CHMP Meeting Highlights October 2022
This month, medicinal products for the following indications have received a positive opinion:
- Cholestatic pruritus in patients with Alagille syndrome
- Epstein-Barr virus positive post-transplant lymphoproliferative disease
- Generalised pustular psoriasis
- Prevention of dengue disease
- Prostate cancer
New medicines recommended for approval:
Ebvallo (tabelecleucel): received a positive opinion for an authorisation under exceptional circumstances for the treatment, as monotherapy, of adult and paediatric patients 2 years of age and older with relapsed or refractory Epstein-Barr virus positive post-transplant lymphoproliferative disease (EBV+ PTLD) who have received at least one prior therapy. For solid organ transplant patients, prior therapy includes chemotherapy unless chemotherapy is inappropriate.
EBV+ PTLD is a rare hematologic malignancy that develops in patients under immunosuppression after organ or haematopoietic cell transplant. Ebvallo is an advanced therapy medicinal product (ATMP) composed of allogeneic anti-EBV cytotoxic T cells. For more information visit the EMA news announcement on Ebvallo. For more information please consult the product for Ebvallo on the EMA website.
Eladynos (abaloparatide): is indicated for the treatment of osteoporosis in postmenopausal women at increased risk of fracture. Osteoporosis is a skeletal disorder characterised by low bone density, which leads to an increased risk of fragility fractures. It is a common disease that develops with advancing age, most commonly in women after menopause. Abaloparatide is a 34 amino acid synthetic analogue of the human parathyroid hormone-related peptide and shows high homology to the naturally occurring human parathyroid hormone, a regulator of calcium homeostasis. Abaloparatide functions as an anabolic agent in the bone. For more information please consult the product for Eladynos on the EMA website.
Livmarli (maralixibat chloride): received a positive opinion for a marketing authorisation under exceptional circumstances for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) 2 months of age and older. Alagille syndrome is a rare genetic disorder caused by mutations in the JAG1 or, much less commonly, in the NOTCH2 gene, both encoding proteins involved in the Notch-signalling cascade. Although it presents with variable penetrance and clinical manifestations, the liver and heart are usually affected. Hepatic manifestations often include cholestatic pruritus, an itchy sensation caused by elevated serum bile acids (sBA) and the most burdensome symptom. By inhibiting the apical sodium–bile acid transporter, maralixibat blocks the reabsorption of bile acids in the intestine, thereby reducing sBA levels. For more information please consult the product for Livmarli on the EMA website.
Pluvicto [lutetium (177Lu) vipivotide tetraxetan]: in combination with androgen deprivation therapy (ADT) with or without androgen receptor (AR) pathway inhibition, is indicated for the treatment of adult patients with progressive prostate-specific membrane antigen (PSMA)-positive metastatic castration-resistant prostate cancer (mCRPC) who have been treated with AR pathway inhibition and taxane based chemotherapy.
Prostate cancer is the second most common cancer in men. The membrane protein PSMA is expressed in the prostate in high levels and further overexpressed in prostate cancer cells and, therefore, serves as a target for prostate cancer therapy and labelling. Pluvicto is a targeted radioligand, consisting of a PSMA-targeting ligand bound to a chelator complexed with 177Lu, a radioactive beta-emitter. Through binding to PSMA and subsequent internalisation, Pluvicto delivers therapeutic radiation to prostate cancer cells.
Furthermore, CHMP has given a positive opinion for the authorisation of the radiolabelling agent Locametz (gozetotide). For more information please consult the product for Pluvicto on the EMA website.
Locametz (gozetotide): is indicated after radiolabelling with gallium 68, for the detection of prostate specific membrane antigen (PSMA) positive lesions with positron emission tomography (PET) in adults with prostate cancer (PCa) in the following clinical settings:
- Primary staging of patients with high risk PCa prior to primary curative therapy;
- Suspected PCa recurrence in patients with increasing levels of serum prostate specific antigen (PSA) after primary curative therapy;
- Identification of patients with PSMA positive progressive metastatic castration resistant prostate cancer (mCRPC) for whom PSMA targeted therapy is indicated.
Locametz is a PSMA-targeting ligand bound to a chelating moiety. Once complexed with the positron-emitting 68Ga isotope, it is used for imaging prostate cancer by PET. By binding to PSMA, the radiopharmaceutical is specifically directed towards prostate cancer cells. For more information please consult the product for Locametz on the EMA website.
Qdenga [dengue tetravalent vaccine (live, attenuated)]: is indicated for the prevention of dengue disease in individuals from 4 years of age. Dengue fever is a mosquito-borne disease caused by the dengue virus, a single-stranded RNA virus of the flaviviridae family. Dengue fever is one of the most common vector-borne diseases globally. Dengue fever usually leads to mild, flu-like symptoms. However, unlike in most other cases, second infections tend to be more severe, with potentially fatal bleeding and organ damage. Antibody-dependent enhancement is presumed to be the main cause of the more severe clinical course of a second infection.
