FDA to Review Doptelet for Treatment of Chronic Immune Thrombocytopenia

The sNDA is supported by results from a 6-month Phase 3 clinical trial
The sNDA is supported by results from a 6-month Phase 3 clinical trial

The Food and Drug Administration (FDA) has accepted for review a supplemental New Drug Application (sNDA) for Doptelet (avatrombopag; Dova) for the treatment of chronic immune thrombocytopenia (ITP) in patients who have had an insufficient response to a previous treatment. 

Doptelet is a thrombopoietin receptor agonist (TPO-RA) that was approved in May 2018 to treat thrombocytopenia in adults with chronic liver disease who are scheduled to undergo a procedure. 

The sNDA is supported by results from a 6-month Phase 3 clinical trial, recently published in the British Journal of Haematology. A total of 49 patients were randomized to receive either avatrombopag (N=32) or placebo (N=17). Results showed that avatrombopag was superior to placebo for the primary endpoint of cumulative number of weeks of platelet response ≥50 × 109/L without rescue therapy; 12.4 weeks in the treatment group vs 0 weeks in the placebo group (P<.0001). 

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Additionally, the secondary endpoint of greater platelet response rate at day 8 was observed for patients treated with avatrombopag vs. placebo (65.63% vs. 0.0%; P<.0001).

The FDA has set a Prescription Drug User Fee Act (PDUFA) date for a decision on the sNDA for June 30, 2019. 

For more information visit Wiley.com.