Nineteen further treatments recommended as CHMP continues active approvals cycle

The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has issued a further nineteen positive recommendations spanning oncology, neurology, infectious disease, dermatology, cardiovascular medicine, diabetes, and rare conditions. The batch includes treatments for conditions ranging from Parkinson’s disease and Rett syndrome to HIV, monkeypox, and multiple myeloma, as well as two biosimilar approvals that will support wider patient access to established therapies.

Oncology and dermatology feature prominently in this cycle, with notable recommendations for novel antibody-drug conjugates, a new JAK inhibitor indication, and an innovative combination approach in multiple myeloma. The cycle also includes three vaccine recommendations addressing chikungunya, meningococcal disease, influenza, and orthopoxvirus infections.

Oncology

Datroway recommended for triple-negative breast cancer

Datopotamab deruxtecan (Datroway), an antibody-drug conjugate developed by Daiichi Sankyo and AstraZeneca, has received a positive CHMP recommendation for triple-negative breast cancer (TNBC). TNBC remains one of the most challenging breast cancer subtypes to treat, given its lack of hormone receptor and HER2 expression. Datroway targets TROP2, a protein overexpressed in many epithelial tumours, and delivers a cytotoxic payload selectively to cancer cells, offering a more targeted approach than conventional chemotherapy.

Tecvayli and Darzalex Faspro combination recommended for multiple myeloma

Johnson & Johnson has received a CHMP recommendation for the combination of teclistamab and daratumumab (Tecvayli and Darzalex Faspro) for multiple myeloma. Teclistamab is a bispecific T-cell engager targeting BCMA and CD3, while daratumumab is a well-established anti-CD38 monoclonal antibody. Combining the two mechanisms in a chemotherapy-free regimen represents a significant advance in the treatment of relapsed or refractory multiple myeloma, a condition that typically requires repeated lines of therapy over time.

Jaypirca recommended for chronic lymphocytic leukaemia

Pirtobrutinib (Jaypirca), a non-covalent BTK inhibitor developed by Loxo Oncology at Lilly and Eli Lilly, has been recommended for chronic lymphocytic leukaemia and small lymphocytic lymphoma (CLL/SLL). Unlike covalent BTK inhibitors such as ibrutinib, pirtobrutinib is designed to remain active in patients who have developed resistance mutations, offering a treatment option for a population with limited alternatives following prior BTK inhibitor therapy.

Denosumab biosimilar recommended for bone complications in cancer

Denosumab Ascend, a biosimilar of denosumab developed by Ascend GmbH, has received a CHMP recommendation for the prevention of skeletal-related events in patients with bone metastases and for giant cell tumour of bone. The availability of a biosimilar version of this widely used bone-targeted agent is expected to improve patient access and reduce costs for health systems across the EU.

Pegfilgrastim biosimilar recommended for neutropenia

Nylaspeg, a biosimilar of pegfilgrastim submitted by Qilu Pharmaceuticals, has received a positive recommendation for chemotherapy-induced neutropenia. Pegfilgrastim is routinely used to stimulate white blood cell production and reduce the risk of febrile neutropenia in cancer patients receiving myelosuppressive chemotherapy. The biosimilar recommendation supports continued access to this supportive care treatment at competitive cost.

Dermatology & Immunology

Rinvoq recommended for vitiligo and alopecia areata

AbbVie’s upadacitinib (Rinvoq), a selective JAK1 inhibitor, has received two separate CHMP recommendations expanding its indications into dermatology: one for vitiligo and one for alopecia areata. Vitiligo is a chronic autoimmune condition causing depigmentation of the skin, while alopecia areata results in patchy or total hair loss driven by immune-mediated attack on hair follicles. Both conditions have historically lacked effective systemic treatments, and these recommendations follow growing clinical evidence supporting JAK inhibition as a mechanism of action in autoimmune skin diseases. Rinvoq is already approved for several inflammatory conditions including rheumatoid arthritis, psoriatic arthritis, and atopic dermatitis.

Opzelura recommended for atopic dermatitis

Ruxolitinib cream (Opzelura), developed by Incyte Corporation and Maruho, has received a CHMP recommendation for atopic dermatitis (eczema). Opzelura is a topical JAK1/JAK2 inhibitor that provides targeted anti-inflammatory activity at the site of application, offering an alternative to topical corticosteroids for patients with mild to moderate disease. The recommendation addresses a highly prevalent condition that places significant quality of life burden on patients, particularly those with disease affecting visible areas of skin.

Neurology & Paediatrics

Daybue recommended for Rett syndrome

Trofinetide (Daybue), developed by Acadia Pharmaceuticals, has received a CHMP recommendation for Rett syndrome, a rare and severe neurodevelopmental disorder caused by mutations in the MECP2 gene, which predominantly affects girls. The condition leads to progressive loss of motor and communication skills and has no approved disease-modifying treatments in the EU. Daybue is a synthetic analogue of the naturally occurring tripeptide GPE (glycine-proline-glutamate) and represents the first pharmacological treatment specifically developed for this condition to receive a positive EU recommendation.

