CHMP Meeting Highlights October 2024
This month, medicinal products for the following indications have received a positive opinion:
- Haemophilia A + B
- Prophylaxis of influenza
- Malignant ascites
- Diagnostic aid for detection of Mycobacterium tuberculosis infection
- Hereditary transthyretin-mediated amyloidosis (ATTRv)
New medicines recommended for approval:
Alhemo (concizumab): has received a positive opinion for routine prophylaxis of bleeding in patients with:
- haemophilia A (congenital factor VIII deficiency) with FVIII inhibitors and of 12 years of age or more.
- haemophilia B (congenital factor IX deficiency) with FIX inhibitors and of 12 years of age or more.
Congenital haemophilia is an X-linked recessive disease caused by mutations in the genes encoding 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.
Concizumab is a monoclonal antibody which inhibits the anticoagulation function of TFPI (tissue factor pathway inhibitor). This inhibition enhances the extrinsic pathway of coagulation and can in part compensate for the deficiencies of coagulation factors VIII or IX which belong to the intrinsic coagulation system. For more information please consult the product for Alhemo on the EMA website.
Fluad (influenza vaccine (surface antigen, inactivated, adjuvanted)): has received a positive opinion for prophylaxis of influenza in adults 50 years of age and older.
Influenza is an infectious disease of the nose, throat and lungs which is caused by the influenza virus. Infection usually occurs by droplet spread from infected people to uninfected people through inhalation. The viral infection can cause fever, cough and breathing problems up to pneumonia in humans. Fluad is an inactivated influenza vaccine. For more information please consult the product for Fluad on the EMA website.
Flucelvax (influenza vaccine (surface antigen, inactivated, prepared in cell cultures)): has received a positive opinion for prophylaxis of influenza in adults and children from 2 years of age.
Influenza is an infectious disease of the nose, throat and lungs which is caused by the influenza virus. Infection usually occurs by droplet spread from infected people to uninfected people through inhalation. The viral infection can cause fever, cough and breathing problems up to pneumonia in humans. Flucelvax is an inactivated influenza vaccine. For more information please consult the product for Flucelvax on the EMA website.
Korjuny (catumaxomab): has received a positive opinion for the intraperitoneal treatment of malignant ascites in adults with epithelial cellular adhesion molecule (EpCAM)-positive carcinomas, who are not eligible for further systemic anticancer therapy.
Ascites describes an accumulation of fluid in the space between the lining of the abdomen and abdominal organs, and is in most cases caused by liver cirrhosis and high blood pressure in the liver vein. Malignant ascites is much less common and can be caused by tumours in various organs, like ovary, lung, pancreas or liver, which produce fluid and release it in the peritoneal cavity.
Catumaxomab is a monoclonal antibody which has three binding sites which address targets on immune cells and cancer cells. Catumaxomab binding results in simultaneous recruitment and activation of different types of immune effector cells directly at the tumour site. This initiates a variety of anti-tumour activities. For more information please consult the product for Korjuny on the EMA website.
Siiltibcy (Mycobacterium tuberculosis derived antigens (rdESAT-6 / rCFP-10)): has received a positive opinion as a diagnostic aid for detection of Mycobacterium tuberculosis infection, including disease, in adults and children aged 28 days or older.
This medicinal product is for diagnostic use only.
Tuberculosis is an infectious disease caused by bacteria called Mycobacterium tuberculosis. It typically affects the lungs and can lead to chronic cough with blood-containing mucus, fever, night sweats, and weight loss.
Diagnosis of active tuberculosis is typically done per chest X-ray, while latent tuberculosis is more difficult to diagnose. Siiltibcy is a diagnostic test which can diagnose latent tuberculosis via a so-called Mantoux test, in which bacterial proteins are applied into the upper layer of the skin. The reaction to this application can give information on the existence of Mycobacterium tuberculosis in the patient’s body. For more information please consult the product for Siiltibcy on the EMA website.
Wainzua (eplontersen): has received a positive opinion for the treatment of hereditary transthyretin-mediated amyloidosis (ATTRv) in adult patients with stage 1 or stage 2 polyneuropathy.
ATTRv amyloidosis is a rare and severe autosomal dominant disease, characterised by multisystem amyloid depositions. The most common clinical presentations include polyneuropathy and cardiomyopathy. ATTRv is caused by mutations in the TTR gene (encoding the transport protein transthyretin), which cause protein misfolding, aggregation and subsequent deposition.
Eplontersen is a short chain consisting of nucleotides which can inhibit the production of the mutated protein via blocking the protein translation in the cell. This leads to slowing of disease progression. Eplontersen is specifically designed to reduce pro-inflammatory side effects which are expected due to the mechanism of action. For more information please consult the product for Wainzua on the EMA website.
