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FDA Expands Use of Eltrombopag (Promacta) in Young Children With Rare Blood Disorder

Treatment approved for adults in 2008

The FDA has given the green light to eltrombopag (Promacta, Novartis) for the treatment of low platelet counts in pediatric patients –– 1 year of age and older –– with chronic immune thrombocytopenic purpura (ITP). Eltrombopag can be used in these patients when they have not achieved an appropriate response using other ITP medications or surgery to remove the spleen.

ITP is a disorder that results in an abnormally low number of platelets. Without enough platelets, bleeding can occur under the skin, in mucous membranes (such as in the mouth), or in other parts of the body.

Eltrombopag helps increase platelet production. It is available as a tablet taken once daily or as a powder that is mixed with liquid for children 1 to 5 years of age to take orally. It was first approved in 2008 to treat adults with ITP.

Eltrombopag should be used only in patients with ITP whose degree of thrombocytopenia and clinical condition increase the risk for bleeding.

The efficacy and safety of eltrombopag in pediatric patients 1 to 17 years of age with chronic ITP were evaluated in two double-blind, placebo-controlled studies involving a total of 159 participants. The primary endpoint of both trials was an increase in platelet counts. In the first study, 67 patients were randomly assigned to receive either eltrombopag or placebo daily for 7 weeks. Of those treated with eltrombopag, 62% showed an improvement in platelet counts without rescue therapy between weeks 1 and 6, compared with 32% of the placebo group.

In the second study, 92 patients received either eltrombopag or placebo daily for 13 weeks. Of those treated with eltrombopag, 41% experienced increased platelet counts for at least 6 out of 8 weeks between weeks 5 and 12 compared with 3% of patients receiving placebo.

In both trials, patients treated with eltrombopag also had less need for other therapies to increase their platelet counts, such as corticosteroids or platelet transfusions. Among patients taking one or more ITP medications at the start of the trials, about half were able to reduce or discontinue their use of these medications, primarily corticosteroids.

The most common adverse effects of treatment with eltrombopag in children 1 year of age and older included infections of the upper respiratory tract or nose and throat (with symptoms that included fever, cough, nasal congestion, runny nose, and sore throat), diarrhea, abdominal pain, rash, and an increase in liver enzymes.

The safety and efficacy of eltrombopag have not been established in pediatric patients younger than 1 year of age with ITP, or in pediatric patients with thrombocytopenia associated with chronic hepatitis C and severe aplastic anemia.

The FDA granted eltrombopag an “orphan drug” designation because it treats a rare disease. This designation provides financial incentives –– such as tax credits, user-fee waivers, and eligibility for market exclusivity –– to promote drug development for rare diseases.

Source: FDA; August 24, 2015.

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