29 July 2019 - The EMA's CHMP recently announced its decision to remove Novartis’ spinal muscular atrophy gene therapy onasemnogene abeparvovec from its accelerated assessment program.

The CHMP did not announce its reasoning behind the decision, which effectively means that the treatment, approved in the US as Zolgensma and launched at a price of more than $2 million, will be reviewed in the EU in 210 days rather than the accelerated 150 days. Novartis confirmed that its gene therapy is now under standard approval.

A Novartis spokesman explained to Focus: “This will give the agency the time they need to review the robust amount of data we are providing to answer their questions." An AveXis (Novartis acquired AveXis) spokesperson also told Focus they are working closely with European regulators and anticipate "a potential approval in Q4 2019.”

Read Regulatory Affairs Professional Society article