In 2023 the European Medicines Agency’s human drugs committee (CHMP) recommended the pan-EU approval of 77 new medicinal products, including 39 that contained a new active substance.
Among the NAS-containing products given a CHMP positive opinion was Vertex Pharmaceuticals/CRISPR Therapeutics’ Casgevy (exagamglogene autotemcel) for the treatment of transfusion-dependent beta-thalassemia and severe sickle cell disease. This is the first advanced therapy medicinal product (ATMP) using the ground-breaking CRISPR/Cas9 gene-editing technology. (Also see "First Gene Editing Therapy Among Seven Drugs On Track For EU-Wide Approval" - Pink Sheet, 15 December, 2023.)
Three vaccines containing an NAS were recommended, two of them to protect against lower respiratory tract disease caused by respiratory syncytial virus (RSV): Pfizer’s Abrysvo and GSK’s Arexvy.
The other was HIPRA’s COVID-19 vaccine Bimervax, which was recommended as a booster in people aged 16 years and older who have previously been vaccinated with an mRNA COVID-19 vaccine. Bimervax was evaluated as part of the OPEN initiative, which started in December 2020 to increase international collaboration on the EU’s review of COVID-19 products.
The data come from the EMA’s highlights of 2023, which show that positive opinions were given to 17 orphan drugs, one ATMP, eight biosimilar products, and 14 generics. However, the orphan status of one product – Tevimbra – was removed after authorization at the request of the marketing authorization holder.
Three of the products OKd by the CHMP went through the EMA’s PRIME (priority medicines) scheme, which is designed to expedite the development and regulatory review of drugs for unmet medical needs. They were Casgevy and two drugs for relapsed and refractory multiple myeloma: Pfizer’s Elrexfio (elranatamab) and Janssen-Cilag’s Talvey (talquetamab).
Three products underwent an accelerated assessment (which is also a feature of the PRIME scheme): Talvey and the two RSV vaccines, Abrysvo and Arexvy.
Eight treatments were recommended for a conditional marketing authorization (with additional data to be provided by the company). One product – Inmedica Pharma’s hyperargininema drug Loargys (pegzilarginase) – was OKd under exceptional circumstances, where comprehensive safety and efficacy data may never be provided, for example because of the rarity of the condition, a lack of scientific knowledge or ethical considerations. (Also see "EMA Backs Approval Of Santhera’s Agamree, Pfizer’s Elrexfio & Several Other New Drugs" - Pink Sheet, 13 October, 2023.)
In addition, a total of 77 extensions of indication for already approved drugs were recommended for approval in 2023, including 38 for pediatric use.
Negative Opinions
All was not rosy on the approvals front though, with the EMA noting that the CHMP issued three negative opinions during the year: MSD’s Lagevrio (molnupiravir), for the treatment of COVID-19 in adults, Amylyx’s Albrioza (sodium phenylbutyrate/ursodoxicoltaurine) for amyotrophic lateral sclerosis, and Ipsen’s Sohonos (palovarotene) for fibrodysplasia ossificans progressiva, a rare genetic disease.
A total of 19 marketing authorization applications were also withdrawn during the year, the EMA noted.
Non-EU Products
The CHMP adopted two positive opinions for medicines for use in countries outside the EU, under the regulatory procedure known as “EU-Medicines for all” (EU-M4All), which is intended to make it easier to approve medicines for use in developing countries.
One of the products is Merck Europe’s Arpraziquantel, a new treatment for the estimated 50 million young children with schistosomiasis, a neglected tropical disease caused by parasitic blood flukes (trematode worms) that can cause damage to organs such as the bladder, the kidneys and the liver.
The other is Fexinidazole Winthrop (fexinidazole) for human African trypanosomiasis (sleeping sickness). In 2018, the medicine received a positive opinion for sleeping sickness caused by the parasite trypanosoma brucei gambiense – the CHMP’s opinion extends its use to include treatment of the more acute and lethal form of the disease caused by trypanosoma rhodesiense. Both parasites are transmitted by the tsetse fly.
An analysis by the Pink Sheet of marketing authorizations issued by the European Commission in 2023 is expected to be published on the website in the coming week.
