CHMP Meeting Highlights April 2026 New
This month, medicinal products for the following indications have received a positive opinion:
- Multiple sclerosis
- 5q spinal muscular atrophy
- familial chylomicronaemia syndrome
New medicines recommended for approval:
Cenrifki (Tolebrutinib): has received a positive opinion for the treatment of adult patients with secondary progressive multiple sclerosis (SPMS) without relapses in the last 2 years.
Multiple sclerosis is a chronic neurodegenerative autoimmune disease of the central nervous system, which leads to irreversible decrease in physical and cognitive function. SPMS is the later stage of MS after the relapsing-remitting multiple sclerosis (RRMS) form.
It is characterized by a steady progression of the disease and approx. 50% of RRMS patient transition into the SPMS stage within 20 years of disease onset. Cenrifki is a brain penetrant Bruton’s tyrosine kinase inhibitor. This reduces the inflammatory BTK activity of B-cell, macrophages and microglia in the central nervous system. For more information please consult the product for Cenrifki on the EMA website.
Itvisma (onasemnogene abeparvovec): has received a positive opinion for the treatment of 5q spinal muscular atrophy (SMA) with a bi-allelic mutation in the SMN1 gene in patients 2 years of age and older.
Spinal muscular atrophy is an autosomal recessive neurodegenerative disease that primarily affects motor neurons in the spinal cord and brainstem. It is caused by mutations in the survival motor neuron (SMN) 1 gene, leading to reduced levels of the SMN protein essential for motor neuron survival. The clinical spectrum ranges from severe infantile onset (type I) to milder adult-onset forms (type IV), with symptoms including progressive muscle weakness, loss of motor function, and, in the most severe cases, respiratory failure.
Itvisma is a non-replicating recombinant AAV vector that utilizes AAV9 capsid to deliver a stable, fully functional human SMN1 transgene. The alternative source of SMN1 gene leads to an increased expression of SMN protein and a better survival and function of transduced motor neurons. Itvisma is an advanced therapy medicinal product (ATMP) and an orphan designation was granted for the treatment of this disease. For more information please consult the product for Itvisma on the EMA website.
Redemplo (plozasiran): has received a positive opinion as an adjunct to diet to reduce triglyceride levels in adult patients with familial chylomicronaemia syndrome (FCS) in whom response to diet and triglyceride lowering therapy has been inadequate.
Familial chylomicronemia syndrome or lipoprotein lipase (LPL) deficiency is a rare inherited disorder of triglyceride metabolism caused by genetic mutations that impair lipoprotein lipase activity. It results in extremely high triglyceride levels and an excess of lipoprotein particles in the bloodstream. Patients may develop eruptive xanthomas, abdominal pain, pancreatitis, and lipemia retinalis.
Redemplo is a small interfering RNA against apolipoprotein C-III-mRNA leading to reduced apolipoprotein C-III (ApoC3) expression. This reduces the ApoC3-mediated inhibition of LPL allowing increased peripheral LPL activity. It also enhances the clearance of triglyceride-rich lipoproteins by the liver.
An orphan designation was granted for the treatment of this disease and a news announcement was released by the EMA for this product. For more information please consult the product for Redemplo on the EMA website.
Recommendations on extensions of therapeutic indication:
Agamree (vamorolone): extension of indication for Agamree to include the treatment of Duchenne muscular dystrophy (DMD) in patients aged 2 years and older. Previously the age limit was 4 years of age. For more information please consult the product for Agamree on the EMA website.
Aquipta (atogepant): extension of indication for Aquipta to include the acute treatment of migraine with or without aura in adults. Aquipta is already authorised for the prophylaxis of migraine. For more information please consult the product for Aquipta on the EMA website.
Comirnaty (COVID-19 mRNA vaccine): change of the indication for all Comirnaty 10 micrograms formulations. Comirnaty dispersion for injection is indicated for active immunisation to prevent COVID-19 caused by SARS-CoV-2 in individuals 6 months of age and older. Previously the age limit for these formulations was 5 years. For more information please consult the product for Comirnaty on the EMA website.
