The U.S. FDA has completed their Priority Review and granted full approval to avatrombopag (Doptelet) for the treatment of thrombocytopenia in adult patients with chronic liver disease who are scheduled to undergo a procedure.
Last night, Dova Pharmaceuticals, Inc. announced that the US Food and Drug Administration (FDA) has completed a Priority Review and granted full approval to avatrombopag (Doptelet) for the treatment of thrombocytopenia in adult patients with chronic liver disease (CLD) who are scheduled to undergo a procedure.
The approval was based on results from the consistent safety and efficacy data reported from a pair of global, multicenter, randomized, double-blind, placebo-controlled phase 3 trials that met all primary and secondary endpoints and is the first for a thrombopoietin (TOP) receptor agonist in the United States.
“We are delighted FDA has approved DOPTELET, which represents a significant milestone for Dova, physicians, and most importantly, patients,” said Alex C. Sapir, President and Chief Executive Officer in a press release. “DOPTELET is the first orally administered treatment option for patients with CLD, allowing the majority of patients to avoid a platelet transfusion prior to a procedure by increasing platelet counts to the target level of greater or equal to 50,000 per microliter.”
Per the National Cancer Institute (NCI) at the National Institute of Health (NIH), there are approximately 70,000 patients with CLD that have a platelet count of less than 50,000/µL. Typically, these patients require 1 to 3 invasive diagnostic and therapeutic procedures per year, and each puts the patient at risk for bleeding.
If not effectively treated, thrombocytopenia can lead to severe uncontrolled bleeding, resulting in prolonged hospitalizations and other post-procedure complications.
“Given the need for patients with CLD to routinely undergo multiple, invasive procedures, the availability of an oral agent that can lead to a measured increase in platelets, to minimize the need for platelet transfusions and risk of bleeding, will facilitate the clinical management of these patients,” said Norah Terrault, MD, MPH., Professor of Medicine at the University of California San Francisco, Division of Gastroenterology, and Principal Investigator for the phase 3 pivotal avatrombopag trials.
Avatrombopag is a second generation, once daily, orally administered TPO receptor agonist, designed to mimic the effects of TPO, the primary regulator of normal platelet production. In the two phase 3 trials — ADAPT-1 [N=231] and ADAPT-2 [N=204] – adults with thrombocytopenia with a platelet count of less than 50,000 µL and CLD were assigned to either 40 mg or 60 mg of abatrombopag daily for 5 days based on their baseline platelet counts (40 to <50,000/mL or <40,000/mL, respectively).
Avatrombopag exhibited superiority to placebo in increasing the proportion of patients not requiring platelet transfusions or rescue procedures for bleeding up to 7 days following a scheduled procedure in both trials in both the 40 mg (ADAPT-1, 88% vs. 38%, p <0.0001; ADAPT-2, 88% vs. 33%; p<0.0001), and 60 mg (ADAPT-1, 66% vs. 23%, p <0.0001; ADAPT-2, 69% vs. 35%; p=0.0006) treatment groups.
Additionally, avatrombopag was superior to placebo at the 2 secondary efficacy endpoints in each trial.
Dova has announced that, given its extensive preparations to date, the company is prepared to launch the drug in June.
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