Recommendations on extensions of therapeutic indication:
Cerdelga (eliglustat): extension of indication to include the treatment of paediatric patients with Gaucher disease type 1 (GD1) who are 6 years and older with a minimum body weight of 15 kg, who are stable on enzyme replacement therapy (ERT), and who are CYP2D6 poor metabolisers (PMs), intermediate metabolisers (IMs) or extensive metabolisers (EMs). Cerdelga is already authorised in adults. For more information please consult the product for Cerdelga on the EMA website.
Hepcludex (bulevirtide): extension of indication to include the treatment of chronic hepatitis delta virus (HDV) infection in plasma (or serum) HDV-RNA positive paediatric patients 3 years of age and older weighing at least 10 kg with compensated liver disease. Hepcludex is already authorised in adults. For more information please consult the product for Hepcludex on the EMA website.
Kevzara (sarilumab): extension of indication to include the treatment of polymyalgia rheumatica (PMR) in adult patients who have had an inadequate response to corticosteroids or who experience a relapse during corticosteroid taper. Kevzara is already authorised for rheumatoid arthritis. For more information please consult the product for Kevzara on the EMA website.
Kisqali (ribociclib): extension of indication to include, in combination with an aromatase inhibitor, the adjuvant treatment of patients with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative early breast cancer at high risk of recurrence.
Kisqali is already authorised for the treatment of advanced or metastatic breast cancer. For more information please consult the product for Kisqali on the EMA website.
Tevimbra (tislelizumab): extension of indication to include the following two indications:
- Tevimbra, in combination with platinum-based chemotherapy, is indicated for the first-line treatment of adult patients with unresectable, locally advanced or metastatic oesophageal squamous cell carcinoma (OSCC) whose tumours express PD-L1 with a tumour area positivity (TAP) score ≥ 5%.
- Tevimbra, in combination with platinum and fluoropyrimidine-based chemotherapy, is indicated for the first-line treatment of adult patients with HER-2-negative locally advanced unresectable or metastatic gastric or gastroesophageal junction (G/GEJ) adenocarcinoma whose tumours express PD-L1 with a tumour area positivity (TAP) score ≥ 5%.
Tevimbra is already authorised for the treatment of various lines of non-small cell lung cancer, as well as for the second-line treatment of OSCC. For more information please consult the product for Tevimbra on the EMA website.
Yselty (linzagolix choline): extension of indication to include the symptomatic treatment of endometriosis in adult women of reproductive age with a history of previous medical or surgical treatment for their endometriosis. Yselty is already authorised for treatment of moderate to severe symptoms of uterine fibroids. For more information please consult the product for Yselty on the EMA website.
Newly published EPARs:
The EPAR (European public assessment report) is the main document where the EMA publishes detailed information on the medicines assessed by the CHMP. Below is a list of the EPARs for recently approved products that have been made available on the EMA homepage:
Anzupgo (delgocitinib): is indicated for the treatment of moderate to severe chronic hand eczema (CHE) in adults for whom topical corticosteroids are inadequate or inappropriate. EPAR Anzupgo.
Iqirvo (elafibranor): is indicated for the treatment of primary biliary cholangitis (PBC) in combination with ursodeoxycholic acid (UDCA) in adults with an inadequate response to UDCA, or as monotherapy in patients unable to tolerate UDCA. EPAR Iqirvo.
Kayfanda (odevixibat): is indicated for the treatment of cholestatic pruritus in Alagille syndrome (ALGS) in patients aged 6 months or older. EPAR Kayfanda.
Loqtorzi (toripalimab): is indicated for the following two indications:
Loqtorzi, in combination with cisplatin and gemcitabine, is indicated for the first-line treatment of adult patients with recurrent, not amenable to surgery or radiotherapy, or metastatic nasopharyngeal carcinoma.
Loqtorzi, in combination with cisplatin and paclitaxel, is indicated for the first-line treatment of adult patients with unresectable advanced, recurrent, or metastatic oesophageal squamous cell carcinoma. EPAR Loqtorzi.
Vyloy (zolbetuximab): in combination with fluoropyrimidine- and platinum-containing chemotherapy, is indicated for the first-line treatment of adult patients with locally advanced unresectable or metastatic HER2-negative gastric or gastro-oesophageal junction (GEJ) adenocarcinoma whose tumours are Claudin (CLDN) 18.2 positive. EPAR Vyloy.
Winrevair (sotatercept): in combination with other pulmonary arterial hypertension (PAH) therapies, is indicated for the treatment of PAH in adult patients with WHO Functional Class (FC) II to III, to improve exercise capacity. EPAR Winrevair.
Yuvanci (tadalafil): is indicated as substitution therapy for the long-term treatment of pulmonary arterial hypertension (PAH) in adult patients of WHO Functional Class (FC) II to III, who are already treated with the combination of macitentan and tadalafil given concurrently as separate tablets. EPAR Yuvanci.
Previous CHMP Meeting Highlights can be accessed at: https://www.basg.gv.at/en/healthcare-professionals/chmp-highlights
