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Regulatory Focus™ > News Articles > EMA Sees Spike in Authorizations in 2017

EMA Sees Spike in Authorizations in 2017

Posted 23 January 2018 | By Michael Mezher 

EMA Sees Spike in Authorizations in 2017

Like the US Food and Drug Administration (FDA), the European Medicines Agency (EMA) saw an increase in the number of new drugs and biologics it recommended for marketing authorization in 2017.

Looking back at the prior year, EMA recommended 35 new active substances (NASs) for marketing authorization, up from 27 in 2016. By comparison, FDA approved 46 new molecular entities (NMEs) in 2017, a 21-year high for the agency.

Including non-NASs such as generic drugs and biosimilars, EMA gave 92 positive opinions and six negative opinions in 2017.

Of the NASs EMA recommended, 11 were new drugs and biologics to treat cancer, five to treat neurological disorders, four each for infectious diseases and immunology/rheumatology. Other therapeutic areas that saw new approvals in 2017 include endocrinology, hematology, nephrology, hepatology and dermatology.

"Many of these medicines represent a significant improvement in their therapeutic areas; they include medicines for children, for rare diseases and advanced therapies," EMA writes.

While the majority of the NASs EMA recommended for marketing authorization in 2017 have already been approved in the US, six of the products EMA recommended have not yet been approved by FDA.

NASs Recommended by EMA Not Approved by FDA
Product Sponsor
Fotivda (tivozanib) AVEO Pharma
Crysvita (burosumab) Ultragenyx/Kyowa Kirin Limited
Spherox CO.DON AG
Oxervate (cenegermin) Dompé Farmaceutici
Lokelma (sodium zirconium cyclosilicate) AstraZeneca
Alofisel (darvadstrocel) TiGenix

EMA also highlighted its use of its expedited programs and incentives to bring products to market more quickly and spur development of products to treat rare diseases or other areas of high unmet medical need.

Program or Designation NASs Total
Orphan Medicine 13 19
Accelerated Assessment 5 7
Conditional Marketing Authorization 2 3
Approval Under Exceptional Circumstances 1 2

For orphan medicines, EMA recommended roughly the same proportion as in 2016, at 37% for both years. The agency also completed a similar amount of accelerated assessments, with five for NASs in 2017 compared to four in 2016.

While EMA recommended fewer NASs for conditional marketing authorization, two in 2017 compared to eight in 2016, the agency recommended one NAS, BioMarin's Brineura (cerliponase alfa) to treat neuronal ceroid lipofuscinosis type 2, for approval under exceptional circumstances.

EMA also recommended two advanced therapies for marketing authorization, Tigenix's Alofisel (darvadstrocel) to treat perianal fistulas in patients with Crohn's disease and CO.DON AG's Spherox for repairing knee cartilage defects, the same number as in 2016.

In 2017, EMA said that good manufacturing practice (GMP) and good clinical practice (GCP) compliance issues resulted in several submissions being withdrawn, and that GCP issues resulted in two of the negative opinions the agency issued.

"Medicine development and manufacturing is global. It is important for regulators to ensure that EU standards are adhered to no matter where clinical trials or manufacturing takes place," EMA writes.



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