Novartis’ Zolgensma Joins Growing List of Medicines to Lose Accelerated Assessment Status in EU

Regulatory NewsRegulatory News | 29 July 2019 |  By 

The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) recently announced its decision to remove Novartis’ spinal muscular atrophy gene therapy onasemnogene abeparvovec from its accelerated assessment program.

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.”

And although it’s rare for CHMP to make such a decision (only six other medicines have lost their accelerated assessment status after it was granted in recent years), many of these reversals have come in the last two months.

This month, five medicines, including four cancer medicines, were taken off the accelerated assessment track.
In addition to Zolgensma, those included Karyopharm’s multiple myeloma treatment selinexor, which was controversially approved in the US following a negative advisory committee decision, Stemline Therapeutics’ rare blood disease treatment tagraxofusp, which was approved in the US as Elzonris last December and Daiichi Sankyo’s acute myeloid leukemia drug quizartinib, which was rejected by FDA in June.

Shionogi’s antibacterial cefiderocol is also no longer being reviewed by CHMP under the accelerated approval program, the committee said on 9 July.

But a decision by CHMP to remove a treatment from an accelerated assessment is not always a negative sign. For instance, Bayer’s Vitrakvi (larotrectinib), which was taken out of the accelerated assessment program in June, then won a recommendation for conditional approval in July.

The other two treatments to lose their accelerated reviews were TaiMed Biologics’ HIV treatment ibalizumab, which was approved by FDA in March 2018 as Trogarzo, and lost its accelerated assessment status in the EU in June, and AMMTeK’s neonatal diabetes drug Amglidia (glibenclamide), which was switched at day 90 at the applicant's request to a standard review and was later recommended by CHMP for a marketing authorization in February 2018.

As far as why CHMP might decide that it is no longer appropriate to conduct an accelerated assessment, an EMA guideline from 2016 points to several scenarios.

“Examples of such situations are when major objections have been identified that cannot be handled in an accelerated timetable, when a longer clock-stop is requested by the applicant (e.g. to prepare for the oral explanation), or when the need for GMP or GCP inspection becomes apparent during the assessment,” the guideline says. “Similarly, in case of a negative trend following the oral explanation, the CHMP may decide to continue the assessment under standard assessment timelines. The new timetable will be communicated to the applicant and the reasons for the change to the standard timetable will be summarised in the CHMP assessment report.”

An EMA official's presentation in 2017 also shows more than a dozen other medicines from 2013 to 2017 that obtained accelerated assessments but later reverted to standard timelines.

Accelerated assessment

Editor's note: Updated on 7/29 with comment from AveXis and a link to the 2017 EMA presentation.


© 2022 Regulatory Affairs Professionals Society.

Discover more of what matters to you

No taxonomy