EMA Sees Spike in Medicines Recommendations in 2018

Regulatory NewsRegulatory News | 04 January 2019 |  By 

In 2018 the European Medicine Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) recommended more new drugs for approval than any year in the past decade with 42 positive opinions for medicines with new active substances (NASs).

"Many of these medicines represent a significant improvement in their therapeutic areas," EMA writes, noting that the new batch of products includes advanced therapies, medicines for children and a number of treatments for rare diseases.

Across the Atlantic, the US Food and Drug Administration (FDA) also set a record for new drugs, approving 59 novel drugs and biologics through its Center for Drug Evaluation and Research (CDER) in 2018.

Of the 42 NASs recommended by CHMP in 2018, 17 (40%) were orphan medicines. Of those, three were advanced therapy medicinal products (ATMPs); four received accelerated assessments; one was authorized conditionally; three were approved under exceptional circumstances; and two were reviewed under EMA's PRIME (PRIority MEdicines) scheme.

In total, CHMP recommended 21 medicines with orphan indications in 2018, four of which were for new indications for already authorized medicines.

The three ATMPs include the first two chimeric antigen receptor (CAR) T-cell therapies recommended in the EU, Novartis' Kymriah (tisagenlecleucel) and Gilead's Yescarta (axicabtagene ciloleucel), and Spark Therapeutics' Luxturna (voretigene neparvovec).

CHMP also issued five negative opinions for new medicines in 2018, three of which have been approved for use in the US. Those three medicines are Portola Pharmaceuticals' Dexxience (betrixaban), which is marketed as Bevyxxa in the US; Radius Health's Eladynos (abaloparatide), which is marketed as Tymlos in the US; and Sarepta Therapeutics' Exondys (eteplirsen), which is known as Exondys 51 in the US.

CHMP also initially issued a negative opinion for Puma Biotechnology's Nerlynx (neratinib), but reversed course and recommended the drug after the sponsor requested a re-examination of the committee's opinion.

EMA, Human Medicines Highlights 2018


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