Sickle cell anemia. New crizanlizumab study shows no “statistical superiority in VOC reduction vs. placebo” Other review in progress

This was announced by Aifa with an “important note” to doctors recommending that they “evaluate the benefit and individual risks of each patient in making therapeutic decisions regarding the use of crizanlizumab”. The drug was approved for reimbursement in January 2022 after showing clinical benefits in a randomized phase II trial. THE AIFA NOTE.

15 FEB – AIFA yesterday issued an “important” information note with which Novartis, in agreement with the European Medicines Agency and the Italian Medicines Agency, informed Italian doctors of the preliminary results of the phase III study CSEG101A2301 ( STAND) on the drug Adakveo (crizanlizumab) indicated for the prevention of recurrent vaso-occlusive crises (VOC) in patients with sickle cell disease aged 16 years and older.

Preliminary results at the time “do not confirm the statistical superiority of crizanlizumab compared to placebo in the reduction of VOCs leading to a doctor’s visit in the first year following randomization”.

The preliminary results also “do not suggest any new safety issues with crizanlizumab. However, higher rates of ≥ Grade 3 treatment-related adverse events were reported with crizanlizumab compared to placebo.”

The company then informs that “a further evaluation of the data from study CSEG101A2301 and their potential impact on the benefit/risk balance of crizanlizumab is currently underway by the EMA” and that “the final conclusions and recommendations will be communicated as soon as the evaluation is completed”.

While this evaluation is ongoing, the note issued by AIFA continues, “doctors must evaluate the benefit and individual risks of each patient in making therapeutic decisions regarding the use of crizanlizumab”.

The drug Adakveo it had been approved for reimbursement by the NHS in January 2022 and may be given as add-on therapy to hydroxyurea/hydroxycarbamide (HU/HC) or as monotherapy in patients for whom treatment with HU/HC is inappropriate or inadequate. Adakveo is currently approved for use at a dose of 5.0 mg/kg.

Crizanlizumab has shown clinical benefit in a randomized phase II study (CSEG101A2201, SUSTAIN), which led to conditional marketing authorization from the European Medicines Agency.

The data from the confirmatory study CSEG101A2301 (STAND) were requested by the EMA as part of the conditions for the marketing authorisation.

The initial analysis of the STAND study was conducted on data from 252 participants enrolled from its inception in 2019 to the August 31, 2022 data cutoff.

February 15, 2023
© breaking latest news

Other articles in Science and Drugs