FDA OKs First Oral Treatment for Spinal Muscular Atrophy FDA OKs First Oral Treatment for Spinal Muscular Atrophy
The FDA has approved risdiplam (Evrysdi) as the first oral treatment for adults and children at least 2 months of age with spinal muscular atrophy.FDA Approvals (Source: Medscape Neurology and Neurosurgery Headlines)
Source: Medscape Neurology and Neurosurgery Headlines - August 7, 2020 Category: Neurology Tags: Neurology & Neurosurgery News Alert Source Type: news
Nusinersen with screening for spinal muscular atrophy cost effective?
(Source: PharmacoEconomics and Outcomes News)
Source: PharmacoEconomics and Outcomes News - June 30, 2020 Category: Drugs & Pharmacology Source Type: news
Roche announces 2-year risdiplam data from SUNFISH and new data from JEWELFISH in infants, children and adults with spinal muscular atrophy (SMA)
Basel, 12 June 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today presented two-year data from Part 1 of its pivotal SUNFISH trial in people aged 2-25 years with Type 2 or 3 spinal muscular atrophy (SMA) at the virtual Cure SMA Annual Conference, 8-12 June, 2020. The results of an exploratory efficacy analysis show risdiplam significantly improved motor function after 24 months of treatment compared to natural history data. In addition, preliminary 12 month data from JEWELFISH, a trial in people with all types of SMA aged 6 months to 60 years previously treated with other SMA therapies, showed that treatment with risdiplam le...
Source: Roche Media News - June 12, 2020 Category: Pharmaceuticals Source Type: news
Roche announces 2-year risdiplam data from SUNFISH and new data from JEWELFISH in infants, children and adults with spinal muscular atrophy (SMA)
Basel, 12 June 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today presented two-year data from Part 1 of its pivotal SUNFISH trial in people aged 2-25 years with Type 2 or 3 spinal muscular atrophy (SMA) at the virtual Cure SMA Annual Conference, 8-12 June, 2020. The results of an exploratory efficacy analysis show risdiplam significantly improved motor function after 24 months of treatment compared to natural history data. In addition, preliminary 12 month data from JEWELFISH, a trial in people with all types of SMA aged 6 months to 60 years previously treated with other SMA therapies, showed that treatment with risdiplam le...
Source: Roche Investor Update - June 12, 2020 Category: Pharmaceuticals Source Type: news
Treat early or wait? Experts ponder best way to manage milder forms of spinal muscular atrophy
(IOS Press) The advent of therapeutic interventions for spinal muscular atrophy (SMA) has increased the importance of presymptomatic diagnosis and treatment. When to start treatment in children with less severe disease remains controversial. Now, in a report published in the Journal of Neuromuscular Diseases, German researchers argue for an earlier start of treatment to prevent permanent nerve damage, challenging recommendations originally proposed by a group of American experts that suggests a strict follow-up strategy for children expected to have less severe disease. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - June 11, 2020 Category: International Medicine & Public Health Source Type: news
New longer-term data reinforce safety of Roche ’s satralizumab in adults and adolescents with neuromyelitis optica spectrum disorder
Basel, 22 May 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) will present new pooled pivotal satralizumab safety results for the treatment of neuromyelitis optica spectrum disorder (NMOSD), a rare, debilitating central nervous system disorder. These data are being presented at the 6th Annual Meeting of the European Academy of Neurology (EAN) and show satralizumab was well-tolerated in a broad patient population – including adolescents, for whom there is no approved medicine. Satralizumab is an investigational humanised monoclonal antibody that targets the interleukin-6 (IL-6) receptor, believed to play a key role in the infla...
Source: Roche Investor Update - May 22, 2020 Category: Pharmaceuticals Source Type: news
New longer-term data reinforce safety of Roche ’s satralizumab in adults and adolescents with neuromyelitis optica spectrum disorder
Basel, 22 May 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) will present new pooled pivotal satralizumab safety results for the treatment of neuromyelitis optica spectrum disorder (NMOSD), a rare, debilitating central nervous system disorder. These data are being presented at the 6th Annual Meeting of the European Academy of Neurology (EAN) and show satralizumab was well-tolerated in a broad patient population – including adolescents, for whom there is no approved medicine. Satralizumab is an investigational humanised monoclonal antibody that targets the interleukin-6 (IL-6) receptor, believed to play a key role in the infla...
Source: Roche Media News - May 22, 2020 Category: Pharmaceuticals Source Type: news
Roche ’s risdiplam shows significant improvement in survival and motor milestones in infants with Type 1 spinal muscular atrophy (SMA)
Basel, 28 April 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today presented 1-year data from FIREFISH Part 2, a pivotal global study evaluating risdiplam in infants aged 1 – 7 months old with symptomatic Type 1 spinal muscular atrophy (SMA). The study met its primary endpoint with 29% of infants (12/41; p<0.0001) sitting without support for five seconds by month 12, as assessed by the Gross Motor Scale of the Bayley Scales of Infant and Toddler Development Third Edition (BSID-III). No infants achieve this milestone in the natural history of Type 1 SMA. In addition, 18 (43.9%) infants were able to hold their head upright...