Qdenga is a live attenuated viral vaccine, which replicates locally and elicits an immune response against the 4 viral serotypes. Qdenga is indicated for both seropositive and seronegative persons. Accordingly, no determination of serostatus is necessary prior to vaccination, at variance with the previously authorised vaccine Dengvaxia®, which is indicated for persons with test-confirmed previous dengue infection only. For more information, visit the EMA news announcement on Qdenga. For more information please consult the product for Qdenga on the EMA website.
Spevigo (spesolimab): received a positive opinion for a conditional marketing authorisation (CMA) for the treatment of flares in adult patients with generalised pustular psoriasis (GPP) as monotherapy. GPP is a potentially fatal rare type of psoriasis, a chronic inflammatory skin disease, characterised by the eruption of pustules on the skin, as well as the presence of systemic symptoms such as fever. Although it is unclear what triggers the flares, mutations in the interleukin-36 receptor gene (IL-36R) have been shown to cause GPP. Spevigo is a monoclonal antibody that targets IL-36R and blocks the binding to its ligands, thereby exerting its immunosuppressive function. For more information please consult the product for Spevigo on the EMA website.
Recommendations on extensions of therapeutic indication:
Brukinsa (zanubrutinib): extension of indication to include the treatment of adult patients with chronic lymphocytic leukaemia, as monotherapy. Brukinsa was already authorised for the treatment of adult patients with Waldenström’s macroglobulinaemia. For more information please consult the product for Brukinsa on the EMA website.
Libtayo (cemiplimab): extension of indication to include the treatment of adult patients with recurrent or metastatic cervical cancer and disease progression on or after platinum-based chemotherapy, as monotherapy. Libtayo was already authorised for the treatment of cutaneous squamous cell carcinoma, basal cell carcinoma and non-small cell lung cancer. For more information please consult the product for Libtayo on the EMA website.
Lyumjev (insulin lispro): extension of indication to include the treatment of diabetes mellitus in children aged 1 year and above. Lyumjev was already authorised in adults. For more information please consult the product for Lyumjev on the EMA website.
Xydalba (dalbavancin): extension of indication to include the treatment of acute bacterial skin and skin structure infections in paediatric patients aged 3 months and older. Xydalba was already authorised in adults. For more information please consult the product for Xydalba 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:
Amvuttra (vutrisiran): is indicated for the treatment of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in adult patients with stage 1 or stage 2 polyneuropathy. EPAR Amvuttra.
Lupkynis (voclosporin): is indicated in combination with mycophenolate mofetil for the treatment of adult patients with active class III, IV or V (including mixed class III/V and IV/V) lupus nephritis. EPAR Lupkynis.
Nulibry (fosdenopterin): is indicated for the treatment of patients with molybdenum cofactor deficiency Type A. EPAR Nulibry.
Rayvow (lasmiditan): is indicated for the acute treatment of the headache phase of migraine attacks, with or without aura in adults. EPAR Rayvow.
Tabrecta (capmatinib): as monotherapy, is indicated for the treatment of adult patients with advanced non-small cell lung cancer harbouring alterations leading to mesenchymal-epithelial transition factor gene exon 14 (METex14) skipping, who require systemic therapy following prior treatment with immunotherapy and/or platinum-based chemotherapy. EPAR Tabrecta.
Tecvayli (teclistamab): 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 Tecvayli.
Tezspire (tezepelumab): is indicated as an add-on maintenance treatment in adults and adolescents 12 years and older with severe asthma who are inadequately controlled despite high dose inhaled corticosteroids plus another medicinal product for maintenance treatment. EPAR Tezspire.
Vabysmo (faricimab): is indicated for the treatment of adult patients with neovascular (wet) age-related macular degeneration or with visual impairment due to diabetic macular oedema. EPAR Vabysmo.
Recently started procedures:
- Aripiprazole - Maintenance treatment of schizophrenia.
- Crisantaspase - Indicated as a component of a multi-agent chemotherapeutic regimen for the treatment of acute lymphoblastic leukaemia and lymphoblastic lymphoma.
- Pirtobrutinib - Orphan - Treatment of mantle cell lymphoma.
- Treprostinil diolamine - Orphan - Treatment of pulmonary arterial hypertension (WHO Group 1) to delay disease progression and to improve exercise capacity.
- Gentamicin sulfate / sargramostim / heparin sodium / insulin human - Human assisted reproductive techniques including in vitro fertilisation procedures.
Other topics of interest:
Update on the following authorised medicines for the prevention of COVID-19:
The COVID-19 vaccine Vaxzevria has been recommended for a switch from CMA to standard authorisation following the submission of data that fulfils the outstanding specific obligations.