The European Medicines Agency (EMA) has issued a series of recommendations for new drug approvals and expanded indications that promise to reshape treatment paradigms across several key therapeutic areas. The Committee for Medicinal Products for Human Use (CHMP) recently convened and recommended the approval of ten new medicines, including therapies for hemophilia, hereditary transthyretin-mediated amyloidosis, and cancer, alongside updates to COVID-19 vaccines. These decisions reflect the EMA's ongoing commitment to facilitating access to innovative treatments for patients across the European Union.
Approvals for Rare and Genetic Diseases
Among the notable recommendations is Alhemo (concizumab), a medicine designed for routine prophylaxis of bleeding in patients with hemophilia A or B with inhibitors, addressing a critical need in managing this rare inherited bleeding disorder. Additionally, Wainzua (eplontersen) received a positive opinion for treating adults with hereditary transthyretin-mediated amyloidosis, a rare and often fatal disease caused by variants in the transthyretin gene, which leads to polyneuropathy.
Hympavzi (marstacimab) also received a positive opinion for the treatment of bleeding episodes in patients aged 12 years and older with severe haemophilia A or B.
Oncology Advancements
In oncology, the CHMP recommended Elahere (mirvetuximab soravtansine) for treating adults with ovarian cancer, fallopian tube cancer, or primary peritoneal cancer. Hetronifly (serplulimab) was approved for the treatment of extensive-stage small cell lung cancer. Pomalidomide Teva (pomalidomide), a generic medicine, received a positive opinion for the treatment of multiple myeloma.
Korjuny (catumaxomab) received a positive opinion for the intraperitoneal treatment of malignant ascites, a build-up of fluid containing cancer cells in the abdomen of cancer patients.
Vaccines and Diagnostics
The CHMP also adopted positive opinions for two influenza vaccines, Fluad and Flucelvax, intended for active immunization against influenza in adults and children, respectively. Siiltibcy (rdESAT-6 / rCFP-10) received a positive opinion as a diagnostic tool for infection with Mycobacterium tuberculosis, addressing a significant global health concern, with 10.6 million new cases estimated and 1.6 million deaths reported in 2021, according to the World Health Organization.
Penbraya (meningococcal groups A, C, W, Y conjugate and group B vaccine (recombinant, adsorbed)) received a positive opinion for immunization against invasive disease caused by Neisseria meningitidis.
Biosimilars and Generics
Further expanding treatment options, the committee recommended granting marketing authorizations for Absimky (ustekinumab) and Imuldosa (ustekinumab), two biosimilar medicines for treating plaque psoriasis, psoriatic arthritis, Crohn’s disease, and ulcerative colitis. Eltrombopag Viatris (eltrombopag), a generic medicine for treating primary immune thrombocytopenia and thrombocytopenia associated with chronic hepatitis C, was also recommended for approval.
Biosimilars Afqlir (aflibercept) and Opuviz (aflibercept) received positive opinions for the treatment of age-related macular degeneration.
Expanded Indications
The CHMP recommended extensions of indication for twelve already authorized medicines, including Cerdelga (eliglustat), Hepcludex (bulevirtide), Kevzara (sarilumab), Kisqali (ribociclib), Tevimbra (tislelizumab), Yselty (linzagolix choline), Imvanex, Aflunov, Buccolam, Darzalex, Dupixent, Esperoct, Fasenra, Keytruda, Otezla, Pravafenix, Synjardy and Zavicefta, broadening their therapeutic applications.
Leadership Transition
In leadership news, Bruno Sepodes has been elected as the new chair of the CHMP, succeeding Dr. Harald Enzmann. Outi Mäki-Ikola was elected as the new vice-chair for a three-year mandate, starting on 15 October 2024.
