Novartis Wins Second FDA Approval for Promacta

Novartis scored its second FDA approval for Promacta since acquiring the drug from GlaxoSmithKline in March, this time to treat children ages 1 to 5 with a rare blood disorder that causes low platelet counts.

The agency approved Promacta (eltrombopag) for children with chronic immune thrombocytopenia purpura who are ages 6 through 17 in June. The drug, which has orphan status, was originally approved in 2008 for adults with the disease.

The FDA based its approval on two double-blind, placebo-controlled trials of 159 patients with a primary endpoint of increased platelet count. Sixty-two percent of children receiving Promacta (eltrombopag) daily for seven weeks saw their platelet counts grow, while 41 percent of those getting the drug daily for 13 weeks improved. In both trials, the Promacta arm had less need for other treatments, such as corticosteroids or platelet transfusions, to increase platelet counts.

ITP affects roughly five in 100,000 children each year, and up to 30 percent of those develop chronic ITP, which puts them at risk of significant bleeding.

Last year’s revenues for Promacta were about $364 million, according to GSK’s 2014 annual report. The drug is marketed under the brand name Revolade in most countries outside the U.S. — Meg Bryant