FDA approves drug to treat rare blood disorder in children

The US Food and Drug Administration (FDA) has approved a label expansion for Novartis’ Promacta (eltrombopag) to treat low blood platelet count in paediatric patients with a rare blood disorder called chronic immune thrombocytopenic purpura (ITP).

Daily RX writes that last Monday, the FDA has announced that it is extending the use of Promacta for children with ITP for the ages one year and above to help curb low blood platelet count. The release from the FDA said without enough platelets, bleeding can occur under the skin, in mucous membranes (such as in the mouth) or in other parts of the body.

The first trial also showed that Promacta boosted and sustained platelet counts among pediatric patients with chronic ITP. In a group of ITP patients receiving a fake pill (placebo), only 32 percent saw an increase.

The extension of the approval has been brought about by the results of two double-blind trials which include a Phase 3 clinical trial in nearly 160 patients.

Ligand, which is entitled to receive royalties on global sales of Promacta, said the updated label also includes a new oral suspension formulation of Promacta that is designed for younger children who may not be able to swallow tablets.

Promacta is a once-daily oral thrombopoietin (TPO) receptor agonist that works by inducing stimulation and differentiation of megakaryocytes from bone marrow stem cells to increase platelet production.

The new approval builds on a recent approval for ages 6 years and up.

The most common side effects in the treatment arm were infections of the upper respiratory tract or nose and throat, diarrhoea, abdominal pain, rash and increase in liver enzymes, the FDA said. A small number of pediatric patients with chronic ITP may be at risk of significant bleeding5. Orphan drug designation provides financial incentives – like tax credits, user fee waivers, and eligibility for market exclusivity – to promote rare disease drug development.

Promacta is produced by Novartis, based in East Hanover, N.J. This is especially vital for children who did not respond well with other drugs.