The gene therapy can be given only once, due to the body’s natural immune reaction and production of antibodies against the modified virus it uses to deliver the gene to cells. In an R&D and Investor update last November, Novartis said that its “one-time, potentially curative” gene therapy could be cost-effective at a list price of $4 million to $5 million. If both Spinraza and Zolgensma are available, doctors, payers, patients, and their families will face tough medical, logistical, and economic decisions. “Because SMA is a rare disease and the trials have short-term follow-up, the long-term effects of Spinraza or Zolgensma will take time to emerge,” ICER stated. Both Spinraza and Zolgensma are high-priced drugs – with Biogen’s drug costing $750,000 in the first year and $375,000 thereafter at US wholesale prices, while Zolgensma’s one-time cost is $2.1m. More information on how to submit comments and what type of information would be most useful can be found here. Ask questions and share your knowledge of SMA in our forums. Recently, Biogen announced plans to launch a Phase 4 clinical trial, called RESPOND, evaluating the benefits of Spinraza in approximately 60 infants and children with SMA who were treated previously with Zolgensma. Zolgensma is designed to restore production of working and full-length SMN protein in motor neurons, which are specialized cells controlling muscle contraction, by delivering a functional copy of the SMN1 gene. We are sorry that this post was not useful for you!

This report will be subject to deliberation during a public meeting of the New England Comparative Effectiveness Public Advisory Council (CEPAC), on March 7 in Boston. Our clients with solve real health problems every... Communiqué Awards 2015: Winners in pictures, Roche’s Tecentriq/Avastin combination approved by European Commission, Roche builds case for SMA drug risdiplam as FDA verdict nears, Biogen proffers new data to support high-priced Spinraza, FDA approves Biogen's Spinraza for spinal muscular atrophy, Roche scores FDA approval for SMA drug risdiplam, now named Evrysdi, Lead Scientific Writer, IME/ CME, UK, circa £65K, Director of Scientific Services – Publications, Medical Communications agency, London/ South East UK – home/ office based, Home based Principal Medical Writer, Medical Communications, UK, FDA sets November date for expert panel review of Biogen’s aducanumab, Pfizer CEO pushes back COVID-19 vaccine submission to November, CureVac reveals early data for mRNA-based COVID-19 vaccine candidate, Russia submits COVID-19 vaccine to WHO for emergency use listing, Eldridge and Crump launch new consultancy to drive patient-centred care, UK agrees further COVID-19 vaccine deals with Pfizer/BioNTech, Valneva, Live Webinar: Improving Patient Care by Removing Barriers to Drug Accessibility, Overcoming Behavioral Health Access Limitations Through Telemedicine, OPEN VIE and Pharmerit Bring the Best of Both Worlds to Virtual ISPOR Europe 2020, EU agrees on supply deal for 200 million doses of Pfizer, BioNTech's COVID-19 vaccine, Envision Pharma Group acquires Two Labs to advance its position as a global strategic partner to pharma and create a unique and unrivaled launch excellence offering, Moderna preparing for interim analysis of late-stage COVID-19 vaccine data, All content copyright © PMGroup Worldwide Ltd 2020. Analyses of therapeutic efficacy were based on mortality, the need for permanent invasive ventilation, motor function, mobility, feeding support and other issues associated with SMA, as well as quality of life. The positive results of Zolgensma’s Phase 1 study were largely the basis of Novartis’ Biological License Application (BLA) to the U.S. Food and Drug Administration (FDA), seeking approval for Zolgensma — previously AVXS-101 — as a gene therapy for SMA type 1, the most common and severe form of the disease.

