CHMP Meeting Highlights September 2024
This month, medicinal products for the following indications have received a positive opinion:
- Ovarian, fallopian tube, or primary peritoneal cancer
- Extensive-stage small cell lung cancer
- Haemophilia A + B
- Radiolabelling with lutetium (177Lu) chloride
- Active immunisation to prevent invasive disease caused by Neisseria meningitidis groups A, B, C, W, and Y
New medicines recommended for approval:
Elahere (mirvetuximab soravtansine): has received a positive opinion for the monotherapy treatment of adult patients with folate receptor-alpha (FRα) positive, platinum-resistant high grade serous epithelial ovarian, fallopian tube, or primary peritoneal cancer who have received one to three prior systemic treatment regimens.
Ovarian epithelial, fallopian tube, or primary peritoneal cancer are a group of cancers which arise in the lining of the above-listed organs. As they derive from the same cells and require similar treatment strategies, they are summarised as epithelial ovarian cancer. Survival rates of epithelial ovarian cancer have increased slightly over the past decade, however it is often diagnosed at an advanced stage which correlates with a poor prognosis.
The folate receptor alpha (FRα) is overexpressed on many epithelial tumours, particularly serous and endometrioid ovarian cancers and serous and endometrioid endometrial cancers, and is associated with DNA synthesis, cell proliferation, DNA repair, and intracellular signalling, all of which are essential for tumorigenesis. Mirvetuximab soravtansine, the active substance of Elahere, is an antibody-drug conjugate (ADC) in which both parts are attached to each other via a cleavable linker. While the monoclonal antibody mirvetuximab binds to FRα and initiates internalisation of the whole conjugate into the cell, the drug component soravtansine is an chemotherapeutic agent which induces cell cycle arrest and cell death. For more information please consult the product for Elahere on the EMA website.
Hetronifly (serplulimab): has received a positive opinion, in combination with carboplatin and etoposide, for the first-line treatment of adult patients with extensive-stage small cell lung cancer (ES-SCLC).
Small-cell lung cancer is an aggressive form of lung cancer and accounts for approximately 15% of all lung cancers. It is diagnosed almost exclusively in smokers. It is characterised by the early development of metastases and thus, prognosis is poor. The median overall survival in extensive-stage disease ranges from 8 to 13 months.
Serplulimab is a monoclonal antibody and novel checkpoint inhibitor, targeting PD-1 (programmed death protein 1), which is expressed on T cells. Many cancer cells in various tissues express PD-L1 or PD-L2 (programmed cell death ligand 1/2), the ligands for PD-1. This overexpression represents an immune evasion mechanism, since activation of PD-1 down-regulates the immune system. Serplulimab blocks the interaction between PD-1 and PD-L1/2 and enhances the T cell responses against the cancer cells. For more information please consult the product for Hetronifly on the EMA website.
Hympavzi (marstacimab): has received a positive opinion for routine prophylaxis of bleeding episodes in patients 12 years of age and older, weighing at least 35 kg, with:
- severe haemophilia A (congenital factor VIII deficiency, FVIII < 1%) without factor VIII inhibitors, or
- severe haemophilia B (congenital factor IX deficiency, FIX < 1%) without factor IX inhibitors
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.
Marstacimab 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 Hympavzi on the EMA website.
Theralugand (lutetium (177Lu) chloride): has received a positive opinion for the following indication:
Theralugand is a radiopharmaceutical precursor, and it is not intended for direct use in patients. It is to be used only for the radiolabelling of carrier molecules that have been specifically developed and authorised for radiolabelling with lutetium (177Lu) chloride.
Radiolabelling is required for certain diagnostic procedures, like positron emission tomography (PET) imaging or single photon emission computed tomography (SPECT) imaging. These imaging techniques are used, e.g., for the diagnosis of several types of cancer.
Lutetium (177Lu) chloride is a radioactive molecule emitting beta-minus and gamma radiation and can be attached to relevant carrier medicines. For more information please consult the product for Theralugand on the EMA website.
Penbraya (meningococcal groups A, C, W, Y conjugate and group B vaccine (recombinant, adsorbed)): has received a positive opinion for active immunisation of individuals 10 years of age and older to prevent invasive disease caused by Neisseria meningitidis groups A, B, C, W, and Y.
Meningococcal disease is a serious bacterial infection of the linings of the brain and the spinal cord (meninges), or the blood. Symptoms include fever, headache, stiff neck and rash. Meningococcal disease can result in organ damage or death.
Penbraya is an active immunisation against meningococcal disease. For more information please consult the product for Penbraya on the EMA website.
