Smaller-cap orphan drug companies provide investment opportunities in the biotech space due to their niche characteristics and pricing freedom, a senior analyst at Global Hunter Securities, LLC, says.
“I think the orphan drug space will be hot, given that there is no pricing pressure for these drugs because of the unmet medical need for the drugs that they’re targeting,” said Dr. Kimberly Lee, D.O., adding that health care reform has had virtually no impact on orphan drug pricing.
Corcept Therapeutics (CORT) is developing Corlux for the treatment of Cushing’s syndrome, an orphan indication with less than 10,000 patients, but which may be priced at $100,000 or more per year.
“Corcept Therapeutics has an interesting story,” Dr. Lee said. “With no major competition, and a pretty good safety profile and some documented cases of efficacy in some major endocrinology journals, we think that there could be significant upside for this company from current levels.”
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