CHMP Meeting Highlights April 2023
This month, medicinal products for the following indications have received a positive opinion:
- Active immunisation against Respiratory Syncytial Virus
- Diffuse large B-cell lymphoma
- Mantle cell lymphoma
- Obstructive hypertrophic cardiomyopathy
- Pompe disease (acid α-glucosidase deficiency)
New medicines recommended for approval:
Arexvy (Respiratory Syncytial Virus (RSV): vaccine [recombinant, adjuvanted]): is indicated for active immunisation for the prevention of lower respiratory tract disease (LRTD) caused by respiratory syncytial virus in adults 60 years of age and older.
RSV is a single-stranded RNA virus that infects people of all ages but does not confer long-term immunity. In vulnerable individuals, such as older adults, re-infections could lead to more severe manifestations of the disease, such as LRTD. Arexvy includes an engineered version of the well conserved RSV F surface glycoprotein, which is required for the entry of the virus into the cell.
Arexvy is the first vaccine authorised to protect adults from RSV. For more information see the EMA news announcement on Arexvy. For more information please consult the product for Arexvy on the EMA website.
Camzyos (mavacamten): is indicated for the treatment of symptomatic (New York Heart Association, NYHA, class II-III) obstructive hypertrophic cardiomyopathy (oHCM) in adult patients.
HCM is a disease in which the heart muscle becomes thickened, impairing the blood-pumping and electrical conduction function of the heart. HCM is classified as obstructive when the outflow of blood from the left ventricle is obstructed. Camzyos selectively, reversibly and allosterically inhibits cardiac myosin, which is responsible for the contraction of the heart. For more information please consult the product for Camzyos on the EMA website.
Columvi (glofitamab): received a positive opinion for a conditional marketing authorisation (CMA) for the treatment, as monotherapy, of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL), after two or more lines of systemic therapy.
DLBCL is an aggressive cancer of the B-lymphocytes and it is the most common form of non-Hodgkin lymphoma. As many mature B-cell lymphomas, DLBCL express the surface antigen CD20. Columvi is a bispecific antibody that targets CD20 and CD3, a subunit of the T-cell receptor complex. Columvi binds CD3, thereby eliciting recruitment of effector T-cells. Upon concurrent binding with CD20, T-cell activation occurs, resulting in T-cell-mediated B-cell lysis. For more information please consult the product for Columvi on the EMA website.
Jaypirca (pirtobrutinib): received a positive opinion for a conditional marketing authorisation (CMA) for the treatment, as monotherapy, of adult patients with relapsed or refractory mantle cell lymphoma (MCL) who have been previously treated with a Bruton’s tyrosine kinase (BTK) inhibitor.
MCL is rare type of non-Hodgkin’s lymphoma. BTK is a soluble tyrosine kinas involved in the development and maturation of B cells, which has been shown to play an important role in the development of various B-cell cancers, such as MCL. Jaypirca is a highly selective small molecule non-covalent BTK inhibitor. For more information please consult the product for Jaypirca on the EMA website.
Lytgobi (futibatinib): received a positive opinion for a conditional marketing authorisation (CMA) for the treatment, as monotherapy, of adult patients with locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or rearrangement that have progressed after at least one prior line of systemic therapy.
CCA is a rare cancer of the bile duct with high genomic diversity. Recent next-generation sequence analyses have shown that many mutations typically found in other cancer types are also present in CCA, including fusions and rearrangements in FGFR2. Indeed, fusions in the FGFR2 gene have been found in around 10 to 15 % of intrahepatic CCA. Lytgobi is a small molecule covalent tyrosine kinase inhibitor that irreversibly binds to and inhibits FGFR1/2/3/4. For more information please consult the product for Lytgobi on the EMA website.
Opfolda (miglustat): is an enzyme stabiliser of cipaglucosidase alfa long-term enzyme replacement therapy in adults with late-onset Pompe disease (acid α-glucosidase deficiency).
