CHMP Meeting Highlights December 2024 New
This month, 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. For more information please consult the product for Andembry on the EMA website.
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. For more information please consult the product for Beyonttra on the EMA website.
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. For more information please consult the product for Kavigale on the EMA website.
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. For more information please consult the product for Nemluvio on the EMA website.
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. For more information please consult the product for Rytelo on the EMA website.
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. For more information please consult the product for Seladelpar Gilead on the EMA website.
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. For more information please consult the product for Welireg on the EMA website.
Recommendations on extensions of therapeutic indication:
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. For more information please consult the product for Blincyto on the EMA website.
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. For more information please consult the product for Bridion on the EMA website.
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. For more information please consult the product for Flucelvax Tetraon the EMA website.
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. For more information please consult the product for Jemperli on the EMA website.
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. For more information please consult the product for Ofev on the EMA website.
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. For more information please consult the product for Omvoh on the EMA website.
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. For more information please consult the product for Rekambys on the EMA website.
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. For more information please consult the product for Vocabria 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:
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. EPAR Elahere.
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. EPAR Ordspono.
Recently started procedures:
- Bifikafusp alfa / Onfekafusp alfa - EMEA/H/C/005651 neoadjuvant treatment of adult patients with locally advanced fully resectable melanoma.
- 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.
- Denosumab - EMEA/H/C/006269, prevention of skeletal related events with advanced malignancies.
- Denosumab - EMEA/H/C/006268 treatment of osteoporosis and bone loss.
- 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.
- 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.
- L-Acetylleucine - EMEA/H/C/006327 is indicated in adults and children from birth for chronic treatment of Niemann-Pick Type C (NPC).
- 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.
- 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.
Previous CHMP Meeting Highlights can be accessed at: https://www.basg.gv.at/en/healthcare-professionals/chmp-highlights