May 24, 2019

FDA approves Novartis' gene therapy

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

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Making sense of the UN's climate conference coronavirus delay

Illustration: Sarah Grillo/Axios

The scuttling of November's pivotal UN climate conference is the starkest sign yet of how coronavirus is throwing a wrench into efforts to combat global warming. But like the wider relationship between the coronavirus and climate initiatives, the ramifications are ... complicated.

Driving the news: UN officials announced Wednesday that the annual summit to be held in Glasgow, Scotland, is postponed until some unknown time next year.

Coronavirus dashboard

Illustration: Aïda Amer/Axios

  1. Global: Total confirmed cases as of 9 a.m. ET: 952,171 — Total deaths: 48,320 — Total recoveries: 202,541Map.
  2. U.S.: Total confirmed cases as of 9 a.m. ET: 216,722 — Total deaths: 5,137 — Total recoveries: 8,672Map.
  3. Stimulus updates: Social Security recipients won't need to file a tax return to receive their checks.
  4. Jobs update: 6.6 million people filed for unemployment last week, a staggering number that eclipses the record set on March 26.
  5. Health updates: The Trump administration won't reopen enrollment for ACA marketplaces this year.
  6. National updates: The Grand Canyon closed after a resident tested positive for coronavirus.
  7. World update: Israeli Prime Minister Benjamin Netanyahu re-entered self-quarantine after his health minister tested positive for coronavirus.
  8. What should I do? Answers about the virus from Axios expertsWhat to know about social distancingQ&A: Minimizing your coronavirus risk.
  9. Other resources: CDC on how to avoid the virus, what to do if you get it.

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The weirdest NBA draft ever

Table: Axios Visuals

The 2020 NBA draft was already shaping up to be the weirdest draft in years, and now that the coronavirus pandemic has disrupted the sports world, it could be the weirdest draft ever.

Why it matters: While most drafts have a clear hierarchy by the time April rolls around, this draft does not. There's no reliable No. 1 pick, almost every top-10 prospect has a glaring weakness and the global sports hiatus has shrouded the whole class in mystery.

Go deeperArrow40 mins ago - Sports