The European Commission has approved Novartis’ Revolade (eltrombopag) as a first-in-class therapy for children aged one and over with chronic idiopathic thrombocytopenic purpura (ITP) that have not responded to other treatments.
In ITP, blood does not clot properly due to a low number of platelets, with sufferers experiencing purple bruises or tiny red or purple dots on the skin as well as nosebleeds, bleeding from the gums during dental work, or other bleeding that's hard to stop. In most cases, the immune system is thought to attack and destroy its own platelets.
“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, managing director and global head of Novartis Oncology Development and Medical Affairs.
Revolade is approved in more than 100 countries worldwide for adults with ITP, in over 45 countries for the treatment of thrombocytopenia (low blood platelet counts) in patients with chronic hepatitis C and, in September 2015, received approval for severe aplastic anaemia.
The paediatric approval includes the use of tablets as well as a new oral suspension formulation of Revolade, which is designed for younger children who may not be able to swallow tablets.
ITP is a rare blood disorder that affects about five in 100,000 children each year. Chronic ITP – defined as ongoing disease more than 12 months after diagnosis – occurs in 13-36 percent of children with ITP. Revolade is a thrombopoietin (TPO) receptor agonist that works by inducing stimulation and differentiation of megakaryocytes (large cells, found especially in bone marrow) to increase platelet production.