PTC Announces Evrysdi Agreement with Royalty Pharma for Up To $1.5 Billion
RAINBOWFISH study met its primary endpoint with 80% of babies sitting without support for at least five seconds after 1 year of Evrysdi treatment without treatment these babies would never be able...
Aug 29 (Reuters) - Britain's state-run national health service will be the first in the world to offer an injection that treats cancer to hundreds of patients in England which could cut treatment times by up to three quarters.
Basel, 21 July 2023 - Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today that the EU Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for the extension of the Evrysdi® (risdiplam) European Union (EU) marketing authorisation, which would include infants with genetically confirmed diagnosis of SMA Type 1, Type 2 or Type 3 or with one to four SMN2 copies, including from birth to below two months.1 The recommendation is based on an interim analysis from the ongoing RAINBOWFISH trial in pre-symptomatic babies with Type 1 SMA from birth to six weeks. In SMA, early treatment is critical to counteract ongoing and irreversible loss of motor neurons.3,4,5 A final decision regarding the approval is expected from the European Commission later this year.
SOUTH PLAINFIELD, N.J., July 21, 2023 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for the extension of the Evrysdi® (risdiplam) marketing authorization to include infants under two months of age in the European Union (EU). A final decision regarding the approval is expected from the European Commission later this year.
SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today new long-term data for Evrysdi® (risdiplam) from the open-label extension (n=50) of the pivotal FIREFISH study, reinforcing its sustained efficacy and safety profile in children with Type 1 spinal muscular atrophy (SMA). FIREFISH is a two-part study in babies aged 1-7 months at the time of enrollment. After four years of treatment with Evrysdi, many of the babies, now young children, continued to improve their ability to sit, stand and walk without support. All the Evrysdi-treated children who were alive at the time of the primary analysis were still alive at month 48.
New four year data for Roche’s Evrysdi reinforce long-term efficacy and safety profile in some of the most severely affected people with types 2 and 3 spinal muscular atrophy (SMA)
Armed with new data from what Genentech is touting as one of the biggest and most diverse studies of previously treated spinal muscular atrophy (SMA) patients, the Roche unit is padding its case for Evrysdi in people who may have already received a pricey therapy for their disease.
Basel, 12 October 2022- Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced new two-year data from the JEWELFISH study evaluating Evrysdi® (risdiplam) in people with Type 1, 2 or 3 SMA aged 6 months to 60 years at time of enrolment. Patients had been previously treated with other approved or investigational SMA-targeting therapies, including nusinersen (Spinraza(R)) or onasemnogene abeparvovec (Zolgensma(R)). Data showed Evrysdi improved or maintained motor function and led to rapid increases in SMN protein levels which were sustained after 2-years of treatment. These data will be presented at the 27th World Muscle Society (WMS) congress, 11-15 October 2022.
The Food and Drug Administration has expanded approval of Roche’s spinal muscular atrophy drug Evrysdi to include infants younger than 2 months old, the company announced Tuesday, providing an oral alternative in that population to a gene therapy from Novartis and a drug from Biogen that is administered through a spinal injection.