CHMP Meeting Highlights March 2024

CHMP Monthly News | 09/04/2024

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

Diabetes mellitus
Various infections (complicated intra-abdominal infection, pneumonia, complicated urinary tract infections)
Paroxysmal nocturnal haemoglobinuria
Neovascular (wet) age-related macular degeneration

New medicines recommended for approval:

Awiqli (insulin icodec): received a positive opinion for the treatment of diabetes mellitus in adults.

Awiqli was developed for the treatment of both type 1 and type 2 diabetes mellitus. Type 1 diabetes is an autoimmune disease in which pancreatic cells are destroyed by the immune system, leading to a decreased insulin production. Type 2 diabetes is a metabolic disease characterised by high blood sugar and insulin resistance and it accounts for around 90% of diabetes cases. Type 2 diabetes occurs primarily as a result of obesity and sedentary lifestyle, and the prevalence increases globally along with obesity and overweight.

The active substance of Awiqli, insulin icodec, is a new, very long-acting human insulin analogue that needs to be injected only once weekly. For more information please consult the product for Awiqli on the EMA website.

Emblaveo (aztreonam / avibactam): received a positive opinion for the treatment of the following infections in adult patients:

Complicated intra-abdominal infection (cIAI)
Hospital-acquired pneumonia (HAP), including ventilator-associated pneumonia (VAP)
Complicated urinary tract infection (cUTI), including pyelonephritis

Emblaveo is also indicated for the treatment of infections due to aerobic Gram-negative organisms in adult patients with limited treatment options.

Emblaveo combines two antibiotics. Aztreonam is a monocyclic beta-lactam agent (monobactam) that inhibits bacterial cell wall synthesis by targeting penicillin-binding proteins. Avibactam is a beta-lactamase inhibitor that prevents a certain class of bacterial enzymes, beta-lactamases, from hydrolysing aztreonam. The combination of these two compounds aims at multidrug-resistant, Gram-negative bacteria, which are estimated to cause 35,000 deaths in the EU every year.

Emblaveo underwent accelerated assessment, recognising that the product is of major interest for public health. The EMA has published a press release on Emblaveo. For more information please consult the product for Emblaveo on the EMA website.

Fabhalta (iptacopan): received a positive opinion for the treatment, as monotherapy, of adult patients with paroxysmal nocturnal haemoglobinuria (PNH) who have haemolytic anaemia.

PNH is a life-threatening blood disorder characterised by the destruction of red blood cells by the complement system, a component of the immune system. This occurs most commonly as a result of a somatic mutation that alters the exterior surface proteins of red blood cells, rendering these susceptible to be attacked by the complement.

Iptacopan is an orally available inhibitor of the alternative complement pathway, and can thus control both extravascular and intravascular haemolysis. The development of Fabhalta was supported through EMA's PRIority MEdicines (PRIME) scheme, which provides early and enhanced scientific and regulatory support.

The EMA has published a press release on Fabhalta. For more information please consult the product for Fabhalta on the EMA website.

Lytenava (bevacizumab gamma): received a positive opinion for the treatment of adult patients with neovascular (wet) age-related macular degeneration (nAMD).

Vascular endothelial growth factor (VEGF) is the main protein responsible for the induction of blood vessel growth and, therefore, it is thought contribute to the pathophysiology of retinal vascular diseases, such as nAMD. Bevacizumab is a monoclonal antibody that targets VEGF, thereby blocking the binding to its receptor on endothelial cells, and preventing the growth of new abnormal blood vessels in the back of the eye. For more information please consult the product for Lytenava on the EMA website.

Recommendations on extensions of therapeutic indication:

Bimzelx (bimekizumab): extension of indication to include the treatment of active moderate to severe hidradenitis suppurativa (HS, acne inversa) in adults with an inadequate response to conventional systemic HS therapy.

Bimzelx is already authorised for the treatment of plaque psoriasis, psoriatic arthritis, and axial spondyloarthritis. For more information please consult the product for Bimzelx on the EMA website.

Nilemdo (bempedoic acid): extension of indication to include the treatment of adults with established or at high risk for atherosclerotic cardiovascular disease to reduce cardiovascular risk by lowering LDL-C levels, as an adjunct to correction of other risk factors:

in patients on a maximum tolerated dose of a statin with or without ezetimibe or,
alone or in combination with ezetimibe in patients who are statin-intolerant, or for whom a statin is contraindicated.

Nilemdo is already authorised for the treatment of hypercholesterolaemia and mixed dyslipidaemia. For more information please consult the product for Nilemdo on the EMA website.

