CHMP Meeting Highlights May 2024
In the month of May 2024, medicinal products for the following indications have received a positive opinion:
- Congenital thrombotic thrombocytopenic purpura
- Acanthamoeba keratitis
- Non-small cell lung cancer
- Haemophilia B
- Prophylaxis of influenza
- Radiolabelling for positron emission tomography (PET) imaging
- Prevention of disease caused by chikungunya virus (CHIKV)
- Severe hypoglycaemia in patients with diabetes mellitus
New medicines recommended for approval
Adzynma (rADAMTS13) has received a positive opinion for a marketing authorisation under exceptional circumstances, as a replacement therapy, for the treatment of ADAMTS13 deficiency in children and adult patients with congenital thrombotic thrombocytopenic purpura (cTTP).
cTTP is a rare disorder of the small blood vessels caused by mutations in the ADAMTS13 gene. It is inherited in an autosomal recessive order. ADAMTS13 is an antithrombocytic enzyme, and its absence leads to a reduced number of platelets in the blood, while the risk for thrombosis is increased. Patients may develop neurological, renal, cardiac and gastrointestinal symptoms due to widespread microvascular thrombosis.
Adzynma is a recombinant protein and is intended to replace the missing ADAMTS13 gene product.
Akantior (polihexanide) has received a positive opinion for the treatment of Acanthamoeba keratitis in adults and children from 12 years of age.
Acanthamoeba keratitis is a rare eye disease which primarily affects contact lens wearers. The infectious disease is caused by acanthamoeba, a certain amoeba species, which can colonise the human eye as a parasite. Symptoms may include pain, decreased vision, light sensitivity and tearing.
Polihexanide, the active substance in Akantior, destroys both the cell membrane and the chromosomes of acanthamoeba. It is administered as an eye drops solution.
Cejemly (sugemalimab) has received a positive opinion for the first-line treatment, in combination with platinum-based chemotherapy, of adults with metastatic non-small cell lung cancer (NSCLC) with no sensitising EGFR mutations, or ALK, ROS1 or RET genomic tumour aberrations.
NSCLC is a serious and often fatal disease that accounts for 80 to 85% of all lung cancers. A significant number of patients present with driver mutations in oncogenes, some of which can be aimed at by targeted therapies. Patients without known driver mutations need broader-acting therapies.
Sugemalimab is a checkpoint inhibitor, targeting PD-L1 (programmed cell death protein 1) on cancer cells. The corresponding receptor, PD-1, is expressed on immune cells. Since activation of PD-1 down-regulates the immune system, blocking of the interaction between PD-1 and PD-L1 enhances the T cell responses against the cancer cells.
Durveqtix (fidanacogene elaparvovec) has received a positive opinion for a conditional marketing authorisation 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.
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. 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.
Fidanacogene elaparvovec, the active substance of Durveqtix, is a gene therapy that aims at enabling the body to produce factor IX itself and prevent and control bleeding. While many of the authorised products for the treatment of haemophilia B require frequent infusions, Durveqtix needs to be administered only once.
Durveqtix was supported through EMA‘s Priority Medicines (PRIME) scheme. The EMA has published a press release on Durveqtix.
Fluenz (influenza vaccine (live attenuated, nasal)) has received a positive opinion for the prevention of influenza in children and adolescents from 24 months to less than 18 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.
Fluenz is a live attenuated influenza vaccine. It will be available as a nasal spray.
GalliaPharm (Germanium (68Ge) chloride / Gallium (68Ga) chloride) has received a positive opinion 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.
PET imaging is a diagnostic procedure which is used, e.g., for several types of cancer. To this end, carrier products need to be labelled with radioactive, positron-emitting molecules like gallium (68Ga).
GalliaPharm contains radioactive Germanium (68Ge) which is used to generate a 68gallium solution for labelling. It is not intended for direct use in patients.
GalliaPharm (Germanium (68Ge) chloride / Gallium (68Ga) chloride) has received a positive opinion 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.
PET imaging is a diagnostic procedure which is used, e.g., for several types of cancer. To this end, carrier products need to be labelled with radioactive, positron-emitting molecules like gallium (68Ga).
GalliaPharm contains radioactive Germanium (68Ge) which is used to generate a 68gallium solution for labelling. It is not intended for direct use in patients.
