CHMP Meeting Highlights May 2025 New
This month, medicinal products for the following indications have received a positive opinion:
- Acute lymphoblastic leukaemia
- Multiple myeloma
- Neurofibromatosis type 1
- Breast cancer
- Maple syrup urine disease
- Multiple sclerosis
New medicines recommended for approval:
Aucatzyl (obecabtagene autoleucel): has received a positive opinion for the conditional marketing authorisation for the treatment of adult patients 26 years of age and above with relapsed or refractory (r/r) B cell precursor acute lymphoblastic leukaemia (B ALL).
An orphan designation was granted for the treatment of this disease. Aucatzyl was supported through EMA‘s Priority Medicines (PRIME) scheme. The EMA has published a press release for this medicine.
ALL is a type of blood cancer impacting the white blood cells, in which large numbers of immature lymphocytes are developed. This reduces the ability of the bone marrow to produce other types of blood cells. Obecabtagene autoleucel is a chimeric antigen receptor T (CAR-T) cell therapy. T Cells – a type of white blood cells - are harvested from the patient and genetically modified to identify and attack the cancer cells. For more information please consult the product for Aucatzyl on the EMA website.
Blenrep (belantamab mafodotin): has received a positive opinion for the treatment of relapsed or refractory multiple myeloma in combination with bortezomib and dexamethasone in patients who have received at least one prior therapy.
It received also a positive opinion for the treatment of relapsed or refractory multiple myeloma in combination with pomalidomide and dexamethasone in patients who have received at least one prior therapy including lenalidomide.
An orphan designation was granted for the treatment of this disease. Multipe myeloma is a type of blood cancer characterised by the malignant proliferation of plasma cells that consistently express the B-cell maturation antigen (BCMA). BCMA is not expressed in normal non-haematopoietic cells. For more information please consult the product for Blenrep on the EMA website.
Ezmekly (mirdametinib): has received a positive opinion for the conditional marketing authorisation for the treatment as monotherapy of symptomatic, inoperable plexiform neurofibromas (PN) in paediatric and adult patients with neurofibromatosis type 1 (NF1) aged 2 years and above. An orphan designation was granted for the treatment of this disease.
Neurofibromatosis type 1 is a neurocutaneous autosomal dominant genetic disorder affecting multiple organs. Neurofibromas are a type of benign tumour of peripheral nerves, which are difficult to remove because they encase the nerve. For more information please consult the product for Ezmekly on the EMA website.
Itovebi (inavolisib): has received a positive opinion for the treatment in combination with palbociclib and fulvestrant, of adult patients with PIK3CA mutated, oestrogen receptor (ER) positive, HER2 negative, locally advanced or metastatic breast cancer, following recurrence on or within 12 months of completing adjuvant endocrine treatment.
Patients previously treated with a CDK 4/6 inhibitor in the (neo)adjuvant setting should have had an interval of at least 12 months between termination of CDK 4/6 inhibitor treatment and the detection of recurrence. In pre/perimenopausal women and in men, endocrine therapy should be combined with a luteinising hormone releasing hormone (LHRH) agonist. For more information please consult the product for Itovebi on the EMA website.
Maapliv (amino acids): has received a positive opinion for marketing authorisation under exceptional circumstances 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.
An orphan designation was granted for the treatment of this disease. MSUD is a rare metabolic disorder in which the patient can’t properly break down branched-chain amino acids. This leads to toxic levels of alpha-ketoacids. Maapliv is a branched-chain amino acid free mix of other amino acids for parenteral use. For more information please consult the product for Maapliv on the EMA website.
Riulvy (tegomil fumarate): has received a positive opinion for the treatment of adult and paediatric patients aged 13 years and older with relapsing remitting multiple sclerosis (RRMS).
Multiple sclerosis is a chronic neurodegenerative autoimmune disease of the central nervous system, which leads to irreversible decrease in physical and cognitive function. For more information please consult the product for Riulvy on the EMA website.
Recommendations on extensions of therapeutic indication:
Imfinzi (durvalumab): extension of indication for Imfinzi to include the treatment of adults with resectable muscle invasive bladder cancer in combination with gemcitabine and cisplatin as neoadjuvant treatment, followed by Imfinzi as monotherapy adjuvant treatment after radical cystectomy. Imfinzi is already authorised for different types of cancer. For more information please consult the product for Imfinzi on the EMA website.
