Novartis announced late Friday that the Food and Drug Administration has approved Adakveo — formerly called SEG101 — to reduce the frequency of vaso-occlusive crises (VOCs), or pain crises, in adult and pediatric patients aged 16 years and older with sickle cell disease.
The drug (crizanlizumab) happens to be the first FDA-blessed drug in sickle cell disease that binds to the protein P-selectin that works in cell adhesion – the process by which cells interact with specific molecules on the cell surface and attach to adjacent cells – and plays a key role in multicellular interactions that can lead to pain crises.
The Swiss company announced that the drug would be available in the coming weeks.
The FDA’s decision to approve Adakveo is based on the results of the year-long randomized, placebo-controlled SUSTAIN trial, which showed that Adakveo significantly lowered the median annual rate of VOCs to 1.63 vs 2.98 compared to placebo (P=.010). That equals a 45% reduction.
Perhaps more notably, these reductions in the frequency of VOCs were seen among patients regardless of sickle cell disease genotype and/or hydroxyurea use. Hydroxyurea is used to treat chronic myeloid leukemia, ovarian cancer, and certain types of skin cancer.
A Deep-dive Into Specialty Pharma
A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.
“We know this drug can decrease the frequency of sickle cell pain crises in a significant and clinically meaningful way,” said Dr. Kenneth Ataga, director, Center for Sickle Cell Disease, University of Tennessee Health Science Center at Memphis, and principal investigator of the SUSTAIN trial, in a news release. “The approval of crizanlizumab is an important advancement for people living with this very difficult condition.”
Novartis also noted other results from the randomized trial:
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A decrease in the median annual rate of days hospitalized to 4 vs 6.87 days when compared with placebo (a 42% reduction)
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Thirty-six percent of patients treated with Adakveo did not experience a VOC, compared to 17% of placebo-treated patients
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The median time to first VOC was 4.1 for Adakveo vs 1.4 months for placebo
The most common negative reaction that patients experienced were nausea (18%), arthralgia (18%), back pain (15%) and pyrexia (fever) (11%).
The approval could especially be a boon for patients who suffer pain crises because the condition is often undertreated out of fear that it might lead patients to be addicted to narcotics. Such pain crises are one of the top reasons for sickle cell disease patients ending up in the emergency room. The genetic disease overwhelmingly affects people of African descent in the U.S. with the total number of patients numbering around 100,000 according to an estimate from the American Society of Hematology.
“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, president, Novartis Oncology, in a news 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.”
Photo: Waldemarus, Getty Images
