CHMP Meeting Highlights September 2026 New
This month, medicinal products for the following indications have received a positive opinion:
- Haemophilia A
- Non-small cell lung cancer
- Generalised myasthenia gravis
- Ovarian cancer
- Hidradenitis suppurativa
- Prophylaxis against rabies
- Short Bowel Syndrome
New medicines recommended for approval:
Frehemgo (denecimig): has received a positive opinion for the routine prophylaxis of bleeding episodes in patients with haemophilia A (congenital factor VIII deficiency):
- with FVIII inhibitors
- without FVIII inhibitors who have,
- severe disease (FVIII < 1%)
- moderate disease (FVIII ≥ 1% and ≤ 5%) requiring prophylaxis treatment.
Haemophilia A is a disease caused by mutations in the genes coding for the coagulation factor VIII.It is characterised by the inability to form blood clots, which results in a higher risk of bruising, internal bleeding and bleeding inside the joints. The disease can be classified as mild, moderate or severe, depending on the endogenous plasma activity levels of clotting factors.
Frehemgo is a bispecific antibody that bind to activated coagulation factors IX and X on the surface of platelets mimicking the missing factor VIII. For more information please consult the product for Frehemgo on the EMA website.
Gevalka (ensartinib): has received a positive opinion for the treatment of adult patients with anaplastic lymphoma kinase (ALK)-positive advanced non-small cell lung cancer (NSCLC) previously not treated with an ALK inhibitor. It also received as positive opinion for the treatment of adult patients with ALK-positive advanced NSCLC previously treated with crizotinib.
NSCLC is a serious and often fatal disease that accounts for 80 to 85% of all lung cancers. A significant number of patients present with driver mutations in oncogenes, some of which can be aimed at by targeted therapies. Patients without known driver mutations need broader-acting therapies. For more information please consult the product for Gevalka on the EMA website.
Klygefa (gefurulimab): has received a positive opinion as add-on to standard therapy for the treatment of generalised myasthenia gravis (gMG) in adults who are anti-acetylcholine receptor (AChR) antibody positive.
gMG is a rare autoimmune disorder that causes muscle weakness. Around 90% of patients have detectable levels of autoantibodies in serum and the most common target AChR at the neuromuscular junction (NMJ), leading to failure of neuromuscular transmission. Klygefa is an immunosuppresant nanobody which targets and blocks the C5 complement protein. For more information please consult the product for Klygefa on the EMA website.
Lifyorli (relacorilant): has received a positive opinion in combination with nab-paclitaxel for the treatment of adult patients with platinum-resistant high-grade epithelial ovarian, fallopian tube, or primary peritoneal cancer who have received one to three prior systemic treatment regimens.
Ovarian epithelial, fallopian tube, or primary peritoneal cancer are a group of cancers which arise in the lining of the above-listed organs. As they derive from the same cells and require similar treatment strategies, they are summarised as epithelial ovarian cancer. Survival rates of epithelial ovarian cancer have increased slightly over the past decade, however it is often diagnosed at an advanced stage which correlates with a poor prognosis.
Lifyorli is glucocorticoid receptor (GR) antagonist. Activation of the GR pathway in cancer cells increases the expression of anti-apoptotic proteins. By blocking this pathway, the cells are more susceptible for chemotherapy. An orphan designation was granted for the treatment of this disease. For more information please consult the product for Lifyorli on the EMA website.
Povofortay (povorcitinib): has received a positive opinion for the treatment of active moderate to severe hidradenitis suppurativa (acne inversa) in adults who have responded inadequately to, or who are intolerant or contraindicated to an anti-TNF therapy.
Hidradenitis suppurativa is a chronic inflammatory skin disorder that affects sweat glands and hair follicles, leading to painful nodules, abscesses primarily in intertriginous areas such as the armpits, groin, and buttocks. It often causes scarring, malodorous discharge, and can significantly impact quality of life if not managed properly.
Povofortay is a JAK1 (Janus kinase 1) inhibitor which is involved in proinflammatory signalling pathways. This inhibition leads to a downregulation of the immune and inflammatory responses elevating the symptoms. For more information please consult the product for Povofortay on the EMA website.
Sepalna (senaparib): has received a positive opinion for the maintenance treatment of adult patients with advanced (FIGO Stages III and IV) epithelial high-grade ovarian, fallopian tube or primary peritoneal cancer who are in response (complete or partial) following completion of first-line platinum-based chemotherapy.
Ovarian epithelial, fallopian tube, or primary peritoneal cancer are a group of cancers which arise in the lining of the above-listed organs. As they derive from the same cells and require similar treatment strategies, they are summarised as epithelial ovarian cancer. Survival rates of epithelial ovarian cancer have increased slightly over the past decade, however it is often diagnosed at an advanced stage which correlates with a poor prognosis.
