European Commission approves Roche’s Evrysdi for babies under two
months old with spinal muscular atrophy (SMA)
- Evrysdi
available to treat people of all ages with SMA in the
European Union, including babies from
birth1
- Approval is based on
interim data from ongoing RAINBOWFISH trial showing majority
of Evrysdi-treated babies were
able to stand and walk within timeframes typical of healthy babies
by 12 months’ treatment2,3
- Evrysdi is
the only non-invasive SMA therapy and is approved in 100 countries
with more than 11,000 patients treated globally
Basel, 29 August 2023 - Roche (SIX: RO, ROG; OTCQX: RHHBY)
announced today that the European Commission has approved the
extension of the Evrysdi® (risdiplam) European Union (EU) marketing
authorisation to include infants with a clinical diagnosis of SMA
Type 1, Type 2 or Type 3 or with one to four SMN2 copies from birth
to below two months.1 Interim data from the ongoing RAINBOWFISH
trial in pre-symptomatic babies from birth to six weeks with Type 1
SMA supported the marketing authorisation extension.
“The SMA community welcomes the European Commission’s decision
to extend the use of Evrysdi from birth,” said Dr Nicole Gusset,
President & CEO of SMA Europe. “Preserving motor neurons from
the earliest age possible and preventing their irreversible loss
can have a substantial impact on a person’s future ability to move
and function. We look forward to continued collaborative efforts to
improve diagnosis, including newborn screening, and ensuring all
individuals living with SMA have access to medicines.”
“With this label extension, we can treat babies soon after birth
with Evrysdi, allowing them the greatest chance to achieve the
milestones of sitting, standing and walking, similar to healthy
children,” said Levi Garraway, M.D., Ph. D., Roche’s Chief Medical
Officer and Head of Global Product Development.
The European Commission approval is based on the RAINBOWFISH
interim analysis (n=18), which included six babies with 2 or 3
copies of the SMN2 gene who completed at least one year of study
assessments. Of these, 100% (6/6) were able to sit after one year
of treatment with Evrysdi, 67% (4/6) could stand and 50% (3/6)
could walk independently. All infants were alive at 12 months
without permanent ventilation.
The RAINBOWFISH data show that the safety profile of Evrysdi in
pre-symptomatic babies is consistent with the safety profile seen
in previous trials with symptomatic SMA patients. The most common
adverse reactions were fever, diarrhoea, rash, upper respiratory
tract infection (including nasopharyngitis, rhinitis), lower
respiratory tract infection (including pneumonia, bronchitis),
constipation, vomiting and cough.
Evrysdi was initially approved in Europe in March 2021 for the
treatment of patients aged two months or older.4 The approval was
based on clinical trial data from the pivotal SUNFISH and FIREFISH
studies.
Roche is currently investigating Evrysdi in combination with an
anti-myostatin molecule targeting muscle growth in the Ph II/III
trial MANATEE for the treatment of SMA.
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 100 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=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.
All trademarks used or mentioned in this release are protected
by law.References[1] European Commission. Evrysdi.
Available
at:https://ec.europa.eu/health/documents/community-register/html/h1531.htm
Accessed August 2023.[2] Finkel RS, et al, on behalf of the
RAINBOWFISH Study Group. RAINBOWFISH: Preliminary efficacy and
safety data in risdiplam-treated infants with presymptomatic SMA.
Poster presented at: Muscular Dystrophy Association Clinical and
Scientific Conference. 2022; Nashville, TN.[3] Day JW, et al.
Advances and limitations for the treatment of spinal muscular
atrophy. BMC Pediatr. 2022;22(1):632.[4] Evrysdi (risdiplam).
Summary of Product Characteristics. Available at:
https://www.ema.europa.eu/en/documents/product-information/evrysdi-epar-product-information_en.pdf
Accessed August 2023.
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