Alexion Wins U.S. Approval for Drug for Rare Bone Disease

  • Analysts expect drug to have sales of $610 million in 2020
  • Drug is company's first U.S. approval after blockbuster

Alexion Pharmaceuticals Inc.’s treatment for a rare children’s bone disease was cleared by U.S. regulators, giving the company its first new-drug approval in the country in eight years.

The Food and Drug Administration cleared the drug, called Strensiq, for use in patients with hypophosphatasia, according to an agency statement Friday. Strensiq was approved in Europe last month and in Japan in July.

Alexion is trying to prove that it’s more than a one-hit wonder. The company made its name selling a single drug for a lot of money for a disease that only affects a small population -- a practice that’s proven lucrative. Last year that drug, Soliris, had $2.23 billion in sales -- and the company has estimated that 10,000 people or fewer in the U.S. and Europe have the disease it treats. So Alexion scours the world for patients and insists on full prices everywhere, a practice it says it will follow again with Strensiq.

"Incidence of HPP is somewhere around 3 patients per million of live births, or 12 newborns per year in the U.S. -- that’s ultra-rare," Chief Executive Officer David Hallal said by phone. "Our expectation would be a slow and steady launch trajectory as patients are diagnosed with this very rare disease."

Strensiq is expected to pull in $610 million in sales in 2020, according to an average of six analysts’ estimates compiled by Bloomberg. More than 70 percent of people born with hypophosphatasia die before their sixth birthday, according to Alexion. The company’s trials have found that those on Strensiq live longer than kids who were observed before the treatment was studied.

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