diagnosed after the onset of symptoms, like abdominal pain or bleeding, or through routine screening of asymptomatic individuals. CRC at metastatic stage has a poor prognosis, with a five-year survival rate of less than 10%.
Fruquintinib is an orally available inhibitor of vascular endothelial growth factor receptors (VEGFRs) 1, 2, and 3. Binding of VEGF to its receptor promotes angiogenesis, the formation of new blood vessels. This is a mechanism cancer cells can take advantage of, as they can be supplied with oxygen and nutrients. Via upregulation of VEGFR, continued growth in solid tumours can be enabled. For more information please consult the product for Fruzaqla on the EMA website.
Jeraygo (aprocitentan): has received a positive opinion for the treatment of resistant hypertension in adult patients in combination with at least three antihypertensive medicinal products.
Hypertension, or high blood pressure, is a leading cause of cardiovascular disease and mortality. About 10% of patients have a difficult-to-control hypertension which means that blood pressure cannot be controlled despite being treated with at least three antihypertensive drugs.
Aprocitentan is an orally available endothelin receptor antagonist. Binding of aprocitentan to the receptor leads to relaxation of small blood vessels thus preventing a rise in blood pressure. For more information please consult the product for Jeraygo on the EMA website.
Obgemsa (vibegron): has received a positive opinion for the symptomatic treatment of adult patients with overactive bladder (OAB) syndrome.
Overactive bladder (OAB) syndrome is a highly-prevalent disease among middle aged and elderly adults. It is estimated that one third of men and women 75 years of age and older are affected. It is characterised by urinary urgency and frequency, with or without the involuntary loss of urine.
Vibegron is a beta-3 adrenergic receptor agonist. Binding of vibegron to the receptor leads to relaxation of the smooth muscle cells of the bladder, thus increasing bladder capacity. For more information please consult the product for Obgemsa on the EMA website.
Truqap (capivasertib): has received a positive opinion, in combination with fulvestrant, for the treatment of adult patients with oestrogen receptor (ER)-positive, HER2-negative locally advanced or metastatic breast cancer with one or more PIK3CA/AKT1/PTEN-alterations following recurrence or progression on or after an endocrine-based regimen.
Breast cancer (BC) is the leading cause of cancer and cancer deaths in women worldwide, especially when diagnosed at an advanced stage. BC is categorised to subtypes according to its histopathology. Oestrogen receptor (ER) and progesterone receptor (PR) positive tumours are referred to as hormone-receptor (HR) positive BC. About 70% of all diagnosed BCs are HR positive and HER2 negative.
Capivasertib is a selective inhibitor of AKT 1, 2 and 3. The AKT serine/threonine protein kinases are key downstream effectors of the PI3K/AKT/PTEN pathway, promoting cell proliferation, survival, metabolism, and gene expression. Several other AKT inhibitors are currently under development for different oncological conditions. For more information please consult the product for Truqap on the EMA website.
Recommendations on extensions of therapeutic indication:
Alecensa (alectinib): extension of indication to include, as monotherapy, the adjuvant treatment following complete tumour resection for adult patients with ALK-positive non-small cell lung cancer (NSCLC) at high risk of recurrence.
Alecensa is already authorised for different, non-adjuvant treatment settings of NSCLC. For more information please consult the product for Alecensa on the EMA website.
Opdivo (nivolumab): extension of indication to include, in combination with cisplatin and gemcitabine, the first-line treatment of adult patients with unresectable or metastatic urothelial carcinoma.
Opdivo is already authorised for a number of solid tumours, including the second-line treatment of urothelial carcinoma. For more information please consult the product for Opdivo on the EMA website.
Rozlytrek (entrectinib): extension of indication to include the treatment of adult and paediatric patients older than 1 month with solid tumours that have a NTRK gene fusion,
• who have a disease that is locally advanced, metastatic or where surgical resection is likely to result in severe morbidity, and
• who have not received a prior NTRK inhibitor
• who have no satisfactory treatment options
Rozlytrek is a tumour-agnostic cancer treatment and has previously been authorised for the treatment of adult and adolescent patients 12 years of age and older with solid, NTRK gene fusion expressing tumours, as well as for the treatment of adult patients with ROS1-positive, advanced non-small cell lung cancer. For more information please consult the product for Rozlytrek on the EMA website.
Rybrevant (amivantamab): extension of indication to include, in combination with carboplatin and pemetrexed, the first-line treatment of adult patients with advanced non-small cell lung cancer (NSCLC) with activating EGFR Exon 20 insertion mutations.
Rybrevant is already authorised for the second-line treatment of EGFR mutated NSCLC. For more information please consult the product for Rybrevant on the EMA website.
Sirturo (bedaquiline): extension of indication to include the use as part of an appropriate combination regimen in adult and paediatric patients (5 years to less than 18 years of age and weighing at least 15 kg) with pulmonary tuberculosis (TB) due to Mycobacterium tuberculosis resistant to at least rifampicin and isoniazid.
Sirturo is already authorised for a different treatment setting in pulmonary TB. For more information please consult the product for Sirturo on the EMA website.
Triumeq (dolutegravir / abacavir / lamivudine): extension of indication to include the treatment of Human Immunodeficiency Virus type 1 (HIV-1) infected children of at least 3 months of age weighing at least 6 kg to less than 25 kg.
Up to now, Triumeq (as dispersible tablet) was authorised for children weighing at least 14 kg, without age restriction. In addition, the separate film-coated tablet formulation remains authorised for children, adolescents and adults weighing at least 25 kg. For more information please consult the product for Triumeq on the EMA website.
Recently started procedures:
- Acoramidis – Orphan - for the treatment of wild-type or variant transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM).
- Diflunisal – Orphan - Treatment of ATTR amyloidosis.
- Lazertinib - treatment of adult patients with advanced non-small cell lung cancer (NSCLC).
- Linvoseltamab - monotherapy for the treatment of adult patients with relapsed or refractory multiple myeloma.
- Nemolizumab - for the treatment of moderate-to-severe atopic dermatitis and for the treatment of prurigo nodularis.
- Pegfilgrastim – paediatric use marketing authorisation (PUMA) - treatment of neutropenia in paediatric patients.
- Tisotumab vedotin - treatment of adult patients with recurrent or metastatic cervical cancer with disease progression on or after systemic therapy.
- Human albumin solution - vitrification of human MII-phase oocytes and embryos for assisted reproductive technology (ART).
Previous CHMP Meeting Highlights can be accessed at: https://www.basg.gv.at/en/healthcare-professionals/chmp-highlights
