CHMP Meeting Highlights December 2021
Information about the Austrian representation on the CHMP and monthly updates can be found at this Link.
This month, medicinal products for the following indications have received a positive opinion:
- Chronic kidney disease associated with type 2 diabetes
- Growth hormone deficiency
- Haemolytic anaemia due to sickle cell disease
- Non-small cell lung cancer
- Prophylaxis against pneumococcal pneumonia
- Symptoms of uterine fibroids
- Systemic lupus erythematosus
- Urothelial cancer
New medicines recommended for approval:
Apexxnar (pneumococcal polysaccharide conjugate vaccine [20-valent, adsorbed]): is indicated as active immunisation for the prevention of invasive disease and pneumonia caused by Streptococcus pneumoniae in individuals 18 years of age and older. Invasive disease and pneumonia caused by an infection with S. pneumoniae result in high morbidity and mortality, especially in the elderly and in patients with comorbidities or immunocompromised. Apexxnar is a conjugate vaccine that contains pneumococcal polysaccharides from 20 serotypes, conjugated to a carrier protein from Corynebacterium diphtheriae. This conjugation induces a T-cell dependent immune response. For more information please consult the product page for Apexxnar on the EMA website.
Kerendia (finerenone): is indicated for the treatment of chronic kidney disease (CKD) (stage 3 and 4 with albuminuria) associated with type 2 diabetes (T2D) in adults. Several factors can contribute to the development of CKD in patients with T2D, including hemodynamic, inflammatory and metabolic factors. Kerendia is a non-steroidal mineralocorticoid receptor (MR) antagonist. Through sodium retention and endothelial dysfunction, an over-active MR can contribute to the effects seen in patients with CKD, such as hypertension, inflammation and organ damage. For more information please consult the product page for Kerendia on the EMA website.
Ngenla (somatrogon): is indicated for the treatment of children and adolescents from 3 years of age with growth disturbance due to insufficient secretion of growth hormone. Somatropin, or human growth hormone (hGH) , is secreted in the somatotropic cells of the pituitary gland and it stimulates the release of insulin-like growth factor 1 (IGF-1), thereby promoting growth in children. Growth hormone deficiency (GHD) results in insufficient IGF-1 levels and manifests with growth failure. The aetiology of GHD can be of congenital, acquired or idiopathic nature. Ngenla is a long-acting glycoprotein with a mechanism of action similar to hGH, exerting its downstream effect by binging to the growth hormone receptor. For more information please consult the product page for Ngenla on the EMA website.
Oxbryta (voxelotor): is indicated for the treatment of haemolytic anaemia due to sickle cell disease (SCD) in adults and paediatric patients 12 years of age and older as monotherapy or in combination with hydroxycarbamide. SCD is a group of autosomal recessive disorders caused by mutations in the beta-globin gene (HBB), which expresses haemoglobin. The most common form of the disease is characterised by the ability of the mutant haemoglobin S (HbS) protein to polymerize when in its deoxygenated state. Erythrocytes containing these polymers present with a distinct sickled shape and are prone to haemolysis, leading to haemolytic anaemia. Oxbryta is a small molecule that inhibits the polymerization of HbS by increasing its affinity for oxygen. For more information please consult the product page for Oxbryta on the EMA website.
Okedi (risperidone): is intended for the treatment schizophrenia in adults for whom tolerability and effectiveness has been established with oral risperidone. Risperidone has been authorised in the EU since 1993 (Risperdal®) for the treatment of schizophrenia, amongst other. Okedi is a psycholeptic antipsychotic with a new injectable prolongued-release formulation containing risperidone in microparticles. The administration should take place every 28 days. For more information please consult the product page for Okedi on the EMA website.
Padcev (enfortumab vedotin): as monotherapy is indicated for the treatment of adult patients with locally advanced or metastatic urothelial cancer (UC) who have previously received a platinum-containing chemotherapy and a programmed cell death‑1 receptor (PD-1) or programmed cell death‑ligand 1 (PD-L1) inhibitor. Urothelial cancer is an aggressive cancer of the bladder or other parts of the urinary tract. Padcev is an antibody-drug conjugate that binds Nectin-4 on the cell surface, forming a complex which is subject to endocytosis. The cleavage of Padcev within the lysosomes releases the drug portion of the conjugate, monomethyl auristatin E, which blocks the polymerisation of tubulin and triggers a cascade of events that culminate in cellular apoptosis. Nectin-4 is expressed by the vast majority of UCs. Although more predictive factors for UC are still being studied, the presence of elevated levels of expression of the adhesion protein Nectin-4 seem to be associated with disease progression and poor prognosis in UC and other cancers. For more information please consult the product page for Padcev on the EMA website.
Saphnelo (anifrolumab): is indicated as an add-on therapy for the treatment of adult patients with moderate to severe, active autoantibody-positive systemic lupus erythematosus (SLE), despite standard therapy. SLE is a multifactorial chronic and multi-systemic autoimmune disease with varied clinical manifestations, although arthritis and photosensitive skin rash are common symptoms. The aetiology of SLE is unknown, although genetic, environmental and hormonal factors are believed to play important roles. The type I interferon cytokine family regulates several immune functions and cellular components. In SLE, several interferon-regulated genes are over-expressed. Anifrolumab is a monoclonal antibody which binds to anti-type I interferon receptor (IFNAR1) and inhibits the binding of type I interferons to their receptor, thereby blocking their activity. For more information please consult the product page for Saphnelo on the EMA website.
Tepmetko (tepotinib): as monotherapy is indicated for the treatment of adult patients with advanced non-small cell lung cancer (NSCLC) harbouring alterations leading to mesenchymal-epithelial transition factor gene exon 14 (METex14) skipping, who require systemic therapy following prior treatment with immunotherapy and/or platinum-based chemotherapy. NSCLC is a serious and often fatal disease that accounts for over 85% of all lung cancers. MET is a proto-oncogene, which encodes a tyrosine kinase receptor. Deletion or skipping of exon 14 in the MET gene confers the enzyme increased stability and renders it constitutively active. Tepmetko hinders MET downstream signalling by inhibiting its activation by phosphorylation. For more information please consult the product page for Tepmetko on the EMA website.
Xevudy (sotrovimab): is indicated for the treatment of COVID-19 in adults and adolescents (from 12 years of age and weighing at least 40 kilograms) who do not require supplemental oxygen and who are at increased risk of the disease becoming severe. COVID-19 or Coronavirus disease 2019, is a contagious disease caused by the Severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2). Although this virus can infect a wide variety of cells, it is most known for causing respiratory tract symptoms, which vary from mild to severe. The virus enters the cells mainly by binding to the angiotensin converting enzyme 2 (ACE2). Xevudy is a monoclonal antibody that binds to a highly conserved region of the receptor-binding domain (RBD) of the Spike protein of the SARS‑CoV-2 virus. Although Xevudy does not impede the attachment to ACE2, it blocks the entry of the virus into the cells and hinders infection. The majority of patients in the study were infected with the original SARS-CoV-2 virus. Some patients were infected with variants including Alpha and Epsilon. Findings from laboratory studies indicate that Xevudy is also expected to be effective against other variants (including Omicron). For more information, visit the press release at the EMA website. For more information please consult the product page for Xevudy on the EMA website.
Yselty (linzagolix choline): is indicated for the treatment of moderate to severe symptoms of uterine fibroids in adult women of reproductive age. Uterine fibroids are benign hormone-sensitive tumours of the uterus. The most troublesome symptoms are heavy menstrual bleeding and pain. Yselty is an antagonist of the gonadotropin-releasing hormone (GnRH) and, therefore, it binds to the GnRH receptors. This suppresses the release of the luteinizing hormone and the follicle-stimulating hormone, which translates into a reduction in oestradiol levels. For more information please consult the product page for Yselty on the EMA website.
Recommendations on extensions of therapeutic indication:
Entyvio (vedolizumab): extension of indication to the treatment of adult patients with moderately to severely active chronic pouchitis, who have undergone proctocolectomy and ileal pouch anal anastomosis for ulcerative colitis, and have had an inadequate response with or lost response to antibiotic therapy. Entyvio is already authorised for the treatment of ulcerative colitis and Crohn’s disease.
