Five-year data for Roche’s Evrysdi show the majority of treated
children with a severe form of spinal muscular atrophy (SMA)
achieved or maintained the ability to sit, stand or walk
- After five years of
treatment, 91% of children were alive — without treatment, children
with Type 1 SMA would not be expected to live past two years of
age
- 96% of Evrysdi-treated
children could swallow, 80% could feed without a feeding tube and
59% could sit without support for at least 30 seconds
- Evrysdi is now approved in
more than 100 countries with over 15,000 patients treated
globally
Basel, 07 June 2024 - Roche (SIX: RO, ROG; OTCQX: RHHBY)
announced today new five-year data confirming the sustained
efficacy and safety profile of Evrysdi® (risdiplam) in children
with Type 1 spinal muscular atrophy (SMA) from the open-label
extension of the pivotal FIREFISH study. By the end of Year 5, 91%
of children treated with Evrysdi were alive, 81% were alive without
permanent ventilation and the majority were able to sit without
support for at least 30 seconds (59%). At the end of year 5, seven
children were able to stand, three with support, four unaided and
six could walk with support. Without disease modifying treatment,
natural history studies indicate that children with Type 1 SMA
would not only never be able to reach such milestones, but also not
typically live past the age of two. The data were presented at the
Cure SMA Research & Clinical Care Meeting, June 5 - 7,
2024.
“These long-term findings confirm the ongoing benefit of Evrysdi
for children with Type 1 SMA,” said Professor Giovanni Baranello,
M.D., UCL Great Ormond Street Institute of Child Health & Great
Ormond Street Hospital, London, UK. “Children treated with Evrysdi
over five years have maintained or improved their ability to sit,
stand and walk - critical skills for development and daily living.
An overwhelming majority also maintained the ability to swallow and
to eat without a feeding tube.”
Motor function abilities, as assessed by the Gross Motor Scale
of the Bayley Scales of Infant and Toddler Development Third
Edition (BSID-III) and Hammersmith Infant Neurological Examination
2 (HINE-2), were maintained or continued to be achieved in those
treated with Evrysdi. The FIREFISH results showed most children
treated with Evrysdi also maintained their feeding and swallowing
abilities; of those assessed at year 5, 96% were able to swallow
and 80% were able to feed without a feeding tube.
“This is the final readout of the FIREFISH study, which has
provided a wealth of insights and data, helping to firmly establish
Evrysdi as an important treatment option, improving the lives of
children across the globe living with SMA,” said Levi Garraway,
M.D., Ph. D., Chief Medical Officer and Head of Global Product
Development. “This would not have been possible without the
commitment and dedication of the children and families who
participated, as well as numerous healthcare professionals and
patient support organisations to whom we are immensely
thankful.”
No treatment-related adverse events (AEs) led to treatment
discontinuation or withdrawal from the study. The overall rate of
AEs decreased by 66% between Year 1 and the final year of study.
The most common AEs were upper respiratory tract infection (64%),
pyrexia (64%) and pneumonia (50%). Hospitalisations declined over
the 5-year treatment period and 22% of children did not require
hospitalisation at all since beginning treatment with Evrysdi.
Evrysdi is the only oral, non-invasive small molecule SMA
treatment designed to be systemically delivered to both the central
nervous system (CNS) & peripheral tissues.
Roche leads the clinical development of Evrysdi as part of a
collaboration with the SMA Foundation and PTC Therapeutics.
About Evrysdi® (risdiplam)
Evrysdi is a survival motor neuron 2 (SMN2) splicing
modifier designed to treat SMA caused by mutations in chromosome 5q
that lead to survival motor neuron (SMN) protein deficiency.
Evrysdi is administered daily at home in liquid form either by
feeding tube or by mouth.
Evrysdi is designed to treat SMA by increasing and sustaining
the production of SMN protein in the CNS and peripheral tissues.
SMN protein is found throughout the body and is critical for
maintaining healthy motor neurons and core motor functions such as
swallowing, speaking, and breathing.
