CHMP Meeting Highlights March 2026 New
This month, medicinal products for the following indications have received a positive opinion:
- non-muscle invasive bladder cancer
- extensive-stage small cell lung cancer
- oedema
- activated phosphoinositide 3 kinase delta syndrome
New medicines recommended for approval:
Adstiladrin (Nadofaragene firadenovec): has received a positive opinion for the conditional marketing authorisation as monotherapy for the treatment of adult patients with Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumours.
Non-muscle invasive bladder cancer (NMIBC) is cancer that has not spread through the bladder wall into the muscle layer. It's the most common type of bladder cancer, typically appearing as flat, early-stage tumours. Adstiladrin is a non-replicating recombinant type 5 adenovirus vector-based gene therapy containing the human IFNα2b transgene. IFNα2b encodes the cytokine interferon α-2b. The virus particles are delivered to the tumour via intravesical instillation, penetrate it, and lead to the expression of interferon. Interferon α reduces tumour growth and activates the immune system. For more information please consult the product for Adstiladrin on the EMA website.
Bopediat (Furosemide): has received a positive opinion for the treatment in children from birth to less than 18 years of oedema of cardiac or renal origin, oedema of hepatic origin, and hypertension in patients with chronic kidney disease.
Oedema or fluid retention is the build-up of water in the body. It can have different reasons like venous insufficiency, heart failure or low protein levels. Furosemide is a loop diuretic and hinders the reabsorption of sodium ions in kidney. This leads to an increased excretion of water. For more information please consult the product for Bopediat on the EMA website.
Imdylltra (Tarlatamab): has received a positive opinion as monotherapy for the treatment of adult patients with extensive-stage small cell lung cancer (ES-SCLC), who require systemic therapy following disease progression on or after first-line treatment with platinum-based chemotherapy. 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.
Imdylltra is a monoclonal antibody and a bispecific T-cell engager (BiTE). It binds to both T cells via the CD3 binding site and delta-like ligand 3 (DLL3)-expressing cancer cells via its DLL3 binding site. Tarlatamab-mediated T-cell activation leads to the release of pro-inflammatory cytokines and T-cell-mediated tumor cell killing. An orphan designation was granted for the treatment of this disease and a news announcement was release by the EMA for this product. For more information please consult the product for Imdylltra on the EMA website.
Joenja (Leniolisib): has received a positive opinion under exceptional circumstances for the treatment of activated phosphoinositide 3 kinase delta syndrome (APDS) in adults and adolescents 12 years of age and older and weighing 45 kg or more.
APDS is a rare, primary immunodeficiency caused by mutations leading to overactive PI3Kδ signalling. This results in immune system dysfunction, causing frequent infections, enlarged lymph nodes, and an increased risk of lymphoma, alongside potential autoimmune features. It's typically diagnosed in childhood and requires lifelong management to control symptoms and complications. Joenja is an inhibitor of the mutated PI3K and downregulate the overactive pathway. An orphan designation was granted for the treatment of this disease. For more information please consult the product for Joenja on the EMA website.
Zepzelca (Lurbinectedin): has received a positive opinion for the maintenance treatment in combination with atezolizumab of adult patients with extensive-stage small cell lung cancer (ES-SCLC) whose disease has not progressed after first-line induction therapy with atezolizumab, carboplatin and etoposide.
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. Zepzelca is an alkylating agent and binds to GC-rich regions in the DNA. This leads to a hindered protein biosynthesis and cell death. An orphan designation was granted for the treatment of this disease. For more information please consult the product for Zepzelca on the EMA website.
Recommendations on extensions of therapeutic indication:
Besponsa (Inotuzumab ozogamicin): extension of indication to include the treatment as monotherapy in paediatric patients 1 year and older with CD22-positive B cell precursor ALL:
- in first relapse after allo-haematopoietic stem cell transplant (HSCT);
- after any first relapse in patients with Very High Risk (VHR) disease;
- after a second or greater relapse;
- and in those with refractory disease.
Patients with Philadelphia chromosome positive (Ph+) disease should have exhausted relevant BCR-ABL targeting treatment options. Besponsa is already authorised for the treatment of adult patients with CD22-positive B cell precursor ALL. For more information please consult the product for Besponsa on the EMA website.
Capvaxive (Pneumococcal polysaccharide conjugate vaccine (21-valent)): extension of indication to include the active immunisation for the prevention of invasive disease and pneumonia caused by Streptococcus pneumoniae in children and adolescents 2 to less than 18 years of age who previously completed a primary paediatric pneumococcal vaccination regimen. Previously it was only authorised for the immunisation of adults. For more information please consult the product for Capvaxive on the EMA website.
Feraccru (Ferric mannitol): extension of indication to include the treatment of iron deficiency in adults and adolescents aged 12 years and older. Previously it was only authorised for the treatment of adults. For more information please consult the product for Feraccru on the EMA website.
Hetronifly (Serplulimab): extension of indication to include the first-line treatment in combination with carboplatin and pemetrexed adult non-squamous NSCLC patients with no EGFR, ALK or ROS1 positive mutations and who have:
- locally advanced NSCLC who are not candidates for surgery or radiotherapy, or
- metastatic NSCLC.
