FDA clears Dova drug for liver-related low blood platelets

Dova Pharmaceuticals has won its first product approval, getting a green light from the FDA for Doptelet as a treatment for low platelet counts (thrombocytopenia) in patients with chronic liver disease who have to undergo a medical procedure.

Doptelet (avatrombopag) – an orally-active thrombopoietin receptor agonist (TRA) – is delivered over a five-day course that prepares patients to have a medical or dental procedure five to eight days thereafter, and is the first drug to be approved by the US regulator in this indication.

At the moment, around two-thirds of patients with liver-related thrombocytopenia have to receive transfusions of platelets ahead of time, which is cumbersome, must occur on the same day of the procedure and carries risks of adverse reactions and antibody development. The company estimates there are about 70,000 patients in the US eligible for treatment with its drug, and that they undergo an average of three procedures per year.

“This drug may decrease or eliminate the need for platelet transfusions, which are associated with risk of infection and other adverse reactions,” said Richard Pazdur, acting director of the Office of Hematology and Oncology Products at the FDA.

Doptelet is in the same class as Novartis’ Promacta/Revolade (eltrombopag), originally developed by GlaxoSmithKline, which is used to treat low platelet levels caused by hepatitis C virus infection as well as the rare diseases idiopathic thrombocytopenia (ITP) and acquired severe aplastic anaemia. The drug is a big earner for Novartis and one of its growth products with sales of $867m last year – up 37% on 2016.

Dova bills its drug as a second-generation TRA with fewer liver-related side effects than its rival, and a reduced tendency to interact with foods. The US biotech says it has Revolade’s territory firmly in its sights, with plans to file for approval in ITP in the second half of the year, and is also starting a pivotal trial of Doptelet in chemotherapy induced thrombocytopenia as well as a study in pre-surgery patients.

In Europe, the company has filed with the EMA for the initial pre-medical procedure indication, and notes the agency has granted a standard review assessment for that application, with a targeted approval date of around June 2019.

The company has spent the last months building a sales team in preparation of a launch next month, and setting up its access programmes, including free supply of the drug for eligible uninsured patients. It’s not communicating the price of the drug yet, saying it will do so on the date of launch.

Dova may not have too long before it faces direct competition in its first indication, however, as Japanese drugmaker Shionogi filed for approval of its lusutrombopag (S-888711) candidate in pre-medical procedure patients in the US and Europe in February. Shionogi has a priority review for its marketing application in the US with a verdict due by August 26.