FDA grants Avatrombopag Orphan Drug Designation for thrombocytopenia

The US Food and Drug Administration (FDA) has granted avatrombopag (Doptelet) Orphan Drug Designation (ODD) for the potential treatment of chemotherapy-induced thrombocytopenia (CIT). Enrolment for the Phase III clinical trial for avatrombopag as a treatment of CIT is ongoing.

CIT results in low platelet levels; patients with CIT frequently require chemotherapy dose reductions or delays and or chemotherapy regimen changes. As a result, these patients may have compromised long-term outcomes from their treatments.

“CIT is a potentially serious complication of chemotherapy with no available treatments.  The granting of ODD highlights the significant need for a drug that could improve chemotherapy-induced low platelet counts and thereby enable patients to better adhere to their chemotherapy regimen,” said Mahmood Ladha, President and Head of Dova Pharmaceuticals Inc.

Doptelet is an oral thrombopoietin (TPO) receptor agonist administered with food. It is already FDA approved for the treatment of:

• thrombocytopenia in adult patients with chronic liver disease (CLD) who are scheduled to undergo a procedure (also European Medicines Agency (EMA) approved for this indication)
• thrombocytopenia in adult patients with chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment.

Chronic ITP is a rare autoimmune bleeding disorder characterised by low number of platelets, affecting approximately 60,000 adults in the US.