CHMP Meeting Highlights July 2025 New
This month, medicinal products for the following indications have received a positive opinion:
- Niemann-Pick type C
- Hereditary angioedema
- Alzheimer’s disease
- Tenosynovial giant cell tumour
- Pre-exposure prophylaxis of HIV-1
- Postpartum depression
New medicines recommended for approval:
Aqneursa (L-Acetylleucine): has received a positive opinion for the treatment of neurological manifestations of Niemann-Pick type C (NPC) disease, in combination with miglustat, or as a monotherapy in patients where miglustat is not tolerated, in adults and children aged 6 years and older and weighing at least 20 kg.
NPC is a rare genetic neurodegenerative disorder caused by a disrupted cholesterol metabolism and accumulation in the lysosomes. L-Acetylleucine is a derivative of the essential amino acid L-leucine, targeting the lysosomal function.An orphan designation was granted for the treatment of this disease and the EMA has published a press release for this medicine. For more information please consult the product for Aqneursa on the EMA website.
Ekterly (Sebetralstat): has received a positive opinion for the symptomatic treatment of acute attacks of hereditary angioedema (HAE) in adults and adolescents aged 12 years and older. Ekterly is the first oral therapy.
HAE is a potentially life-threatening genetic disorder that involves recurrent attacks of severe swelling of different body parts. Sebetralstat is an inhibitor of plasma kallirein and therefore reduces the production of bradykinin. The dysregulation of bradykinin is the cause of the swelling attacks in HAE. An orphan designation was granted for the treatment of this disease. For more information please consult the product for Ekterly on the EMA website.
Kisunla (Donanemab): has received a positive opinion for the treatment of adult patients with a clinical diagnosis of mild cognitive impairment and mild dementia due to Alzheimer’s disease (Early symptomatic Alzheimer’s disease) who are apolipoprotein E ε4 (ApoE ε4) heterozygotes (who have only one copy of the gene of the apolipoprotein) or non-carriers (who do not have a copy of the gene of the apolipoprotein) with confirmed amyloid pathology.
Alzheimer’s disease is a progressive brain disorder that destroys memory and thinking skills to a point that simplest tasks are impossible. Donanemab is directed against insoluble N-truncated pyroglutamate amyloid beta (Aβ), which is found in the brain amyloid plaques that contribute to the pathophysiology of Alzheimer’s disease. For more information please consult the product for Kisunla on the EMA website.
Romvimza (Vimseltinib): has received a positive opinion for the treatment of adult patients with symptomatic tenosynovial giant cell tumour (TGCT) associated with clinically relevant physical function deterioration and in whom surgical options have been exhausted or would induce unacceptable morbidity or disability.
TGCT is a rare benign tumour often arise from the synovium of joints, bursae and tendon sheaths. Main cause of the tumour is a mutation and overexpression of the colony stimulating factor 1. This attracts tumour-associated macrophages, which provide anti-inflammatory immune responses that support tumour survival. Vimseltinib is a selective inhibitor of the colony-stimulating factor 1 receptor and inhibits the tumour-promoting functions of the microenvironment. An orphan designation was granted for the treatment of this disease. For more information please consult the product for Romvimza on the EMA website.
Tryngolza (Olezarsen): has received a positive opinion as an adjunct to diet in adult patients for the treatment of genetically confirmed familial chylomicronemia syndrome (FCS). FCS is a rare genetic disease of the triglyceride and fat metabolism. This leads to increased plasma triglyceride levels. Other symptoms are fat deposids in the skin, enlargement of the liver and spleen and pancreatitis. Olezarsen is a GalNAc3-conjugated antisense oligonucleotide that reduces apolipoprotein C-III (APOC3) production which is responsible of elevated levels of chylomicrons and triglycerides.An orphan designation was granted for the treatment of this disease. For more information please consult the product for Tryngolza on the EMA website.
Voranigo (Vorasidenib): has received a positive opinion as monotherapy for the treatment of predominantly non-enhancing Grade 2 astrocytoma or oligodendroglioma with an IDH1 R132 or IDH2 R172 mutation in adult and adolescent patients aged 12 years and older and weighing at least 40 kg who only had surgical intervention and are not in immediate need of radiotherapy or chemotherapy.
Astrocytoma and Oligodendroglioma are two types of brain tumours that are mainly caused by the mutation of the isocitrate dehydrogenase (IDH). Vorasidenib is an inhibitor of the IDH1 and 2. An orphan designation was granted for the treatment of this disease. For more information please consult the product for Voranigo on the EMA website.
Yeytuo (Lenacapavir): has received a positive opinion for the pre-exposure prophylaxis (PrEP) in combination with safer sex practices to reduce the risk of sexually acquired HIV-1 infection in adults and adolescents with increased Human Immunodeficiency Virus (HIV)-1 acquisition risk, weighing at least 35 kg.
HIV-1 is a retrovirus that causes infection in humans, leading to a progressive failure of the immune system if the infection progresses. Immunocompromised patients are susceptible of contracting life-threatening infections and of developing cancer. Without treatment, an infection with HIV-1 can lead to the development of the acquired immunodeficiency syndrome (AIDS) and subsequent death. By binding to a structural protein of the capsid, lenacapavir inhibits viral replication.
