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 2 hours ago - Politics & Policy

Coronavirus dashboard

Illustration: Aïda Amer/Axios

  1. Global: Total confirmed cases as of 11:30 a.m. ET: 11,304,534 — Total deaths: 531,659 — Total recoveries — 6,111,195Map.
  2. U.S.: Total confirmed cases as of 11:30 a.m. ET: 2,841,124 — Total deaths: 129,689 — Total recoveries: 894,325 — Total tested: 34,858,427Map.
  3. States: Photos of America's pandemic July 4 ICU beds in Arizona hot spot near capacity.
  4. Public health: U.S. coronavirus infections hit record highs for 3 straight days.
  5. Politics: Trump extends PPP application deadlineNew Jersey gov. wants national face mask requirement
  6. World: Mexican leaders call for tighter border control as infections rise in U.S.
  7. Sports: 31 MLB players test positive as workouts resume.
  8. 1 📽 thing: Drive-in movie theaters are making a comeback.
3 hours ago - Sports

Sports return stalked by coronavirus

Tampa Bay Rays left fielder Austin Meadows bumps elbows Friday during a workout at Tropicana Field. Photo: Kim Klement/USA Today Sports via Reuters

When MLB teams arrived at the ballpark this weekend for the first summer workouts of 2020, the comforting sounds of baseball brought smiles to players' faces.

Between the lines: Even the loudest crack of the bat couldn't mask the eerie silence or distract from the ever-present coronavirus threat.

4 hours ago - Health

239 scientists call on WHO to recognize coronavirus as airborne

People walk at the boardwalk in Venice Beach. Photo: Apu Gomes/AFP via Getty Images

A group of 239 scientists in 32 countries is calling for the World Health Organization to revise its recommendations to account for airborne transmission as a significant factor in how the coronavirus spreads, the New York Times reports.

The big picture: The WHO has said the virus mainly spreads via large respiratory droplets that fall to the ground once they've been discharged in coughs and sneezes. But the scientists say evidence shows the virus can spread from smaller particles that linger in air indoors.