CHMP Meeting Highlights January 2024

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

• Several infections (urinary tract, pyelonephritis and pneumonia) and associated bacteraemia
• Reduction of neutropenia in patients treated with cytotoxic chemotherapy for malignancy

New medicines recommended for approval:

Exblifep (cefepime / enmetazobactam): received a positive opinion for the treatment of the following infections in adults:

• Complicated urinary tract infections (cUTI), including pyelonephritis
• Hospital-acquired pneumonia (HAP), including ventilator associated pneumonia (VAP),

and for the treatment of patients with bacteraemia that occurs in association with, or is suspected to be associated with, any of the infections listed above.

Cefepime and enmetazobactam are two antibiotics. Cefepime is a cephalosporin and directly inhibits bacterial cell wall synthesis, while enmetazobactam inhibits a bacterial enzyme called beta-lactamase. This enzyme is able to hydrolyse cephalosporins. Thus, as a combination treatment, enmetazobactam protects cefepime from being destroyed by beta-lactamase, enabling its anti-bacterial activity. For more information please consult the product for Exblifep on the EMA website.

Ryzneuta (efbemalenograstim alfa): received a positive opinion for the reduction in the duration of neutropenia and the incidence of febrile neutropenia in adult patients treated with cytotoxic chemotherapy for malignancy (with the exception of chronic myeloid leukaemia and myelodysplastic syndromes).

Cytotoxic chemotherapy is often accompanied by neutropenia, a decrease in a certain kind of blood cells which play an important role in the immune system. Neutropenia is a severe side effect of chemotherapy, which leads to an increased risk of infection. The active substance in Ryzneuta is a haematopoietic growth factor which increases the production and differentiation of mature and functionally active neutrophils from bone marrow precursor cells. For more information please consult the product for Ryzneuta on the EMA website.

Recommendations on extensions of therapeutic indication:

Abecma (idecabtagene vicleucel): extension of indication to include treatment of adult patients with relapsed and refractory multiple myeloma (RRMM) who have received at least two prior therapies, including an immunomodulatory agent, a proteasome inhibitor and an anti CD38 antibody and have demonstrated disease progression on the last therapy.

Abecma is already authorised for a later line of treatment of RRMM. For more information please consult the product for Abecma on the EMA website.

Aspaveli (pegcetacoplan): extension of indication to include the treatment, as monotherapy, of adult patients with paroxysmal nocturnal haemoglobinuria (PNH) who have haemolytic anaemia.

Aspaveli is already authorised for the second-line treatment of anaemic PNH patients. For more information please consult the product for Aspaveli on the EMA website.

Prevenar 20 / previously Apexxnar (pneumococcal polysaccharide conjugate vaccine (20-valent, adsorbed)): extension of indication to include the active immunisation for the prevention of invasive disease, pneumonia, and acute otitis media caused by Streptococcus pneumoniae in infants, children, and adolescents from 6 weeks to less than 18 years of age.

Prevenar 20 / Apexxnar is already authorised for the active immunisation of adults. For more information please consult the product for Prevenar 20 on the EMA website.

Retsevmo (selpercatinib): extension of indication to include the treatment of adults and adolescents 12 years and older with advanced RET fusion-positive thyroid cancer who are radioactive iodine-refractory (if radioactive iodine is appropriate).

Retsevmo is already authorised for the treatment of advanced RET fusion-positive thyroid cancer in a different second-line setting, and the first-line treatment of medullary thyroid cancer (MTC) and non-small cell lung cancer (NSCLC). For more information please consult the product for Retsevmo 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:

Agamree (vamorolone): is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients aged 4 years and older. EPAR Agamree.

Elrexfio (elranatamab): is indicated as monotherapy for the treatment of adult patients with relapsed and refractory multiple myeloma, who have received at least three prior therapies, including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 antibody and have demonstrated disease progression on the last therapy. EPAR Elrexfio.

Loargys (pegzilarginase): is indicated for the treatment of arginase 1 deficiency (ARG1-D), also known as hyperargininemia, in adults, adolescents and children aged 2 years and older. EPAR Loargys.

Rezzayo (rezafungin): is indicated for the treatment of invasive candidiasis in adults. EPAR Rezzayo.

Spexotras (trametinib): is indicated 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.
EPAR Spexotras.

Veoza (fezolinetant): is indicated for the treatment of moderate to severe vasomotor symptoms (VMS) associated with menopause. EPAR Veoza.

Recently started procedures:

• Clascoterone - indicated for the topical treatment of acne vulgaris in adults and adolescents
• Elafibranor - Orphan - treatment of primary biliary cholangitis (PBC)
• Eplontersen - Orphan - indicated for the treatment of adult patients with polyneuropathy associated with hereditary transthyretin-mediated amyloidosis (ATTRv)
• Marstacimab - Orphan - is indicated for routine prophylaxis of bleeding episodes in patients with haemophilia A or haemophilia B
• Mirvetuximab soravtansine - Orphan - treatment of ovarian, fallopian tube, or primary peritoneal cancer
• Sotatercept - Orphan - Accelerated review - treatment of pulmonary arterial hypertension in adults
• Tiratricol - Orphan - treatment of monocarboxylate transporter 8 (MCT8) deficiency
• Troriluzole - Orphan - is indicated for the treatment of adult patients with spinocerebellar ataxia genotype 3 (SCA3)

Further inquiry note

basg_anfragen@basg.gv.at