Nustendi (bempedoic acid / ezetimibe): extension of indication to include the treatment of adults with established risk of, or at high risk for atherosclerotic cardiovascular disease to reduce cardiovascular risk by lowering LDL-C levels, as an adjunct to correction of other risk factors:

in patients on a maximum tolerated dose of a statin and not adequately controlled with additional ezetimibe treatment or,
in patients who are either statin-intolerant, or for whom a statin is contraindicated, and not adequately controlled with ezetimibe treatment or,
in patients already being treated with the combination of bempedoic acid and ezetimibe as separate tablets.

Nustendi is already authorised for the treatment of hypercholesteraemia and mixed dyslipidaemia. For more information please consult the product for Nustendi on the EMA website.

Onivyde pegylated liposomal (irinotecan hydrochloride trihydrate): extension of indication to include, in combination with oxaliplatin, 5-fluorouracil (5-FU) and leucovorin (LV), the first-line treatment of adult patients with metastatic adenocarcinoma of the pancreas.

Onivyde pegylated liposomal is already authorised in a second-line setting for pancreatic cancer, in combination with 5-FU and LV. For more information please consult the product for Onivyde on the EMA website.

Retsevmo (selpercatinib): extension of indication to include the treatment of adult patients with advanced RET fusion-positive solid tumours, when treatment options not targeting RET provide limited clinical benefit, or have been exhausted.

Retsevmo is already authorised, based on a conditional marketing authorisation, for the treatment of RET fusion positive non-small cell lung cancer and thyroid cancer. For more information please consult the product for Retsevmo on the EMA website.

Xtandi (enzalutamide): extension of indication to include, as monotherapy or in combination with androgen deprivation therapy, the treatment of adult men with high risk biochemical recurrent (BCR) non-metastatic hormone sensitive prostate cancer (nmHSPC) who are unsuitable for salvage radiotherapy.

Xtandi is already authorised for the treatment of metastatic hormone-sensitive prostate cancer (mHSPC) in combination with androgen deprivation therapy, and for various subtypes of castration-resistant prostate cancer (CRPC). For more information please consult the product for Xtandi 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:

Casgevy (exagamglogene autotemcel): is indicated for the following indications:

β-thalassemia
Casgevy is indicated for the treatment of transfusion-dependent β-thalassemia (TDT) in patients 12 years of age and older for whom haematopoietic stem cell (HSC) transplantation is appropriate and a human leukocyte antigen (HLA)-matched related HSC donor is not available.

Sickle cell disease
Casgevy is indicated for the treatment of severe sickle cell disease (SCD) in patients 12 years of age and older with recurrent vaso-occlusive crises (VOCs) for whom haematopoietic stem cell (HSC) transplantation is appropriate and a human leukocyte antigen (HLA)-matched related HSC donor is not available.

EPAR Casgevy.

Exblifep (cefepime / enmetazobactam): is indicated 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. EPAR Exblifep.

Krazati (adagrasib): as monotherapy is indicated 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. EPAR Krazati.

Rystiggo (rozanolixizumab): is indicated 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. EPAR Rystiggo.

Skyclarys (omaveloxolone): is indicated for the treatment of Friedreich’s ataxia in adults and adolescents aged 16 years and older. EPAR Skyclarys.

Recently started procedures:

Autologous cartilage-derived articular chondrocytes, in-vitro expanded – ATMP – Repair of symptomatic, localised, full-thickness cartilage defects of the knee joint grade III or IV
Belzutifan – Treatment of adult patients with advanced renal cell carcinoma (RCC) and treatment of adult patients with von Hippel-Lindau (VHL) disease
Beremagene geperpavec – Orphan – ATMP – Treatment of patients from birth with dystrophic epidermolysis bullosa (DEB) with mutation(s) in the collagen type VII alpha 1 chain (COL7A1) gene
Chikungunya virus, strain CHIKV LR2006-OPY1, live attenuated – Accelerated review – Prevention of disease caused by chikungunya (CHIKV) virus
Garadacimab – Orphan – Routine prevention of attacks of hereditary angioedema (HAE)
Govorestat – Orphan – treatment of adults and children aged 2 years and older with a confirmed diagnosis of classic galactosemia
Odevixibat – Treatment of cholestatic pruritus in Alagille syndrome (ALGS)
Repotrectinib – Treatment of ROS1-positive locally advanced or metastatic non-small cell lung cancer (NSCLC) and for solid tumours
Vorasidenib – Orphan – Accelerated review – Treatment of predominantly non-enhancing astrocytoma or oligodendroglioma with a IDH1 R132 mutation or IDH2 R172 mutation