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The FDA on Friday approved Zolgensma, a gene replacement therapy from 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 the $2.1 million, 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. Vaccines: U.S. ahead of pace on vaccines.
  2. Health: Lessons for trapping the next pandemic.
  3. Tech: "Fludemic" model accurately maps COVID hotspotsVirtual doctor's visits and digital health tools take off.
  4. Politics: Harris breaks tie as Senate proceeds with lengthy debate on COVID relief bill — Republican governor of West Virginia says there's no plan to lift mask mandate.
  5. World: Canada vaccine panel recommends 4 months between doses — In AstraZeneca spat, EU fights hard for a vaccine its hardly using.
Dave Lawler, author of World
2 hours ago - World

In AstraZeneca spat, EU fights hard for a vaccine it's hardly using

Macron, Merkel and European Council President Charles Michel (R) at a summit in October. Photo: Yves Herman/Pool/AFP via Getty

Italy on Thursday blocked the export of 250,000 AstraZeneca doses to Australia, becoming the first EU country to exercise an export ban due to a vaccine shortfall in the bloc.

Why it matters: The controversial step exposes multiple major challenges to distributing vaccines — even among the world’s richest countries.

Dave Lawler, author of World
2 hours ago - World

Global freedom continues steady decline: report

The global erosion of democracy has continued for a 15th consecutive year, according to an annual report from Freedom House.

Zoom in: The report calls particular attention to India, which slipped from “free” to “partly free” due to the government's “scapegoating of Muslims” and “crackdown on critics.” Prime Minister Narendra Modi is, according to the report, “driving India itself toward authoritarianism.”

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