EMA Approved Fewer New and Orphan Drugs in 2019

The EMA authorized fewer new active substances and orphan drugs in 2019 than in the previous year, according to the agency.

The agency gave its blessing for marketing authorization for 30 new active substances in 2019, 12 fewer than for 2018. It also recommended only seven orphan drugs, a sharp decrease from the 21 orphan medications that received a thumbs up the year before.

The EMA chose to approve a number of drugs that were shot down by the FDA, including Sanofi and Lexicon’s diabetes treatment Zynquista (sotagliflozin) and Akcea and Ionis’ familial chylomicronaemia syndrome treatment Waylivra (volanesorsen), both of which received complete response letters from the FDA.

Bluebird’s Zynteglo, a $1.8 million gene therapy for a rare blood disease, was also approved by the EMA and awaits an FDA decision.