Nov 21, 2019

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

Axios

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.

What they're saying: Alnylam CEO John Maraganore told Bloomberg that the company will adjust the drug's price based on its results.

  • "If patients respond well, we will be fully paid. If they don't have an acceptable response to the drug, we will give a rebate back to the payer," he said.

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Bob Herman

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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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 deeperArrowNov 20, 2019
Caitlin Owens

CRISPR-based gene editing treatments benefit patients with blood disorders

A researcher observes a CRISPR/Cas9 process through a stereomicroscope. Photo: Gregor Fischer/picture alliance via Getty Images

The first two patients to receive CRISPR-based treatments for their blood disorders benefited from the therapy, the two companies developing the treatment announced yesterday — a sign that gene editing may be a safe and effective way to cure blood diseases, STAT reports.

Why it matters: The two blood disorders, sickle cell disease and beta thalassemia, can have a drastic impact on patients' lives. A cure would be transformative.

Go deeperArrowNov 20, 2019