spinal muscular atrophy

NICE reaches a deal with Roche on access to oral SMA drug Evrysdi

Roche’s oral treatment Evrysdi for the rare genetic disease spinal muscular atrophy (SMA) will be made available on the NHS in England, after NICE reached a three-year access agreement with the company. The cost-effectiveness agency had turned down Evrysdi (risdiplam) in draft guidance published in June, saying it was too expensive, but after negotiations with …

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Roche builds evidence for Evrysdi’s benefits younger SMA patients

Roche has highlighted new data with its oral treatment for spinal muscular atrophy (SMA) – Evrysdi – showing its benefits when given to pre-symptomatic babies with the rare disease. The updated results come from the open-label RAINBOWFISH study, which investigate the effects of Evrysdi (risdiplam) in children from birth to six weeks who had yet …

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Novartis cleared to restart trials of new Zolgensma delivery route

The FDA has relaxed a clinical hold on clinical trials of Novartis’ intrathecal formulation of spinal muscular atrophy (SMA) gene therapy Zolgensma, allowing a new phase 3 trial to get underway. Zolgensma (onasemnogene abeparvovec) is already approved as an intravenous treatment in babies and very young children for the rare muscle-wasting disease, but the new …

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Roche Reports New Interim Data of JEWELFISH & RAINBOWFISH Studies for Evrysdi (risdiplam) to Treat Spinal Muscular Atrophy

Shots: The new interim data from two studies i.e JEWELFISH open-label study evaluating Evrysdi in people aged 1 to 60 yrs previously treated with another SMA-targeting therapy & RAINBOWFISH study evaluating Evrysdi in babies from birth to 6 wks. with pre-symptomatic SMA The JEWELFISH showed stabilization in motor function as measured by change from baseline …

Roche Reports New Interim Data of JEWELFISH & RAINBOWFISH Studies for Evrysdi (risdiplam) to Treat Spinal Muscular Atrophy Read More »

NICE ‘no’ to Roche’s risdiplam for SMA not unexpected, says patient group

NICE has rejected routine NHS funding for Roche’s Evrysdi (risdiplam) for spinal muscular atrophy drug in draft guidance, a decision which doesn’t come as a surprise to patient association SMA UK. The cost-effectiveness agency’s initial assessment is that Evrysdi is simply too expensive at its current price to be provided to the roughly 1,500 people …

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Roche’s Evrysdi (risdiplam) Receives Health Canada’s Approval for Spinal Muscular Atrophy in Adults and Children

Shots: The approval is based on two studies i.e. FIREFISH & SUNFISH. The FIREFISH study involves assessing Evrysdi in infants aged 2-7mos. with symptomatic Type 1 SMA, and the SUNFISH study assesses Evrysdi in children & young adults aged 2-25yrs. with Type 2 or 3 SMA Evrysdi demonstrated improvement in motor function, 29.3% of participants …

Roche’s Evrysdi (risdiplam) Receives Health Canada’s Approval for Spinal Muscular Atrophy in Adults and Children Read More »

Novartis says £1.8m Zolgensma is model for gene therapy pricing

Novartis has said its gene therapy pricing model, involving a steep one-off payment, could be a template for the hundreds of similar treatments that could follow its Zolgensma for Spinal Muscular Atrophy (SMA).  Zolgensma (onasemnogene abeparvovec) has a list price of £1,795,000 excluding VAT in the UK, but Novartis says the price is justified by …

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Chugai Reports NDA Submission of Risdiplam to the MHLW as the First Oral Drug for SMA in Japan

Shots: The NDA submission is based on FIREFISH study assessing Risdiplam in infants with symptomatic SMA Type 1 & SUNFISH study in children and young adults with SMA Type 2 or 3 FIRESISH study results: improvement in survival and motor milestones in infants. SUNFISH study results: improvement in motor function in people aged 2-25 with …

Chugai Reports NDA Submission of Risdiplam to the MHLW as the First Oral Drug for SMA in Japan Read More »

FDA’s Ok to Roche’s Oral SMA Therapy; Roche’s Etrolizumab Mixed Results in Ulcerative Colitis; Secura receives rights to Copiktra from Verastem

Roche Set to Compete With Biogen and Novartis in the SMA Market With its First Oral Therapy  The US FDA has recommended the use of Evrysdi (risdiplam), a candidate of Genentech, a Roche company, for spinal muscular atrophy (SMA), an inherited neuromuscular disease resulting in muscle atrophy, in adults and children two months of age …

FDA’s Ok to Roche’s Oral SMA Therapy; Roche’s Etrolizumab Mixed Results in Ulcerative Colitis; Secura receives rights to Copiktra from Verastem Read More »

Biogen to launch Phase IV study of spinal muscular atrophy drug in post-gene therapy patients

The company plans to enroll 60 patients who have received its drug, Spinraza, following suboptimal response to Novartis’ Zolgensma, which was approved last year. The trial, RESPOND, is anticipated to start enrolling in the first quarter of 2021, pending regulatory approval.