Nov 21, 2019

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.

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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Biden formally secures Democratic presidential nomination

Joe Biden speaks at Delaware State University's student cente on June 5. Photo: Jim Watson/AFP via Getty Images

Former Vice President Joe Biden became the formal Democratic presidential nominee on Friday evening, per AP.

The big picture: Biden has been the presumptive frontrunner to take on President Trump since Sen. Bernie Sanders suspended his campaign in early April.

Updated 6 hours ago - Politics & Policy

Coronavirus dashboard

Illustration: Sarah Grillo/Axios

  1. Global: Total confirmed cases as of 9 p.m. ET: 6,724.516 — Total deaths: 394,018 — Total recoveries — 2,996,832Map.
  2. U.S.: Total confirmed cases as of 9 p.m. ET: 1,894,753 — Total deaths: 109,042 — Total recoveries: 491,706 — Total tested: 19,231,444Map.
  3. Public health: WHCA president says White House violated social-distancing guidelines to make reporters "a prop" — Jailing practices contribute to spread.
  4. Sports: How coronavirus could reshuffle the sports calendar.
  5. Jobs: Better-than-expected jobs report boosts stock market.
  6. Media: The Athletic lays off 8% of staff, implements company-wide pay cut.

Scoop: German foreign minister to travel to Israel with warning on annexation

Heiko Maas. Photo: Michael Kappeler/picture alliance via Getty Images

German Foreign Minister Heiko Maas is expected to travel to Israel next week to warn that there will be consequences if Israeli leaders move forward with plans to annex parts of the West Bank, Israeli officials and European diplomats tell me.

Why it matters: Israeli and European officials agree that if Israel goes ahead with unilateral annexation, the EU will respond with sanctions.