EMA Carries 2014 Momentum, Recommends 39 New Drugs and Sets Orphan Record

Regulatory NewsRegulatory News | 12 January 2016 |  By 

In 2015, the European Medicines Agency (EMA) maintained its momentum from 2014, granting 93 positive opinions, 39 of which were for new active substances. The agency also recommended 18 orphan drugs for approval, breaking last year's record of 17.

Notably, EMA trailed just behind the US Food and Drug Administration (FDA)'s Center for Drug Evaluation and Research (CDER) for the year. In 2015, CDER approved 45 new drugs, 21 of which had been granted orphan drug designation by the agency.

While the EU is still slightly behind the US in terms of orphan drug approvals, it has closed the gap significantly in the 16 years since it established incentives for orphan drugs under its orphan regulation, known as Regulation (EC) No 141/2000.

EMA and FDA (CDER) 2015 Activities
New Drugs (NAS/NCE)3945
Orphan Designation18 (46%)21 (46%)
Expedited Review5 (13%)27 (60%)

One major difference between the two regulators is the number of drugs that benefited from an expedited review. In 2015, EMA recommended five drugs for approval under its accelerated assessment procedure, which reduces the assessment period for a drug from 210 days to 150. However, in 2015, FDA approved 27 drugs using one or more of the agency's four types of expedited review: fast track, breakthrough, priority review and accelerated approval.

In 2015, EMA recommended three drugs for conditional marketing authorization, Blincyto, Tagrisso and Zykadia. In the EU, a conditional marketing authorization is granted to drugs that fill an unmet need and treat rare or life-threatening diseases using less clinical data than would typically be required, under the condition that the sponsor will complete certain post-authorization study requirements.

EMA also recommended authorization for three drugs, Obizur, Raxone and Strensiq "under exceptional circumstances." This type of authorization allows patients to access drugs for conditions that are too rare to study in a traditional manner, or would be unethical to study. Similar to conditional marketing authorization, drugs authorized under exceptional circumstances generally must follow "specific post-authorization obligations."

EMA Human Medicines Highlights 2015


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