Novartis gets FDA nod for Adakveo

Novartis has received the Food and Drug Administration’s green light for Adakveo (crizanlizumab), a treatment to reduce the frequency of vaso-occlusive crisis – a common and painful complication of sickle cell disease that occurs when blood circulation is obstructed by sickled red blood cells – for patients age 16 years old and older.

“Hope has never been higher for people living with sickle cell disease and their families and supporters, with a pipeline of new treatments on the horizon, like the one being approved today, and several initiatives underway to better utilize current tools in the battle against the painful and deadly blood disorder,” said Acting FDA commissioner Adm. Brett Giroir. “The opportunity before us in the coming months and years is profound and historic.”

“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,” Susanne Schaffert, Novartis Oncology president said. “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.

Sickle cell disease is an inherited blood disorder in which the red blood cells are abnormally shaped (in a crescent or "sickle" shape), which restricts the flow in blood vessels and limits oxygen delivery to the body’s tissues, leading to severe pain and organ damage. It also is characterized by severe chronic inflammation that results in a vaso-occlusive crisis where patients experience episodes of extreme pain and organ damage. According to the Centers for Disease Control and Prevention, sickle cell disease affects approximately 100,000 Americans. The disease occurs most often in African-Americans, where 1 out of every 365 babies born have the disease.

“Adakveo is the first targeted therapy approved for sickle cell disease, specifically inhibiting selectin, a substance that contributes to cells sticking together and leads to vaso-occlusive crisis,” said Richard Pazdur, director of the FDA’s oncology center of excellence and acting director of the office of oncologic diseases in the FDA’s Center for Drug Evaluation and Research. “Vaso-occlusive crisis can be extremely painful and is a frequent reason for emergency department visits and hospitalization for patients with sickle cell disease.”