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… Policymakers will have the results of the Zolgensma versus Spinraza modeling to support their own judgment of value,” the report stated. However, SMA type 1 babies were about a month older in Spinraza’s ENDEAR (NCT02193074) and CHERISH (NCT02292537) trials than in Zolgensma’s pivotal Phase 1 study, ICER noted. In a Dec. 20 Reuters story, ICER chief medical officer David Rind noted that although a good value usually lies within $100,000 to $150,000 per QALY, it can go higher for rare diseases such as SMA. Interestingly, the two children receiving the gene therapy the latest (23 months, nearly 2 years) also showed asymptomatic low levels of platelets, which “could possibly be explained by a more “experienced” immune system posing a robust immune response,” the researchers wrote. “We therefore rate the evidence to be insufficient.” However, ICER noted that these conclusions may be updated in case additional safety and efficacy data become available. Four children were treated first with Spinraza and then with Zolgensma, and three of them continued Spinraza treatment after that. This represents a cost of $240,000 per QALY in patients with symptomatic type 1 SMA, ICER said. However, further studies are needed to better characterize combination therapies’ safety and determine whether it is more beneficial than single therapy, especially considering their high cost, researchers noted. SMA patients have a defective SMN1, leading to significantly lower levels of working SMN protein and loss of motor neurons, as well as muscle weakness and atrophy (shrinkage). The FDA is currently reviewing the BLA, specifically covering intravenous delivery in infants up to 9 months old. Be the first to rate this post. Further studies involving more patients are needed to better understand the effects of combination therapy in liver function and to “determine whether there are circumstances in which combination therapy would be more efficacious than either monotherapy,” the them concluded.

var arr2 = [ 'Roche', 'Risdiplam', 'Spinraza', 'Zolgensma', 'Spinal muscular atrophy' ]; After the combination therapy, all patients continued to show motor function improvements, as assessed by the Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders and the Hammersmith Infant Neurological Examination Part 2. Indeed, ICER addressed the number of patients treated with Zolgensma as a limitation for this cost-effectiveness comparison. It reviewed existing evidence while taking uncertainty and patient-centered considerations into account. “As in prior reports, we feel it is inappropriate for a therapy to appear cost-effective simply by offsetting costs of a recently introduced very expensive alternative. While treatment combination aiming at increasing or sustaining motor function improvements is likely to occur in these patients, there is limited data on its safety and effectiveness. Suite 354 The child who received Zolgensma after Spinraza experienced no side effects. The report is open to public comment until Jan. 31. In contrast, Zolgensma is administered directly into the bloodstream and delivers a functional copy of SMN1 to cells. Significant safety and longevity concerns still remain for Zolgensma, which may restrict its future use in the clinic. Patients, who were 17 to 29 months of age at last assessment, received first treatment between 1.5 and 7 months and second therapy between 9 and 23 months. Your email address will not be published. So far, Spinraza has far more data to support it. Email: [email protected] One issue with Zolgensma is the therapy is less effective in patients who have already been exposed to the virus used to deliver it, which becomes more common with age. Last month, a trial of Zolgensma involving the intrathecal injection route for SMA type 2 patients was halted after animal studies pointed to a possible risk with the drug. According to ICER’s preliminary estimate, Spinraza represents a cost of $728,000 per QALY (higher than Zolgensma’s) in presymptomatic patients. However, ICER noted that more must be known regarding Zolgensma’s U.S. price and both therapies’ long-term benefits. The data, which included children treated with Zolgensma at older ages than those reported in clinical trials, suggested that older patients may be at a higher risk of developing gene therapies’ known side effects, such as liver dysfunction and low platelet counts.

Novartis AG is confident that Zolgensma, its closely watched gene therapy for spinal muscular atrophy, will quickly take over from Biogen Inc.'s Spinraza as standard of care for the disease.

For the preliminary report, ICER, a nonprofit organization whose assessments increasingly influence medicines’ price negotiations in the U.S., spoke with patients and their families, clinicians, researchers, insurers, representatives from SMA patient advocacy groups, and the manufacturers of the two medicines.

Combining Spinraza (nusinersen) with the gene therapy Zolgensma (onasemnogene abeparvovec-xioi) is generally well-tolerated and sustains motor improvements in children with spinal muscular atrophy (SMA) type 1, according to a case series study. Risdiplam is a closer competitor to Spinraza in terms of its target age range, and could have one advantage in that it is an orally-active treatment, while Spinraza has to be injected into the spine (intrathecally). The report stated that both treatments provide substantial clinical benefit, as reflected in extended survival and improved motor function compared with SMA’s natural history or placebo. The assessment — announced last August — compared the clinical effectiveness and economic impact of Zolgensma and Spinraza versus supportive care for SMA patients of all ages and types. Novartis said it would like comparisons against an ultra-rare threshold of $500,000 per QALY and against Spinraza, according to company spokesman Eric Althoff. We use cookies to ensure that we give you the best experience on our website. The researchers suspected that this rise in liver enzymes was related mainly to Zolgensma and noted that the fast reintroduction of Spinraza after Zolgensma may result in cumulative liver toxicity.

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