FDA Expands Use for Rare Blood Disorder Drug

Promacta use now includes young children with chronic immune thrombocytopenia.

The FDA yesterday approved an expanded use for a rare blood disorder treatment that will now include younger children.

The updated label for the chronic immune thrombocytopenia (ITP) treatment eltrombopag (Promacta) now will include children aged 1 year or older who did not have a sufficient response to corticosteroids, immunoglobulins, or splenectomy. The expanded use for Promacta also includes a new oral suspension formulated for younger children who can’t swallow tablets.

The drug was approved by the FDA in tablet form in June 2015 for children aged 6 years and older, following its initial approval in 2008 for adult patients with ITP. ITP is characterized by a low platelet count and affects as many as 5 in 100,000 children each year. As many as 30% of these children experience the disease for more than 6 months and are diagnosed with chronic ITP, putting them at ongoing risk for significant bleeding.

“It’s challenging and often very emotional for parents of a baby or toddler affected by a rare condition to manage their child’s disease with limited treatment options,” said Bruno Strigini, president of Novartis Oncology, in a press release. “Today’s label expansion for Promacta provides a new disease management option for families affected by chronic ITP and highlights our commitment to providing treatments for even the youngest children with rare diseases.”

ITP, which is characterized by a low platelet count, afflicts up to 5 in 100,000 children annually. Chronic ITP is classified as an ongoing disease more than a year following diagnosis, occurs between 13 and 36% of children with ITP. A minority of pediatric patients with chronic ITP may face a significant bleeding risk.

The expanded label for Promacta followed 2 double-blind, placebo-controlled trials, which includes the largest phase 3 clinical trial in ITP patients. Promacta was found to significantly increase and sustain platelet counts in pediatric patients with chronic ITP who previously had an insufficient response to chronic ITP therapies.

Certain patients administered concomitant ITP medications were able to decrease or halt use of chronic ITP medications, specifically corticosteroids. Use of Promacta should be limited to patients whose degree of thrombocytopenia and clinical condition increases bleeding risk.