- The European Medicines Agency’s (EMA) newly-released “Human Medicines: Highlights 2020” report shows that in 2020, the European Union (EU) drug regulator issued 97 positive opinions for new medicines, a substantial increase of 31 on the previous year, despite the pandemic
- Of the 91 approvals, 39 contained a new active substance that had never been authorized in the EU until now, and 12 were biosimilar versions of already-approved drugs
- In a year unlike any other, unpredictable conditional marketing authorizations were also issued for Gilead’s Veklury (remdesivir) for COVID-19 treatment in June, as well as for Comirnaty later in the year, the COVID-19 vaccine co-developed by Pfizer and BioNTech
AMSTERDAM, The Netherlands – The European Medicines Agency’s (EMA) newly-released “Human Medicines: Highlights 2020” report shows that in 2020, the European Union (EU) drug regulator issued 97 positive opinions for new medicines, a substantial increase of 31 on the previous year, despite the pandemic.
Of the 91 approvals, 39 contained a new active substance that had never been authorized in the EU until now, and 12 were biosimilar versions of already-approved drugs.
2020 was a big year for cell and gene therapies, and the EMA recommended three – Novartis’ Zolgensma (onasemnogene abeparvovec), Kite’s Tecartus (autologous anti-CD19 transduced CD3+ cells) and Orchard’s lentiviral vector-based Libmeldy (autologous CD34+ cells encoding the ARSA gene) – for approval.
Referred to in the report as advanced therapy medicinal products (ATMPs), the three pivotal recommendations surpass the one recommendation that was made for a gene therapy the previous year – as bluebird bio’s Zynteglo (betibeglogene autotemcel) was the only drug of its kind to make the cut in 2019’s report.
In a year unlike any other, unpredictable conditional marketing authorizations were issued for Gilead’s Veklury (remdesivir) for COVID-19 treatment in June, as well as for Comirnaty later in the year, the COVID-19 vaccine co-developed by Pfizer and BioNTech.
Both conditional marketing authorizations included obligations for the companies to fulfill; Pfizer was asked to continue to provide results from the ongoing clinical trial as well as carrying out studies to provide additional assurance on the pharmaceutical quality of the vaccine as the manufacturing continues to be scaled up.
Gilead already satisfied its promise and provided the EMA with more data on the quality, effectiveness and safety of the medicine and the final reports of studies with Veklury before the end of 2020
Since the cut-off point of the report, the EMA has also recommended granting conditional marketing authorization for COVID-19 Vaccine Moderna for the prevention of Coronavirus disease (COVID-19) in people aged 18 years and up.
The report also notes that over the course of 2020, eight meds were recommended for approval under the EMA’s Priority Medicines Scheme (PRIME):
- Blenrep (belantamab mafodotin)
- Rozlytrek (entrectinib)
- Givlaari (givosiran)
- Hepcludex (bulevertide)
- Idefirix (imlifidase)
- Oxlumo (lumasiran)
Among the 97 medicines recommended for marketing authorization in 2020, 22 had their orphan designation confirmed by the end of the year, ready to be reviewed by the Committee for Orphan Medicinal Products (COMP).
Designations of note included Elzonris (tagraxofusp), Libmeldy, Hepcludex and Oxlumo, as they all provide a new treatment option for an indication that doesn’t have any approved products so far.
The Orphan Drug Legislation is currently under re-evaluation after the European Commission released a document analyzing the benefits and outputs of the scheme.
The Commission’s release, which notes both the positive outcomes and the shortcomings of the legislation, concludes that for the most part, the orphan regulation has had a positive impact by adding 210,000-440,000 quality-adjusted life years for patients in the EU, despite increasing costs by €23 billion from 2000-2017.
On the release of the report, the Regulatory Affairs Professionals Society (RAPS) reminds that 142 orphan medicines have been authorized in the EU since 2000, 131 of which are still on the market.
Before the release of the document, critics had already suggested that re-opening the Legislation may prove to be risky for both the pharma industry and patients, with the European Federation of Pharmaceutical Industries and Associations (EFPIA) previously stating that “we need to further incentivize the research and development of new treatments for patients where, currently, no treatment options exist.”
The EMA’s Committee for Medical Products for Human Use (CHMP) did, however, issue two negative opinions over the course of 2020.
Sobi’s Gamifant (emapalumab) as a treatment for primary haemophagocytic lymphohistiocytosis (HLH) was given a final rejection after the Committee initially turned away the drug in July, due to an “unconvincing” study.
It also voted against Daiichi Sankyo’s Turalio (pexidartinib), which was to be used to treat tenosynovial giant cell tumors, citing concern for small improvement in clinical outcomes and uncertainty about the duration of effect, as well as potentially life-threatening adverse effects.
The Future of Access
In December the EMA also published a joint strategy on medicine availability and access with the Heads of Medicines Agencies (HMA) for the next five years, in which the groups highlight six priority areas, including specific goals that will be translated into “concrete actions” over the next five years.
According to Emer Cooke, EMA’s Executive Director, “The COVID-19 pandemic has highlighted the pivotal role of medicine regulation for the protection of public health. Lack of availability of medicines, either because they are not marketed or due to supply disruptions, has shown to pose serious threats to patient and animal health, animal disease control programs and sustainable livestock production. This strategy ensures that we join forces across the EU to effect tangible improvements for citizens.”
Their strategy discusses how these regulators will enable the supply of safe and effective medicines that meet patients’ needs considering the “ever-accelerating developments” in science, medicine, digital technologies, globalization, and emerging health threats like the COVID-19 pandemic.
The network will review its strategy after 18 months to gauge its feasibility and efficacy and will make adjustments if necessary.
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