The European Medicines Agency (EMA) announced on October 18, 2024, that its Committee for Medicinal Products for Human Use (CHMP) has issued positive recommendations for 10 new medicines. These include five new drugs, one orphan drug, two biosimilars, and two generic drugs, marking a significant step in expanding treatment options for a variety of conditions. The CHMP also recommended extensions of indications for six existing drugs.
Novel Therapies Recommended
Among the novel therapies receiving positive opinions are Alhemo (concizumab) for the routine prophylaxis of bleeding in patients with hemophilia A or B with inhibitors. Hemophilia, a genetic bleeding disorder, often requires frequent infusions of clotting factors to prevent life-threatening bleeds. Concizumab offers a new approach to managing this condition.
Another notable recommendation is for Wainzua (eplontersen), an orphan drug designated for treating adults with hereditary transthyretin-mediated amyloidosis (ATTR). ATTR amyloidosis is a rare, progressive disease caused by the misfolding and aggregation of the transthyretin protein, leading to organ damage. Eplontersen aims to address this unmet need by targeting the underlying cause of the disease.
Korjuny (catumaxomab) was recommended to treat intraperitoneal malignant ascites, a build-up of fluid containing cancer cells in the abdomen of cancer patients. Siiltibcy (rdESAT-6/rCFP-10) was recommended to diagnose Mycobacterium tuberculosis infection. Buprenorphine Neuraxpharm (buprenorphine) was recommended to treat opioid addiction.
Biosimilars and Generics
The CHMP also granted positive opinions for two biosimilars, Absimky (ustekinumab) and Imuldosa (ustekinumab), indicated for the treatment of plaque psoriasis in children and adults, psoriatic arthritis, Crohn’s disease, and ulcerative colitis. Ustekinumab, a monoclonal antibody targeting interleukin-12 and interleukin-23, has proven effective in managing these immune-mediated inflammatory diseases. The availability of biosimilars offers a more cost-effective alternative to the originator product.
Eltrombopag Viatris (eltrombopag), a generic drug, was recommended for treating children and adults with primary immune thrombocytopenia and thrombocytopenia associated with chronic hepatitis C. Eltrombopag stimulates the production of platelets, helping to reduce the risk of bleeding in these patients.
Expanded Indications
In addition to new drug recommendations, the CHMP recommended extending the indications for several existing medications, including Cerdelga (eliglustat), Hepcludex (bulevirtide), Kevzara (sarilumab), Kisqali (ribociclib), Tevimbra (tislelizumab), and Yselty (linzagolix choline). These extensions aim to broaden the use of these drugs to benefit a wider range of patients.
Rejection of Masitinib Confirmed
Notably, the CHMP confirmed its earlier decision to refuse marketing authorization for Masitinib AB Science (masitinib) for treating amyotrophic lateral sclerosis (ALS). The agency cited concerns regarding the reliability of study data and the inability to convincingly demonstrate the drug's benefits. Despite a re-examination of the application and consultation with experts, the CHMP maintained its opinion that the benefits of masitinib did not outweigh its risks.
Two drug applications, Apremilast Viatris (apremilast) and Epixram (levetiracetam), were withdrawn from consideration.
