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Regulatory Focus™ > News Articles > 2020 > 1 > EMA Recommends Fewer New and Orphan Drugs in 2019

EMA Recommends Fewer New and Orphan Drugs in 2019

Posted 09 January 2020 | By Michael Mezher 

EMA Recommends Fewer New and Orphan Drugs in 2019

The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) recommended 30 new active substances (NAS) for marketing authorization in 2019, down from 42 the previous year.
The agency also recommended just six new orphan medicines for authorization, the fewest in recent years and a drop from the 17 new orphan medicines recommended in 2018.
In comparison, the US Food and Drug Administration’s (FDA) Center for Drug Evaluation and Research (CDER) approved 48 novel drugs in 2019, down from 59 in 2018, 21 of which were orphan products.
While most of the products EMA recommended in 2019 have also been approved by FDA, four have not been approved in the US.
Those four products are Lexicon’s type 1 diabetes drug Zynquista (sotagliflozin), which received a complete response letter from FDA in March 2019; Akcea and Ionis’ familial chylomicronemia syndrome drug Waylivra (volanesorsen), which FDA rejected in August 2018; Bluebird Bio’s gene therapy Zynteglo (autologous CD34+ cells encoding βA-T87Q-globin gene), which is expected to be submitted to FDA for review this year; and Recordati’s Cushing syndrome treatment Isturisa (osilodrostat), which is pending FDA review.
EMA’s count also includes some products that were approved by FDA’s Center for Biologics Evaluation and Research (CBER) in the US, including Merck’s Ebola virus vaccine Ervebo and Novo Nordisk’s Hemophilia A treatment Esperoct (turoctocog alfa pegol).
In 2019, EMA recommended eight medicines for conditional marketing authorization, tying with 2016 for the most recommendations for conditional marketing authorization in a single year since the pathway was created.
Three of the recommended medicines, Zynteglo, Ervebo and Roche’s B-cell lymphoma drug Polivy (polatuzumab vedotin) were granted PRIority MEdicines (PRIME) designation, with both Zynteglo and Ervebo also receiving an accelerated assessment. The only other drug CHMP recommended following an accelerated assessment in 2019 was Astellas’ acute myeloid leukaemia drug Xospata (gilteritinib).
Additionally, EMA recommended five biosimilars for marketing authorization in 2019, compared to 10 biosimilars approved by FDA.


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