Crysvita (burosumab): extension of indication for Crysvita to include treatment of X-linked hypophosphataemia in infants from 1 month to 1 year of age with hypophosphatemia, in children and adolescents aged 1 to 17 years with radiographic evidence of bone disease, and in adults. Previously the age limit was 1 year. For more information please consult the product for Crysvita on the EMA website.
Inaqovi (cedazuridine, decitabine): extension of indication for Inaqovi in combination with venetoclax to include the treatment of adult patients with newly diagnosed acute myeloid leukaemia (AML) who are ineligible for standard induction chemotherapy. Inaqovi is already authorised for the treatment of this disease as monotherapy. For more information please consult the product for Inaqovi on the EMA website.
Opdivo (nivolumab): extension of indication for Opdivo to include in combination with doxorubicin, vinblastine and dacarbazine (AVD) the treatment of adults and adolescents 12 years of age and older with previously untreated Stage III or IV classical Hodgkin lymphoma. Opdivo is already authorised for the treatment of different types of cancer. For more information please consult the product for Opdivo on the EMA website.
Privigen (human normal immunoglobulin): extension of indication for Privigen to include measles pre-/post-exposure prophylaxis for susceptible adults, children and adolescents (0 to 18 years) in whom active immunisation is contraindicated or not advised. Privigen is approved for the replacement therapy in adult and paediatric patients with Primary immunodeficiencies (PID) and Secondary immunodeficiencies (SID). For more information please consult the product for Privigen on the EMA website.
Skyrizi (risankizumab): extension of indication for Skyrizi 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. Previously it was only authorised in adults. For more information please consult the product for Skyrizi on the EMA website.
Venclyxto (venetoclax): extension of indication for Venclyxto to include the treatment of adult patients with previously untreated chronic lymphocytic leukaemia (CLL):
- in combination with ibrutinib
- in combination with acalabrutinib with or without obinutuzumab
Venclyxto is already authorised for the treatment of previously untreated CLL in combination with obinutuzumab. For more information please consult the product for Venclyxto 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:
Acoziborole Winthrop: is indicated for the treatment of both first-stage (hemo-lymphatic) and second stage (meningo-encephalitic), including severe second-stage with ≥ 100 White Blood Cell (WBC)/μL with or without trypanosomes in cerebrospinal fluid (CSF), human African trypanosomiasis (HAT) due to Trypanosoma brucei gambiense (g-HAT) in adolescents ≥ 12 years old with body weight ≥ 40 kg, and in adults. EPAR Acoziborole Winthrop.
Fylrevy: Hormone replacement therapy (HRT) for oestrogen deficiency symptoms in hysterectomised postmenopausal women. Hormone replacement therapy (HRT) for oestrogen deficiency symptoms in non-hysterectomised postmenopausal women with at least 12 months since last menses. EPAR Fylrevy.
Kayshild: is indicated in conjunction with diet and exercise for the treatment of adults with non cirrhotic metabolic dysfunction-associated steatohepatitis (MASH) with moderate to advanced liver fibrosis (fibrosis stages F2 to F3). EPAR Kayshild.
Kygevvi: is indicated for the treatment of paediatric and adult patients with genetically confirmed thymidine kinase 2 deficiency (TK2d) with an age of symptom onset on or before 12 years. EPAR Kygevvi.
Rezurock: is indicated for the treatment of adults and paediatric patients (12 years and older with a body weight of at least 40 kg) with chronic graft-versus-host disease (cGVHD) when other treatment options provide limited clinical benefit, are not suitable or have been exhausted. EPAR Rezurock.
Supemtek: is indicated for active immunisation for the prevention of influenza disease in adults and children from 9 years of age and older. EPAR Supemtek.
Previous CHMP Meeting Highlights can be accessed at: https://www.basg.gv.at/en/healthcare-professionals/chmp-highlights
