news
Roche’s Evrysdi® shows long-term efficacy in spinal muscular atrophy
New data demonstrates the long-term safety and efficacy of Evrysdi® (risdiplam) in a broad population of people with spinal muscular atrophy (SMA).
List view / Grid view
Spinal muscular atrophy (SMA)
news
Roche awarded 2022 Drug Discovery of the Year for Evrysdi®
The development team behind Roche’s Evrysdi® (risdiplam), the first mRNA splicing modifier to be approved, have been honoured with the BPS’s Drug Discovery of the Year Award 2022.
news
NICE recommends risdiplam for spinal muscular atrophy
The new draft guidance recommends Evrysdi (risdiplam) to treat spinal muscular atrophy as part of a managed access agreement (MAA).
news
Babies with SMA able to swallow with Evrysdi® treatment
All five pre-symptomatic babies with spinal muscular atrophy (SMA) were able to swallow and feed orally after 12 months of treatment.
article
Taking cell and gene therapies from idea to commercialisation
David Vincent, Process Development Lead at eXmoor Pharma, details some of the critical steps that form the manufacturing strategy for providers of cell and gene therapies.
news
Collaboration to enable standardisation of AAV quality measurement
US Pharmacopeia to collaborate on assessment of analytical methods for adeno-associated viruses (AAVs) to facilitate the development of reference standards.
news
Zolgensma® meaningfully improves spinal muscular atrophy in patients
Asymptomatic and symptomatic children treated with Zolgensma® (onasemnogene abeparvovec) gene therapy achieved motor milestones never observed in the natural history of spinal muscular atrophy.
article
Curative cell and gene therapies and healthcare system disruption
In this article, experts from Charles River Associates discuss how the introduction of cell and gene therapies will impact upon various aspects of the healthcare system, from provision of care to delivery and supply, and pricing and market access.
news
Evrysdi™ approved for treatment of spinal muscular atrophy in Europe
The European Commission has granted marketing authorisation to Roche’s Evrysdi™ (risdiplam) as a treatment for spinal muscular atrophy in infants, children and adults.
news
NICE approves gene therapy for spinal muscular atrophy
Zolgensma, a one-off, potentially curative treatment for type 1 spinal muscular atrophy, is set to become the most expensive drug ever approved for use on the UK’s NHS.
news
EMA recommends six medications for approval in February meeting
The EMA’s committee adopted positive opinions of six medications, including two orphan drugs, and concluded its review of the use of REGN-COV2 – an investigational antibody therapy for COVID-19.
article
EMA’s transformative treatments of 2020
In this article, we summarise the trial findings for the European Medicines Agency’s 13 most transformative therapies of 2020.
news
SMA drug Zolgensma® has “significant therapeutic benefit”, according to interim data
Treatment with Zolgensma® (onasemnogene abeparvovec) resulted in rapid and sustained improvement in motor function in spinal muscular atrophy (SMA) patients.
news
EMA accepts marketing application for Spinal Muscular Atrophy treatment
The European Medicines Agency (EMA) has accepted the marketing authorisation application for Evrysdi™ (risdiplam) as a treatment of spinal muscular atrophy (SMA).
news
Study suggests Zolgensma will generate $2.5bn of global sales by 2025
According to researchers, Zolgensma, made by Novartis, will lead the global spinal muscular atrophy market and generate billions in sales.