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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.

Go deeper

Alayna Treene, author of Sneak Peek
28 mins ago - Politics & Policy

Minority Mitch still setting Senate agenda

Illustration: Aïda Amer/Axios

Chuck Schumer may be majority leader, yet in many ways, Mitch McConnell is still running the Senate show — and his counterpart is about done with it.

Why it matters: McConnell rolled over Democrats unapologetically, and kept tight control over his fellow Republicans, while in the majority. But he's showing equal skill as minority leader, using political jiujitsu to convert a perceived weakness into strength.

Go deeper (2 min. read)Arrow
Axios
Updated 30 mins ago - Politics & Policy

Coronavirus dashboard

Illustration: Eniola Odetunde/Axios

  1. Health: Pandemic fatigue quantified — Coronavirus could be worsening childhood obesity.
  2. Vaccine: Biden floats prospect of 1.5 million vaccinations a day — Moderna says vaccine appears to protect against new COVID-19 variants.
  3. Politics: Deborah Birx went "on the road" for local COVID efforts to avoid White House censorship — Sanders says Democrats will push coronavirus relief package through with simple majority.
  4. Business: Budweiser skips Super Bowl ads in favor of vaccine education campaign.
  5. World: First U.S. case of COVID-19 variant from Brazil confirmed in Minnesota — Mexican President López Obrador tests positive for coronavirus.
  6. 🎧 Podcast: Google's chief health officer Karen DeSalvo on vaccinating America.
Go deeper (1 min. read)Arrow
Hans Nichols, Alayna Treene
31 mins ago - Politics & Policy

Schumer rattles reconciliation saber

More than an aisle separates Mitch McConnell and Chuck Schumer, seen in the Senate Chamber after the Capitol siege. Photo: Drew Angerer/Getty Images

Chuck Schumer is expected to telegraph, as soon as tonight, that he will use his political muscle to pass some of his party’s priorities — like President Biden's $1.9 trillion stimulus package.

Why it matters: While the Senate majority leader wants to work with Republicans on key legislation, advisers say, he will make clear that using the simple majority vote inherent in the budget reconciliation process is one of the big sticks at his disposal.

Go deeper (1 min. read)Arrow