EU Accelerated Assessment Tracker

Pharming’s leniolisib has lost its accelerated assessment status at the European Medicines Agency while Krystal Biotech’s beremagene geperpavec will be fast-tracked once a filing has been made.

This tracker provides information on accelerated assessment requests for planned marketing authorization applications (MAAs) that have come before the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) for a decision. It also tracks whether companies retain fast-track status for MAAs as they make their way through the EU centralized drug evaluation procedure.

This latest update (see table at the end of this article) reflects recent developments.

Specifically it records the fact that the MAA for leniolisib, Pharming's potential treatment for adolescents and adults with activated phosphoinositide 3-kinase delta syndrome (APDS), has lost its accelerated assessment status and has reverted to standard review timelines.

It also shows that AstraZeneca has asked the EMA to fast-track its planned MAA for danicopan, for treating paroxysmal nocturnal hemoglobinuria. This request was considered during the CHMP’s latest monthly meeting, which took place in January. The outcome of the request has not yet been made public. A spokesperson for Alexion, AstraZeneca Rare Disease told the Pink Sheet: "AstraZeneca regularly updates on the status of regulatory submissions or acceptances during quarterly results. We will present data from the ALPHA Phase III trial at a forthcoming medical meeting and intend to proceed with regulatory submissions in the coming months."

Other more recent updates to the tracker show that the agency granted accelerated assessment for Krystal Biotech’s planned MAA for beremagene geperpavec and Janssen’s MAA for talquetamab, but rejected the fast-track status for the MAAs for Pfizer’s elranatamab, Novo Nordisk’s concizumab, and Biogen/Eisai’s lecanemab.

Leniolisib

Pharming’s MAA for leniolisib reverted to the standard review timelines in January. The review of the MAA began on 27 October 2022.

In the US, the Food and Drug Administration is conducting a priority review of a new drug application for leniolisib as a treatment for adolescents and adults with APDS, and has assigned a Prescription Drug User Fee Act goal date of 29 March.

The Process

The CHMP decides whether an MAA will be evaluated under accelerated or standard review timelines before the MAA is filed. When an accelerated assessment request reaches the stage of being listed for a decision on the agenda of a CHMP monthly meeting, it is usually a sign that an MAA filing will be made in the following few months.

The accelerated assessment mechanism is reserved for drugs of potential major public health interest, particularly from the point of view of therapeutic innovation. It can cut the time it takes the EMA to evaluate an MAA under the EU centralized procedure from up to 210 days to up to 150 days (not counting clock stops when applicants have to provide additional information). In practice, it can cut months off the approximately 12 months it generally takes to review an application at the EMA under standard EU centralized drug evaluation procedure timelines. 

The outcomes of accelerated assessment requests are published in the minutes of the CHMP meeting at which the requests are decided on. It takes at least six weeks for CHMP meeting minutes to be published. Some companies choose to disclose the outcome in the interim, but most do not. Companies sometimes submit their MAA before the minutes are issued, at which point it usually becomes clear whether or not they have been granted accelerated assessment.