Zolgensma vs. Spinraza. $2.15m? Copyright © 2013-2020 All rights reserved. The decision by the National Institute for Health and Care Excellence (NICE) comes as a boost to the Swiss drugmaker, which had come under regulatory scrutiny for data discrepancy issues related to its $2 million gene therapy, Zolgensma. Our Standards: The Thomson Reuters Trust Principles.

It is used as a one-time injection into a vein with at least two months of corticosteroids. “We are immensely excited about the prospects of a gene therapy for SMA. That’s an even more acute question for the NHS. Rucinski told SMA News Today that a large proportion of Europeans still have no access to Spinraza, and that four of the EU’s 28 member states — Bulgaria, Estonia, Ireland, and Latvia — still have not agreed to make the therapy available through public healthcare. Patryk Marczuk, scientific information coordinator of the Polish patient advocacy group Fundacja SMA, says that theoretically at least, Zolengsma is “everyone’s dream come true.” Yet he has doubts over both the therapy’s long-term effectiveness and EU governments’ willingness to pay for it. That’s the staggering price of a drug produced by the pharmaceutical giant Novartis that has just come on the market. Zolgensma illuminates the astonishing medical breakthroughs now possible – and asks profound questions about the economic and social systems within which medical research and healthcare are embedded. “Zolgensma is far more expensive, but if you look 10 years ahead and compare Spinraza versus a one-time gene therapy, it’s a no-brainer what will end up being cheaper over time,” he said. No one has ever proven that gene therapy would last for 10 years.

It also costs $2.15 million a treatment which is, I … “SMA Europe would regard it as unethical if the price would preclude any patient in any country from receiving this potentially lifesaving therapy.”. The only other FDA-approved treatment for SMA is Spinraza (nusinersen), an injectable medication developed by Biogen. “In Europe, the approval was for the entire spectrum of SMA, but Biogen did not have supporting data. The case of Zolgensma raises questions, too, about priorities. We are excited to share that the National Institute of Health and Care Excellence has begun the appraisal process of Zolgensma, the first gene therapy in SMA.

That’s the. “The issue with many of these drugs is that they are expensive but also life-changing for those who need them,” observes Michael Sherman, chief medical officer of the US insurance company Harvard Pilgrim Health Care. She found out about Zolgensma, ... per patient per year.

Wales and Northern Ireland … Particularly so as more than 60 gene therapies are expected on the market by 2024. To see the exact status of Spinraza access in each country, please click here for a detailed map.

We use cookies to ensure that we give you the best experience on our website. Novartis’ Zolgensma was approved by the US Food and Drug Administration (FDA) in May 2019 for treatment of spinal muscular atrophy (SMA) in pediatric patients less than two years of age. Does Zolgensma, a revolutionary one-off treatment for spinal muscular atrophy, really need to cost so much? The NICE committee met on 8th October 2020 and is scheduled to report on 25th March 2021. “We have not shared any further information beyond that time frame.”. “In many ways, the introduction of Zolgensma offsets those costs of treating someone with SMA,” Dave Lennon, president of AveXis, told POLITICO.

Does Zolgensma, a revolutionary one-off treatment for spinal muscular atrophy, really need to cost so much?

... 5 What a Joe Biden win would mean for the UK October 30, 2020 7:57 pm Advertisement. Tagged AveXis, Biogen, Centogene, European Medicines Agency, Fundacja SMA, gene therapy, NICE, Novartis, SMA Europe, Spinal Muscular Atrophy UK, Spinraza (nusinersen), Zolgensma. We are sorry that this post was not useful for you!

We never use your cookies for creepy ad retargeting that follows you around the web. Zolgensma (onasemnogene abeparvovec-xioi) is being evaluated under an accelerated assessment, which slashes the time frame for the review from 210 to 150 days.

In 1983, the US Congress passed the Orphan Drug Act, designed to encourage research into rare diseases, by offering tax breaks and a lower bar for market approval. How much is a life worth? Be the first to rate this post. It now anxiously awaits a decision from the EMA and further development of the intrathecal formulation to address the needs of older patients,” it stated in a May 24 note on its website. That fate was something Matilde’s mom couldn’t accept. Novartis has been expanding into gene and cell therapies for rare diseases and licensed Luxturna last year, for $105 million upfront, up to another $65 million in milestone payments, and royalties on sales.

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