CHMP recommends revoking Adakveo’s EU conditional approval
Final decision on the therapy in Europe expected in about 2 months
According to the therapy’s developer Novartis, a final decision from the European Commission is expected in about two months. In the meantime, no new patients in the European Union will be started on Adakveo, and those who are currently receiving the therapy are advised to contact their healthcare team to discuss alternative treatment options.
Adakveo has been conditionally approved in Europe since 2020 as a treatment to reduce the risk of vasco-occlusive crises (VOCs) in people with sickle cell, ages 16 and older. The therapy had received a similar approval in the U.S. in 2019. VOCs are painful episodes caused by blood vessel blockage.
Adakveo’s original approvals were based on data from a Phase 2 trial called SUSTAIN (NCT01895361), which suggested the therapy could reduce the risk of VOCs. However, available data at the time were limited, and there were questions about the efficacy of the therapy. As part of the conditional approval in Europe, the European Medicines Agency (EMA) requested Novartis conduct additional testing.
The company sponsored a Phase 3 clinical trial called STAND (NCT03814746), which is testing Adakveo against a placebo in more than 250 people with SCD.
Preliminary data from the trial, announced earlier this year, showed the therapy failed to lower the rate of VOCs compared with a placebo. No new safety issues related to Adakveo have been identified in STAND.
The CHMP, which is part of the EMA, conducted a review of the available data from these clinical trials, as well as other studies and real-world data on Adakveo. Considering the findings, the CHMP concluded the potential benefits of Adakveo do not outweigh its risks and has recommended the therapy’s conditional approval be revoked.
“While we are disappointed by the recommendation to revoke the conditional MA [marketing authorization] for [Adakveo] in the European Union, we acknowledge that the STAND study did not meet one of the specific obligations of the conditional MA,” Novartis stated in a press release.
The company did not specifically state whether or how results of the STAND trial will impact Adakveo’s approval in the U.S., noting that it will “continue to coordinate with health authorities globally.”