Recommendations on extensions of therapeutic indication:
Aflunov (zoonotic influenza vaccine (H5N1) (surface antigen, inactivated, adjuvanted)): extension of indication to include the active immunisation against H5N1 subtype of Influenza A virus in individuals 6 months of age and above.
Aflunov is already authorised in adults. For more information please consult the product for Aflunov on the EMA website.
Buccolam (midazolam): extension of indication to include the treatment of prolonged, acute, convulsive seizures in adults.
Buccolam is already authorised for the treatment of seizures in children from 3 months to < 18 years. For more information please consult the product for Buccolam on the EMA website.
Darzalex (daratumumab): extension of indication to include, in combination with bortezomib, lenalidomide and dexamethasone, the treatment of adult patients with newly diagnosed multiple myeloma who are eligible for autologous stem cell transplant.
Darzalex is already authorised for the treatment of multiple myeloma in different lines of therapy and different combination regimens, as well as for the treatment of light chain (AL) amyloidosis. For more information please consult the product for Darzalex on the EMA website.
Dupixent (dupilumab): extension of indication to include the treatment of eosinophilic esophagitis in children aged 1 year and older, weighing at least 15 kg, who are inadequately controlled by, are intolerant to, or who are not candidates for conventional medicinal therapy.
Dupixent is already authorised for the treatment of eosinophilic esophagitis in adults and adolescents, as well as for the treatment of atopic dermatitis, asthma, chronic rhinosinusitis with nasal polyposis (CRSwNP), prurigo nodularis (PN), and chronic obstructive pulmonary disease (COPD). For more information please consult the product for Dupixent on the EMA website.
Esperoct (turoctocog alfa pegol): extension of indication to include treatment and prophylaxis of bleeding in patients below 12 years with haemophilia A (congenital factor VIII deficiency).
Esperoct is already authorised for the use in patients from 12 years and in adults. For more information please consult the product for Esperoct on the EMA website.
Fasenra (benralizumab): extension of indication to include the add-on treatment for adult patients with relapsing or refractory eosinophilic granulomatosis with polyangiitis.
Fasenra is already authorised for the treatment of asthma. For more information please consult the product for Fasenra on the EMA website.
Imvanex (modified vaccinia Ankara - Bavarian Nordic (MVA-BN) virus): extension of indication to include the active immunisation against smallpox, monkeypox and disease caused by vaccinia virus in individuals 12 years of age and older.
Imvanex is already authorised for the use in adults. EMA has published a press release on this extension of indication. For more information please consult the product for Imvanex on the EMA website.
Keytruda (pembrolizumab): extension of indication to include the following two indications:
- Keytruda, in combination with chemoradiotherapy (external beam radiation therapy followed by brachytherapy), is indicated for the treatment of FIGO 2014 Stage III - IVA locally advanced cervical cancer in adults who have not received prior definitive therapy.
- Keytruda, in combination with carboplatin and paclitaxel, is indicated for the first-line treatment of primary advanced or recurrent endometrial carcinoma in adults who are candidates for systemic therapy.
Keytruda is already authorised for a broad variety of solid tumours, including different treatment regimens in cervical and endometrial cancer. For more information please consult the product for Keytruda on the EMA website.
Otezla (apremilast): extension of indication to include the treatment of moderate to severe plaque psoriasis in children and adolescents from the age of 6 years and weighing at least 20 kg who are candidates for systemic therapy.
Otezla is already authorised for the treatment of psoriasis in adults, as well as the treatment of psoriatic arthritis and Behçet’s disease.
For more information please consult the product for Otezla on the EMA website.
Pravafenix (fenofibrate / pravastatin sodium): extension of the existing indication to include maintenance treatment with moderate-intensity statins other than pravastatin. The full indication will be as follows:
Pravafenix is indicated as an adjunct to diet and other non-pharmacological treatment (e.g. exercise, weight reduction) for the treatment of mixed hyperlipidaemia in adult patients at high cardiovascular risk to reduce triglycerides and increase HDL-C when LDL-C levels are adequately controlled while on a treatment with pravastatin 40 mg monotherapy or on another moderate-intensity statin regimen. For more information please consult the product for Pravafenix on the EMA website.
Synjardy (empagliflozin / metformin): extension of indication to include children aged 10 years and above for the treatment of type 2 diabetes mellitus as an adjunct to diet and exercise:
- in patients insufficiently controlled on their maximally tolerated dose of metformin alone
- in combination with other medicinal products for the treatment of diabetes, in patients insufficiently controlled with metformin and these medicinal products
- in patients already being treated with the combination of empagliflozin and metformin as separate tablets.
Synjardy is already authorised in adults
For more information please consult the product for Synjardy on the EMA website.
