US agency approves new drug for treatment of sickle cell disease

The Food and Drug Administration (FDA), an agency of the US department of health and human services, has approved Crizanlizumab-tmca, a new drug to treat extremely painful sickle cell disease flare-ups.

The new drug, which comes as the first-ever of its kind therapy targeted for the disease, was approved on Friday, as FDA gave Novartis, a multinational pharmaceutical company, the go-ahead to market Adakveo, a brand of it, in the country.

Adakveo is targeted at reducing the frequency of vaso-occlusive crisis — a common and painful complication of the disease that occurs when blood circulation is obstructed by sickled red blood cells — for patients of age 16 years and older.

According to FDA, the approval came after a randomized clinical trial enrolling 198 sickle cell disease patients with a history of vaso-occlusive crisis, where those who received Adakveo as against a placebo reported fewer healthcare visits.

Speaking at the event of the approval, Brett Giroir, acting commissioner at FDA, said the opportunity before medical experts working towards a cure for people living with the SCD “in the coming months and years is profound and historic.”

“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,” he said.

In SCD, the red blood cells are abnormally shaped and this restricts the flow in blood vessels, limits oxygen delivery to the body’s tissues, and leads to severe pain and organ damage.

According to Richard Pazdur, director of the FDA’s Oncology Center of Excellence, Adakveo will clamp down on frequent hospitalization of patients.

“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. It can be extremely painful and is a frequent reason for emergency department visits and hospitalization for patients with sickle cell disease,” said Pazdur, who  doubles as acting director of the Office of Oncologic Diseases in the FDA’s Center for Drug Evaluation and Research.

On the cost of the drug, Novartis said it will be priced between $84,852 and $113,136 per year for most patients who will be infused by a healthcare provider with three and four vials monthly — projecting that annual sales of it will top $1 billion.

Approximately 100,000 Americans are currently suffering from sickle cell disease, according to the Center for Disease Control and Prevention (CDC).

With over 300,000 babies born with the disease annually, about 250 million worldwide carry the gene. 75 percent of all patients with SCD live in Sub-Saharan Africa — Nigeria alone accounting for more than 100,000 new births annually.