FDA Expands Age Indication for Eltrombopag (Promacta) in ITP

August 24, 2015

The US Food and Drug Administration (FDA) announced today that it has expanded the indication of eltrombopag (Promacta, Novartis) for a rare blood disorder called chronic immune thrombocytopenic purpura (ITP) to include children as young as 1 year old.

The news falls on the heels of the FDA's decision in June to extend the drug's indication to children 6 years of age and older with the disorder.

Patients with ITP have abnormally low levels of platelets. "Without enough platelets, bleeding can occur under the skin, in mucous membranes (such as the mouth), or in other parts of the body," the agency said in a news release.

Eltrombopag, first approved for ITP in 2008, is intended only for patients with this condition who have not responded to corticosteroids, immunoglobulins, or splenectomy. As an oral thrombopoietin receptor agonist, the drug induces the proliferation and differentiation of bone marrow stem cells to boost blood cell production.

Today's decision by the FDA to extend the indication of eltrombopag for ITP in patients as young as 1 year was based on two double-blind, placebo-controlled trials involving 159 patients. In the first trial, which lasted 7 weeks, 62% of those taking eltrombopag had higher platelet counts between weeks 1 and 6 without rescue therapy compared with 32% of patients receiving a placebo. In the second trial, lasting 13 weeks, 41% of patients treated with eltrombopag experienced higher platelet counts compared with 3% of patients receiving a placebo.

Infections of the upper respiratory tract, diarrhea, abdominal pain, rash, and an increase in liver enzymes were the most common adverse events reported.

The FDA has not established the safety or efficacy of eltrombopag for ITP in patients younger than 1 year.

More information on today's decision is available on the FDA website.


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