CHMP Meeting Highlights December 2024
In the month of December 2024, medicinal products for the following indications have received a positive opinion:
- hereditary angioedema
- transthyretin amyloidosis
- COVID-19
- atopic dermatitis
- anaemia
- primary biliary cholangitis
- renal cell carcinoma
- von Hippel-Lindau disease
New medicines recommended for approval
Andembry (garadacimab) has received a positive opinion for the marketing authorisation for the prevention of recurrent attacks of hereditary angioedema (HAE). HAE is a rare genetic disorder that leads to recurrent attacks of severe swelling, which affects arms, legs, face, the intestinal tract and airways.
Beyonttra (acoramidis) has received a positive opinion for the marketing authorisation for the treatment of wild-type or variant transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM). ATTR-CM is characterized by the deposition of insoluble amyloid fibrils in the heart muscle, which leads to cardiomyopathy and a reduced function of the heart.
Kavigale (sipavibart) has received a positive opinion for the marketing authorisation for the prevention of COVID-19 in immunocompromised people aged 12 years and older.
A statement of the EMA emergency task force about the activity of monoclonal antibodies against emerging SARS-CoV-2 variants, was published.
Nemluvio (nemolizumab) has received a positive opinion for the marketing authorization for the treatment of atopic dermatitis (AD). AD is a chronic and complex disease, characterised by dry and pruritic skin. The main contributors seem to be an altered permeability of the skin due to perturbations in the epidermal barrier and an inappropriate immune response, which results in skin inflammation and contributes to the epidermal barrier disruption.
Rytelo (imetelstat) has received a positive opinion for the marketing authorization for the treatment of adult patients with transfusion dependent anaemia due to very low, low or intermediate risk myelodysplastic syndromes (MDS). MDS is a group of cancers of the bone marrow. The blood cells do not mature into healthy blood cells, which can lead to anaemia. An orphan designation was granted for the treatment of this disease.
Seladelpar Gilead (seladelpar lysine dihydrate) has received a positive opinion for the conditional marketing authorization for the treatment of primary biliary cholangitis (PBC) in combination with ursodeoxycholic acid (UDCA) in adults who have an inadequate response to UDCA alone, or as monotherapy in those unable to tolerate UDCA . PBC is an autoimmune disease of the liver causing the slow destruction of the small bile ducts. This leads to cholestasis and damages the liver further. An orphan designation was granted for the treatment of this disease.
Welireg (belzutifan) has received a positive opinion for the conditional marketing authorization for the treatment as monotherapy of adult patients with von Hippel-Lindau disease who require therapy for associated, localised renal cell carcinoma (RCC), central nervous system (CNS) haemangioblastomas, or pancreatic neuroendocrine tumours (pNET), and for whom localised procedures are unsuitable. Von Hippel-Lindau is a rare genetic disease that causes the growth of cysts and tumours in different parts of the body including eyes, brain, spine, kidneys and pancreas.
Welireg is the first medicinal product for this disease and a separate news was published by the EMA.
It also received a positive opinion for the treatment as monotherapy of adult patients with advanced clear cell renal cell carcinoma that progressed following two or more lines of therapy that included a PD-(L)1 inhibitor and at least two VEGF-targeted therapies.
Recommendations on extensions of therapeutic indication
Ofev (nintedanib): extension of indication for Ofev to include the treatment of children and adolescents from 6 to 17 years old of clinically significant, progressive fibrosing interstitial lung diseases (ILDs). Ofev is the first medicinal product for this disease in children and a separate news was published by the EMA
Ofev also received a positive opinion for the treatment of systemic sclerosis associated interstitial lung disease (SSc‑ILD) in children aged 6 and older.
Ofev was already approved for the treatment of SSc-ILD in adults.
Omvoh (mirikizumab): extension of indication for Omvoh to include the treatment of adult patients with moderately to severely active Crohn's disease who have had an inadequate response with, lost response to, or were intolerant to either conventional therapy or a biologic treatment.
Omvoh is already approved for the treatment of ulcerative colitis.
Blincyto (blinatumomab): extension of indication for Blincyto to include the treatment of paediatric patients from 1 month or older with Philadelphia chromosome-negative CD19 positive B cell precursor ALL which is refractory or in relapse after receiving at least two prior therapies or in relapse after receiving prior allogeneic haematopoietic stem cell transplantation.
Blincyto also received a positive opinion as monotherapy as part of consolidation therapy for the treatment of adult patients with newly diagnosed Philadelphia chromosome negative CD19 positive B-cell precursor ALL.
Previously the minimum age was 1 year.
Bridion (sugammadex): extension of indication for Bridion to include the treatment of paediatric patients from birth to 17 years of reversal of rocuronium induced neuromuscualar blockade.
