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  • At $2.1 million, Novartis gene therapy for SMA will be world’s most expensive

    Posted by Jean-Pierre Le Rouzic on May 25, 2019 at 1:24 am

    A good summary about the business aspect is available in Statnews and for patients there is a good article in Smanewstoday.

    Why it matters to pALS? Well SMA is a disease with some similarities with ALS, but when ALS strikes older people, SMA strikes children or even toddlers.

    It matters also because Zolgensma is very effective (more than Spinraza) and if the same kind of therapy was used against TARDPB, the gene that secretes the protein TDP-43, it would be applicable to 95% of the ALS cases and one third of the Alzheimer cases.

    It matters also because the price asked by Novartis is incredibly high, so we could expect the same for an ALS genetic therapy. Zolgensma was not developped by Novartis, it was developped under the name AVXS-101 by AvXis a R&D biotech that Novartis bought recently (the main researcher left). I estimate the cost of a generic version of this treatment to be a few thousand Dollars at most.

    I made a few months ago a plea for a genetic therapy for ALS based on Zolgensma but aiming at TDP-43. Some scientists answered but no pharmaceutical companies replied. I hope that someone in Cuba/China/Iran will develop a low cost gene therapy for ALS.

    Dagmar replied 5 years ago 2 Members · 1 Reply
  • 1 Reply
  • Dagmar

    May 25, 2019 at 3:11 pm

    Thank you for sharing this news Jean-Pierre!

    The SMA article link is very helpful in explaining the “what/why/how” of this breakthrough treatment for SMA.

    ALS treatments are so focused on symptom management. Gene therapy will get to the pre-ALS and post-ALS corrective phase.

    Yes, we hope a scientist out there has interest in the applications for ALS – – and a low cost one as well.

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