Nov 20, 2019

FDA approves high-priced drug for rare genetic disorder

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.

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Axios

FDA approves drug with annual cost of $442,000 for rare genetic disorder

Photo: Al Drago/CQ Roll Call

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 1 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 to treat very rare diseases.

Go deeperNov 21, 2019

Caitlin Owens

Global health regulators testing carcinogen levels of popular diabetes drug

Illustration: Eniola Odetunde/Axios

Health regulators around the world — including the FDA — are investigating whether metformin, a popular drug used to treat Type 2 diabetes, contains unsafe amounts of NDMA, a carcinogen, Bloomberg reports.

What they're saying: "The agency is in the beginning stages of testing metformin," an FDA spokesman told Bloomberg in a statement. "However, the agency has not confirmed if NDMA in metformin is above the acceptable daily intake (ADI) limit of 96 nanograms in the U.S."

Go deeperDec 5, 2019

Caitlin Owens

Researchers criticize $225,000 annual price of Pfizer's new heart-failure drug

Illustration: Rebecca Zisser/Axios

Some of the researchers who helped develop a new heart-failure drug are criticizing its high price, Bloomberg reports.

The intrigue: Pfizer, which makes the drug, says that it's targeted at a small population, justifying the high price. But critics say that the condition isn't that rare, setting up Pfizer to reap in a fortune from the medication.

Go deeperNov 20, 2019