Zavicefta (ceftazidime / avibactam): extension of indication to include paediatric patients from birth for the treatment of the following infections:
- Complicated intra-abdominal infection (cIAI)
- Complicated urinary tract infection (cUTI), including pyelonephritis
- Hospital-acquired pneumonia (HAP), including ventilator associated pneumonia (VAP)
Zavicefta is already authorised for the treatment of the infections listed above in paediatric patients from 3 months of age and in adults, as well as for the treatment of adult patients with bacteraemia that occurs in association with, or is suspected to be associated with, any of the infections listed above. For more information please consult the product for Zavicefta 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:
Adzynma (rADAMTS13): is an enzyme replacement therapy (ERT) indicated for the treatment of ADAMTS13 deficiency in children and adult patients with congenital thrombotic thrombocytopenic purpura (cTTP). Adzynma can be used for all age groups. EPAR Adzynma.
Balversa (erdafitinib): as monotherapy is indicated for the treatment of adult patients with unresectable or metastatic urothelial carcinoma (UC), harbouring susceptible FGFR3 genetic alterations who have previously received at least one line of therapy containing a PD-1 or PD-L1 inhibitor in the unresectable or metastatic treatment setting. EPAR Balversa.
Durveqtix (fidanacogene elaparvovec): is indicated for the treatment of severe and moderately severe haemophilia B (congenital factor IX deficiency) in adult patients without a history of factor IX inhibitors and without detectable antibodies to variant AAV serotype Rh74. EPAR Duveqtix.
The sterile eluate (gallium (68Ga) chloride solution) from the radionuclide generator GalliaPharm is indicated for in vitro radiolabelling of various kits for radiopharmaceutical preparation developed and approved for radiolabelling with such eluate, to be used for positron emission tomography (PET) imaging. This radionuclide generator is not intended for direct use in patients. EPAR GalliaPharm.
EURneffy (epinephrine): is indicated in the emergency treatment of allergic reactions (anaphylaxis) due to insect stings or bites, foods, medicinal products and other allergens as well as idiopathic or exercise induced anaphylaxis. Treatment is indicated for adults and children with a body weight ≥ 30 kg. EPAR EURneffy.
mResvia (Respiratory Syncytial Virus (RSV) mRNA vaccine): is indicated for active immunisation for the prevention of lower respiratory tract disease (LRTD) caused by Respiratory Syncytial Virus in adults 60 years of age and older. EPAR mResvia.
Piasky (rovalimab): as monotherapy is indicated for the treatment of adult and paediatric patients 12 years of age or older with a weight of 40 kg and above with paroxysmal nocturnal haemoglobinuria (PNH):
• In patients with haemolysis with clinical symptom(s) indicative of high disease activity.
• In patients who are clinically stable after having been treated with a complement component 5 (C5) inhibitor for at least the past 6 months.
EPAR Piasky.
Tauvid (flortaucipir (18F)): is a radiopharmaceutical indicated for positron emission tomography (PET) imaging of the brain to assess the neocortical distribution of aggregated tau neurofibrillary tangles (NFTs) in adult patients with cognitive impairment who are being evaluated for Alzheimer’s disease (AD). Flortaucipir (18F) is an adjunct to clinical and other diagnostic evaluations. This medicinal product is for diagnostic use only. EPAR Tauvid.
Zegalogue (dasiglucagon): is indicated for the treatment of severe hypoglycaemia in adults, adolescents, and children aged 6 years and over with diabetes mellitus. EPAR Zegalogue.
Recently started procedures:
- Atropine sulfate - treatment of progression of myopia in children aged 3 to 18 years
- Ferric citrate coordination complex - treatment of iron deficiency anaemia in adult chronic kidney disease (CKD) patients with elevated serum phosphorus levels
- Human normal immunoglobulin - replacement therapy (primary immunodeficiency syndromes and secondary hypogammaglobulinemia), immunomodulation (in primary immune thrombocytopenic purpura, Guillain Barré syndrome, Kawasaki disease and Multifocal Motor Neuropathy).
- Mozafancogene autotemcel - Orphan, ATMP - treatment of paediatric patients with Fanconi Anaemia Type A
- Obecabtagene autoleucel - Orphan, ATMP - treatment of patients with relapsed or refractory B cell precursor acute lymphoblastic leukaemia (ALL)
Deutetrabenazine - treatment of tardive dyskinesia - Pneumococcal polysaccharide conjugate vaccine (21-valent) - for active immunisation for the prevention of invasive disease and pneumonia caused by Streptococcus pneumonia
- Sargramostim – Accelerated review - treatment for exposure to myelosuppressive doses of radiation
- Tegomil fumarate - treatment of multiple sclerosis
Previous CHMP Meeting Highlights can be accessed at: https://www.basg.gv.at/en/healthcare-professionals/chmp-highlights
