FDA approves first TPO agonist for chronic liver disease
- Following a priority review, the Food and Drug Administration has approved Dova Pharmaceuticals' Doptelet for the treatment of thrombocytopenia in adult patients with chronic liver disease scheduled to undergo a procedure.
- The approval is based on safety and efficacy data from two Phase 3 trials, where the drug met all of its primary and secondary endpoints. Doptelet was better than placebo at reducing the proportion of patients needing platelet transfusions or rescue up to a week after a scheduled procedure.
- Dova expects to launch Doptelet on the U.S. market in June 2018. The pricing will be announced at the time of launch, and sales force training is under way.
Dova, which floated in mid-2017, has a focus on thrombocytopenia therapeutics, and Doptelet (avatrombopag) is its first drug to reach the market. The drug's initial indication, thrombocytopenia (a reduction in platelet numbers), is a common complication in chronic liver disease, and affects around 70,000 people. Thrombocytopenia means a higher risk of bleeding after diagnostic and therapeutic procedures unless people have platelet infusions.
"Doptelet is the first orally administered treatment option for patients with chronic liver disease, 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," said Alex Sapir, president and CEO, in a statement.
The drug aims to reduce the need for these additional interventions by mimicking the action of thrombopoietin, and therefore triggering platelet production.
"[Doptelet] 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 in the FDA's Center for Drug Evaluation and Research, in a statement.
Eun Yang, equity analyst at Jefferies, predicts U.S. sales of around $76.3 million in 2019, rising to peak sales of around $400 million or more for Doptelet.
"In [the] EU, we assume an EU partner and a [royalty of around] 30% on EU sales of Doptelet to Dova, [with] EU peak sales of [around] $110 million."
Rival Shionogi’s lusutrombopag has an FDA action date of Aug. 26.
Comparing the two, Yang suggested Dova's comes with a potentially better clinical profile and easier dosing, in an addition to the first-to-market advantage.
Dova is continuing development for related indications, and forthcoming milestones include: a Phase 3 trial in chemotherapy-induced thrombocytopenia (CIT) due to start in the second quarter of 2018, with a readout in mid-2020; a supplemental New Drug Application planned for Idiopathic thrombocytopenic purpura (ITP) in the second half of 2018, with a potential approval in 2019; and a Phase 3 data readout in a broader surgical population in the second half of 2019.
While expected, Leerink analyst Geoffrey Porges said it was surprising the Dova was not ready to go out of the gate.
"Surprisingly the company does not seem prepared to immediately launch the product, instead deferring its initial sales by several weeks," he wrote.
The analyst suggested the only remaining question was the price, which he estimated at $6,000.
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