FDA announces BLA for Zolgensma may contain manipulated data

Senator Elizabeth Warren , Senator Bernie Sanders , Senate Minority Leader Chuck Schumer , Senator Tammy Baldwin

Senator Elizabeth Warren , Senator Bernie Sanders , Senate Minority Leader Chuck Schumer , Senator Tammy Baldwin

The Food and Drug Administration had revealed Tuesday that some data from a potency assay used to test Zolgensma (onasemnogene abeparvovec-xioi) in mice had been manipulated.

The concerns are limited to a small portion of the product testing data that was used to support the development of its production process for the product, the FDA said. The FDA is now investigating the situation and if this does reveal compromised data from human trials, ICER could update its pricing of the novel gene therapy.

"We made the decision to progress our quality investigation prior to informing FDA and other regulatory authorities so that we could provide the best information and technical analysis, which we did promptly on completion on June 28", Novartis Chief Executive Vasant Narasimhan said on a conference call with analysts.

"The assays in question were used for initial product testing and are not now used for commercial product release".

The data manipulation issue is also the reason why the European Medicines Agency dropped Zolgensma from accelerated assessment, Lennon said, stating that the volume of questions the company had received and the time needed to address them meant Zolgensma would lose accelerated assessment.

"We maintain that the totality of the evidence demonstrating the product's effectiveness and its safety profile continue to provide compelling evidence supporting an overall favorable benefit-risk profile", Novartis said in an emailed statement, CBS News reported.

The integrity of the product testing data used in the development of the product's manufacturing process is "still a matter that we are continuing to evaluate and take very seriously", Marks said. "We remain fully capable of releasing high-quality, fully compliant Zolgensma to patients in need" (Johnson, AP/New York Times, 8/6; Thomas, New York Times, 8/6; McGinley, Washington Post, 8/6; Thomas, New York Times, 8/7; Sagonowsky, FiercePharma, 8/7).

Salman Khan & Madhuri Dixit celebrated 25th anniversary of 'Hum Aapke Hain Koun.!'
Salman Khan and Madhuri Dixit Nene's Hum Aapke Hain Koun .! continues to be one of the most loved Bollywood films of all time. Madhuri Dixit looked gorgeous at the screening of Hum Aapke Hain Koun . "Nisha will always hold a special place in my heart".

"First and foremost, we are fully confident in the safety, quality and efficacy of Zolgensma", AveXis said.

Zolgensma was approved in May as a treatment for children younger than two years old who have been diagnosed with SMA with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene. SMA is a leading genetic cause of infant mortality.

The FDA recently inspected Zolgensma's production process, Marks said, and information gathered will be part of a "thorough assessment" now underway.

Novartis in a statement said that once AveXis learned of the data manipulation, the company immediately began to investigate, and once it had "interim conclusions", it shared them with FDA. He also said the therapy will be "life-changing" for the patients, as well as their families. The disease can lead to death.

Marks said "AveXis became aware of the issue of the data manipulation that created inaccuracies in their [biologics license application] before the FDA approved the product, yet did not inform the FDA until after the product was approved". "We understand the agency has a different perspective which we can respect", he said.

Those test results are "not now used for commercial product release", AveXis said in a statement, adding it informed the FDA of the manipulation once it and Novartis had "interim conclusions from our investigations".

Recommended News

  • EU's Juncker tells Britain: no-deal Brexit will hurt you the most

    EU's Juncker tells Britain: no-deal Brexit will hurt you the most

    Jeremy Corbyn has urged a top civil servant to block Boris Johnson from pushing through a no-deal Brexit if an election is called. If the Government was to fall, an alternative Government could take power - either a Labour or a national unity administration.
    Airline hits back over 'backless seat' photo

    Airline hits back over 'backless seat' photo

    Replying to the interaction, one social media user joked: "I'm sorry you found evidence of our negligence". However, the airline says the image was misleading as the seats were out of order and nobody used them.
    You can now slow down or speed up Amazon Alexa

    You can now slow down or speed up Amazon Alexa

    It looks like the feature has only gone live in the USA so far - as we couldn't get a UK-based Alexa smart speaker to recreate it. If users ever hoped that Alexa spoke faster, then Amazon's announcement about the speed of Alexa's speech is a wish come true.
  • According to Infinity Ward, Call of Duty: Modern Warfare is not political

    According to Infinity Ward, Call of Duty: Modern Warfare is not political

    But you could have a game that takes place in revolutionary America talk about those exact same concepts". From there, Minkoff says their goal is to present different perspectives in the game.
    Sonia, Rahul Gandhi pull out of panels to choose Congress chief

    Sonia, Rahul Gandhi pull out of panels to choose Congress chief

    Approval of his resignation is still pending with the CWC, showing the obvious unwillingness of the party to look beyond a Gandhi. A week after the Congress' second worst loss in national elections, Rahul Gandhi resigned from the post of party president.
    Taylor Swift explains why she stayed quiet ahead of 2016 presidential election

    Taylor Swift explains why she stayed quiet ahead of 2016 presidential election

    Please enjoy this totally snake-free palette cleanser in the form of Anna Wintour ONCE AGAIN expressing her hatred of leggings. The reason her fans are big on using Tumblr instead of another social media platform is that it is the one Swift uses the most.
  • Eid Al-Adha 2019: History, significance, date and importance of Bakr-Id

    Eid Al-Adha 2019: History, significance, date and importance of Bakr-Id

    The statement reads: "The All Progressives Congress (APC) celebrates with Muslim faithfuls on the commemoration of Eid ul-Adha". God then gave Prophet Ibrahim the option to substitute his son for a lamb instead and continue performing his sacrifice.
    YouTuber Marina Joyce Is Found 10 Days After She Went Missing

    YouTuber Marina Joyce Is Found 10 Days After She Went Missing

    But fortunately, not even 24 hours after that public appeal, Joyce was reportedly located - and she's since been termed as safe. Police have launched an appeal to find British YouTuber Marina Joyce who has been missing for ten days.
    Spectacular weekend forecast with sunshine, low humidity

    Spectacular weekend forecast with sunshine, low humidity

    Today , it will be partly sunny with a high near 87 and an east wind of 5 miles per hour , the weather service said. Saturday night , spotty storms will be possible once again, mainly in north central and northeastern Kansas.
  • Cornwall picked in West Indies squad for India series

    Cornwall picked in West Indies squad for India series

    He has 260 wickets from 55 first-class matches and finished as the leading bowler in the most recent regional four-day tournament. Rahkeem Cornwall has received a surprise maiden Test call up for West Indies for their upcoming series against India .
    How major U.S.  stock indexes fared Wednesday

    How major U.S. stock indexes fared Wednesday

    The Nasdaq composite, which is heavily weighted with technology stocks, vaulted 176.33 points, or 2.2%, to 8,039.16. Global Investors, told ABC News, referring to larger-than-expected rate cuts by India, Thailand and New Zealand.
    Hamilton praises Mercedes strategy

    Hamilton praises Mercedes strategy "risk" in Budapest win

    Both drivers are starting to put in good performances, so they're there, and it is clearly a very good vehicle . But I came second and got fastest lap, so it was a good weekend.

We are pleased to provide this opportunity to share information, experiences and observations about what's in the news.
Some of the comments may be reprinted elsewhere in the site or in the newspaper.
Thank you for taking the time to offer your thoughts.