By Adria Calatayud
AstraZeneca reported mixed results from three late-stage clinical trials of an experimental treatment for a rare metabolic disease, but remains confident the drug can generate annual peak sales of $3 billion to $5 billion.
The U.K. drugmaker expects to be able to launch the medicine, efzimfotase alfa, in more markets than its predecessor treatment, Strensiq, the chief executive of AstraZeneca's rare-disease unit Alexion, Marc Dunoyer, said in an interview Tuesday. It plans to submit data to regulators as soon as possible, Dunoyer added.
AstraZeneca said the results of the studies support the drug's potential to transform the treatment of hypophosphatasia, a rare, chronic disease caused by deficient activity of an enzyme that is important for building healthy bones and supporting proper muscle function.
The company conducted three late-stage trials: two with infant patients showed positive results, and a third that studied the medicine in adults and adolescents didn't deliver a statistically significant outcome for its primary objective.
The drug was well tolerated across the three studies, the company said.
Alexion began to work on efzimfotase alfa after it launched Strensiq over a decade ago, seeking to develop a product that required lower frequency of administration and a lower dosage to facilitate wider adoption, Dunoyer said.
"We believe that efzimfotase will give us another great opportunity to reach patients in most countries of the world with a much easier administration product than Strensiq was," Dunoyer said. "The regional expansion is probably the biggest driver of growth for this product."
Write to Adria Calatayud at adria.calatayud@wsj.com
(END) Dow Jones Newswires
03-31-26 0820ET