Hetronifly also received a positive opinion for the extension of indication to include the first-line treatment in combination with fluoropyrimidine- and platinum-based chemotherapy of adult patients with unresectable, locally advanced/recurrent or metastatic oesophageal squamous cell carcinoma whose tumours express PD-L1 with a CPS ≥ 5. Hetronifly is already authorised in combination with carboplatin and etoposide for the first-line treatment of adult patients with extensive-stage small cell lung cancer (ES-SCLC). For more information please consult the product for Hetronifly on the EMA website.
Hympavzi (Marstacimab): extension of indication to include the routine prophylaxis of bleeding episodes in patients 12 years of age and older, weighing at least 35 kg, with:
- Haemophilia A (congenital factor VIII deficiency):
- Without factor VIII inhibitors who have severe disease (FVIII < 1%),
- With factor VIII inhibitors
- Haemophilia B (congenital factor IX deficiency):
- Without factor IX inhibitors who have severe disease (FIX < 1%).
- With factor IX inhibitors
Previously it was only authorised for the treatment of severe haemophilia A and B without inhibitors. For more information please consult the product for Hympavzi on the EMA website.
Imcivree (Setmelanotide): extension of indication to include the treatment of obesity and the control of hunger in adults and children 4 years of age and above with acquired hypothalamic obesity (aHO) due to hypothalamic injury or impairment.
Imcivree is already authorised for the treatment of obesity and the control of hunger associated with genetically confirmed Bardet-Biedl syndrome (BBS), loss-of-function biallelic pro-opiomelanocortin (POMC), including PCSK1, deficiency or biallelic leptin receptor (LEPR) deficiency in adults and children 2 years of age and above. For more information please consult the product for Imcivree on the EMA website.
Lojuxta (Lomitapide): extension of indication to include as an adjunct to a low-fat diet and other lipid-lowering medicinal products with or without low density lipoprotein (LDL) apheresis the treatment of adult and paediatric patients aged 5 years and older with homozygous familial hypercholesterolaemia (HoFH). Previously it was only authorised for the treatment of adults. For more information please consult the product for Lojuxta on the EMA website.
Mekinist (Trametinib): extension of indication to include the treatment as monotherapy or in combination with dabrafenib of adults and adolescents aged 12 years and older with unresectable or metastatic melanoma with a BRAF V600 mutation. Mekinist also received a positive opinion for the extension of indication to include in combination with dabrafenib the adjuvant treatment of adults and adolescents aged 12 years and older with Stage III melanoma with a BRAF V600 mutation, following complete resection.
Previously it was only authorised for the treatment of adults. Mekinist also received a positive opinion for the extension of indication to include in combination with dabrafenib the treatment of adult patients with locally advanced or metastatic differentiated thyroid cancer with a BRAF V600E mutation, refractory to or not eligible for radioactive iodine (RAI) who have progressed during or after prior systemic therapy. For more information please consult the product for Mekinist on the EMA website.
mResvia (Respiratory syncytial virus mRNA vaccine (nucleoside modified)): extension of indication to include the active immunisation for the prevention of lower respiratory tract disease (LRTD) caused by Respiratory Syncytial Virus (RSV) in adults 18 years of age and older. Previously it was only authorised only for the immunisation of adults over 60 years or adults at increased risk for LRTD. For more information please consult the product for mResvia on the EMA website.
Namuscla (Mexiletine): extension of indication to include the treatment of myotonia in patients with non-dystrophic myotonic disorders. Namuscla is indicated in adults, children and adolescents aged 6 years and older. Previously it was only authorised for the treatment of adults. For more information please consult the product for Namuscla on the EMA website.
Retsevmo (Selpercatinib): extension of indication to include the treatment of paediatric patients 2 years and older with:
- Advanced RET fusion-positive thyroid cancer who are radioactive iodine-refractory,
- Advanced RET-mutant medullary thyroid cancer,
- Advanced RET fusion-positive solid tumours, when treatment options not targeting RET provide limited clinical benefit, or have been exhausted, patients who harbour a RET fusion-positive solid tumor, i.e. agnostic indication.
Previously the age cut-off was 12 years of age for thyroid cancer and adulthood for the tumour agnostic indication. Sotyktu (Deucravacitinib): extension of indication to include alone or in combination with methotrexate (MTX), the treatment of active psoriatic arthritis (PsA) in adults who have had an inadequate response to, or who have been intolerant of, prior DMARD therapy. Sotyktu is already authorised for the treatment of moderate to severe plaque psoriasis in adults. For more information please consult the product for Retsevmo on the EMA website.
Tafinlar (Dabrafenib): extension of indication to include the treatment as monotherapy or in combination with dabrafenib of adults and adolescents aged 12 years and older with unresectable or metastatic melanoma with a BRAF V600 mutation.
Tafinlar also received a positive opinion for the extension of indication to include in combination with dabrafenib the adjuvant treatment of adults and adolescents aged 12 years and older with Stage III melanoma with a BRAF V600 mutation, following complete resection. Previously it was only authorised for the treatment of adults. For more information please consult the product for Tafinlar 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:
Anktiva: in combination with Bacillus Calmette-Guérin (BCG) is indicated for the treatment of adult patients with BCG-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumours. EPAR Anktiva.
Exdensur: is indicated as add-on maintenance treatment for severe asthma with type 2 inflammation characterised by blood eosinophil count in adults and adolescents 12 years and older who are inadequately controlled despite high dose inhaled corticosteroids (ICS) plus another asthma controller. EPAR Exdensur.
Previous CHMP Meeting Highlights can be accessed at: https://www.basg.gv.at/en/healthcare-professionals/chmp-highlights