Rezolsta (darunavir / cobicistat): extension of indication for Rezolsta to include in combination with other antiretroviral medicinal products, the treatment of human immunodeficiency virus 1 (HIV 1) infection in adults and paediatric patients (aged 6 years and older, weighing at least 25 kg). Previously the age limit was 12 years weighing at least 40 kg. For more information please consult the product for Rezolsta on the EMA website.
Saxenda (liraglutide): extension of indication for Saxdena to include as an adjunct to healthy nutrition and increased physical activity for weight management in children from the age of 6 to <12 years with
- obesity (Body Max Index -BMI- ≥95th percentile) and
- body weight ≥45 kg
Treatment with Saxenda should be discontinued and re-evaluated if patients have not lost at least 4% of their BMI or BMI z score after 12 weeks on the 3.0 mg/day or maximum tolerated dose.
Saxenda is already authorised for weight management in adolescents over 12 years of age and adults. For more information please consult the product for Saxenda on the EMA website.
Tevimbra (tislelizumab): extension of indication for Tevimbra to include in combination with platinum and fluoropyrimidine-based chemotherapy, the first-line treatment of adult patients with HER-2-negative locally advanced unresectable or metastatic gastric or gastroesophageal junction (G/GEJ) adenocarcinoma whose tumours express PD L1 with a tumour area positivity (TAP) score ≥ 5%.
Tevimbra is already approved for the treatment of Non-small cell lung cancer and Oesophageal squamous cell carcinoma. For more information please consult the product for Tevimbra on the EMA website.
Other topics:
The CHMP finished an Article 31 referral procedure for Azithromycin-containing medicinal products for systemic use. The CHMP recommends changing the approved indications to the treatment of the following infections in adults and adolescents weighing at least 45 kg:
- Acute streptococcal tonsillitis and pharyngitis
- Acute bacterial sinusitis
- Acute bacterial otitis media
- Community-acquired pneumonia (CAP)
- Acute bacterial skin and skin structure infections (ABSSSI)
- Erythema migrans (early localised Lyme disease)
- Periodontal abscesses and periodontitis
- Urethritis and cervicitis caused by Chlamydia trachomatis
- Urethritis and cervicitis caused by Neisseria gonorrhoeae, in combination with another appropriate antibacterial agent (e.g. ceftriaxone)
- Chronic prostatitis caused by Chlamydia trachomatis
- Chancroid
- Disseminated Mycobacterium avium complex (DMAC) infection in people living with advanced HIV infection, in combination with ethambutol
Azithromycin is also indicated for the prophylaxis of Mycobacterium avium complex (MAC) infection in people living with HIV with inadequate immune restoration.
Azithromycin is indicated for the treatment of adult patients with acute exacerbation of chronic bronchitis or with pelvic inflammatory disease, the latter always in combination with other appropriate antibacterial agent(s) (e.g. metronidazole).
Consideration should be given to official guidance on the appropriate use of antibacterial agents.
The CHMP recommends removing the indications for:
- gastro-duodenal infections caused by Helicobacter pylori
- Treatment of (moderate) acne vulgaris
- Prevention of exacerbations of eosinophilic and non-eosinophilic asthma
The EMA has published a press release for this referral procedure.
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:
Datroway: as monotherapy is indicated for the treatment of adult patients with unresectable or metastatic hormone receptor (HR)-positive, HER2-negative breast cancer who have received endocrine therapy and at least one line of chemotherapy in the advanced setting. EPAR Datroway.
Ivermectin/Albendazole: orodispersible tablets are indicated in adults, adolescents and children ≥ 5 years of age for the treatment of:
- Soil-transmitted helminth infections, caused by one or more of the following parasites (see section 5.1): hookworm (Ancylostoma duodenale, Necator americanus), roundworm (Ascaris lumbricoides), whipworm (Trichuris trichiura) and Strongyloides stercoralis.
- Proven or suspected microfilaraemia in patients with lymphatic filariasis caused by Wuchereria bancrofti.
Vyjuvek: is indicated for the treatment of wounds in patients with dystrophic epidermolysis bullosa (DEB) with mutation(s) in the collagen type VII alpha 1 chain (COL7A1) gene, from birth. EPAR Vyjuvek.
Previous CHMP Meeting Highlights can be accessed at: https://www.basg.gv.at/en/healthcare-professionals/chmp-highlights
