Treatment with Zolgensma® (onasemnogene abeparvovec) resulted in rapid and sustained improvement in motor function in spinal muscular atrophy (SMA) patients. “Our approach in using IV early in infants is to try to replicate the biological situation by giving high levels of SMN expression throughout the body,” Lennon said.
Other infants who received Zolgensma in both a U.S. study and European study achieved other treatment-related developmental milestones that will be discussed at the conference. AAN data demonstrate efficacy of Zolgensma® across broad spectrum of SMA Presymptomatic Ph 3, open-label, single-arm, multi-center trial to evaluate safety and efficacy of IV Zolgensma® in pre-symptomatic SMA patients with 2 or 3 copies of SMN2 <6 weeks Type 1 Ph 3, open-label, single-arm, single-dose, multi-center trial to evaluate efficacy and The cookie is used to remember the user consent for the cookies under the category "Performance". All rights reserved. Suite 354 His work has ranged from the association of central cardiovascular and pain control to the neurobiological basis of hypertension, and the molecular pathways driving Alzheimer’s disease. If you continue to use this site we will assume that you are happy with it. It boosts the ability of a related gene — called SMN2 — to generate full-length and stable SMN protein. Website development by e-Motive Media Limited. Zolgensma uses a genetically modified virus, called AAV9, to deliver a normal copy of SMN1 to motor neurons, allowing for healthy SMN protein production. Overall, no new safety signals have been identified and the reported adverse events are consistent with the cumulative safety profile with Zolgensma. But opting out of some of these cookies may have an effect on your browsing experience. On Thursday, AveXis presented new data from two Phase III trials, the SPR1NT trial in pre-symptomatic patients as well as interim data from the ongoing Phase III STR1VE clinical program for Zolgensma that showed positive outcomes demonstrating age‑appropriate major milestone gain with pre‑symptomatic treatment and prolonged event-free survival in patients with SMA Type 1.
While the approval of AveXis’ gene therapy for spinal muscular atrophy has been tainted by the revelation of early data manipulation, the Novartis subsidiary presented some long-term data at a conference that backs up the treatment’s efficacy. Because the potential for additional motor gains with Spinraza is still “an open question” in the SMA community, however, “I’m sure we’ll continue to look at how we might be able to study if Spinraza can add benefit,” Lennon concluded. The cookie is used to store and identify a users' unique session ID for the purpose of managing user session on the website. This cookie is set by YouTube and registers a unique ID for tracking users based on their geographical location. Olga Santiago, AveXis’ chief medical officer, said the data being presented at EPNS “demonstrate the life-changing impact Zolgensma can have on children with SMA Type 1.”.
The report also indicates 65.6 percent achieved motor milestones not observed in the natural history of SMA Type 1. This session cookie is served by our membership/subscription system and controls whether you are able to see content which is only available to logged in users.
The results also found that 32 of 33 patients were reported to have at least one adverse event (AE), of which six patients experienced serious adverse events that were related to Zolgensma. These cookies will be stored in your browser only with your consent. One patient discontinued the study because of a serious adverse event – hypoxic-ischemic brain damage and respiratory distress that resulted in death; however, this was considered to be unrelated to treatment with Zolgensma based on autopsy findings. There was one death in the European arm of the STR1VE trial. “We are seeing further evidence of the potential of Zolgensma to effectively halt motor neuron loss… In STR1VE-EU, patients achieved rapid improvements in motor function following treatment with Zolgensma and most have already achieved motor milestones not observed in the natural history of SMA Type 1,” said Professor Dr Eugenio Mercuri, Department of Pediatric Neurology, Catholic University, Italy.
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“We are seeing strong efficacy … very rapidly.
