Avatrombopag ITP Phase 3 PHASE3 COMPLETED n=49
Drug: Doptelet · SOBI
Study Design
DesignRandomized, double-blind, placebo-controlled
Randomization2:1
BlindingDouble-blind
Enrollment49
Duration26 weeks
Treatment Arms
Avatrombopag 20 mg 20 mg oral once daily n=32
Placebo Placebo daily n=17
Primary Endpoints
[{"id":"dotp-itp-ep1","name":"Cumulative weeks of platelet response (>=50K) without rescue","type":"PRIMARY","unit":"weeks","p_value":"<0.0001","results":[{"arm":"dotp-itp","label":"12.4 weeks (median)","value":12.4},{"arm":"dotp-pbo","label":"0.0 weeks (median)","value":0}],"timepoint":"Over 26 weeks","description":"Total weeks with platelets above 50,000/uL (the threshold for adequate hemostasis) without needing rescue therapy. ITP patients live with bleeding risk from persistently low platelets."}]
Safety Results
Headache 37.5% (vs 11.8% placebo). Contusion, URTI, arthralgia, epistaxis, fatigue, gingival bleeding, petechiae, nasopharyngitis all >=10%. No hepatotoxicity. Oral dosing with no food restriction.
Assessment
Small trial (n=49) but strong efficacy: 87.5% response at Week 26 vs 5.9% placebo. Rapid onset (65.6% by Day 8). Oral with no food restriction = compliance advantage. Pediatric ITP approval July 2025 with Sprinkle formulation expands addressable market.
Background & Context
Oral TPO receptor agonist for chronic ITP. Key advantage vs Nplate (romiplostim): oral vs SC injection. Key advantage vs Promacta (eltrombopag): no food restriction, no hepatotoxicity monitoring.
Data from Supabase · Updated 2026-03-24