FDA Approves Novartis Drug for Sickle Cell Disease Pain Treatment

Novartis announced on Nov.15, 2019 that FDA approved Adakveo (crizanlizumab-tmca), previously known as SEG101, to reduce the frequency of vaso-occlusive crises (VOCs) in adults and pediatric patients aged 16 years and older with sickle cell disease, a genetic blood disorder.

Adakveo is the first FDA-approved medicine for sickle cell disease that binds to P-selectin, a cell adhesion protein that is crucial for the multicellular interactions that can lead to vaso-occlusion, according to Novartis.

According to a company press release, the approval was based on a 52-week, randomized, placebo-controlled trial, which concluded that the drug reduced the median annual rate of VOCs to 1.63 vs 2.98 compared to placebo (P=.010), a 45% reduction. The reductions were indifferent to sickle cell disease genotype and hydroxyurea use.

“We know this drug can decrease the frequency of sickle cell pain crises in a significant and clinically meaningful way,” said Kenneth Ataga, MD, director, Center for Sickle Cell Disease, University of Tennessee Health Science Center at Memphis, and principal investigator for the trial, in the press release. “The approval of crizanlizumab is an important advancement for people living with this very difficult condition.”

Other results from the trial included:

• A 42% reduction in the median annual rate of days hospitalized to 4 vs 6.87 days when compared with placebo.

• Of patients treated with the drug, 36% did not experience a VOC, compared to 17% of placebo-treated patients.

• The median time to first VOC was 4.1 for Adakveo vs 1.4 months for placebo

“The approval of Adakveo marks a new era in the treatment of sickle cell disease, a genetic condition that places an extraordinary burden of unpredictable pain crises on patients and their families,” said Susanne Schaffert, PhD, president, Novartis Oncology, in the press release. “The stories we have heard from patients about their sickle cell pain crises are devastating. We are pleased to help reimagine medicine together with the sickle cell community and offer new hope for fewer VOCs.”

“Patients with sickle cell disease often face unique challenges, and have long suffered silently through unimaginable pain crises,” added Beverley Francis-Gibson, president and CEO of the Sickle Cell Disease Association of America, in the press release. “We are excited to have a new medicine that may help many of the thousands of people living with sickle cell disease by reducing the frequency of these potentially dangerous and painful episodes.”

Source: Novartis