Ixchiq (Chikungunya vaccine (live)) has received a positive opinion for active immunisation for the prevention of disease caused by chikungunya virus (CHIKV) in individuals 18 years and older. The use of this vaccine should be in accordance with official recommendations.
Chikungunya is a viral disease caused by the Chikungunya virus, which is transmitted to humans by infected mosquitoes. Symptoms include fever and severe joint pain, muscle pain, headache nausea and fatigue. Chikungunya is a debilitating disease and can lead to multiorgan failure in a small proportion of patients.
Ixchiq is the first vaccine in the EU to protect adults from Chikungunya infection. It was supported through EMA‘s Priority Medicines (PRIME) scheme and reviewed under the OPEN (Opening procedures at EMA to non-EU authorities) framework to promote global public health.
The EMA has published a press release on Ixchiq.
Zegalogue (dasiglucagon) has received a positive opinion for the treatment of severe hypoglycaemia in adults, adolescents, and children aged 6 years and over with diabetes mellitus.
There are two types of diabetes mellitus. Type 1 diabetes is an autoimmune disease in which pancreatic cells are destroyed by the immune system, leading to a decreased insulin production. Type 2 diabetes is a metabolic disease characterised by high blood sugar and insulin resistance and it accounts for around 90% of diabetes cases. Type 2 diabetes occurs primarily as a result of obesity and sedentary lifestyle, and the prevalence increases globally along with obesity and overweight.
In patients with diabetes mellitus, insulin treatment can sometimes lead to hypoglycaemia, which is a too low glucose level in the blood.
Dasiglucagon, the active substance in Zegalogue, acts on a receptor relevant for glucose metabolism and is intended to increase plasma glucose levels. It is administered as subcutaneous injection.
Recommendations on extensions of therapeutic indication
Dupixent (dupilumab): extension of indication to include, as add-on maintenance treatment in adults, the treatment of uncontrolled chronic obstructive pulmonary disease (COPD) characterised by raised blood eosinophils on a combination of an inhaled corticosteroid (ICS), a long-acting beta2-agonist (LABA), and a long-acting muscarinic antagonist (LAMA), or on a combination of a LABA and a LAMA if ICS is not appropriate.
Dupixent is already authorised for atopic dermatitis, asthma, chronic rhinosinusitis with nasal polyposis, prurigo nodularis, and eosinophilic esophagitis.
Eliquis (apixaban): extension of indication to include the treatment of venous thromboembolism (VTE) and prevention of recurrent VTE in paediatric patients from 28 days to less than 18 years of age.
Eliquis is already authorised for the prevention of VTE in adults, as well as the prevention of stroke and systemic embolism, the treatment of deep vein thrombosis (DVT) and pulmonary embolism (PE), and prevention of recurrent DVT and PE in adults.
Kinpeygo (budesonide): extension of indication to include the treatment of adults with primary immunoglobulin A nephropathy (IgAN) with a urine protein excretion ≥1.0 g/day (or urine protein-to-creatinine ratio ≥0.8 g/g).
Kinpeygo is already authorised for IgAN patients with worse urine protein-to-creatinine ratios.
Livmarli (maralixibat): extension of indication to include the treatment of progressive familial intrahepatic cholestasis (PFIC) in patients 3 months of age and older.
Livmarli is already authorised for the treatment of cholestatic pruritus in patients with Alagille syndrome.
Skyrizi (risankizumab): extension of indication to include the treatment of adult patients with moderately to severely active ulcerative colitis who have had an inadequate response to, lost response to, or were intolerant to conventional therapy or a biologic therapy.
Skyrizi is already authorised for the treatment of plaque psoriasis, psoriatic arthritis and Crohn’s disease.
Tagrisso (osimertinib): extension of indication to include, in combination with pemetrexed and platinum-based chemotherapy for the first-line treatment of adult patients with advanced non-small cell lung cancer (NSCLC) whose tumours have EGFR exon 19 deletions or exon 21 (L858R) substitution mutations.
Tagrisso is already authorised for different treatment settings in NSCLC.
Tevimbra (tislelizumab): extension of indication to include several treatment settings of non-small cell lung cancer.
