CHMP Meeting Highlights October 2025
This month, medicinal products for the following indications have received a positive opinion:
- non-cystic fibrosis bronchiectasis
- immune thrombocytopenia
New medicines recommended for approval:
Brinsupri (brensocatib): has received a positive opinion for the treatment of non-cystic fibrosis bronchiectasis (NCFB) in patients 12 years of age and older with two or more exacerbations in the prior 12 months.
NCFB is a chronic condition where the airways in your lungs become abnormally widened and damaged by repeated infections and inflammation. This makes it hard to clear mucus, leading to chronic infections, coughing, and shortness of breath. It can by triggered by several causes including respiratory infections, autoimmune diseases and immunodeficiency disorders.
An underlying cause is the repeated activation of neutrophils. Neutrophils are a type of white blood cells and release a protein called neutrophil serine protease (NSP). NSP causing the airway wall damage, excessive mucus, sustained inflammation and impaired functioning of the immune system. Brinspuri prevents the neutrophil activation by inhibiting the dipeptidyl peptidase 1 (DPP1).
Brinsupri was reviewed under EMA’s accelerated assessment programme. 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 Brinsupri on the EMA website.
Wayrilz (rilzabrutinib): has received a positive opinion for the treatment of immune thrombocytopenia (ITP) in adult patients who are refractory to other treatments.
ITP is an autoimmune disease in which the immune system attacks the platelets. This leads to a low platelet count and an increased bleeding risk. Wayrilz is a Bruton's tyrosine kinase (BTK) inhibitor and modulates the immune system by inhibiting B-cell activation. An orphan designation was granted for the treatment of this disease. For more information please consult the product for Wayrilz on the EMA website.
Recommendations on extensions of therapeutic indication:
Breyanzi (lisocabtagene maraleucel): extension of indication for Breyanzi to include the treatment adult patients with relapsed or refractory mantle cell lymphoma (MCL) after at least two lines of systemic therapy including a Bruton’s tyrosine kinase (BTK) inhibitor. Breyanzi is already authorized for the treatment of other types of lymphoma. For more information please consult the product for Breyanzi on the EMA website.
Cejemly (sugemalimab): extension of indication for Cejemly to include the treatment of unresectable stage III non-small cell lung cancer (NSCLC) with no sensitising EGFR mutations, or ALK, ROS1 genomic tumour aberrations in adults whose tumours express PD-L1 on ≥ 1% of tumour cells and whose disease has not progressed following platinum-based chemoradiotherapy. Cejemly is already authorised for the treatment of NSCLC in combination with platinum-based chemotherapy. For more information please consult the product for Cejemly on the EMA website.
Gazyvaro (obinutuzumab): extension of indication for Gazyvaro to include the treatment of adult patients with active Class III or IV, with or without concomitant Class V, lupus nephritis (LN) in combination with mycophenolate mofetil. Gazyvaro is already authorised for the treatment of Chronic lymphocytic leukaemia (CLL) and Follicular lymphoma (FL). For more information please consult the product for Gazyvaro on the EMA website.
Libtayo (cemiplimab): extension of indication for Libtayo to include the adjuvant treatment of adult patients with cutaneous squamous cell carcinoma (CSCC) at high risk of recurrence after surgery and radiation. Libtayo is already authorised for the treatment of different types of cancer. For more information please consult the product for Libtayo on the EMA website.
Paxlovid (nirmatrelvir / ritonavir): extension of indication for Paxlovid to include the treatment of coronavirus disease 2019 (COVID-19) in adults and paediatric patients 6 years of age and older weighing at least 20 kg who do not require supplemental oxygen and who are at increased risk for progressing to severe COVID-19. Previously Paxlovid was only authorised for the treatment of adults. For more information please consult the product for Paxlovid on the EMA website.
Pyrukynd (mitapivat): extension of indication for Pyrukynd to include the treatment of anaemia associated with transfusion-dependent and non-transfusion-dependent alpha- or beta-thalassaemia in adult patients. Pyrukynd is already authorised for the treatment of pyruvate kinase deficiency in adult patients. For more information please consult the product for Pyrukynd on the EMA website.
Scemblix (asciminib): extension of indication for Scemblix to include the treatment of adult patients with Philadelphia chromosome-positive chronic myeloid leukaemia in chronic phase (Ph+ CML-CP). Previously Scemblix was only authorised for the treatment of Ph+ CML-CP after two or more tyrosine kinase inhibitors. For more information please consult the product for Scemblix on the EMA website.
Tremfya (guselkumab): extension of indication for Tremfya to include the treatment of moderate to severe plaque psoriasis in children and adolescents from the age of 6 years who are candidates for systemic therapy. Tremfya is already authorised for the treatment of plaque psoriasis in adult patients. For more information please consult the product for Tremfya 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:
Alyftrek: tablets are indicated for the treatment of cystic fibrosis (CF) in people aged 6 years and older who have at least one non-Class I mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. EPAR Alyftrek.
Ekterly: is indicated for symptomatic treatment of acute attacks of hereditary angioedema (HAE) in adults and adolescents aged 12 years and older. EPAR Ekterly.
Maapliv: is indicated for the treatment of maple syrup urine disease (MSUD) presenting with an acute decompensation episode in patients from birth who are not eligible for an oral and enteral branched chain amino acids free (BCAA- free) formulation. EPAR Maapliv.
Ogsiveo: as monotherapy is indicated for the treatment of adult patients with progressing desmoid tumours who require systemic treatment. EPAR Ogsiveo.
Riulvy: is indicated for the treatment of adult and paediatric patients aged 13 years and older with relapsing remitting multiple sclerosis (RRMS). EPAR Riulvy.
Romvimza: is indicated for 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. EPAR Romvimza.
Tryngolza: is indicated as an adjunct to diet in adult patients for the treatment of genetically confirmed familial chylomicronemia syndrome (FCS). EPAR Tryngolza.
Voranigo: as monotherapy is indicated 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. EPAR Voranigo.
Yeytuo: injection is indicated in combination with safer sex practices for pre-exposure prophylaxis (PrEP) to reduce the risk of sexually acquired HIV-1 infection in adults and adolescents with increased HIV-1 acquisition risk, weighing at least 35 kg. EPAR Yeytuo.
Zemcelpro: is indicated for the treatment of adult patients with haematological malignancies requiring an allogeneic haematopoietic stem cell transplantation following myeloablative conditioning for whom no other type of suitable donor cells is available. EPAR Zemcelpro.
Zurzuvae: is indicated for the treatment of postpartum depression (PPD) in adults following childbirth. EPAR Zurzuvae.
Previous CHMP Meeting Highlights can be accessed at: https://www.basg.gv.at/en/healthcare-professionals/chmp-highlights
