FDA Grants Priority Review for New Leukemia Option

The new indications include treatment of pediatric CML as well as a powder for oral suspension formulation of the agent

The US Food and Drug Administration (FDA) has granted priority review status for two new indications of dasatinib (Sprycel, Bristol-Myers Squibb), according to the drug’s developer. The new indications include treatment of pediatric patients with chronic myeloid leukemia (CML), as well as a powder for oral suspension (PFOS) formulation of the agent.

“[Dasatinib]… has the potential to be the first drug in its class approved for pediatric use with an oral solution,” said Murdo Gordon, executive vice president and chief commercial officer of Bristol-Myers Squibb.

Dasatinib, a tyrosine kinase inhibitor, is currently approved to treat newly diagnosed Philadelphia chromosome–positive CML in the chronic phase in adult patients, and Philadelphia chromosome–positive CML in patients intolerant to imatinib, as well as for treatment of acute lymphoblastic leukemia.

The priority review, which sets a goal of the FDA taking action within 6 months instead of the usual 10 months under standard review, is based on data from an ongoing phase II trial known as CA180-226. Results from that study were presented on June 5 at the American Society of Clinical Oncology (ASCO) 2017 Annual Meeting, held in Chicago, by Lia Gore, MD, of the University of Colorado School of Medicine (abstract 10511).

In that study, 113 pediatric patients were treated in three cohorts: 29 CML patients were resistant or intolerant of imatinib and were treated with dasatinib tablets 60 mg/m²; 51 newly diagnosed CML patients were treated with the same dasatinib regimen; and 33 newly diagnosed CML patients were treated with dasatinib 72 mg/m² PFOS.

For the imatinib-resistant/intolerant patients, major cytogenetic response (MCyR) was achieved in 90% of patients, and complete cytogenetic response (CCyR) was achieved in 83% of patients. Major molecular response (MMR) was achieved in 55% of patients.

Both the tablet and PFOS forms of dasatinib yielded good results in the newly diagnosed patients as well. In both those groups, 96% achieved MCyR, and 94% achieved CCyR, while 70% achieved MMR. The estimated 48-month progression-free survival rate was above 75% for the imatinib-resistant/intolerant patients, and above 90% for the newly diagnosed CML patients. The toxicity of dasatinib was consistent with that seen in adults.

The priority review has an action date of November 9, 2017.