The US Food and Drug Administration (FDA) has given Novartis’ investigational sickle cell medicine, crizanlizumab (SEG101), Priority Review.
The governing body accepted the company's Biologics License Application (BLA) which was submitted for the prevention of vaso-occlusive crises (VOCs) in patients with sickle cell disease (SCD) and was granted Breakthrough Therapy designation in December 2018.
The submission is supported by Phase II results from the SUSTAIN study, which showed that crizanlizumab (5 mg/kg) reduced the median annual rate of VOCs leading to health care visits by 45.3% compared with placebo.
Priority Review is granted to therapies that the FDA determines have the potential to provide significant improvements in the treatment, diagnosis, or prevention of serious conditions. The designation is intended to shorten the FDA review period to six months from the standard ten months.
"The FDA's decision to give crizanlizumab priority review reflects the impact that this medicine could have for the many thousands of US sickle cell adult patients who experience painful vaso-occlusive crises," said John Tsai, head of global drug development and chief medical officer, Novartis.
He continued, "We are looking forward to the opportunity, if crizanlizumab is approved, to reimagine medicine in sickle cell disease for patients who live with this condition every day of their lives."
Sickle cell disease is a debilitating inherited genetic blood disorder that affects the shape of the red blood cells and can make blood cells and blood vessels stickier than usual.
When blood cells stick to one another, they can form multicellular adhesion clusters in the bloodstream. These clusters can reduce and block the flow of blood and oxygen, which can cause damage to the blood vessels and lead to acute and chronic complications.