This medicine was part of the “EU-Medicines for all” programme and a press release was published. Under the name of Sunlenca, lenacapavir is already approved to treat adults infected with HIV-1. For more information please consult the product for Yeytuo on the EMA website.
Zurzuvae (Zuranolone): has received a positive opinion for the treatment of postpartum depression (PPD) in adults following childbirth.
PPD is one of the most common complications of pregnancy and childbirth with a prevalence of around 15% in the EU. Common symptoms are mood swings, anxiety, sadness, feeling overwhelmed, crying, appetite problems and trouble with sleeping. Zuranolone is a neuroactive steroid of gamma-aminobutyric acid type A (GABAA) receptors. It works by enhancing the activity of GABA to reduce neuronal excitability, leading to a relaxing effect. For more information please consult the product for Zurzuvae on the EMA website.
Recommendations on extensions of therapeutic indication:
Alhemo (Concizumab): extension of indication for Alhemo to include the routine prophylaxis of bleeding in patients 12 years of age or more with:
- severe haemophilia A (congenital factor VIII deficiency, FVIII <1%) without FVIII inhibitors.
- moderate/severe haemophilia B (congenital factor IX deficiency, FIX ≤2%) without FIX inhibitors.
Alhemo is also authorised for the prophylaxis of haemophilia A and B with FVIII or FIX inhibitors. For more information please consult the product for Alhemo on the EMA website.
Baqsimi (Glucagon): extension of indication for Baqsimi to include the treatment of severe hypoglycaemia in adults, adolescents, and children aged 1 year and over with diabetes mellitus.
Previously the age-limit was 4 years. For more information please consult the product for Baqsimi on the EMA website.
Clopidogrel Zentiva (Clopidogrel): extension of indication for Clopidogrel Zentiva to include the prevention of atherothrombotic events in adult patients suffering from acute coronary syndrome: ST segment elevation acute myocardial infarction, in combination with ASA in patients undergoing percutaneous coronary intervention (including patients undergoing a stent placement) or medically treated patients eligible for thrombolytic/fibrinolytic therapy. Clopidogrel Zentiva is also authorised for the preventions of atherothrombotic events with other cardiac diseases and conditions. For more information please consult the product for Clopidogrel Zentiva on the EMA website.
Invokana (Canagliflozin): extension of indication for Invokana to include the treatment of adults and children aged 10 years and older with insufficiently controlled type 2 diabetes mellitus as an adjunct to diet and exercise:
- as monotherapy when metformin is considered inappropriate due to intolerance or contraindications
- in addition to other medicinal products for the treatment of diabetes.
Previously it was only authorised for the treatment of adults. For more information please consult the product for Invokana on the EMA website.
mRESVIA (Respiratory syncytial virus mRNA vaccine (nucleoside modified)): extension of indication for mRESVIA to include the active immunisation for the prevention of lower respiratory tract disease (LRTD) caused by Respiratory Syncytial Virus (RSV) in:
- adults 60 years of age and older;
- adults 18 through 59 years of age who are at increased risk for LRTD caused by RSV.
Previously it was only authorised for the treatment of adults 60 years and older. For more information please consult the product for mRESVIA on the EMA website.
Sirturo (bedaquiline): extension of indication for Sirturo to include the treatment as part of an appropriate combination regimen in adult and paediatric patients (2 years to less than 18 years of age and weighing at least 7 kg) with pulmonary tuberculosis (TB) due to Mycobacterium tuberculosis resistant to at least rifampicin and isoniazid.
Previously it was authorised for patients from 5 years of age weighing at least 15 kg. For more information please consult the product for Sirturo on the EMA website.
Taltz (Ixekizumab): extension of indication for Taltz to include the treatment, alone or in combination with methotrexate, of active Juvenile psoriatic arthritis in patients 6 years of age and older and with a body weight of at least 25 kg, who have had an inadequate response to, or who are intolerant of, conventional therapy.
It received also a positive opinion for the treatment, alone or in combination with methotrexate, of active Enthesitis-related arthritis in patients 6 years of age and older and with a body weight of at least 25 kg, who have had an inadequate response to, or who are intolerant of, conventional therapy. Taltz is already authorised for the treatment of different other types of arthritis and psoriasis. For more information please consult the product for Taltz on the EMA website.
Tevimbra (Tislelizumab): extension of indication for Tevimbra to include in combination with platinum-containing chemotherapy as neoadjuvant treatment and then continued as monotherapy as adjuvant treatment, the treatment of adult patients with resectable NSCLC at high risk of recurrence. Tevimbra is already authorised for the treatment of NSCLC in other settings and for the treatment of other types of cancer. For more information please consult the product for Tevimbra 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:
Leqembi: is indicated for the treatment of adult patients with a clinical diagnosis of mild cognitive impairment and mild dementia due to Alzheimer’s disease (Early Alzheimer’s disease) who are apolipoprotein E ε4 (ApoE ε4) non-carriers or heterozygotes with confirmed amyloid pathology. EPAR Leqembi.
Previous CHMP Meeting Highlights can be accessed at: https://www.basg.gv.at/en/healthcare-professionals/chmp-highlights
