EU regulator recommends revoking approval for Novartis’ sickle cell disease drug

The EMA’s human medicines committee concluded that the benefits of Adakveo did not outweigh its risks

The European Medicines Agency’s (EMA) human medicines committee has recommended revoking the conditional marketing authorisation for Novartis’ sickle cell disease (SCD) drug Adakveo (crizanlizumab).

The recommendation follows a review by the Committee for Medicinal Products for Human Use (CHMP) that concluded the benefits of the drug, which is indicated for preventing painful crises in SCD patients aged 16 years and older, did not outweigh its risks.

The CHMP’s review looked at results from the phase 3 STAND study, which compared the safety and efficacy of Adakveo with placebo.

While the study did not raise new safety concerns, it showed a higher rate of severe and serious treatment-related side effects for Adakveo compared with placebo, the regulator said.

The study also showed that Adakveo did not reduce the number of painful crises leading to a healthcare visit, with patients in the Adakveo group experiencing an average of 2.5 over the first year of treatment, compared with 2.3 in the placebo cohort.

Additionally, the average number of crises requiring a healthcare visit or treatment at home was 4.7 with Adakveo compared with 3.9 for those receiving placebo.

When the EMA granted the conditional marketing authorisation of Adakveo in October 2020, supporting data showed that the drug was effective at reducing the number of painful crises in patients with SCD.

However, due to the data being limited and some uncertainty about the size of Adakveo’s effect, the EMA requested the STAND study as a condition for the authorisation.

“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 in a statement.

Affecting approximately 100,000 people in the US, SCD is a life-long, incurable genetic disease causing red blood cells to take a distinct crescent shape, which can block blood vessels and affect the way oxygen is carried around the body.

The disease can cause serious health problems, including anaemia, fatigue, episodes of pain and chronic end-organ damage.