EMA's CHMP Recommends 10 New Medicines, Including Novel Therapies and Biosimilars

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查看药品详情 (concizumab查看药品详情) for the routine prophylaxis of bleeding in patients with hemophilia A (搜索)搜索该疾病 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查看药品详情 (eplontersen查看药品详情), an orphan drug designated for treating adults with hereditary transthyretin-mediated amyloidosis (搜索)搜索该疾病 (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查看药品详情 (catumaxomab查看药品详情) was recommended to treat intraperitoneal malignant ascites (搜索)搜索该疾病, a build-up of fluid containing cancer cells in the abdomen of cancer patients. Siiltibcy (搜索)搜索该药品 (rdESAT-6/rCFP-10 (搜索)搜索该药品) was recommended to diagnose Mycobacterium tuberculosis infection. Buprenorphine Neuraxpharm查看药品详情 (buprenorphine查看药品详情) was recommended to treat opioid addiction (搜索)搜索该疾病.

Biosimilars and Generics

The CHMP also granted positive opinions for two biosimilars, Absimky查看药品详情 (ustekinumab查看药品详情) and Imuldosa查看药品详情 (ustekinumab), indicated for the treatment of plaque psoriasis (搜索)搜索该疾病 in children and adults, psoriatic arthritis (搜索)搜索该疾病, Crohn’s disease (搜索)搜索该疾病, and ulcerative colitis (搜索)搜索该疾病. 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查看药品详情 (eltrombopag查看药品详情), a generic drug, was recommended for treating children and adults with primary immune thrombocytopenia (搜索)搜索该疾病 and thrombocytopenia (搜索)搜索该疾病 associated with chronic hepatitis C (搜索)搜索该疾病. 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查看药品详情 (eliglustat查看药品详情), Hepcludex查看药品详情 (bulevirtide查看药品详情), Kevzara查看药品详情 (sarilumab查看药品详情), Kisqali查看药品详情 (ribociclib查看药品详情), Tevimbra查看药品详情 (tislelizumab查看药品详情), and Yselty查看药品详情 (linzagolix choline查看药品详情). 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 (搜索)搜索该词条 (masitinib查看药品详情) for treating amyotrophic lateral sclerosis (搜索)搜索该疾病 (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 (搜索)搜索该药品 (apremilast查看药品详情) and Epixram (搜索)搜索该药品 (levetiracetam查看药品详情), were withdrawn from consideration.