Orphan drugs

Orphan drug approvals have skyrocketed under Trump

llustration of medication bottle, pills, dollar, syringe, and price arrow
Illustration: Sarah Grillo/Axios

The past 2 years have seen a surge in the number of prescription drugs approved through a special process that was designed to help foster new treatments for rare diseases.

Why it matters: Rapid scientific advances have helped fuel this rise. But the sheer volume of rare-disease approvals is also sparking a debate about whether they're too easy to obtain, or whether they give drugmakers a way to game the system.

Roche buying Spark Therapeutics for $4.8 billion

A building with the Roche logo.
Roche is betting on gene therapies. Photo: Fabrice Coffrini/AFP via Getty Images

Roche, the Swiss-based pharmaceutical conglomerate, is acquiring Spark Therapeutics in a $4.8 billion all-cash deal. The Wall Street Journal first reported the buyout.

Why it matters: Pharmaceutical companies see promise in gene therapies and are willing to pay a large premium for them, but the treatments also come with record-high price tags. Spark has an $850,000 gene therapy to treat a rare eye condition, and a separate product is in the works for hemophilia.

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