FDA Committee Votes Against AstraZeneca’s Anemia Treatment

(Bloomberg) -- AstraZeneca Plc was dealt a blow as an advisory committee of the Food and Drug Administration said it doesn’t support the approval of treatment for anemia in patients with chronic kidney disease.

The Cardiovascular and Renal Drugs Advisory Committee voted 13 to 1 that the benefits and risk of AstraZeneca’s roxadustat don’t support approval for non-dialysis dependent patients, the company said in a statement. Similarly, the vote was 12 to 2 against the use of roxadustat in dialysis-dependent patients.

The decision comes after the company and its partner FibroGen Inc. were accused of overstating the safety profile of the anemia pill earlier this year. FibroGen issued a statement in April clarifying the results, saying the cardiovascular safety analyses had included “post-hoc changes,” but that the drug still held up against its comparator and had a strong benefit-risk profile.

Analysts project U.S. sales of about $500 million for the drug by 2025 if approved, which would be divided between the companies and only equates to a small percentage of AstraZeneca’s overall revenue.

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AstraZeneca said the safety and efficacy of roxadustat, an oral treatment, has been demonstrated in the phase 3 program, including more than 8,000 patients and published in five peer-reviewed journal articles.

Roxadustat is already approved in a number of countries including China, Japan, Chile and South Korea. The treatment is under regulatory review in other jurisdictions, including the European Union, where it’s known as evrenzo and recently received a positive opinion from the European Medicines Agency’s Committee for Medicinal Products for Human Use.

Glaxo’s Drug

GlaxoSmithKline Plc also published results Friday for its equivalent anemia drug -- daprodustat -- with positive headline data from five advanced-stage trials. The study met its primary endpoint with improved hemoglobin levels in untreated patients and showed it wasn’t inferior to the current standard treatment option in terms of its risk profile for major adverse cardiovascular events, a key concern for patients with anemia due to chronic kidney disease.

Astra said the FDA will consider the vote as well as independent recommendations from experts and is not bound by the committee’s decision. The FDA hasn’t announced when it will make a final decision.

“New solutions are needed for the six million people in the U.S. affected by anemia of chronic kidney disease,” said Mene Pangalo, executive vice president of biopharmaceuticals at AstraZeneca. “Although we are disappointed by today’s outcome, we will continue to work closely with our partner FibroGen and the FDA to determine the path forward for roxadustat.”

Anemia can cause significant fatigue, cognitive dysfunction and decreased quality of life. It is also associated with increased risk of hospitalization and death.

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