Crexont recommended for Parkinson’s disease

A prolonged-release combination of carbidopa and levodopa (Crexont), submitted by Amneal Pharmaceuticals and Zambon, has been recommended for Parkinson’s disease. Levodopa remains the cornerstone of Parkinson’s pharmacotherapy, but standard formulations are associated with motor fluctuations and wearing-off effects as the disease progresses. The extended-release formulation aims to provide more consistent plasma drug levels throughout the day, potentially reducing the frequency and severity of motor complications in patients with advanced disease.

Stelara recommended for paediatric ulcerative colitis

Ustekinumab (Stelara), developed by Johnson & Johnson, has received a CHMP recommendation for paediatric ulcerative colitis. Ustekinumab is an interleukin-12 and interleukin-23 inhibitor already established in adult inflammatory bowel disease and psoriasis. This paediatric recommendation addresses a significant unmet need in younger patients with moderate to severe ulcerative colitis who have an inadequate response to conventional therapy.

Infectious Diseases & Vaccines

Ixchiq recommended for chikungunya fever

Ixchiq, a live-attenuated chikungunya vaccine developed by Valneva SE and Instituto Butantan, has received a positive CHMP recommendation. Chikungunya is a mosquito-borne viral illness characterised by fever and severe joint pain, with outbreaks increasingly reported in southern Europe due to the northward spread of the Aedes albopictus mosquito. The approval of a preventive vaccine represents an important public health tool as the geographical range of the disease continues to expand.

MenQuadfi recommended for meningococcal disease

MenQuadfi, a conjugate vaccine targeting meningococcal serogroups A, C, W, and Y, developed by Sanofi, has received a CHMP recommendation. Meningococcal disease can cause life-threatening bacterial meningitis and septicaemia, particularly in infants, young children, and adolescents. The conjugate formulation is designed to produce a robust and long-lasting immune response and is suitable for use in paediatric populations.

Imvanex recommended for monkeypox and orthopoxvirus infections

Bavarian Nordic’s Imvanex, a modified vaccinia Ankara-based vaccine, has received a CHMP recommendation for protection against monkeypox (mpox) and other orthopoxvirus infections. Imvanex was already authorised for smallpox prevention in the EU and was used extensively during the 2022 mpox outbreak. This recommendation formalises its indication for mpox and broader orthopoxvirus prophylaxis, reflecting the continued public health relevance of the product.

Aujemflu recommended for influenza

Aujemflu, an adjuvanted trivalent influenza vaccine developed by Seqirus/CSL, has received a CHMP recommendation for the prevention of influenza. The adjuvanted formulation is designed to enhance immunogenicity, particularly in older adults and immunocompromised populations where vaccine responses may be suboptimal. The recommendation adds to the existing portfolio of seasonal influenza vaccines available to EU member states.

Symtuza and Prezcobix recommended for HIV

Two HIV antiretroviral regimens have received CHMP recommendations. Symtuza — a single-tablet combination of darunavir, emtricitabine, cobicistat, and tenofovir alafenamide (TAF), developed by Johnson & Johnson and Gilead Sciences — offers a complete one-tablet-once-daily regimen for HIV-1 infection. Separately, Prezcobix — a combination of darunavir and cobicistat developed by Janssen Therapeutics — provides a boosted protease inhibitor backbone for use in combination with other antiretrovirals. Both recommendations likely reflect updated indications or label revisions for these established regimens.

Cardiovascular & Diabetes

Leqvio recommended for hypercholesterolaemia

Inclisiran (Leqvio), a small interfering RNA (siRNA) therapy developed by Novartis, has received a CHMP recommendation for hypercholesterolaemia. Inclisiran works by inhibiting the production of PCSK9, a protein that reduces the liver’s ability to clear LDL cholesterol from the blood. Administered just twice yearly by subcutaneous injection, inclisiran offers a highly differentiated dosing schedule compared with daily oral statins or monthly PCSK9 inhibitor antibodies, which may improve adherence in patients requiring long-term lipid-lowering therapy.

Onswik recommended for type 2 diabetes

Insulin efsitora alfa (Onswik), a once-weekly basal insulin analogue developed by Eli Lilly, has received a positive CHMP recommendation for type 2 diabetes. The treatment represents a significant step forward in insulin therapy, offering a weekly injection as an alternative to the daily or twice-daily basal insulin regimens that many patients find burdensome. A reduced injection frequency is expected to support better adherence and patient quality of life, particularly in the early stages of insulin initiation.

All nineteen decisions listed carry CHMP Recommended status. CHMP recommendations are subject to final marketing authorisation by the European Commission. This article is based on data from the European Medicines Agency CHMP opinion cycle.