Novartis' approved sickle cell disease drug fails to beat placebo in PhIII
Novartis’ sickle cell drug, approved in 2019 and branded as Adakveo, has failed an ongoing Phase III, according to preliminary results.
The Swiss pharma giant unveiled early data from the ongoing STAND Phase III study on Friday, saying that crizanlizumab showed no statistically significant difference between the drug at two different dose levels compared to placebo in annualized rates of vaso-occlusive crises that lead to a healthcare visit over the first year since being randomized into the trial.
Vaso-occlusive crises are highly painful events that occur when sickled red blood cells block blood flow to the point that tissues become deprived of oxygen. It can also cause organ damage and increase chances of dying.
Novartis said in a statement that the findings are incongruous with previous trial results, pointing to the SUSTAIN trial that showed crizanlizumab at 5.0mg/kg as superior to placebo.
Crizanlizumab was approved by the FDA in November 2019 to reduce the frequency of vaso-occlusive crises. The drug was picked up from its Selexys buyout for up to $665 million in 2016. Almost a year after the FDA approval, the EMA granted conditional marketing authorization to prevent recurrences of these events in patients aged 16 years and older with sickle cell disease.
A Novartis spokesperson told Endpoints News in an email:
Our priority now is to thoroughly review the full data set of the STAND study and we are working with regulators globally, including the EMA and FDA, and trial investigators to determine the appropriate next steps. Any decisions on the overall crizanlizumab development program will be made in coordination with trial investigators and regulatory authorities. Currently, all other ongoing studies are continuing as per protocol.
Sickle cell disease has been attracting more and more attention in recent years, but it’s had its pitfalls. GlycoMimetics, a former Pfizer partner, had a candidate that failed to meet both the primary and secondary endpoints in a Phase III trial in 2019, resulting in the alliance getting cut.
More recently in December, Editas Medicine reported some early-stage data in two patients suffering from sickle cell disease, noting that neither patient suffered vaso-occlusive events in initial months of followup after being treated with a cell therapy candidate.