Sepalna is a poly ADP ribose polymerase (PARP) inhibitor. PARPs are involved in many cellular processes including DNA repair and stability and apoptosis. Sepalna blocks DNA-associated PARPs, thereby inhibiting DNA repair and promoting the formation of double strand breaks in replicating cells. For more information please consult the product for Sepalna on the EMA website.
VaxRabeo (Rabies vaccine (inactivated) for human use): has received a positive opinion for pre-exposure and post-exposure prophylaxis against rabies in individuals of all age groups.
Rabies is an acute, progressive viral infection that targets the central nervous system, usually acquired from the bite or scratch of an infected animal. It leads to severe neurological symptoms such as confusion, agitation and seizures. Even with intensive care rabies is fatal once clinical signs appear. For more information please consult the product for VaxRabeo on the EMA website.
Zeydovio (glepaglutide): has received a positive opinion for the treatment of adults with Short Bowel Syndrome (SBS). Patients should be stable following a period of intestinal adaptation after surgery.
SBS is a malabsorptive disorder that occurs when large portions of the small intestine are surgically removed or become non‑functional, leaving insufficient length for adequate nutrient, fluid, and electrolyte absorption. The resulting symptoms include chronic diarrhea, weight loss, dehydration, and deficiencies in vitamins and minerals, often necessitating parenteral nutrition or specialized enteral formulas to maintain health.
Zeydovio is an analogue of the glucagon-like peptide-2 (GLP-2). It promotes intestinal mucosal growth, increases villus height and crypt depth, slows transit time, and enhances nutrient absorption. This reduces the reliance on parental support. For more information please consult the product for Zeydovio on the EMA website.
Recommendations on extensions of therapeutic indication:
Abiraterone Mylan (abiraterone acetate): extension of indication for Abiraterone Mylan to include the treatment of newly diagnosed mHSPC in adult patients in combination with ADT and docetaxel. It also received a positive opinion for the treatment of newly diagnosed high risk non-metastatic hormone sensitive prostate cancer (HSPC) in adult patients in combination with ADT and radiotherapy. Both new indications are in combination with prednisone or prednisolone. For more information please consult the product for Abiraterone Mylan on the EMA website.
Anzupgo (delgocitinib): extension of indication for Anzupgo to include the treatment of moderate to severe chronic hand eczema (CHE) in adults and adolescents 12 years and older for whom topical corticosteroids are inadequate or inappropriate. Previously it was only authorised for the treatment of adults. For more information please consult the product for Anzupgo on the EMA website.
Cosentyx (secukinumab): extension of indication for Cosentyx to include the treatment of polymyalgia rheumatica in adults who have had an inadequate response to glucocorticoids or who experience a relapse during glucocorticoid taper. Cosentyx is already authorised for the treatment of different auto-immune diseases. For more information please consult the product for Cosentyx on the EMA website.
Enhertu (trastuzumab deruxtecan): extension of indication for Enhertu to include the adjuvant treatment of adult patients with resected HER2-positive breast cancer who have residual invasive disease after neoadjuvant taxane-based and HER2-targeted treatment.
Enhertu is already authorised for the treatment of breast cancer in different settings and lines of treatment. For more information please consult the product for Enhertu on the EMA website.
Jyseleca (filgotinib): extension of indication for Jyseleca to include the treatment of active non-radiographic axial spondyloarthritis in adult patients with objective signs of inflammation as indicated by elevated C-reactive protein (CRP) and/or magnetic resonance imaging (MRI), who have responded inadequately to nonsteroidal anti-inflammatory drugs (NSAIDs).
It also received a positive opinion for the treatment of active ankylosing spondylitis (radiographic axial spondyloarthritis) in adult patients who have responded inadequately to conventional therapy. Jyselca is already authorised for the treatment of rheumatoid arthritis and ulcerative colitis. For more information please consult the product for Jyseleca on the EMA website.
Keytruda (pembrolizumab): extension of indication for Keytruda to include in combination with enfortumab vedotin as neoadjuvant treatment and then continued after radical cystectomy as adjuvant treatment, the treatment of adults with resectable muscle invasive bladder cancer (MIBC).
Previously it was only authorised for patients with MIBC who were ineligible for cisplatin containing chemotherapy. Keytruda is also authorised for the treatment of various types of cancer. For more information please consult the product for Keytruda on the EMA website.
