CHMP Meeting Highlights December 2023

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

• Transfusion-dependent β-thalassemia and sickle cell disease
• Friedreich’s ataxia
• Ulcerative colitis
• Schistosomiasis

New medicines recommended for approval:

Arpraziquantel (arpraziquantel): received a positive opinion for the treatment of schistosomiasis in young children aged 3 months to 6 years. The application for marketing authorisation was submitted under the EU-M4all procedure for high-priority medicines for human use intended for markets outside the EU.

Schistosomiasis is a parasitic disease caused by trematode worms, and causes damage of several organs, as the bladder, the kidneys and the liver. The parasite infection occurs in tropical and subtropical regions and mainly affects people without access to clean water. Arpraziquantel is a derivative of praziquantel, the standard treatment of Schistosomiasis since decades. Arpraziquantel has been developed to provide an age-appropriate formulation for young children, as it can be administered as a dispersible tablet. For more information please consult the product for Arpraziquantel on the EMA website.

Casgevy (exagamglogene autotemcel): received a positive opinion, recommending the granting of a conditional marketing authorisation (CMA), for the treatment of transfusion-dependent β-thalassemia (TDT) and sickle cell disease (SCD). The full indication is:

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.
β-thalassemia and sickle cell disease are two inherited disorders of the red blood cells. Different mutations lead to impaired production of haemoglobin and thus, to problems in red blood cell function. Casgevy is a cellular therapy, consisting of the patient’s own haematopoietic stem cells which are genetically modified to foster normal gene expression in red blood cells. Casgevy needs to be administered only once. For more information please consult the product for Casgevy on the EMA website.

Skyclarys (omaveloxolone): received a positive opinion for the treatment of Friedreich’s ataxia in adults and adolescents aged 16 years and older.

Friedreich’s ataxia is a hereditary progressive degenerative disorder and is caused by mutations in a gene encoding a mitochondrial protein, frataxin. The impaired production of this protein results in progressive neurological symptoms like movement and speech disorders, and sensory loss. Skyclarys activates a protein involved in the cellular response to oxidative stress, which is involved in the pathophysiological mechanism of the disease. For more information please consult the product for Skyclarys on the EMA website.

Velsipity (etrasimod): received a positive opinion for the treatment of patients 16 years of age and older with moderately to severely active ulcerative colitis (UC) who have had an inadequate response, lost response, or were intolerant to either conventional therapy, or a biological agent.

UC is a chronic inflammatory disease that affects the colon. Although the aetiology is not fully understood, many factors seem to be involved in the pathogenesis of the disease, including altered immune responses, environmental and genetic factors. Velsipity is a selective immunosuppressant. It blocks receptors expressed on lymphocytes and thus reduces lymphocyte migration into sites of inflammation. For more information please consult the product for Velsipity on the EMA website.

Recommendations on extensions of therapeutic indication:

Fexinidazole Winthrop (fexinidazole): extension of indication for the treatment of both first-stage (haemo-lymphatic) and second-stage (meningo-encephalitic) of human African trypanosomiasis (HAT) due to Trypanosoma brucei rhodesiense (r-HAT) in adults and children ≥ 6 years old and weighing ≥ 20 kg.

Fexinidazole Winthrop is already authorised for the treatment of HAT due to Trypanosoma brucei gambiense (g‑HAT). For more information please consult the product for Fexinidazole Winthrop on the EMA website.

HyQvia (human normal immunoglobulin): extension of indication to include HyqVia as an immunomodulatory therapy in adults, children and adolescents (0 to 18 years) in:

• Chronic inflammatory demyelinating polyneuropathy (CIDP) as maintenance therapy after stabilization with IVIg.

Hyqvia is already authorised as a replacement therapy in patients with primary and secondary immunodeficiency syndromes. For more information please consult the product for HyQvia on the EMA website.

Metalyse (tenectaplase): extension of indication to include the thrombolytic treatment of acute ischaemic stroke (AIS) within 4.5 hours from last known well and after exclusion of intracranial haemorrhage.
Metalyse is already authorised, with a different strength, for the thrombolytic treatment of suspected myocardial infarction. For more information please consult the product for Metalyse on the EMA website.

VeraSeal (human fibrinogen / human thrombin): extension of indication to include the supportive treatment in children of all age groups where standard surgical techniques are insufficient:

• for improvement of haemostasis
• as suture support: in vascular surgery

VeraSeal is already authorised for the treatment of adult patients in the same indications. For more information please consult the product for VeraSeal on the EMA website.

Zinplava (bezlotoxumab): extension of indication to include the preventive treatment of paediatric patients 1 year of age and older at high risk for recurrence of Clostridioides difficile infection (CDI).

Zinplava is already authorised for the treatment of adult patients in the same indication. For more information please consult the product for Zinplava 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:

Aqumeldi (enalapril maleate): is indicated for the treatment of heart failure in children from birth to less than 18 years. EPAR Aqumeldi.

Ebglyss (lebrikizumab): is indicated for the treatment of moderate-to-severe atopic dermatitis in adults and adolescents 12 years and older with a body weight of at least 40 kg who are candidates for systemic therapy. EPAR Ebglyss.

Finlee (dabrafenib): has the following indications:

Low-grade glioma
Finlee in combination with trametinib 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
Finlee in combination with trametinib 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 Finlee.

Inaqovi (decitabine / cedazuridine): is indicated for the treatment, as monotherapy, of adult patients with newly diagnosed acute myeloid leukaemia (AML) who are ineligible for standard induction chemotherapy. EPAR Inaqovi.

Jaypirca (pirtobrutinib): is indicated 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. EPAR Jaypirca.

Vanflyta (quizartinib): is indicated in combination with standard cytarabine and anthracycline induction and standard cytarabine consolidation chemotherapy, followed by Vanflyta single-agent maintenance therapy for adult patients with newly diagnosed acute myeloid leukaemia (AML) that is FLT3-ITD (Feline McDonough Sarcoma-like tyrosine kinase 3 internal tandem duplication) positive. EPAR Vanflyta.

Yorvipath (palopegteriparatide): is a parathyroid hormone (PTH) replacement therapy indicated for the treatment of adults with chronic hypoparathyroidism.
EPAR Yorvipath.

Zilbrysq (zilucoplan): 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) antibody positive. EPAR Zilbrysq.

Recently started procedures:

• Amino acids – Orphan – treatment of decompensation episodes in maple syrup urine disease (MSUD) patient
• Aztreonam / avibactam – treatment of infections (cIAI, HAP, VAP, and cUTI), and aerobic Gram-negative infections with limited treatment options
• Erdafitinib – treatment of adult patients with locally advanced unresectable or metastatic urothelial carcinoma (UC
• In vitro diagnostic medical device – next generation sequencing (NGS) assay for tumour mutation profil
• Imetelstat – Orphan – treatment of adult patients with transfusion-dependent anaemia due to very low, low and intermediate-risk myelodysplastic syndromes (MDS) with ring sideroblasts

Further inquiry note

basg_anfragen@basg.gv.at