Several Orphan Drugs Among Medicines Recommended for Approval by EMA in December

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The European Medicines Agency’s (EMA’s) Committee for Medicinal Products for Human Use (CHMP) recommended 17 drugs for market authorization at its December meeting, EMA announced on Dec. 13, 2024. The approvals include five orphan drug recommendations and six biosimilar recommendations.

Among the approval recommendations is a monoclonal antibody, Kavigale (sipavibart), for COVID-19 prevention in immunocompromised people aged 12 and older. “Following the recommendation for approval of Kavigale, EMA’s Emergency Task Force (ETF) has issued an updated statement on the loss of activity of anti-spike protein monoclonal antibodies due to emerging SARS-CoV-2 variants and recommends healthcare professionals to check the current epidemiological situation in their region,” the agency stated in a press release (1). Kostaive (zapomeran), a self-amplifying messenger RNA vaccine, was also recommended for the prevention of COVID-19.

Orphan drugs recommended by CHMP include the following:

• Andembry(garadacimab), which prevents recurrent attacks of hereditary angioedema
• Beyonttra (acoramidis), which treats a wild-type or variant transthyretin amyloidosis in adult patients with cardiomyopathy
• Rytelo(imetelstat), which treats adults with transfusion-dependent anemia due to very low, low, or intermediate risk myelodysplastic syndromes
• Seladelpar Gilead(seladelpar lysine dihydrate), which treats primary biliary cholangitis
• Emcitate(tiratricol), which treats peripheral thyrotoxicosis in patients with Allan-Herndon-Dudley syndrome.

The following six biosimilars were recommended for authorization:

• Avtozma (tocilizumab) was given positive recommendation for treating rheumatoid arthritis, systemic juvenile idiopathic arthritis, polyarticular juvenile idiopathic arthritis, giant cell arteritis, chimeric antigen T cell (CAR-T cell)-induced severe or life-threatening cytokine release syndrome and COVID-19.
• Eydenzelt (aflibercept) treats eye diseases such as neovascular age-related macular degeneration, macular oedema secondary to retinal vein occlusion, diabetic macular oedema, and myopic choroidal neovascularization.
• Osenvelt (denosumab) prevents bone complications in adults with advanced bone cancer and treats adults and skeletally mature adolescents with giant cell bone tumors.
• Stoboclo (denosumab) treats osteoporosis in menopausal women, bone loss linked to hormone ablation in men at increased risk of fractures, and bone loss associated with long-term treatment with systemic glucocorticoid.
• Yesintek (ustekinumab) treats adults and children with plaque psoriasis and adults with psoriatic arthritis or Crohn’s disease.
• Zefylti (filgrastim) treats neutropenia and the mobilization of peripheral blood progenitor cells.

Other positive opinions given by CHMP include:

• Nemluvio (nemolizumab), which treats atopic dermatitis and prurigo nodularis
• Welireg(belzutifan), to treat tumors associated with von Hippel-Lindau disease and advanced clear cell renal cell carcinoma
• Paxneury (guanfacine), which treats children with attention-deficit hyperactivity disorder
• Tuzulby (methylphenidate hydrochloride) to treat children with attention-deficit hyperactivity disorder.

The December recommendations bring the total of drugs recommended for approval by CHMP for 2024 to 114.

CHMP also extended recommendations for several drugs, including Ofev for the treatment of progressive fibrosing interstitial lung diseases (ILDs) in children and adolescents, Blincyto, Bridion, Flucelvax Tetra, Jemperli, Omvoh, Rekambys, and Vocabria.

Also during the December meeting, CHMP chose to withdraw Alofisel (darvadstrocel) from the market after data showed its effectiveness was not what had been anticipated. Alofisel had been approved to treat complex anal fistulas in adults with Crohn’s disease.

Reference

1. EMA. Meeting highlights from the Committee for Medicinal Products for Human Use(CHMP) 9-12 December 2024. Press Release. Dec. 13, 2024.