Phase III Trial Fails to Confirm Efficacy of Novartis’ SCD Therapeutic

Harold Cunningham/Getty Images

Harold Cunningham/Getty Images

Novartis’ SCD therapeutic Adakveo is under review by the EMA after failing to replicate previous efficacy results in a Phase III trial.

Courtesy of Harold Cunningham/Getty Images

Novartis’ sickle cell disease therapeutic Adakveo (crizanlizumab) is under review by the European Medicines Agency’s Committee for Medicinal Products for Human Use after failing to replicate previous efficacy results in a Phase III trial.

Adakveo was evaluated for its efficacy in reducing rates of vaso-occlusive crises (VOCs) leading to healthcare visits in patients with SCD.

Preliminary results indicated there was no statistically significant difference between placebo and two different dosages of Adakveo in reducing annualized rates of VOCs leading to a healthcare visit over the first year after randomization.

Adakveo was granted conditional marketing authorization by the EMA in October 2020 for the prevention of recurrent VOCs in SCD patients above the age of 16. Data from the Phase III STAND trial was requested as a condition of the regulatory nod.

The EMA will review the trial results to determine the effect of continued use of the therapeutic.

Results of the trial do not indicate new safety concerns with Adakveo, and the overall safety profile remains consistent with the commercially available dose, Novartis stated in a press release Friday.

Adakveo, an anti-P-selectin antibody, was approved by the FDA in November 2019 to reduce the frequency of VOCs in SCD patients aged 16 and older

The approval was based on data from the SUSTAIN clinical trial which demonstrated that the therapeutic reduced the annual rate of VOCs by 45%. Novartis also reported that more than one-third of patients did not experience a VOC while taking Adakveo.

Additionally, patients treated with Adakveo were hospitalized for fewer days over the course of a year, showing a 42% reduction in comparison to patients treated with placebo.

Friday, Novartis stated it will work with regulators, including the FDA, to determine the appropriate next steps. Further assessment of the trial data is ongoing.