Medicine

FDA approves high-priced drug for rare genetic disorder

Alnylam CEO Dr. John Maraganore poses for a portrait with lab equipment behind.
Alnylam CEO John Maraganore. Photo: Lane Turner/The Boston Globe via Getty Images

The FDA has approved the drug Givlaari, made by Alnylam Pharmaceuticals, for adults who have acute hepatic porphyria, a genetic disorder that leads to severe abdominal pain and affects just one in 25,000 people.

The big picture: Alnylam set Givlaari's net price, after discounts to insurers, at $442,000 per year, or about $39,000 per vial. It's a lofty price tag, but those prices are common among drugs that use new scientific techniques and that treat very rare diseases.

The fall of a major specialty pharmacy

Data: Money.net; Chart: Axios Visuals

Diplomat Pharmacy, which sells medications to people with complex conditions and acts as a drug benefit middleman, is a shell of itself. The company was worth more than $3 billion in its heyday in 2015, but is now worth a little more than $200 million after a disastrous third quarter.

The bottom line: Larger specialty drug players — owned by Cigna, CVS Health and UnitedHealth Group — have crushed Diplomat with their size. Now, Diplomat is running out of cash and is being forced to sell assets, or the entire company, because it has "substantial doubt surrounding our ability to continue,” the company said in its earnings report.