Source: Roche Media News - April 28, 2020 Category: Pharmaceuticals Source Type: news
Roche ’s risdiplam shows significant improvement in survival and motor milestones in infants with Type 1 spinal muscular atrophy (SMA)
Basel, 28 April 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today presented 1-year data from FIREFISH Part 2, a pivotal global study evaluating risdiplam in infants aged 1 – 7 months old with symptomatic Type 1 spinal muscular atrophy (SMA). The study met its primary endpoint with 29% of infants (12/41; p<0.0001) sitting without support for five seconds by month 12, as assessed by the Gross Motor Scale of the Bayley Scales of Infant and Toddler Development Third Edition (BSID-III). No infants achieve this milestone in the natural history of Type 1 SMA. In addition, 18 (43.9%) infants were able to hold their head upright...
Source: Roche Investor Update - April 28, 2020 Category: Pharmaceuticals Source Type: news
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Brittany Stineman worries her 3-year-old son, Nash, won't survive Covid-19 if he catches it. He has a rare form of spinal muscular atrophy, and she is fighting to protect him. Watch more human stories of the pandemic. (Source: CNN.com - Health)
Source: CNN.com - Health - April 9, 2020 Category: Consumer Health News Source Type: news
U.S. regulators delay decision on Roche SMA drug risdiplam to Aug. 24
U.S. regulators delayed a decision on Roche's spinal muscular atrophy drug risdiplam, the Swiss drugmaker said on Tuesday, after the company submitted more data in February on the medicine against the genetic muscle-wasting disease. (Source: Reuters: Health)
Source: Reuters: Health - April 7, 2020 Category: Consumer Health News Tags: healthNews Source Type: news
Roche provides regulatory update on risdiplam for the treatment of spinal muscular atrophy (SMA)
Basel, 07 April 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY), today announced that the U.S. Food and Drug Administration (FDA) has extended the Prescription Drug User Fee Act (PDUFA) date for its review of the New Drug Application (NDA) of risdiplam with a decision expected by August 24, 2020. The extension is a result of the recent submission of additional data by Roche, including data from the pivotal SUNFISH Part 2 study, in close collaboration with the FDA. These data were recently presented at the 2nd International Scientific and Clinical Congress on Spinal Muscular Atrophy. In November 2019, the FDA granted Priority Rev...
Source: Roche Media News - April 7, 2020 Category: Pharmaceuticals Source Type: news
Roche provides regulatory update on risdiplam for the treatment of spinal muscular atrophy (SMA)
Basel, 07 April 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY), today announced that the U.S. Food and Drug Administration (FDA) has extended the Prescription Drug User Fee Act (PDUFA) date for its review of the New Drug Application (NDA) of risdiplam with a decision expected by August 24, 2020. The extension is a result of the recent submission of additional data by Roche, including data from the pivotal SUNFISH Part 2 study, in close collaboration with the FDA. These data were recently presented at the 2nd International Scientific and Clinical Congress on Spinal Muscular Atrophy. In November 2019, the FDA granted Priority Rev...
Source: Roche Investor Update - April 7, 2020 Category: Pharmaceuticals Source Type: news
How should pharma approach patient collaboration in the 2020s?
From the growing range of patient entities through to new possibilities stemming from technology and patient-generated data, pharma has more and more ways to innovate when it comes to collaborating with patients.
Now pharma needs to identify the ones that will really move the dial. “Clearly there is a shift from patient engagement as a nice-to-have, feel-good activity, to something which is more strategic,” says Vanessa Pott, Director, Patient Advocacy& Strategic Partnerships at Merck. “It’s really important for us to stay abreast of all the external trends.” To stay ahead of the curve in the coming...
Source: EyeForPharma - April 1, 2020 Category: Pharmaceuticals Authors: Lucy Fulford Source Type: news
How should pharma approach patient collaboration in the 2020s?
From the growing range of patient entities through to new possibilities stemming from technology and patient-generated data, pharma has more and more ways to innovate when it comes to collaborating with patients.
Now pharma needs to identify the ones that will really move the dial. “Clearly there is a shift from patient engagement as a nice-to-have, feel-good activity, to something which is more strategic,” says Vanessa Pott, Director, Patient Advocacy& Strategic Partnerships at Merck. “It’s really important for us to stay abreast of all the external trends.” To stay ahead of the curve in the coming...
Source: EyeForPharma - April 1, 2020 Category: Pharmaceuticals Authors: Lucy Fulford Source Type: news
