批准日期：2018年5月23日 公司：AkaRx Inc.
血小板计数（x109/L） 每日剂量一次 持续时间
不到40 60毫克（3片） 5天
40到不到50 40毫克（2片） 5天
Avatrombopag Tablet Approved by FDA for Patients with Chronic Liver Disease
The FDA has announced its approval of avatrombopag (Doptelet, AkaRx) tablets to treat low blood platelet count in adults with chronic liver disease (CLD) who are scheduled to undergo a medical or dental procedure. This is the first drug approved for this use by the FDA.
According to Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research, patients with chronic liver disease who have low platelet counts—thrombocytopenia—and who require a medical or dental procedure are at increased risk of bleeding.
“Doptelet was demonstrated to safely increase the platelet count. This drug may decrease or eliminate the need for platelet transfusions, which are associated with risk of infection and other adverse reactions,” Pazdur said, in a statement.
The drug is expected to be made available in June, according to Dova Pharmaceuticals, the parent company of AkaRx.2 “Doptelet is the first orally administered treatment option for patients with CLD, allowing a 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,” said Alex Sapir, president and CEO of Dova Pharmaceuticals, in a statement.
When patients have moderately to severely reduced platelet counts, serious or life-threatening bleeding can occur, especially during invasive procedures.
Patients with significant thrombocytopenia typically receive platelet transfusions immediately prior to a procedure to increase the platelet count.
The safety and efficacy of avatrombopag was studied in a pair of Phase 3 trials (ADAPT-1 and ADAPT-2) involving 435 patients with chronic liver disease and severe thrombocytopenia, who were scheduled to undergo a procedure that would typically require platelet transfusion. The trials investigated 2 dose levels of avatrombopag administered orally over 5 days, as compared to placebo. The trial results showed that for both dose levels of avatrombopag, a higher proportion of patients had increased platelet counts, and did not require platelet transfusion or any rescue therapy on the day of the procedure, and up to 7 days following the procedure, as compared to those treated with placebo.
“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, principal investigator for the pivotal pair of Phase 3 avatrombopag trials, and a professor of medicine at the University of California San Francisco’s Division of Gastrointerology, in a statement.
The most common adverse effects reported by clinical trial participants who received avatrombopag were fever, stomach pain, nausea, headache, fatigue, and swelling in the hands or feet. People with CLD, and people with certain blood clotting conditions may have an increased risk of developing blood clots when taking avatrombopag.
This product was granted Priority Review, under which the FDA’s goal is to take action on an application within 6 months where the agency determines that the drug, if approved, would significantly improve the safety or effectiveness of treating, diagnosing or preventing a serious condition.