Pompe disease is an autosomal recessive metabolic disease caused by a reduced or absent activity of the lysosomal alpha-glucosidase. As a consequence, glycogen accumulates in lysosomes, causing damage in the muscle (including heart) and nerve cells throughout the body. Opfolda has to be administered in combination with Pombiliti (cipaglucosidase alfa), a recombinant human alpha-glucosidase intended for enzyme replacement, which is conjugated with synthetic mannose 6-phosphate-rich glycans to enhance receptor targeting. Opfolda is an enzyme stabiliser that improves stability of cipaglucosidase in the bloodstream.
Opfolda is a hybrid medicine of Zavesca (miglustat), which is authorised in the EU since November 2002 for the treatment of type 1 Gaucher disease. Although both contain the same active substance, miglustat, Opfolda has a lower strength and a different indication. For more information please consult the product for Opfolda on the EMA website.
Recommendations on extensions of therapeutic indication:
Adempas (riociguat): extension of indication to the treatment of pulmonary arterial hypertension (PAH) in paediatric patients aged less than 18 years of age and body weight ≥ 50 kg with WHO Functional Class II to III in combination with endothelin receptor antagonists. Adempas was already authorised for the treatment of PAH and chronic thromboembolic pulmonary hypertension in adults. For more information please consult the product for Adempas on the EMA website.
Bimzelx (bimekizumab): has received a positive opinion for the following extension of indications:
• For the treatment of adults with active non-radiographic axial spondyloarthritis with objective signs of inflammation as indicated by elevated C-reactive protein and/or magnetic resonance imaging (MRI) who have responded inadequately or are intolerant to non-steroidal anti-inflammatory drugs;
• For the treatment of adults with active ankylosing spondylitis who have responded inadequately or are intolerant to conventional therapy; and
• For the treatment, alone or in combination with methotrexate, of active psoriatic arthritis in adults who have had an inadequate response or who have been intolerant to one or more disease-modifying antirheumatic drugs.
Bimzelx was already authorised for the treatment of plaque psoariasis. For more information please consult the product for Bimzelx on the EMA website.
Cosentyx (secukinumab): extension of indication to the treatment of active moderate to severe hidradenitis suppurativa (acne inversa) in adults with an inadequate response to conventional systemic hidradenitis suppurativa therapy. Cosentyx was already authorised for the treatment of plaque psoriasis, psoriatic arthritis, axial spondyloarthritis and juvenile idiopathic arthritis. For more information please consult the product for Cosentyx on the EMA website.
Opdivo (nivolumab): extension of indication to adolescents 12 years of age and older:
• For the treatment of advanced (unresectable or metastatic) melanoma, as monotherapy or in combination with ipilimumab;
• For the adjuvant treatment of melanoma with involvement of lymph nodes or metastatic disease who have undergone complete resection, as monotherapy.
Opdivo was already authorised in these indications in adults. Furthermore, Opdivo was already authorised for the treatment of non-small cell lung cancer, malignant pleural mesothelioma, renal cell carcinoma, classical Hodgkin lymphoma, squamous cell cancer of the head and neck, urothelial carcinoma, mismatch repair deficient or microsatellite instability-high colorectal cancer, oesophageal squamous cell carcinoma, oesophageal or gastro-oesophageal junction (GEJ) cancer and gastric, GEJ or oesophageal adenocarcinoma. For more information please consult the product for Opdivo on the EMA website.
Orkambi (lumacaftor / ivacaftor): extension of indication for Orkambi granules to the treatment of cystic fibrosis in patients from 1 year of age who are homozygous for the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Orkambi granules was already authorised in patients aged 2 and older. For more information please consult the product for Orkambi on the EMA website.
Revestive (teduglutide): extension of indication to the treatment of patients 4 months corrected gestational age and above with Short Bowel Syndrome. Patients should be stable folllowing a period of intestinal adaptation after surgery. Revestive was already authorised for patients aged 1 year and older. For more information please consult the product for Revestive on the EMA website.
