Week's news analysis

2020 European approvals in good health

By The Editor - 13 January 2021

Year-to-date tally at the European Medicines Agency (EMA) suggests the trend for new drug approvals in 2020 hasn't suffered from the Covid-19 pandemic. At the end of the 11 Committee for Medicinal Products for Human Use (CHMP) meetings, some 96 new medicines were approved in Europe throughout the year. The pace of approvals was stable between the first and the second half of the year, with 48 approvals. In most cases, the European Commission follows the CHMP's positive opinions by granting final approval to market a new medicine EU-wide.

Such a strong result means that new drug launches during 2021 are likely to happen, with potential blockbusters, along with exciting news on the Covid-19 front. January 12, EMA received an application for conditional marketing authorisation (CMA) for a Covid-19 vaccine developed by AstraZeneca and Oxford University. The assessment of the vaccine candidate will proceed under an accelerated timeline. An opinion on the marketing authorisation could be issued by January 29, during the meeting of EMA's CHMP, "provided that the data submitted on the quality, safety and efficacy of the vaccine are sufficiently robust and complete and that any additional information required to complete the assessment is promptly submitted", the organization stated yesterday.

Even if all eyes are trained on the more than 200 vaccines in development for the pandemic, Big Pharma have succeed in bringing to the market a drug treatment specifically for Covid-19 (Gilead's remdesivir). The European approval lists included several gene and cell therapies. In October, it approved Orchard Therapeutics' Libmeldy (cryopreserved autologous CD34+ cells encoding the arylsulfatase-A gene) to treat metachromatic leukodystrophy (MLD). MLD is a rare inherited metabolic disease that affects the nervous system and causes the progressive loss of motor function and cognitive ability and, ultimately, death. Still in October, it also granted Gilead's Tecartus (autologous anti-CD19-transduced CD3+ cells) for the treatment of adult patients with a rare cancer of white blood cells called mantle cell lymphoma. Tecartus addresses an unmet medical need.

In the fields of haematological cancers, EMA granted several treatments: Phelinun (melphalan), a hybrid medicine for the treatment of certain haematological and other cancers received a marketing authorization in September; Blenrep (belantamab mafodotin), a new antibody-drug conjugate for adult patients with relapsed and refractory multiple myeloma who no longer respond to treatment with an immunomodulatory agent, a proteasome inhibitor and a CD-38 monoclonal antibody in July; Calquence (acalabrutinib) for the treatment of chronic lymphocytic leukaemia in July too.

In the fields of rare diseases, the big event happened in March with a conditional marketing authorisation for Zolgensma (onasemnogene abeparvovec), for the treatment of babies and young children with spinal muscular atrophy. Later in October, Fintepla (fenfluramine) received a positive opinion for the treatment of seizures associated with Dravet syndrome. Earlier in 2020, in January, the Committee had also recommended granting a marketing authorisation for Givlaari (givosiran), the first treatment for acute hepatic porphyria in adults and adolescents aged 12 years and older. Acute hepatic porphyria is a rare genetic condition in which the liver cannot properly produce a substance called haem.

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