EU regulator recommends revoking authorisation of Novartis' sickle-cell drug Adakveo
26 May 2023
Clinical ResultPhase 3Drug ApprovalAccelerated ApprovalAcquisition
The European Medicines Agency (EMA) on Friday said it is recommending that the marketing authorisation for Novartis' sickle-cell disease (SCD) drug Adakveo (crizanlizumab) be revoked. The stance follows a review by the EMA's Committee for Medicinal Products for Human Use (CHMP) that concluded that the benefits of the anti-P-selectin antibody did not outweigh its risks.
The CHMP started a review of Adakveo earlier this year following preliminary results from a late-stage study that raised questions over the medicine's effectiveness. The drug was conditionally approved in Europe in 2020 to prevent recurrent vaso-occlusive crises (VOCs) in SCD patients aged 16 years and older based on Phase II data from the SUSTAIN trial showing it significantly lowered the median annual rate of VOCs by 45% compared to placebo. US regulators cleared the drug for the same indication in 2019.
However, Novartis reported earlier this year that neither of the two doses tested in the confirmatory Phase III STAND trial significantly outperformed placebo at reducing pain crises. At the time, Novartis indicated that it was working with regulators to determine "appropriate next steps" for the drug.
The STAND study showed that patients given Adakveo experienced an average 2.5 VOCs with a subsequent healthcare visit over the first year of treatment, versus 2.3 crises for placebo, the EMA said. The agency also noted that the average number of crises requiring a healthcare visit or treatment at home was 4.7 with Adakveo compared with 3.9 with placebo.
Higher rate of serious side effects
The CHMP review also took into account data from other studies, a managed access programme and real-world data, but these had "several limitations…and could not be used to show the effect of Adakveo or counterbalance the negative results of the STAND study," the EMA said. Further, while the trial did not raise new safety concerns, the agency pointed to "a higher rate of severe and serious treatment-related side effects" with Adakveo compared to placebo.
"As the STAND study results do not confirm the benefits previously seen with Adakveo, the CHMP has now concluded that the benefits do not outweigh the risks and recommended the revocation of its authorisation in the EU," the EMA said. Novartis acquired the drug through its $665-million purchase of Selexys Pharmaceuticals in 2016.
Speaking to FirstWord shortly after the STAND trial results were announced, David Rees – paediatric haematologist at King's College London and King's College Hospital – said "I don't think that most people are very surprised that it hasn’t come out strongly positive. It seems like a marginally effective drug that maybe helps some people, and perhaps doesn’t help others." With regards removing it from the market, Rees commented "it is not a good thing…but I don't think we should have ineffective drugs on the market just because there aren't any others available."
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