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"They are essentially buying an orphan drug portfolio," Yaron Werber, an analyst at TD Cowen, told Reuters.

The FDA gave Tepezza “orphan drug” status, which comes with financial incentives and exclusive marketing, benefits that are intended to encourage drugmakers to develop rare disease treatments.

Libmeldy is a so-called orphan drug - a pharmaceutical agent developed to treat a medical condition which, because it is so rare, may not be profitable to produce without some form of government assistance.

Two years after this child got her diagnosis, the Food and Drug Administration approved an orphan drug designed specifically for this disorder, called burosumab.

Spark Therapeutics, the company that makes Luxturna, estimates that about 6,000 people worldwide and between 1,000 and 2,000 in the U.S. may be eligible for its treatment—few enough that Luxturna was granted “orphan drug” status, a designation that the FDA uses to incentivize development of treatments for rare diseases.