CHMP Meeting Highlights November 2023

CHMP Monthly News | 21/11/2023

This month, medicinal products for the following indications have received a positive opinion:

  • Non-small cell lung cancer
  • Primary myelofibrosis, post polycythaemia vera myelofibrosis and post essential thrombocythaemia myelofibrosis
  • Generalised myasthenia gravis
  • Low-grade and high-grade glioma

New medicines recommended for approval:

Krazati (adagrasib): as monotherapy has received a positive opinion for a conditional marketing authorisation (CMA) for the treatment of adult patients with advanced non-small cell lung cancer (NSCLC) with KRAS G12C mutation and disease progression after at least one prior systemic therapy.
NSCLC is a serious and often fatal disease that accounts for over 85% of all lung cancers. KRAS-G12C is a driver mutation in many cancers and it represents about 13 % of all mutations in NSCLC. KRAS is a GTPase encoded by a proto-oncogene and the mutated KRAS-G12C is a constitutively active form of the enzyme. Krazati binds KRAS-G12C and prevents downstream signalling by locking the mutated protein in an inactive state. For more information please consult the product for Krazati on the EMA website.

Omjjara (momelotinib): received a positive opinion for the treatment of disease-related splenomegaly or symptoms in adult patients with moderate to severe anaemia who have primary myelofibrosis, post polycythaemia vera myelofibrosis or post essential thrombocythaemia myelofibrosis and who are Janus Kinase (JAK) inhibitor naïve or have been treated with ruxolitinib.
Myelofibrosis is an uncommon type of myeloproliferative neoplasm, in which fibrous tissue in the bone marrow interferes with normal blood cell production. This results in extramedullary movement to other organs, such as the spleen and liver, and results in enlargement of these organs. Symptoms of the disease include bone pain, tiredness, weakness, weight loss, fever and bleeding.

Primary myelofibrosis occurs de novo, while secondary forms of myelofibrosis arise from the myeloproliferative diseases essential thrombocythemia or polycythemia vera.

Myelofibrosis is associated with an aberrant expression of the tyrosine kinase JAK, which is involved in cytokine signalling pathways. This leads to the increased production of immature blood cells. Omjjara is an inhibitor of JAK 1 and 2. For more information please consult the product for Omjjara on the EMA website.

Rystiggo (rozanolixizumab): received a positive opinion as an add-on to standard therapy for the treatment of generalised myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) 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. Rystiggo is a monoclonal antibody which targets the neonatal Fc receptor (FcRn). Thus, the binding of IgG antibodies to FcRn is suppressed and the intracellular degradation of the pathogenic autoantibodies is stimulated. For more information please consult the product for Rystiggo on the EMA website.

Spexotras (trametinib): has received a positive opinion for the following indications:

Low-grade glioma
Spexotras in combination with dabrafenib is indicated for the treatment of paediatric patients aged 1 year and older with low-grade glioma (LGG) with a BRAF V600E mutation who require systemic therapy.

High-grade glioma
Spexotras in combination with dabrafenib is indicated for the treatment of paediatric patients aged 1 year and older with high-grade glioma (HGG) with a BRAF V600E mutation who have received at least one prior radiation and/or chemotherapy treatment.

Gliomas are a group of primary tumours of the central nervous system with glial origin. LGG are rare and HGG even rarer tumours in the paediatric population. The BRAF V600E mutation is present in around 17% of paediatric LGG cases and in 6% of paediatric and young adult cases of HGG. Spexotras is a small molecule allosteric inhibitor of the mitogen-activated protein kinase, MEK, which is an important part of a signalling pathway regulating proliferation, differentiation and apoptosis in cancer cells. Dabrafenib is a small molecule inhibitor of the V600-mutant BRAF kinase. For more information please consult the product for Spexotras on the EMA website.

Recommendations on extensions of therapeutic indication:

Ayvakyt (avapritinib): extension of indication to include the treatment of adult patients with indolent systemic mastocytosis (ISM) with moderate to severe symptoms inadequately controlled on symptomatic treatment.

