Novartis receives EU approval for Revolade as first-in-class therapy for children aged 1 year and above with chronic ITP

Two formulations approved: once-daily tablet and oral suspension formulation designed for younger children who may not be able to swallow tablets.

"For the families and caregivers of children affected by a rare disease, having a new treatment option can be game-changing in managing the disease," said Alessandro Riva, MD, Global Head, Novartis Oncology Development and Medical Affairs.

The approval is based on data from two double-blind, randomized, placebo-controlled trials, including the largest Phase III clinical trial in this patient population. In these studies, patients in the treatment and placebo arms were permitted to use some stable maintenance ITP therapies, per local treatment practices. Treatment with Revolade significantly increased and sustained platelet counts among pediatric patients with chronic ITP who were refractory to or had relapsed after prior chronic ITP therapies, and some patients taking concomitant ITP medications were able to reduce or discontinue their use of these medications, primarily corticosteroids.

ITP is a rare blood disorder that affects about five in 100,000 children each year and is characterized by a low platelet count. Because people with ITP have a low number of platelets, they may bruise easily and experience bleeding that is hard to stop. Chronic ITP, defined as ongoing disease more than 12 months after diagnosis, occurs in 13-36% of children with ITP. A small number of pediatric patients with chronic ITP may be at risk of significant bleeding.

The EC approval applies to all 28 EU member states, plus Iceland, Norway and Liechtenstein.