Amid pandemic, EMA's 2020 authorizations ticked upward

Regulatory NewsRegulatory News | 21 January 2021 |  By 

Despite the pandemic, the European Medicines Agency (EMA) issued many more positive recommendations for human medicines in 2020 than in the previous year, according to a new report from the agency highlighting the year’s regulatory activities.
In 2020, 97 new medicines were recommended for authorization by EMA; of these, 39 contain a new active substance (NAS). In 2019, EMA issued 66 positive opinions for medicines, 30 of which contained a NAS. The total numbers of new recommendations include conditional marketing authorizations such as the ones issued for the COVID-19 treatment Veklury (remdesivir) and Comirnaty, the COVID-19 vaccine co-developed by Pfizer and BioNTech.
The European regulator issued a negative opinion for Gamifant (emapalumab-Izsg), a drug approved by the US Food and Drug Administration (FDA in 2019 to treat patients with primary hemophagocytic lymphohistiocytosis. EMA also gave a thumbs down to Turalio (pexidartinib), which the FDA also approved in 2019 to treat a rare joint tumor.
Twenty-two medicines were recommended under EMA’s orphan designation, while eight were granted Priority Medicines (PRIME) designation. In 2019, just six new orphan medicines were recommended for authorization and three fell under the PRIME scheme. (RELATED: EMA recommends fewer new and orphan drugs in 2019, Regulatory Focus 09 January 2020)
Some medicines, such as the cancer therapy Blenrep (belantamab mafodotin-blmf), received both designations on their way to accelerated assessment and eventual conditional marketing authorization.
Most other conditional marketing authorization recommendations were granted to cancer therapies, though Zolgensma (onasemnogene abeparvovec-xioi), a one-time gene therapy to treat spinal muscular atrophy, the sickle cell disease therapy Adakveo (crizanlizumab-tmca), and two anti-infectives also received conditional marketing authorizations.
For each medicine, conditional authorization comes with specific post-authorization obligations on the part of the sponsor which are detailed in the report. For example, Pfizer and BioNTech must continue to update EMA about ongoing clinical trial results, to include providing efficacy data in specified subgroups; the firms must also carry out additional pharmaceutical quality studies as production scales up to meet global demand.
In 2020, five recommendations for authorization were issued “under exceptional circumstances.” The report explains that this route “allows patients’ access to medicines that cannot be approved under a standard authorization as comprehensive data cannot be obtained, either because there are only very few patients with the disease, or the collection of complete information on the efficacy and safety of the medicine would be unethical, or there are gaps in the scientific knowledge.”
An authorization under exceptional circumstances also carries post-authorization reporting and monitoring obligations. Two oncologic hematology therapies, an inhalational anthrax anti-infective, and two components of a vaccine against Zaire ebolavirus infection were the medicines authorized by this route. The Ebola virus vaccine also received accelerated assessment.
In all, 12 biosimilars were recommended for authorization and counted in EMA’s total. Though therapeutic biologics were included in the year-end tally of new drug approvals by the FDA’s Center for Drug Evaluation and Research (CDER), some medicines falling under EMA’s NAS column, such as vaccines, are not counted as new molecular entities (NMEs) by FDA.
The US regulator issued 53 novel drug approvals in 2020; of these, 31 received orphan designation, and 36 novel drugs (68%) used at least one of FDA’s expedited approval pathways.


© 2023 Regulatory Affairs Professionals Society.

Discover more of what matters to you