EMA Looks Back at Medicines Approved in 2023

The European Medicines Agency (EMA) published Human Medicines Highlights 2023 on Jan. 16, 2024, which gives an overview of the medicines that received recommendations for marketing authorization in 2023. The agency recommended 77 medicines, 39 of which were new active substances to the European Union. Two vaccines were recommended to protect respiratory syncytial virus (RSV). In addition, a new drug that uses clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9 was recommended to treat two rare blood disorders.

Recommended approvals of specific note include the following:

• Nine cancer treatments including Omjjara (momelotinib), the first treatment for myelofibrosis; Columvi (glofitamab) and Tepkinly (epcoritamab) for the treatment of diffuse large B-cell lymphoma; and Elrexfio (elranatamab) for the treatment of adult patients with relapsed or refractory multiple myeloma
• Aqumeldi (enalapril maleate), which treats heart failure in children up to 18 years
• Skyclarys (omaveloxolone) for the treatment of Friedreich’s ataxia

• Arexvy (recombinant, adjuvanted) to protect adults aged 60 years and older against lower respiratory tract disease caused by RSV.

Seventeen orphan medicines were recommended for authorization that included treatments for cancer, chronic hypoparathyroidism, sickle cell disease, Duchenne muscular dystrophy, and a variety of other conditions.

Three medicines were recommended under the agency’s Priority Medicines (PRIME) program: cancer treatments, Elrexfio and Talvey; and Casgevy for the treatment of sickle cell disease.

There were 77 extensions for indications, which included 38 drugs for pediatrics.

Three medicines received negative approval from the agency in 2023: Albrioza (sodium phenylbutyrate/ursodoxicoltaurine) for the treatment of amyotrophic lateral sclerosis; Lagevrio (molnupiravir) for the treatment of COVID-19 in adults; and Sohonos (palovarotene), to treat fibrodysplasia ossificans progressive.

The agency monitors authorized medications after approval for quality and safety and provides industry with advice. Agency advice given in 2023 includes, but is not limited to, the following:

1. It was recommended that Adakveo (crizanlizumab) no longer be used to “prevent painful crises in patients aged 16 years and older with sickle cell disease, a genetic condition in which the red blood cells become rigid and sticky and change from being disc-shaped to being crescent-shaped (like a sickle).”
2. EMA reminded industry to use measures to reduce the risk involved in the use of fluoroquinolone antibiotics.
3. A lower dose of Olumiant (baricitinib) was recommended for patients “at higher risk of blood clots, cardiovascular conditions, and cancer in line with the dosing recommendations for other JAK [Janus kinase] inhibitors used to treat several chronic inflammatory disorders.”

EMA also suspended marketing authorization for more than 350 generic drugs that were tested by the contract research organization, Synapse Labs Pvt. Ltd., after a good clinical practice inspection found that supporting data were lacking or insufficient to show bioequivalence.

Source: EMA