Wednesday, January 17, 2024

EMA - Orphan Designation and Approvals in 2023

European Medicines Agency (EMA) 's an important agency that plays a vital role in safeguarding public health in the European Union. 

In 2023, EMA recommended 77 medicines for marketing authorization. Of these, 39 had a new active substance which had never been authorized in the European Union (EU) before. Among these are a number of medicines that stand out due to their contribution to address public health needs or the innovation they represent. Among these 77 positive opinions, 39 are new active substances, 3 prime, 17 orphan medicines, 1 advanced therapy medicinal product, 8 biosimilars, 14 generics, 3 accelerated assessments, 8 conditional marketing authorization, and 1 approval under exceptional circumstances.

The Agency recommended two vaccines to protect against lower respiratory tract disease caused by respiratory syncytial virus (RSV), and the first advanced therapy medicinal product using a ground-breaking gene-editing technology known as CRISPR/Cas9 to treat two rare blood disorders. EMA also adopted two positive opinions for medicines for use in countries outside the EU. 

Authorization of new medicines is essential to advancing public health. Below is a selection of medicines recommended for approval in 2023:

CANCER: Columvi (glofitamab) and Tepkinly (epcoritamab), Elrexfio (elranatamab), Finlee (dabrafenib), Jaypirca (pirtobrutinib), Krazati (adagrasib), Lytgobi (futibatinib), Omjjara (momelotinib), Pedmarqsi (sodium thiosulfate), Talvey (talquetamab).

CARDIOVASCULAR: Aqumeldi (enalapril maleate), Camzyos (mavacamten).

HEMATOLOGY: Casgevy (exagamglogene autotemcel)

METABOLISM: Loargys (pegzilarginase)

NEUROLOGY: Skyclarys (omaveloxolone)

VACCINES: Abrysvo (bivalent, recombinant), Arexvy (recombinant, adjuvanted)

Three medicines received a recommendation for marketing authorization following an accelerated assessment. It allows for faster assessment of eligible medicines by the European Medicines Agency's scientific committees (within a maximum of 150 days rather than 210 days) - Talvey (talquetamab) Abrysvo (bivalent, recombinant) and Arexvy (recombinant, adjuvanted)

Three PRIME-designated medicines were recommended - Elrexfio (elranatamab), Talvey (talquetamab) and Casgevy (exagamglogene autotemcel). 

17 medicines had their orphan designation confirmed by the end of 2023 - Columvi (glofitamab), Finlee (dabrafenib), Omjjara (momelotinib), Spexotras (trametinib), Talvey (talquetamab), Tepkinly (epcoritamab), Tevimbra8 (tislelizumab), Tibsovo (ivosidenib), Hyftor (sirolimus), Yorvipath (palopegteriparatide), Casgevy (exagamglogene autotemcel), Rezzayo (rezafungin), Loargys (pegzilarginase), Agamree (vamorolone), Rystiggo (rozanolixizumab), Skyclarys (omaveloxolone) and Ztalmy (ganaxolone),

Here's a summary of European Medicines Agency's key functions:

  • Scientific evaluation of medicines: The EMA is responsible for assessing the safety, efficacy, and quality of medicines before they can be marketed in the EU. This involves a rigorous review process that includes examining clinical trial data, manufacturing processes, and potential risks and benefits.
  • Supervision of medicines: The EMA also monitors the safety of medicines once they are on the market. This includes collecting and analyzing reports of side effects, investigating potential safety concerns, and taking appropriate action if necessary.
  • Providing information: The EMA provides information about medicines to healthcare professionals, patients, and the public. This includes information on the benefits and risks of medicines, how to use them safely, and how to report side effects.

    • By carrying out these functions, the EMA helps to ensure that the medicines available in the EU are safe, effective, and of high quality. This is essential for protecting public health and improving patient outcomes.

    Here are some additional details about the EMA:

Please check with European Medicines Agency’s (EMA) website for details and accuracy.