Evrysdi was granted PRIME designation by the European Medicines
Agency (EMA) in 2018 and Orphan Drug Designation by the U.S. Food
and Drug Administration in 2017. In 2021, Evrysdi was awarded Drug
Discovery of the Year by the British Pharmacological Society as
well as the Society for Medicines Research Award for Drug
Discovery. Evrysdi is currently approved in more than 100
countries, and the dossier is under review in a further 13
countries.
Evrysdi is currently being, or has been, evaluated in five
global multicentre trials in people with SMA:
- FIREFISH (NCT02913482) – an
open-label, two-part pivotal clinical trial in infants with Type 1
SMA. Infants were approximately 5.5 months of age (median) at the
time of enrollment and of the 58 infants that completed the first
year of treatment, 52 entered the open-label extension study.The
study met its primary endpoint.
- SUNFISH (NCT02908685) – a two-part,
double-blind, placebo-controlled pivotal study in people aged 2-25
years with Types 2 or 3 SMA. The study met its primary
endpoint.
- JEWELFISH (NCT03032172) – an
open-label exploratory trial designed to assess the safety,
tolerability, pharmacokinetics and pharmacodynamics in people with
SMA aged 6 months to 60 years who received other investigational or
approved SMA therapies for at least 90 days prior to receiving
Evrysdi. The study has completed recruitment (n=174).
- RAINBOWFISH (NCT03779334) – an
open-label, single-arm, multicentre study, investigating the
efficacy, safety, pharmacokinetics, and pharmacodynamics of Evrysdi
in babies (n=26), from birth to six weeks of age (at first dose)
with genetically diagnosed SMA who are not yet presenting with
symptoms. The study met its primary endpoint.
- MANATEE (NCT05115110) – a phase 2/3
clinical study to evaluate the safety and efficacy of GYM329
(RG6237), an anti-myostatin molecule targeting muscle growth, in
combination with Evrysdi for the treatment of SMA in patients 2-10
years of age. The FDA Office of Orphan Products Development granted
GYM329 Orphan Drug Designation for the treatment of patients with
SMA in December 2021. The study is currently recruiting.
About SMA
SMA is a severe, progressive neuromuscular disease that can be
fatal. It affects approximately one in 10,000 babies and is the
leading genetic cause of infant mortality. SMA is caused by a
mutation of the survival motor neuron 1 (SMN1) gene, which
leads to a deficiency of SMN protein. This protein is found
throughout the body and is essential to the function of nerves that
control muscles and movement. Without it, nerve cells cannot
function correctly, leading to muscle weakness over time. Depending
on the type of SMA, an individual’s physical strength and their
ability to walk, eat or breathe can be significantly diminished or
lost.
About Roche in Neuroscience
Neuroscience is a major focus of research and development at Roche.
Our goal is to pursue groundbreaking science to develop new
treatments that help improve the lives of people with chronic and
potentially devastating diseases.
Roche is investigating more than a dozen medicines for
neurological disorders, including multiple sclerosis, spinal
muscular atrophy, neuromyelitis optica spectrum disorder,
Alzheimer’s disease, Huntington’s disease, Parkinson’s disease and
Duchenne muscular dystrophy. Together with our partners, we are
committed to pushing the boundaries of scientific understanding to
solve some of the most difficult challenges in neuroscience
today.
About Roche
Founded in 1896 in Basel, Switzerland, as one of the first
industrial manufacturers of branded medicines, Roche has grown into
the world’s largest biotechnology company and the global leader in
in-vitro diagnostics. The company pursues scientific excellence to
discover and develop medicines and diagnostics for improving and
saving the lives of people around the world. We are a pioneer in
personalised healthcare and want to further transform how
healthcare is delivered to have an even greater impact. To provide
the best care for each person we partner with many stakeholders and
combine our strengths in Diagnostics and Pharma with data insights
from the clinical practice.
In recognising our endeavour to pursue a long-term perspective
in all we do, Roche has been named one of the most sustainable
companies in the pharmaceuticals industry by the Dow Jones
Sustainability Indices for the fifteenth consecutive year. This
distinction also reflects our efforts to improve access to
healthcare together with local partners in every country we
work.
Genentech, in the United States, is a wholly owned member of the
Roche Group. Roche is the majority shareholder in Chugai
Pharmaceutical, Japan.
For more information, please visit www.roche.com.
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