FDA accepts for review sBLA for Doptelet to treat chronic immune thrombocytopenia (ITP) in patients who had an insufficient response to a previous treatment. -Dova Pharma + Salix Pharma

Dova Pharmaceuticals, Inc. a pharmaceutical company focused on acquiring, developing, and commercializing drug candidates for diseases where there is a high unmet need, announced the FDA has accepted for review the supplemental New Drug Application (sNDA) for Doptelet (avatrombopag) for a new indication, the treatment of chronic immune thrombocytopenia (ITP) in patients who have had an insufficient response to a previous treatment.

ITP is an autoimmune bleeding disorder characterized by thrombocytopenia, i.e., an abnormally low level of platelets. The Prescription Drug User Fee Act (PDUFA) goal date for an FDA decision on the sNDA is June 30, 2019. Doptelet, a second generation, orally administered thrombopoietin receptor agonist (TPO-RA), was approved by FDA in May 2018 for the treatment of thrombocytopenia in adult patients with chronic liver disease (CLD) who are scheduled to undergo a procedure. The ITP sNDA is supported by safety and efficacy data from one pivotal randomized, placebo-controlled Phase III clinical trial in the target indication that met its primary (number of weeks with a platelet count greater than 50x109/L in the absence of rescue therapy) and first secondary (proportion of subjects with platelet counts greater than 50�109/L on Day 8) efficacy endpoints with high statistical significance (P<0.0001). data from the phase iii clinical trial has been recently published online br j haematol. 2018 sep 7. doi: 10.1111 bjh.15573. epub ahead of print and will be included in an upcoming volume of the british journal of haematology. additional supportive efficacy data for the itp snda are provided by two phase ii itp clinical trials as well as the two phase iii trials for the treatment of thrombocytopenia in patients with cld. data from all 24 studies in the avatrombopag clinical development program support the safety and tolerability of avatrombopag across multiple indications.>