Ayvakyt is already authorised for the treatment of gastrointestinal stromal tumours (GIST), aggressive systemic mastocytosis (ASM), systemic mastocytosis with an associated haematological neoplasm (SM-AHN) and mast cell leukaemia (MCL). For more information please consult the product for Ayvakyt on the EMA website.

Evkeeza (evinacumab): extension of indication, as an adjunct to diet and other low-density lipoprotein-cholesterol (LDL-C) lowering therapies, for the treatment paediatric patients aged 5 years and older with homozygous familial hypercholesterolaemia (HoFH).

Evkeeza is already authorised for the treatment of adults and adolescents from 12 years of age. For more information please consult the product for Evkeeza on the EMA website.

Fluad tetra (influenza vaccine (surface antigen, inactivated, adjuvanted)): extension of indication to include prophylaxis of influenza in adults 50 years of age and older.
Fluad tetra is already authorised for adults from 65 years of age. For more information please consult the product for Fluad tetra on the EMA website.

Jardiance (empagliflozin): extension of indication to include children aged 10 years and above for the treatment of insufficiently controlled type 2 diabetes mellitus as an adjunct to diet and exercise,

  • as monotherapy when metformin is considered inappropriate due to intolerance
  • in addition to other medicinal products for the treatment of diabetes

Jardiance is already authorised for the treatment of insufficiently controlled diabetes mellitus in adult patients. Moreover, it is authorised for the treatment of symptomatic chronic heart failure and chronic kidney disease in adults. For more information please consult the product for Jardiance on the EMA website.

Keytruda (pemprolizumab): extension of indication to include Keytruda, in combination with gemcitabine-based chemotherapy, for the first-line treatment of locally advanced unresectable or metastatic biliary tract carcinoma in adults.
Keytruda is already authorised for the treatment of various types of solid cancer. In September and October 2023, Keytruda received positive opinions for the adjuvant treatment of adults with non-small cell lung carcinoma who are at high risk of recurrence following complete resection and platinum-based chemotherapy, as well as for the first-line treatment, in combination with fluoropyrimidine and platinum-containing chemotherapy, of locally advanced unresectable or metastatic HER2-negative gastric or gastro-oesophageal junction adenocarcinoma in adults whose tumours express PD-L1 with a CPS ≥ 1. Both EC decisions are pending. For more information please consult the product for Keytruda on the EMA website.

Mounjaro (tirzepatide): extension of  indication to include, as an adjunct to a reduced-calorie diet and increased physical activity for weight management, including weight loss and weight maintenance, in adults with an initial Body Mass Index (BMI) of

  • ≥ 30 kg/m2 (obesity) or
  • ≥ 27 kg/m2 to < 30 kg/m2 (overweight) in the presence of at least one weight-related comorbid condition (e.g., hypertension, dyslipidaemia, obstructive sleep apnoea, cardiovascular disease, prediabetes, or type 2 diabetes mellitus).

Mounjaro is already authorised for the treatment of adults with insufficiently controlled type 2 diabetes mellitus. For more information please consult the product for Mounjaro on the EMA website.

Nexobrid (concentrate of proteolytic enzymes enriched in bromelain): extension of indication to include all age groups for removal of eschar in patients with deep partial- and full-thickness thermal burns.
Nexobrid is already authorised for the treatment of adult patients with the same indication. For more information please consult the product for Nexobrid on the EMA website.

Talzenna (talazoparib): extension of indication to include, in combination with enzalutamide, the treatment of adult patients with metastatic castration-resistant prostate cancer (mCRPC) in whom chemotherapy is not clinically indicated.
Talzenna is already authorised for the treatment of breast cancer. For more information please consult the product for Talzenna on the EMA website.

Veltassa (patiromer): extension of indication to include the treatment of hyperkalaemia in adolescents aged 12 to 17 years.
Veltassa is already authorised for the treatment of adult patients with the same indication. For more information please consult the product for Veltassa on the EMA website.

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