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Raptor Drug Wins U.S. Approval to Treat Rare Disease

Raptor Pharmaceutical Corp. won U.S. approval for its drug to treat a rare metabolic disorder that attacks the kidneys.

The drug called Procysbi, previously known as RP103, may generate sales of $77 million in 2015, according to the average of two analysts’ estimates compiled by Bloomberg. Raptor, based in Novato, California, said in a statement today it will receive a $25 million payment from investment firm HealthCare Royalty Partners for the approval.

Procysbi treats nephropathic cystinosis, which affects about 3,000 people worldwide, including 500 in the U.S., the Food and Drug Administration said in a statement. The disease is an inherited condition in which the body accumulates the amino acid cystine within the cells, according to the National Institutes of Health.

The condition is usually diagnosed in the first year of life and can be fatal in the first decade, Raptor said. Children with nephropathic cystinosis usually experience poor growth, vision problems and severe kidney damage.

Raptor gained 5.3 percent to $6.90 at the close in New York.

Procysbi is a delayed-release version of cysteamine, the active ingredient in Mylan Inc.’s Cystagon. Raptor’s drug worked as well as Cystagon in a clinical trial with fewer doses and higher tolerability related to gastrointestinal side effects, the company said in a July 2011 statement.

“Sustaining appropriate levels of cysteamine in the body is the key to maintaining organ function and lowering the likelihood of kidney transplantation,” Craig Langman, head of kidney diseases at Ann & Robert H. Lurie Children’s Hospital of Chicago, said today in the statement released by Raptor.

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