Data from these two trials, as well as long-term follow-up data to the Phase I START study will be presented at the 2019 European Pediatric Neurology Society (EPNS) Congress. According to the autopsy report, the cause of death was ruled as hypoxic-ischemic brain damage and respiratory distress. These were considered unrelated to the gene therapy by the investigator. And a three-point [increase] is considered clinically meaningful,” Lennon said. STR1VE-EU is designed to evaluate the efficacy and safety of a single, one-time IV infusion of Zolgensma in patients with SMA Type 1 who are less than six months of age, with one or two copies of the SMN2 backup gene and who have bi-allelic SMN1 gene deletion or point mutations. This cookie is set by GDPR Cookie Consent WordPress Plugin. “We’re very confident that Zolgensma can have benefit certainly for type 2 patients and, by extrapolation, potentially for type 3 patients also,” Lennon said. “What we’re seeing very consistently is efficacy in type 1 patients is replicated in efficacy in type 2 and efficacy in type 3 patients,” David Lennon, president of AveXis — the Novartis-owned company that developed Zolgensma said in a phone interview with SMA News Today.. You can adjust your cookie and associated data processing preferences at any time via our "Cookie Settings". When you press play, Vimeo will drop third party cookies to enable the video to play and to see how long a viewer has watched the video. The cookie is set by GDPR cookie consent to record the user consent for the cookies in the category "Advertising & Targeting". Positive outcomes here will inform a further trial, to be called REACH, planned for type 1 to 3 patients up to age 18. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. This website uses cookies to enable, optimise and analyse site operations, as well as to provide personalised content and allow you to connect to social media. It is used by cache busting in Advanced Ads Pro when the appropriate visitor conditions are used. Spinal muscular atrophy is a rare genetic disease that leads to progressive muscle weakness and paralysis. ZOLGENSMA ® (onasemnogene abeparvovec-xioi) is a prescription gene therapy used to treat children less than 2 years old with spinal muscular atrophy (SMA). “With more than 600 patients now treated, including some more than five years post-treatment and more than five years old, these data further reinforce the transformative benefit a one-time dose of Zolgensma has on SMA patients.”. Free membership: sign up today to access all of our exclusive content, Webinar now available on-demand from Thermo Fisher Scientific – ‘Delivering value: how LIMS can enable data integrity in pharma manufacturing’.
With the U.S. Food and Drug Administration (FDA) expected to soon decide whether Zolgensma (onasemnogene abeparvovec) can treat people with spinal muscular atrophy (SMA) type 1, clinical trials of the gene therapy are showing rapid benefits across disease types. ‘Pharmacopoeia changes – what to check for on UV/Vis spectrometers’.
It does not provide medical advice, diagnosis or treatment. Likewise, 24-month results from the START long-term extension study (NCT03421977) showed no loss of motor function in 10 patients given proposed therapeutic high dose in the pivotal Phase 1 trial. Many of the infant patients also gained greater muscle control. Early and positive results at one year of follow-up were presented for a first time at the AAN meeting. All subscriptions include online membership, giving you access to the journal and exclusive content. This cookie does not track individuals.
The cookie is used to remember the user consent for the cookies under the category "Analytics". Clinical Trials, Drug Safety, Gene therapy, Regulation & Legislation, Therapeutics, Novartis, US Food and Drug Administration (FDA), Dr Shephard Mpofu, Professor Dr Eugenio Mercuri, By Hannah Balfour (European Pharmaceutical Review).
To confirm that Zolgensma boosts levels, investigators assessed protein production in the brain, spinal cord, and peripheral organs of an infant who had died in the Phase 3 trial, STR1VE (NCT03306277), of respiratory failure considered unrelated to treatment. This session cookie is served by our membership/subscription system and controls which types of content you are able to access. “It is critical to diagnose SMA and begin treatment as early as possible in order to stop irreversible motor neuron loss and make the achievement of major motor milestones such as crawling, sitting and walking a possibility,” Santiago said in a statement. At the close of the 24-month study, all 12 patients in the cohort that received a targeted therapeutic dose were alive and free of permanent ventilation.
The cookie is a session cookies and is deleted when all the browser windows are closed. Have you registered for our Charles River webinar on 15 October at 15.00 BST to learn more on ‘The analytical reality of recombinant endotoxin technologies’? … In our minds, there’s not a great story yet to suggest there’s added benefit from putting these therapies on top of each other.”. The mean Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) score at baseline was 28. It targets the genetic root cause of SMA with a one-time-only dose and replaces the function of the missing or nonworking survival motor neuron 1 (SMN1) gene with a new, working copy of a human …
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