May 28, 2019

The most expensive drug in the world

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

FEC commissioner fact-checks Trump's voter fraud claims

Federal Election Commission Ellen Weintraub during a committee hearing in the Capitol in 2017. Photo: Joe Raedle/Getty Images

Federal Election Commission commissioner Ellen Weintraub posted an extensive fact-checking thread to Twitter late Wednesday refuting claims by President Trump and some Republicans that mail-in voting can lead to fraud.

Why it matters: Weintraub weighed in after Trump threatened to take action against Twitter for fact-checking him on his earlier unsubstantiated posts claiming mail-in ballots in November's election would be fraudulent, and she directly addressed Twitter's fact-checkin of the president in her post.

China approves Hong Kong national security law

Hong Kong riot police round up a group of protesters during a demonstration on Wednesday. Photo: Willie Siau/SOPA Images/LightRocket via Getty Images

Chinese lawmakers approved a plan on Thursday for a sweeping national security law for Hong Kong that would criminalize sedition, foreign influence and secession in the Asian financial hub.

Why it matters: China bypassed Hong Kong's legislature and chief executive to introduce the law, prompting Secretary of State Mike Pompeo to announce Wednesday that the city is no longer autonomous from the Chinese mainland and does not warrant special treatment under U.S. law.

Go deeper (1 min. read)ArrowUpdated 39 mins ago - World

Minneapolis unrest as hundreds protest death of George Floyd

Protesters and police clash during demonstration on Wednesday over the death of George Floyd in custody outside the Third Police Precinct. Photo: Kerem Yucel/AFP via Getty Images

A man died in a Minneapolis shooting during a second night of clashes between police and protesters in the city over the death of George Floyd, an African American man who died in police custody, per AP.

The latest: Police said officers were responding to reports of a stabbing just before 9:30 p.m. and found a man lying in "grave condition on the sidewalk" with a gunshot wound, CBS Minnesota reports. On man is in custody over the incident.