1.) Tevimbra in combination with pemetrexed and platinum-containing chemotherapy is indicated for the first-line treatment of adult patients with non-squamous NSCLC whose tumours have PD-L1 expression on ≥50% of tumour cells with no EGFR or ALK positive mutations and who have:
- locally advanced NSCLC and are not candidates for surgical resection or platinum-based chemoradiation, or
- metastatic NSCLC.
2.) Tevimbra in combination with carboplatin and either paclitaxel or nab-paclitaxel is indicated for the first-line treatment of adult patients with squamous NSCLC who have:
- locally advanced NSCLC and are not candidates for surgical resection or platinum-based chemoradiation, or
- metastatic NSCLC.
3.) Tevimbra as monotherapy is indicated for the treatment of adult patients with locally advanced or metastatic NSCLC after prior platinum-based therapy. Patients with EGFR mutant or ALKK positive NSCLC should also have received targeted therapies before receiving tislelizumab.
Tevimbra is already authorised for the treatment of oesophageal squamous cell carcinoma.
Newly published EPARs
The European public assessment report (EPAR) 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:
Ryzneuta (efbemalenograstim alfa) is indicated for the reduction in the duration of neutropenia and the incidence of febrile neutropenia in adult patients treated with cytotoxic chemotherapy for malignancy (with the exception of chronic myeloid leukaemia and myelodysplastic syndromes).
Celldemic (zoonotic influenza vaccine (H5N1) (surface antigen, inactivated, adjuvanted, prepared in cell cultures)) is indicated for active immunisation against H5N1 subtype of Influenza A virus in adults and infants from 6 months of age and above
Filspari (sparsentan) is indicated for the treatment of adults with primary immunoglobulin A nephropathy (IgAN) with a urine protein excretion ≥1.0 g/day (or urine protein-to-creatinine ratio ≥ 0.75 g/g, see section 5.1 in the Summary of Product Characteristics).
Incellipan (pandemic influenza vaccine (H5N1) (surface antigen, inactivated, adjuvanted, prepared in cell cultures) is indicated for active immunisation against influenza in an officially declared pandemic.
Tizveni (tislelizumab) is indicated for the following indications of non-small cell lung cancer:
1.) Tizveni in combination with pemetrexed and platinum-containing chemotherapy is indicated for the first-line treatment of adult patients with non-squamous non-small cell lung cancer whose tumours have PD-L1 expression on ≥50% of tumour cells with no EGFR or ALK positive mutations and who have:
- locally advanced NSCLC and are not candidates for surgical resection or platinum-based chemoradiation, or
- metastatic NSCLC .
2.) Tizveni in combination with carboplatin and either paclitaxel or nab-paclitaxel is indicated for the first-line treatment of adult patients with squamous non-small cell lung cancer who have:
- locally advanced NSCLC and are not candidates for surgical resection or platinum-based chemoradiation, or
- metastatic NSCLC.
3.) Tizveni as monotherapy is indicated for the treatment of adult patients with locally advanced or metastatic non-small cell lung cancer after prior platinum-based therapy. Patients with EGFR mutant or ALK positive NSCLC should also have received targeted therapies before receiving tislelizumab.
Voydeya (danicopan) is indicated as an add-on to ravulizumab or eculizumab for the treatment of adult patients with paroxysmal nocturnal haemoglobinuria (PNH) who have residual haemolytic anaemia.
Zynyz (retifanlimab) is indicated as monotherapy for the first-line treatment of adult patients with metastatic or recurrent locally advanced Merkel cell carcinoma (MCC) not amenable to curative surgery or radiation therapy.
Awiqli (insulin icodec) is indicated for the treatment of diabetes mellitus in adults.
Emblaveo (aztreonam / avibactam) is indicated for the treatment of the following infections in adult patients:
- Complicated intra-abdominal infection (cIAI)
- Hospital-acquired pneumonia (HAP), including ventilator-associated pneumonia (VAP)
- Complicated urinary tract infection (cUTI), including pyelonephritis
Emblaveo is also indicated for the treatment of infections due to aerobic Gram-negative organisms in adult patients with limited treatment options.
Lytenava (bevacizumab gamma) is indicated in adults for treatment of neovascular (wet) age-related macular degeneration (nAMD).