EMA committee recommends revoking approval for Novartis sickle cell drug

2023-05-30

上市批准临床3期加速审批临床结果

As promised, the EMA's Committee for Medicinal Products for Human Use (CHMP) has taken a closer look at Novartis' sickle cell disease drug, and is now recommending that the European regulator revokes the drug's marketing authorization.

Novartis broke the bad news earlier this year in January, sharing that preliminary results from the phase 3 STAND study indicated no statistically significant difference between either dose of crizanlizumab, branded Adakveo, and placebo in annualized rates of vaso-occlusive crises (pain crises) leading to a health care visit.

The drugmaker informed regulatory authorities, and the EMA's CHMP began its review.

Novartis has picked up the drug, an anti-P-selectin antibody, through a 2016 $665 million acquistion of Selexys Pharmaceuticals. Adakveo was first approved by the FDA in November 2019 for reduction in frequency of vaso-occlusive crises, making it the first of several proposed new therapies designed to offer relief for patients with the debilitating blood disease to get U.S. regulatory clearance. In October 2020, the EMA followed suit, granting conditional Marketing Authorization for the drug, but requesting a follow-up study.

The initial approvals were based on results from SUSTAIN trial, during which Adakveo cut sickle cell pain crises nearly by half — from 2.98 incidents annually in those getting a placebo to 1.63 incidents annually.

Now, after looking at the results from the STAND study, CHMP says the med’s benefits did not outweigh its risks.

Novartis is working with regulators globally, including the U.S. FDA, for a path forward for the drug that it says represents the first FDA-approved medicine in sickle cell disease that binds to P-selectin — a cell adhesion protein that plays a central role in the multicellular interactions that can lead to vaso-occlusion.

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