CHMP Meeting Highlights June 2025 New
This month, medicinal products for the following indications have received a positive opinion:
- Tardive dyskinesia
- Acute Radiation Syndrome
- Desmoid tumours
- Noncirrhotic metabolic dysfunction-associated steatohepatitis
- Haematological malignancies
- New medicines recommended for approval:
Austedo (deutetrabenazine): has received a positive opinion for the treatment of moderate to severe tardive dyskinesia in adults. Tardive dyskinesia is characterized by involuntary repetitive body movements. The condition is caused by long-term usage of dopamine-receptor blocking drugs like antipsychotics. Deutetrabenazine is a deuterated variant of tetrabenazine - a monoamine-depleting agent - in which some of the hydrogen atoms are exchanged with the heavier deuterium isotopes. The positive opinion is currently under re-examination by request of the applicant. Für weitere Informationen siehe CHMP summary of positive opinion for Austedo.
Imreplys (sargramostim): has received a positive opinion for marketing authorisation under exceptional circumstances for the treatment of patients of all ages acutely exposed to myelosuppressive doses of radiation with Haematopoietic Sub-syndrome of Acute Radiation Syndrome [H-ARS]. Sargramostim is a granulocyte macrophage colony-stimulating factor (GM-CSF) and functions as an immunostimulator. It is primarily used after bone marrow transplantation. Acute Radiation Syndrome or radiation sickness is caused by high doses of ionizing radiation. Für weitere Informationen siehe CHMP summary of positive opinion for Imreplys.
Ogsiveo (nirogacestat): has received a positive opinion for the treatment of adult patients with progressing desmoid tumours who require systemic treatment. Desmoid tumours or aggressive fibromatosis is a rare type of cancer of the fibroblasts. In most cases the cancer is non-lethal but causes pain. Nirogacestat is an inhibitor of the gamma secretase. An orphan designation was granted for the treatment of this disease. Für weitere Informationen siehe CHMP summary of positive opinion for Ogsiveo.
Rezdiffra (resmetirom): has received a positive opinion in conjunction with diet and exercise for the treatment of adults with noncirrhotic metabolic dysfunction-associated steatohepatitis (MASH) with moderate to advanced liver fibrosis (fibrosis stages F2 to F3). MASH is a chronic liver disease characterized by excessive fat build-up in the liver and subsequent scarring of the liver. Resmetirom is an agonist of the thyroid hormone receptor beta and lowers the liver fat. The EMA has published a press release for this medicine. Für weitere Informationen siehe CHMP summary of positive opinion for Rezdiffra.
Zemcelpro: (Dorocubicel / Allogeneic umbilical cord-derived CD34- cells, non-expanded) has received a positive opinion for the conditional marketing authorisation for the treatment of adult patients with haematological malignancies requiring an allogeneic haematopoietic stem cell transplantation following myeloablative conditioning for whom no other type of suitable donor cells is available. An orphan designation was granted for the treatment of this disease. Zemcelpro was supported through EMA‘s Priority Medicines (PRIME) scheme and a press release for this medicine was released. Für weitere Informationen siehe CHMP summary of positive opinion for Zemcelpro.
Recommendations on extensions of therapeutic indication:
Benlysta (belimumab): extension of indication for Benlysta to include the add-on treatment of patients aged 5 years and older with active, autoantibody-positive systemic lupus erythematosus (SLE) with a high degree of disease activity (e.g., positive anti-dsDNA and low complement) despite standard therapy. Previously the usage was restricted to adult patients. Für weitere Informationen siehe CHMP summary of positive opinion for Benlysta.
Cabometyx (cabozantinib): extension of indication for Cabometyx to include the treatment of adult patients with unresectable or metastatic, well differentiated extra-pancreatic (epNET) and pancreatic (pNET) neuroendocrine tumours who have progressed following at least one prior systemic therapy other than somatostatin analogues. Cabometyx is already approved for the treatment of renal cell, hepatocellular and thyroid carcinoma. Für weitere Informationen siehe CHMP summary of positive opinion for Cabometyx.
Dapivirine Vaginal Ring 25 mg (dapivirine): lowering the age limit from 18 to 16 years of age. The indication wording is as follows:
Reducing the risk of HIV-1 infection via vaginal intercourse in HIV-uninfected women 16 years and older in combination with safer sex practices when oral PrEP is not/cannot be used or is not available.
Dapivirine Vaginal Ring 25 mg is part of the EU-Medicines for all program and is intended for use outside the EU. Für weitere Informationen siehe CHMP summary of positive opinion for Dapivirine.
Darzalex (daratumumab): extension of indication for Darzalex to include the treatment of adult patients with smouldering multiple myeloma at high risk of developing multiple myeloma.
Darzalex is already approved for the treatment of multiple myeloma in different settings. Für weitere Informationen siehe CHMP summary of positive opinion for Darzalex.
Imbruvica (ibrutinib): extension of indication for Imbruvica to include the treatment in combination with rituximab, cyclophosphamide, doxorubicin, vincristine, and prednisolone (Imbruvica + R-CHOP) alternating with R-DHAP (or R-DHAOx) without Imbruvica, followed by Imbruvica monotherapy, is indicated for the treatment of adult patients with previously untreated mantle cell lymphoma (MCL) who would be eligible for autologous stem cell transplantation (ASCT). Imbruvica is already approved for the treatment of relapsed or refractory MCL as monotherapy. It is also approved for the treatment of chronic lymphocytic leukaemia and Waldenström’s macroglobulinaemia. Für weitere Informationen siehe CHMP summary of positive opinion for Imbruvica.
Nubeqa (darolutamide): extension of indication for Nubeqa to include the treatment of adult men with metastatic hormone‑sensitive prostate cancer (mHSPC) in combination with androgen deprivation therapy. Nubeqa is already approved for the treatment of non‑metastatic castration resistant prostate cancer and metastatic hormone‑sensitive prostate cancer. Für weitere Informationen siehe CHMP summary of positive opinion for Nubeqa.
Sarclisa (isatuximab): extension of indication for Sarclisa to include the treatment in combination with bortezomib, lenalidomide, and dexamethasone, for the induction treatment of adult patients with newly diagnosed multiple myeloma who are eligible for autologous stem cell transplant. Sarclisa is already approved for the treatment of multiple myeloma in different settings. Für weitere Informationen siehe CHMP summary of positive opinion for Sarclisa.
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:
Duvyzat: is indicated for the treatment of Duchenne muscular dystrophy (DMD) in ambulant patients, aged 6 years and older, and with concomitant corticosteroid treatment. EPAR Duvyzat.
Oczyesa: is indicated for maintenance treatment in adult patients with acromegaly who have responded to and tolerated treatment with somatostatin analogues. EPAR Oczyesa.
Tepezza: is indicated in adults for the treatment of moderate to severe thyroid eye disease (TED). EPAR Tepezza.
Ziihera: as monotherapy is indicated for the treatment of adults with unresectable locally advanced or metastatic HER2-positive (IHC3+) biliary tract cancer (BTC): previously treated with at least one prior line of systemic therapy. EPAR Ziihera.
Previous CHMP Meeting Highlights can be accessed at: https://www.basg.gv.at/en/healthcare-professionals/chmp-highlights