Ocrevus (ocrelizumab): extension of indication for Ocrevus to include the treatment of adult patients and paediatric patients aged 10 years and older with relapsing forms of multiple sclerosis (RMS) with active disease defined by clinical or imaging features. Previously it was only authorised for the treatment of adults. For more information please consult the product for Ocrevus on the EMA website.
Padcev (enfortumab vedotin): extension of indication for Padcev to include in combination with pembrolizumab, as neoadjuvant treatment and then continued after radical cystectomy as adjuvant treatment, the treatment of adult patients with resectable muscle invasive bladder cancer (MIBC).
Previously it was only authorised for patients with MIBC who were ineligible for cisplatin containing chemotherapy. Padcev is also authorised for the treatment of metastatic urothelial cancer. For more information please consult the product for Padcev on the EMA website.
Pepaxti (melphalan flufenamide): extension of indication for Pepaxti in combination with dexamethasone to include the treatment of adult patients with multiple myeloma who have received at least two prior lines of therapies, whose disease is refractory to lenalidomide and the last line of therapy. For patients with a prior autologous stem cell transplantation, the time to progression should be at least 3 years from transplantation.
Previously it was authorised for the treatment of multiple myeloma after three prior lines of therapy. For more information please consult the product for Pepaxti on the EMA website.
Sogroya (somapacitan): extension of indication for Sogroya to include the treatment of Children with idiopathic short stature with persistent growth disturbance (current height SDS < -2.5, HV SDS < 0 during the last year and parental adjusted height SDS < -1).
Sogroya is already authorised for the treatment of growth hormone deficiency in paediatric and adult patients. For more information please consult the product for Sogroya on the EMA website.
Tecvayli (teclistamab): extension of indication for Tecvayli to include the treatment of adult patients with relapsed or refractory multiple myeloma, who have received at least one prior therapy.
Previously it was authorised for the treatment of relapsed or refractory multiple myeloma after three prior lines of therapy. For more information please consult the product for Tecvayli on the EMA website.
Conclusion of Referrals
Ipidacrine containing medicinal products
The CHMP concluded an article 31 referral on ipidacrine containing medicinal products. The committee concluded that the benefit-risk-balance remains positive but recommended to restrict and better define the approved indications.
The new indications are:
symptomatic treatment of acquired mononeuropathies and polyneuropathies and adjunctive symptomatic treatment of myasthenia gravis
adjunctive symptomatic treatment of bulbar dysfunction during the recovery period following ischaemic stroke affecting the brainstem
adjunctive symptomatic treatment of motor impairment during the recovery period following ischaemic or haemorrhagic stroke, or traumatic brain injury
symptomatic treatment of mild to moderately severe Alzheimer’s disease with or without vascular dementia
symptomatic treatment of intestinal atony
Sodium oxybate
The CHMP concluded on an article 31 referral on sodium oxybate . The committee concluded that the benefit-risk-balance remains positive for the treatment of acute alcohol withdrawal syndrome and to support long-term abstinence in people with alcohol dependence.
The CHMP recommended that the marketing authorisation holder should perform new studies to further characterise the efficacy of the medicines as there have been advances in scientific and methodological standards since the studies supporting the initial authorisations were carried out.
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:
Aujemflu: Prophylaxis of influenza in adults 50 years of age and older. Aujemflu should be used in accordance with official recommendations.
Boey: is indicated for the temporary improvement in the appearance of moderate to severe lines between the eyebrows seen at maximum frown (glabellar lines), when these have an important psychological impact in adult patients.
Colchicine AGEPHA Pharma: is indicated for secondary prevention of atherothrombotic events in adult patients with coronary disease stable for at least 6 months.
Daybu: is indicated for the treatment of neurobehavioural symptoms of Rett syndrome in adults and paediatric patients aged 5 years and older.
Hopledo: is indicated for the treatment of adult patients with Parkinson’s disease and moderate to severe motor fluctuations who have not been sufficiently stabilised with oral levodopa/dopa decarboxylase (DDC) inhibitor-based treatment regimens.
Icotyde: is indicated for the treatment of moderate to severe plaque psoriasis in adults, and adolescents 12 years of age and older and weighing at least 40 kg, who are candidates for systemic therapy.
Vijoice: is indicated for the treatment of adult and paediatric patients aged 2 years and older with severe or life-threatening manifestations of PIK3CA-related overgrowth spectrum (PROS) who require systemic therapy.
Zokovea: is indicated for the post-exposure prophylaxis of COVID-19 in adults and adolescents aged 12 years and older. Zokovea should be used in accordance with official recommendations.
Previous CHMP Meeting Highlights can be accessed at: https://www.basg.gv.at/en/healthcare-professionals/chmp-meeting-highlights-basg
