Novartis headquarters in Switzerland. Photo: Fabrice Coffrini/AFP via Getty Images

The FDA has approved Zolgensma, a gene replacement therapy from pharmaceutical company Novartis that treats spinal muscular atrophy, for use in children younger than 2.

Why it matters: The treatment attacks a debilitating genetic disease that often kills infants, and it will come with a price tag of more than $2.1 million, making Zolgensma the most expensive drug on the planet.

Details: Novartis said it will allow health insurance companies to pay for Zolgensma's $2.1 million price, which does not factor in potential rebates or discounts, over 5 years.

  • That puts Zolgensma's annual list price at $425,000, which Novartis said is less expensive than Spinraza, a competing therapy for spinal muscular atrophy made by Biogen.

Between the lines: The Institute for Clinical and Economic Review, a group that evaluates drug pricing and effectiveness, said in a statement that an appropriate all-in price range for Zolgensma would be between $1.1 million and $1.9 million — below what Novartis set. Other ICER estimates say the price should be even lower, between $310,000 and $900,000.

  • Novartis acquired AveXis, the biotech firm that developed this gene therapy, for $8.7 billion last year, so investors want a return for that investment.

The bottom line: Zolgensma is emblematic of the new scientific advances that treat people with crippling diseases and of the debate society will have over how it should pay for these types of therapies.

Go deeper: The drug pricing debate is stuck in the past

Go deeper

Updated 26 mins ago - Politics & Policy

Coronavirus dashboard

Illustration: Sarah Grillo/Axios

  1. Global: Total confirmed cases as of 8 p.m. ET: 33,495,373 — Total deaths: 1,004,314 — Total recoveries: 23,259,632Map.
  2. U.S.: Total confirmed cases as of 8 p.m ET: 7,186,527 — Total deaths: 205,895 — Total recoveries: 2,809,674 — Total tests: 103,155,189Map.
  3. Health: Americans won't take Trump's word on the vaccine, Axios-Ipsos poll finds.
  4. States: NYC's coronavirus positivity rate spikes to highest since June.
  5. Sports: Tennessee Titans close facility amid NFL's first coronavirus outbreak.
  6. World: U.K. beats previous record for new coronavirus cases.
  7. Work: United States of burnout — Asian American unemployment spikes amid pandemic

Misinformation thrives on social media ahead of presidential debate

Joe Biden speaking in Wilmington, Delaware, on Sept. 27. Photo: Roberto Schmidt/AFP via Getty Images

A baseless conspiracy theory that Joe Biden would wear an electronic device in his ear during the first presidential debate on Tuesday went viral on social media hours before the event.

Why it matters: The conspiracy originated on social media before appearing in a text message sent by President Trump’s re-election campaign to supporters. It was then regurgitated by media outlets like Fox News and New York Post, who cited the Trump campaign, throughout the day, according to NBC News.

Amy Coney Barrett says Trump offered her nomination 3 days after Ginsburg's death

Barrett speaks after being nominated to the US Supreme Court by President Trump in the Rose Garden of the White House. Photo:; Olivier Douliery/AFP

Amy Coney Barrett said in a questionnaire released by the Senate Judiciary Committee Tuesday that President Trump offered her the Supreme Court nomination on Sept. 21, five days before he announced the pick to the public.

Why it matters: According to the questionnaire, Trump offered Barrett the nomination just three days after Ruth Bader Ginsburg died, suggesting that the president knew early on that Barrett was his pick. Minutes after offering Barrett the nomination, however, Trump told reporters that he had not made up his mind and that five women were on the shortlist.