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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World coronavirus updates

Data: The Center for Systems Science and Engineering at Johns Hopkins; Map: Axios Visuals

The European Union is considering an $826 billion coronavirus rescue package to fund recovery efforts for all member states, European Commission President Ursula von der Leyen said Wednesday.

By the numbers: More than 5.6 million people have tested positive for COVID-19 and over 2.3 million have recovered from the virus. Over 355,500 people have died globally. The U.S. has reported the most cases in the world (over 1.6 million from 15.1 million tests).

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U.S. coronavirus updates

Data: The Center for Systems Science and Engineering at Johns Hopkins; Map: Andrew Witherspoon/Axios. This graphic includes "probable deaths" that New York City began reporting on April 14.

More than 100,000 Americans have died of the coronavirus, according to data from Johns Hopkins — a milestone that puts the death toll far beyond some of the most tragic events in U.S. history.

By the numbers: Over 1.6 million have tested positive in the U.S. Nearly 354,000 Americans have recovered and over 15.1 million tests have been conducted. California became the fourth state with at least 100,000 reported cases of the coronavirus on Wednesday, along with Illinois, New Jersey and New York.

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Coronavirus dashboard

Illustration: Sarah Grillo/Axios

  1. Global: Total confirmed cases as of 8 p.m. ET: 5,682,389 — Total deaths: 354,944 — Total recoveries — 2,337,385 — Map.
  2. U.S.: Total confirmed cases as of 8 p.m. ET: 1,697,459 — Total deaths: 100,276 — Total recoveries: 391,508 — Total tested: 15,192,481 — Map.
  3. Public health: Fauci says data is "really quite evident" against hydroxychloroquine — Nearly half of Americans say someone in their household has delayed medical care.
  4. States: California hospitals strained by patients in Mexico — Texas Supreme Court blocks mail-in expansion to state voters.
  5. Business: MGM plans to reopen major Las Vegas resorts in June — African American business owners have seen less relief from PPP, Goldman Sachs says.
  6. Tech: AI will help in the pandemic — but it might not be in time for this one.
  7. 1 🎶 thing: Local music venues get rocked by coronavirus.
  8. 🎧 Podcast: Trump vs. Twitter ... vs. Trump.
  9. What should I do? When you can be around others after contracting the coronavirus — Traveling, asthma, dishes, disinfectants and being contagious — Masks, lending books and self-isolating — Exercise, laundry, what counts as soap — Pets, moving and personal health — Answers about the virus from Axios experts — What to know about social distancing — How to minimize your risk.
  10. Other resources: CDC on how to avoid the virus, what to do if you get it, the right mask to wear.

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Updated 1 hour ago - Politics & Policy