Four-year follow up data for Roche’s Evrysdi show continued
increase in number of children with a severe form of spinal
muscular atrophy (SMA) able to sit, stand and walk
- Data from ongoing FIREFISH
study confirm long-term efficacy and safety profile of
Evrysdi in children with Type 1
SMA
- Ninety-one percent of
children were alive at month 48
- More than 95%
maintained the ability to swallow
- without treatment they would have required feeding
support and majority would have died within 2 years
- Evrysdi is
now approved in 99 countries with more than 8,500 patients treated
globally
Basel, 30 June 2023 - Roche (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 enrolment. 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.
Additionally, the majority of infants maintained their ability to
feed by mouth and swallow up to month 48. Motor function was
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) and abilities were
either maintained or improved over four years of Evrysdi treatment.
Without treatment, children with Type 1 SMA are not expected to
live past two years of age and are never able to sit without
support. These data were presented at the Cure SMA Research &
Clinical Care Meeting, June 28 – 30, 2023.Among the infants treated
with Evrysdi (n=58), 37 were able to sit without support for at
least 5 seconds at month 48, compared to 35 at month 24 (BSID-III).
In addition, 36 infants were able to sit without support for at
least 30 seconds at month 48, up from 23 infants at month 24.
Between month 24 and month 48, three infants gained the ability to
stand alone and one infant gained the ability to walk
alone.“Evrysdi’s oral route of administration allows the medicine
to be distributed throughout the body, systemically increasing SMN
protein production, the lack of which is the underlying cause of
SMA,” said Dr Giovanni Baranello, UCL Great Ormond Street Institute
of Child Health & Great Ormond Street Hospital, London, UK.
“This has shown to help in delivering a meaningful impact on
important functions of daily living including motor milestones,
feeding and swallowing, which were maintained or improved in this
long-term study, while also offering a tolerable safety
profile.”Evrysdi is the first and only small molecule pre-mRNA
splicing modifier that targets survival motor neuron-2 (SMN2) for
the treatment of SMA, and can be administered at home in liquid
form by mouth or by feeding tube. “The independence that comes with
sitting, standing and walking is transformational for children with
SMA, and their families, and we are very encouraged by how these
skills increased over four years of Evrysdi treatment for many
children in this study,” said Levi Garraway, M.D., Ph. D., Chief
Medical Officer and Head of Global Product Development. “Nine out
of 10 patients in our studies remain on Evrysdi long-term and these
data underscore its importance as an option for people with SMA
across a broad range of age and disease types.”No treatment-related
adverse events (AEs) led to treatment discontinuation or withdrawal
from the study, and the rate of AEs decreased by 71% between the
first and fourth 12 month periods. The most common AEs were pyrexia
(62%), upper respiratory tract infection (62%) and pneumonia (48%).
The rate of hospitalisations decreased over the study period.
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 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 other
functions such as swallowing, speaking, breathing and movement.
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 99 countries and the
dossier is under review in a further 18 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=25), 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.
About SMASpinal Muscular
Atrophy (SMA) is a severe, progressive neuromuscular disease that
can be fatal. It affects approximately one in 10,000 babies and is
among 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 other functions such as swallowing, speaking,
breathing 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, speak or breathe can be significantly diminished or
lost.Spinal Muscular Atrophy (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 other functions such as
swallowing, speaking, breathing 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, speak 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.sel, 02 June 2021 -
Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that
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 endeavor 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.
All trademarks used or mentioned in this release are protected
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