CHMP Meeting Highlights November 2025
This month, medicinal products for the following indications have received a positive opinion:
- hereditary angioedema
- breast cancer
- type 1 diabetes
- pertussis vaccine
- Wiskott-Aldrich syndrome
New medicines recommended for approval:
Dawnzera (donidalorsen): has received a positive opinion for the prevention of recurrent attacks of hereditary angioedema (HAE) in adults and adolescents aged 12 years and older.
HAE is a rare genetic disorder that leads to recurrent attacks of severe swelling, which affects arms, legs, face, the intestinal tract and airways. Dawnzera is an antisense oligonucleotide which leads to the degradation of prekallikrein-mRNA thus less prekallikrein is produced. Prekallikrein is converted to plasma kallikrein, which induces the release of bradykinin, the cause of the inflammation and swelling in HAE. An orphan designation was granted for the treatment of this disease. For more information please consult the product for Dawnzera on the EMA website.
Inluriyo (imlunestrant): has received a positive opinion for the treatment of adult patients with oestrogen receptor (ER)-positive, HER2-negative, locally advanced or metastatic breast cancer with an activating ESR1-mutation, who have disease progression following prior treatment with an endocrine based regimen as monotherapy. For more information please consult the product for Inluriyo on the EMA website.
Teizeild (teplizumab): has received a positive opinion for the treatment to delay the onset of stage 3 type 1 diabetes (T1D) in adult and paediatric patients 8 years of age and older with stage 2 T1D.
Type 1 diabetes is a chronic autoimmune disease in which the immune system destroys the insulin-producing beta cell in the pancreas. Insulin is needed to regulate the blood sugar, and the patients are dependent on daily insulin injections. With the progression to stage 3 T1D the patients start to have symptoms like thirst, hunger, frequent urination, weight loss and tiredness.
It was supported through EMA‘s Priority Medicines (PRIME) scheme and a press release for this medicine was released. For more information please consult the product for Teizeild on the EMA website.
VacPertagen (pertussis vaccine (recombinant, acellular, component, adsorbed)): has received a positive opinion for the booster immunisation against pertussis of individuals 12 years of age and older and the passive protection against pertussis in early infancy following maternal immunisation during pregnancy.
Pertussis or whooping cough is a disease caused by the bacterium Bordetella pertussis and is an airborne disease which spreads through coughs and sneezes. Early symptoms are similar to a common cold like coughing, mild fever and runny nose. Later stage symptoms are paroxysmal cough, inspiratory whoop, and fainting, or vomiting after coughing. For more information please consult the product for VacPertagen on the EMA website.
Waskyra (Autologous CD34+ haematopoietic stem cells transduced ex vivo with a lentiviral vector encoding human Wiskott-Aldrich syndrome protein): has received a positive opinion for the treatment of patients aged 6 months and older with Wiskott-Aldrich Syndrome (WAS) who have a mutation in the WAS gene for whom haematopoietic stem cell (HSC) transplantation is appropriate and no suitable human leukocyte antigen (HLA)-matched related haematopoietic stem cell donor is available.
Wiskott-Aldrich syndrome is a rare, inherited disease that affects blood cells and cells of the immune system. It is caused by a mutation in the gene encoding for the WAS protein. This protein is needed for the development of blood cells. Waskyra is a gene therapy. Stem cells of the patient are genetically modified with a non-mutated gene in a lab and transplanted into the patient.
An orphan designation was granted for the treatment of this disease and a press release for this product was released. For more information please consult the product for Waskyra on the EMA website.
Recommendations on extensions of therapeutic indication:
Koselugo (selumetinib): extension of indication for Koselugo to include the treatment of symptomatic, inoperable plexiform neurofibromas (PN) in patients with neurofibromatosis type 1 (NF1) aged 1 year to less than 7 years and for older patients with swallowing difficulties.
This extension of indication is accompanied by a new pharmaceutical form: granules in capsules for opening. Koselugo is already authorised as hard capsules for patients 3 years and older with neurofibromatosis type 1. For more information please consult the product for Koselugo on the EMA website.
Minjuvi (tafasitamab): extension of indication for Minjuvi to include in combination with lenalidomide and rituximab for the treatment of adult patients with relapsed or refractory follicular lymphoma (FL) (Grade 1-3a) after at least one line of systemic therapy. Minjuvo is already authorised for the treatment of diffuse large B-cell lymphoma. For more information please consult the product for Minjuvi on the EMA website.
Veyvondi (vonicog alfa): extension of indication for Veyvondi to include the treatment of haemorrhage in children (aged less than 18 years) with von Willebrand disease (VWD), when desmopressin (DDAVP) treatment alone is ineffective or contraindicated. Previously Veyvondi was only authorised for the treatment of adult patients. For more information please consult the product for Veyvondi on the EMA website.
Xerava (eravacycline): extension of indication for Xerava to include the treatment of complicated intra-abdominal infections (cIAI) in adolescents from the age of 12 years weighing at least 50 kg, and in adults. Previously Xerava was only authorised for the treatment of adult patients. For more information please consult the product for Xerava 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:
Imreplys: is indicated for treatment of patients of all ages acutely exposed to myelosuppressive doses of radiation with Haematopoietic Sub-syndrome of Acute Radiation Syndrome (H-ARS). EPAR Imreplys.
Kisunla: Donanemab 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 symptomatic Alzheimer’s disease) who are apolipoprotein E ε4 (ApoE ε4) heterozygotes or non-carriers with confirmed amyloid pathology. EPAR Kisunla.
Rezdiffra: is indicated in conjunction with diet and exercise for the treatment of adults with noncirrhotic metabolic dysfunction-associated steatohepatitis (MASH) with moderate to advanced liver fibrosis (fibrosis stages F2 to F3). EPAR Rezdiffra.
Xoanacyl: is indicated for the treatment of concomitant elevated serum phosphorous and iron deficiency in adult patients with chronic kidney disease (CKD). EPAR Xoanacyl.
Previous CHMP Meeting Highlights can be accessed at: https://www.basg.gv.at/en/healthcare-professionals/chmp-highlights
