FDA Grants Avatrombopag Orphan Drug Designation for the Treatment of Chemotherapy-induced Thrombocytopenia

Publisher: The Insight Partners

Dova Pharmaceuticals, Inc., a wholly owned subsidiary of Swedish Orphan Biovitrum AB (publ) (Sobi™) has been granted Orphan Drug Designation[1] (ODD) by the US Food and Drug Administration (FDA) for avatrombopag for the potential treament of Chemotherapy-Induced Thrombocytopenia (CIT). Enrollment remains ongoing for the phase 3 clinical study for the treatment of patients with CIT. 

CIT, a potentially serious complication of chemotherapy, results in low platelet levels and can lead to chemotherapy dose reductions, chemotherapy dose delays, or changes to chemotherapy regimens. For cancer patients receiving chemotherapy with curative intent, alterations in their chemotherapy regimen due to low platelets may compromise their long-term outcomes. Approximately 10 percent of US cancer patients per year experience CIT2 which may require chemotherapy regimen modifications. Currently there are no approved treatments available for CIT in the US or EU.

"We are very pleased with the orphan drug designation from FDA for avatrombopag within CIT. Chemotherapy-induced low platelet counts are a crucial impediment for patients to be able to adhere to their chemotherapy regimen. Avatrombopag is currently being studied in a phase 3 study for this severe medical condition," said Milan Zdravkovic, Head of Research and Development and Chief Medical Officer at Sobi.

Avatrombopag is approved as Doptelet® in both the US and the EU for the treatment of thrombocytopenia in adult patients with chronic liver disease (CLD) who are scheduled to undergo a procedure, and in the US for the treatment of thrombocytopenia in adult patients with chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment.