Nearly all patients treated with dasatinib (Sprycel) achieved a complete cyotgenic response.
The FDA has granted approval to dasatinib (Sprycel) to treat pediatric patients with Philadelphia chromosome-positive (Ph+) chronic myeloid leukemia (CML), according to a press release.
“While chronic myeloid leukemia is rare in children, accounting for less than 3% of all pediatric leukemias, it is often more aggressive in younger patients than in adults and until recently, there have been few available treatment options,” said Vickie Buenger, president, Coalition Against Childhood Cancer. “The FDA’s decision to approve the expanded use of Sprycel in children with Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase may bring new hope to these patients and their families.”
The approval was based on positive findings from phase 1 and phase 2 clinical trials, which included 97 children with chronic phase Ph+ CML. In the phase 2 trial, 51 patients were newly-diagnosed and 46 patients from both trials were resistant or intolerant to treatment with imatinib.
Most patients were treated with dasatinib 60-mg/m2 daily until disease progression or unacceptable toxicity.
After 24 months of dasatinib therapy, 96.1% of newly-diagnosed patients and 82.6% of patients resistant or intolerant to imatinib achieved a complete cyotgenic response (CCyR).
The investigators said that the median duration of CCyR, major cytogenic response, and major molecular response could not be estimated since more than 50% of patients had not progressed at the time of data cut-off, according to the release.
The most common adverse reactions in pediatric patients were headache, nausea, diarrhea, skin rash, vomiting, pain in extremity, abdominal pain, fatigue, and arthralgia, according to the FDA.
“Our decision to pursue an expanded indication for Sprycel is indicative of our commitment to exploring pediatric applications within our broad development program,” said Johanna Mercier, head, US Commercial, Bristol-Myers Squibb. “We are pleased this option is now available for appropriate pediatric chronic phase CML patients and their physicians.”
Previously, dasatinib received priority review and orphan drug designations for pediatric patients with Ph+ CML. Dasatinib has been previously approved to treat Ph+ CML who are resistant or intolerant to prior therapy.
“Options for pediatric patients with chronic myeloid leukemia are limited, and it is challenging to conduct clinical trials investigating potential new treatments in this small patient population,” said Lia Gore, MD, University of Colorado School of Medicine and Children’s Hospital Colorado. “Dasatinib is an important new option to help address the unmet needs of children with Philadelphia chromosome-positive CML in chronic phase.”