For more information please consult the product for Entyvio on the EMA website.
Keytruda (pembrolizumab): extension of indication for Keytruda as monotherapy, to the adjuvant treatment of adults with renal cell carcinoma at increased risk of recurrence following nephrectomy, or following nephrectomy and resection of metastatic lesions. Keytruda is already authorised for the treatment of melanoma, non-small cell lung carcinoma, classical Hodgkin lymphoma, urothelial carcinoma, head and neck squamous cell carcinoma, renal cell carcinoma, colorectal cancer, oesophageal carcinoma, triple-negative breast cancer and endometrial carcinoma.
For more information please consult the product for Keytruda on the EMA website.
Kineret (anakinra): extension of indication to the treatment of COVID-19 in adult patients with pneumonia requiring supplemental oxygen (low or high flow oxygen) and who are at risk of developing severe respiratory failure. Kineret is already authorised for the treatment of various inflammatory conditions. For more information please consult the product for Kineret on the EMA website.
Lorviqua (lorlatinib): extension of indication for Lorviqua as monotherapy, to the treatment of adult patients with anaplastic lymphoma kinase (ALK)-positive advanced non-small cell lung cancer previously not treated with an ALK inhibitor. For more information please consult the product for Lorviqua on the EMA website.
Teysuno (tegafur / gimeracil / oteracil): extension of indication for Teysuno as monotherapy or in combination with oxaliplatin or irinotecan, with or without bevacizumab, to the treatment of patients with metastatic colorectal cancer for whom it is not possible to continue treatment with another fluoropyrimidine due to hand-foot syndrome or cardiovascular toxicity that developed in the adjuvant or metastatic setting. Teysuno is already authorised for the treatment of gastric cancer. For more information please consult the product for Teysuno on the EMA website.
Veklury (remdesivir): extension of indication to the treatment of adults 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 Veklury on the EMA website.
Newly published EPARs:
The EPAR (European public assessment report) is the main document where the EMA publishes the detailed information from the medicines assessed by the CHMP. Below is a list of the EPARs for recently approved products that have been made available at the EMA homepage:
Aspaveli (pegcetacoplan): for the treatment of patients with paroxysmal nocturnal haemoglobinuria who are anaemic. EPAR Aspaveli.
Brukinsa (zanubrutinib): for the treatment of adult patients with Waldenström’s macroglobulinaemia. EPAR Brukinsa.
Cibinqo (abrocitinib): for the treatment of moderate to severe atopic dermatitis. EPAR Cibinqo.
Trodelvy (sacituzumab govitecan): for the treatment of triple-negative breast cancer. EPAR Trodelvy.
Recently started procedures:
- Vutrisiran - Orphan - Treatment of hereditary transthyretin-mediated amyloidosis.
- Mavacamten - Treatment of symptomatic obstructive hypertrophic cardiomyopathy.
- Maralixibat - Orphan - Treatment of cholestatic liver disease in patients with Alagille syndrome, 1 year of age and older.
- Relatlimab / nivolumab – For the first-line treatment of advanced (unresectable or metastatic) melanoma in adults and adolescents (12 years and older and weighing at least 40 kg).
- Efbemalenograstim alfa - Reduction in the duration of neutropenia and the incidence of febrile neutropenia.
Referral procedure under Article 5(3) for Paxlovid (nirmatrelvir and ritonavir):
A referral is a procedure in which EMA is asked to evaluate scientifically a medicine or class of medicines on behalf of the European Union. These type of procedures are intended to resolve certain safety or benefit-risk balance concerns. Learn more about referral procedures.
The CHMP issued advice on the use of the antiviral Paxlovid for the treatment of adults with COVID-19 who do not require supplemental oxygen and who are at increased risk of progressing to severe disease. This advice is intended to support national authorities who may decide on possible early use of the medicine prior to marketing authorisation. A rolling review has started in parallel, before an application for a marketing authorisation can be submitted.
For more information on the referral procedure for Paxlovid visit the full article at the EMA website.