Treatments for rare diseases — key to the success of several Bay State companies — surged nationally last year, a trend that experts say will likely only continue.
Seventeen of the 41 novel new drugs the Food and Drug Administration approved in 2014 treat rare or "orphan" diseases that affect 200,000 or fewer Americans, offering new hope to patients who previously had few or no drugs available to treat their conditions.
"This really is a time of great interest in orphan drugs," said Mary Dunkle, vice president for educational initiatives at the National Organization for Rare Disorders. "There were a few companies who took a risk early on and made this a successful business model."
An orphan drug designation by the FDA means its developer qualifies for tax benefits and seven years of exclusivity, compared to five years for other drugs.
"Companies are understanding the great value of pushing into new areas where there has not yet been an attempt to develop a drug," said Irving Adler, a spokesman for Alexion Pharmaceuticals, a Connecticut-based firm with operations in Cambridge.
Alexion has one approved orphan drug Soliris, which treats two rare, life-threatening diseases.
Other local companies that have developed orphan drugs include Burlington-based Dyax Corp. and Cambridge-based Genzyme.
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