Majority of newborn babies with spinal muscular atrophy (SMA)
treated with Roche’s Evrysdi able to sit independently after 1 year
of treatment
- 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 to sit
- All babies were able to
swallow and feed orally and none required permanent
ventilation
- Evrysdi is the only
non-invasive SMA therapy and is approved in over 100 countries with
more than 11,000 patients treated globally
Basel, 04 October 2023 - Roche (SIX: RO, ROG; OTCQX: RHHBY)
today presented positive results from the primary analysis of the
ongoing RAINBOWFISH study assessing the efficacy and safety of
Evrysdi® (risdiplam) in babies with pre-symptomatic SMA (n=26),
aged from birth to six weeks. The data were presented at the 28th
World Muscle Society (WMS) Congress, 3-7 October 2023.
“Evrysdi is the only non-invasive SMA treatment and can be used
within hours of birth, potentially allowing these babies to sit,
stand and walk, similar to healthy individuals,” said Levi
Garraway, M.D., Ph. D., Roche’s Chief Medical Officer and Head of
Global Product Development. “Evrysdi has now demonstrated its
safety and efficacy in babies, children and adults and these
compelling data continue to reinforce our confidence in this
treatment.’’
Clinical studies show that the loss of motor neurons may begin
before symptoms start so initiating treatment early is critical for
better outcomes. The RAINBOWFISH study included babies with two or
more copies of the SMN2 gene. Generally, the lower the number, the
more severe the disease.
The study met its primary endpoint with 80% of the primary
efficacy population (n=5) sitting without support for at least five
seconds after 1 year of Evrysdi treatment, assessed by Bayley
Scales of Infant and Toddler Development, third edition (BSID-III).
The primary efficacy population included babies with two SMN2
copies and a CMAP amplitude of ≥1.5 mV at baseline. CMAP amplitude
measures the muscle response to a stimulus, and a low score
correlates with symptom onset in SMA patients and worse functional
outcomes. Of the 26 babies in the study, 81% could sit
independently for 30 seconds, including all patients with low CMAP
amplitude at baseline (<1.5 mV) and the majority were standing
and walking. Without treatment, children with Type 1 SMA would
never be expected to sit.
RAINBOWFISH was the first trial to assess cognition with a
standardised scale (BSID) as an exploratory endpoint and results
showed cognitive skills typical of normal child development after 1
year of Evrysdi treatment, assessed by BSID-III.
Adverse events (AEs) were more reflective of the age of the
babies than underlying SMA. The majority of AEs were not considered
treatment-related, and there were no deaths or AEs leading to
withdrawal or treatment discontinuation. The most common AEs were
teething, COVID-19, pyrexia, gastroenteritis, eczema and
constipation. The AEs observed in the RAINBOWFISH primary analysis
are generally consistent with those AEs seen in other Evrysdi
trials in SMA.
“These data reinforce the value of beginning treatment for SMA
before symptoms appear, with the goal of preserving motor neurons
while they are still abundant,” said Richard Finkel, M.D.,
RAINBOWFISH Principal Investigator and Director of the Experimental
Neuroscience Program at St. Jude Children’s Research Hospital.
“Coupled with widespread newborn screening programs, early
treatment could counteract the effects of the disease to give
babies with pre-symptomatic SMA the best possible start in
life.”
Roche is also investigating Evrysdi in combination with an
anti-myostatin molecule, which is designed to promote muscle
growth, among SMA patients 2-10 years of age in the Phase 2/3
MANATEE trial. 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 and non-invasively by
mouth or by feeding tube.
Evrysdi is designed to treat SMA by increasing and sustaining
the production of SMN protein in the central nervous system (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 over 100 countries, and
the dossier is under review in a further 15 countries.
Evrysdi is currently being evaluated in five multicentre trials
in people with SMA:
- FIREFISH (NCT02913482) – an open-label, two-part pivotal
clinical trial in infants with Type 1 SMA. 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
is fully enrolled.
- MANATEE (NCT05115110) – a global 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.
In addition to bringing Evrysdi to people around the world,
Roche also leads its clinical development as part of a
collaboration with the SMA Foundation and PTC Therapeutics.
About SMASMA 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
NeuroscienceNeuroscience 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 thirteenth 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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