Ronapreve (casirivimab / imdevimab): extension of indication to the treatment of COVID-19 in adults and adolescents aged 12 years and older weighing at least 40 kg and receiving supplemental oxygen, who have a negative SARS-CoV-2 antibody test result. Ronapreve was already authorised for the prevention of COVID-19 and for treatment of COVID-19 in patients who do not require supplemental oxygen and who are at increased risk of progressing to severe COVID-19. For more information please consult the product for Ronapreve on the EMA website.
Spikevax bivalent Original/Omicron BA.4-5 (elasomeran): extension of indication to the active immunisation to prevent COVID-19 in individuals 6 years of age and older who have previously received at least a primary vaccination course against COVID-19. Spikevax bivalent Original/Omicron BA.4-5 was already authorised in patients aged 12 years of age and older. For more information please consult the product for Spikevax on the EMA website.
Vemlidy (tenofovir alafenamide): extension of indication to the treatment of chronic hepatitis B (CHB) in adults and paediatric patients 6 years of age and older weighing at least 25 kg. Vemlidy was already authorised in adolescents aged 12 years and older. For more information please consult the product for Vemlidy on the EMA website.
Yervoy (ipilimumab): extension of indication to the treatment of adolescents 12 years of age and older with advanced (unresectable or metastatic) melanoma, in combination with nivolumab. Yervoy was already authorised in this indication as monotherapy. Furthermore, Yervoy was also already authorised for the treatment of renal cell carcinoma, non-small cell lung cancer, malignant pleural mesothelioma, mismatch repair deficient or microsatellite instability-high colorectal cancer and oesophageal squamous cell carcinoma. For more information please consult the product for Yervoy 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:
Opzelura (ruxolitinib): is indicated for the treatment of non-segmental vitiligo with facial involvement in adults and adolescents from 12 years of age. EPAR Opzelura.
Recently started procedures:
- Bevacizumab - Treatment of neovascular (wet) age-related macular degeneration.
- Cefepime / enmetazobactam - Treatment of:
- complicated urinary tract infections (including pyelonephritis);
- hospital-acquired pneumonia (HAP), including ventilator associated pneumonia;
- patients with bacteraemia that occurs in association with, or is suspected to be associated with, any of the infections listed above and
- infections due to aerobic Gram-negative organisms in adults with limited treatment options.
- Concizumab - Routine prophylaxis to prevent or reduce the frequency of bleeding in patients with:
- haemophilia A (congenital factor VIII deficiency) with FVIII inhibitors ≥ 12 years of age;
- haemophilia B (congenital factor IX deficiency) with FIX inhibitors of any age.
- Elranatamab - Orphan - Treatment of adult patients with relapsed or refractory multiple myeloma.
- Exagamglogene autotemcel - ATMP - Treatment of transfusion-dependent β-thalassemia and sickle cell disease.
- Germanium (68ge) chloride / gallium (68ga) chloride - In vitro radiolabelling of specific carrier molecules to be used for positron emission tomography imaging.
- Influenza virus a/turkey/turkey/1/2005 (h5n1) nibrg-23 strain, ha surface antigen - Prophylaxis of influenza.
- Lecanemab - A disease modifying treatment in adult patients with Mild Cognitive Impairment due to Alzheimer’s disease and Mild Alzheimer’s disease (Early Alzheimer’s disease)
- Omaveloxolone - Orphan - Treatment of Friedreich’s ataxia.
- Omecamtiv mecarbil - Treatment of adult patients with symptomatic chronic heart failure and reduced ejection fraction less than 30%.
- Pandemic influenza vaccine (surface antigen, inactivated, adjuvanted) - Active immunisation for the prevention of disease caused by the influenza A virus H5N1 subtype contained in the vaccine.
- Pegcetacoplan - Treatment of geographic atrophy secondary to age-related macular degeneration.
- Respiratory syncytial virus vaccine - Prevention of respiratory tract disease.
- Talquetamab - Orphan - Monotherapy treatment of adult patients with relapsed and refractory multiple myeloma.