Previously the minimum age was 2 years.
Flucelvax Tetra (influenza vaccine (surface antigen, inactivated, prepared in cell cultures)): extension of indication for Bridion to include the prophylaxis of influenza in adults and children from 6 months of age.
Flucelvax Tetra should be used in accordance with official recommendations.
Previously the minimum age was 2 years.
Jemperli (dostarlimab): extension of indication for Jemperli to include the treatment of adult patients with mismatch repair deficient (dMMR)/ microsatellite instability‑high (MSI‑H) recurrent or advanced endometrial cancer that has progressed on or following prior treatment with a platinum‑containing regimen as monotherapy.
Jemperli was previously approved for the treatment of endometrial cancer in combination with carboplatin and paclitaxel.
Rekambys (rilpivirine): extension of indication for Rekambys to include in combination with cabotegravir the treatment of human immunodeficiency virus type 1 (HIV‑1) infection in adolescents (at least 12 years of age and weighing at least 35 kg) who are virologically suppressed (HIV-1 RNA < 50 copies/mL) on a stable antiretroviral regimen without present or past evidence of viral resistance to, and no prior virological failure with, agents of the non-nucleoside reverse transcriptase inhibitor (NNRTI) and integrase inhibitor (INI) class.
Rekambys is already approved for the treatment of adult patients.
Vocabria (cabotegravir): extension of indication for Vocabria to include in combination with rilpivirine the treatment of human immunodeficiency virus type 1 (HIV‑1) infection in adolescents (at least 12 years of age and weighing at least 35 kg) who are virologically suppressed (HIV-1 RNA < 50 copies/mL) on a stable antiretroviral regimen without present or past evidence of viral resistance to, and no prior virological failure with, agents of the non-nucleoside reverse transcriptase inhibitor (NNRTI) and integrase inhibitor (INI) class.
Vocabria is already approved for the treatment of adult patients.
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:
Ordspono
Ordspono as monotherapy is indicated for the treatment of adult patients with relapsed or refractory follicular lymphoma (r/r FL) after two or more lines of systemic therapy.
Ordspono as monotherapy is indicated for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (r/r DLBCL) after two or more lines of systemic therapy.
Elahere
ELAHERE as monotherapy is indicated for the treatment of adult patients with folate receptor-alpha (FRα) positive, platinum-resistant high grade serous epithelial ovarian, fallopian tube, or primary peritoneal cancer who have received one to three prior systemic treatment regimens.
Recently started procedures
Every month, new marketing authorization applications are submitted to EMA under the centralised authorisation procedure. The CHMP carries out a scientific assessment of the applications and gives a recommendation. The CHMP has started the assessment of the following submitted applications:
Initial marketing authorisation applications:
- L-Acetylleucine - EMEA/H/C/006327 is indicated in adults and children from birth for chronic treatment of Niemann-Pick Type C (NPC).
- Denosumab - EMEA/H/C/006269, prevention of skeletal related events with advanced malignancies
- Denosumab - EMEA/H/C/006268 treatment of osteoporosis and bone loss
- Delandistrogene moxeparvovec - EMEA/H/C/005293, Orphan, ATMP Roche Registration GmbH, treatment of ambulatory patients aged 3 to 7 years old with Duchenne muscular dystrophy
- Emtricitabine / Tenofovir alafenamide - EMEA/H/C/006469 for the treatment of human immunodeficiency virus type 1 (HIV-1)
- Influenza vaccine (surface antigen, inactivated, adjuvanted) - EMEA/H/C/006538 Prophylaxis of influenza in adults 50 years of age and older
- Influenza vaccine (surface antigen, inactivated, prepared in cell cultures) - EMEA/H/C/006532, Article 28 Prophylaxis of influenza in adults and children from 2 years of age.
- Sipavibart - EMEA/H/C/006291 indicated for the pre-exposure prophylaxis of COVID-19 in adults and adolescents 12 years of age and older Accelerated review
- Bifikafusp alfa / Onfekafusp alfa - EMEA/H/C/005651 neoadjuvant treatment of adult patients with locally advanced fully resectable melanoma.
- Dorocubicel / Allogeneic umbilical cord derived CD34- cells, non-expanded - EMEA/H/C/005772, Orphan, ATMP Cordex Biologics International Limited, treatment of adult patients with haematological malignancies Accelerated review
- Zanidatamab - EMEA/H/C/006380, Orphan Jazz Pharmaceuticals Ireland Limited, Treatment of biliary tract cancer
- ATROPINE SULFATE PH. EUR. - EMEA/H/C/006385, PUMA, treatment of myopia in children aged 3
More information
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