After trial flop, EU officials take a closer look at Novartis' Adakveo approval

Working to grow the reach of its sickle cell disease medicine Adakveo, Novartis has more than just Pfizer's Oxbryta to worry about.

After a phase 3 trial miss, the European Medicines Agency’s (EMA's) Committee for Medicinal Products for Human Use (CHMP) says it will be taking a closer look at its prior approval for Adakveo.

In October 2020, the European agency approved the drug to prevent pain crises in sickle cell disease patients ages 16 and older. But, in preliminary results from the STAND trial, the drug failed to outperform placebo in reducing the rates of pain crises leading to a healthcare visit after one year, Novartis said Friday.

After the result, Novartis said it's "working with regulators globally" to determine the "appropriate next steps."

Novartis noted that these results do not suggest any new safety concerns for the drug. The results are "inconsistent" with results from a prior study, called SUSTAIN, which showed a benefit for the medicine compared with placebo, the company said.

Administered once a month, Novartis' drug is intended to reduce pain crises associated with sickle cell disease by inhibiting the P-selectin protein. Novartis got its hands on the medicine through its $665 million acquisition of Selexys Pharmaceuticals in 2016. The drug's 2019 FDA approval was supported by results showing it could lower the median annual rate of the crises by 45% compared with placebo.

Despite “high uncertainty” about Adakveo’s long-term effectiveness and cost-effectiveness, England's National Institute for Health and Care Excellence backed the therapy in 2021 through a special arrangement. 

Meanwhile, Novartis is facing off against Pfizer in sickle cell disease as the New York drugmaker recently bought Global Blood Therapeutics, the maker of Oxbryta. Oxbryta's specific label is different from Adakveo, though, as it's approved in Europe to treat hemolytic anemia in sickle cell disease patients 12 and older.