FDA Extends Use of Rare Blood Disorder Drug to Very Young Children

The FDA has approved Promacta (eltrombopag) to treat low blood platelet count in pediatric patients – ages one year and older – with a rare blood disorder called chronic immune thrombocytopenic purpura (ITP). Promacta can be used in these children when they have not achieved an appropriate response using other ITP medicines or surgery to remove the spleen.


Idiopathic thrombocytopenic purpura (ITP) is a disorder that results in an abnormally low number of platelets, the cells that help your blood clot, and may have no symptoms. When signs and symptoms do occur, they may include easy or excessive bruising (purpura); superficial bleeding into the skin that appears as a rash of pinpoint-sized reddish-purple spots (petechiae), usually on the lower legs; prolonged bleeding from cuts; spontaneous bleeding from the nose; bleeding gums, especially after dental work; and fatigue.


“Today’s approval of Promacta emphasizes the FDA’s commitment to fully developing treatments in areas of pediatric hematology and oncology,” said Richard Pazdur, M.D., director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research. “This new use in ages one and up builds on a recent approval for ages six years and up, and fills an unmet need for young children whose disease has progressed after use of other available treatments.”


Promacta helps increase blood platelet production and is available as a tablet taken once-daily or as a powder that is mixed with liquid for children ages one to five to take orally. It was first approved in 2008 to treat adult patients with the same condition as the new pediatric indication. Promacta should be used only in patients with ITP whose degree of thrombocytopenia and clinical condition increase the risk for bleeding.


The most common side effects of treatment with Promacta in children ages one and older were infections of the upper respiratory tract or nose and throat (symptoms including fever, cough, nasal congestion, runny nose, and sore throat), diarrhea, abdominal pain, rash and increase in liver enzymes.


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


The FDA granted Promacta orphan drug designation because it treats a rare disease. 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 manufactured by Novartis in East Hanover, New Jersey.


See the FDA Announcement


See also Medical Law Perspectives, August 2015 Report: Pediatrician Liability for Childhood Disease Complications


See also Medical Law Perspectives, May 2013 Report: Drugs, Dosage, and Damage: Physician Liability for Prescribing or Administering Medication