CHMP recommends Roche’s Evrysdi for babies under two months old with spinal muscular atrophy (SMA)
21 Julho 2023 - 10:30AM
CHMP recommends Roche’s Evrysdi for babies under two months old
with spinal muscular atrophy (SMA)
- Positive recommendation is
based on interim data from ongoing RAINBOWFISH trial which showed
majority of Evrysdi-treated
babies were able to stand and walk within timeframes typical of
healthy babies by 12 months’
treatment1,2
- If approved by the European
Commission, Evrysdi will be
available to treat people of all ages with SMA in the European
Union, including babies from birth
- Evrysdi is
now approved in 100 countries with more than 8,500 patients treated
globally
Basel, 21 July 2023 - Roche (SIX: RO, ROG; OTCQX: RHHBY)
announced today that the EU Committee for Medicinal Products for
Human Use (CHMP) has adopted a positive opinion for the extension
of the Evrysdi® (risdiplam) European Union (EU) marketing
authorisation, which would include infants with genetically
confirmed diagnosis of SMA Type 1, Type 2 or Type 3 or with one to
four SMN2 copies, including from birth to below two months.1 The
recommendation is based on an interim analysis from the ongoing
RAINBOWFISH trial in pre-symptomatic babies with Type 1 SMA from
birth to six weeks. In SMA, early treatment is critical to
counteract ongoing and irreversible loss of motor neurons.3,4,5 A
final decision regarding the approval is expected from the European
Commission later this year.“Treating babies with SMA early helps
them to carry out daily activities such as sitting, standing, and
walking,” said Levi Garraway, M.D., Ph. D., Chief Medical Officer
and Head of Global Product Development. “This CHMP recommendation
is an important step towards treating babies from birth with an
oral formulation, and is a testament to Evrysdi’s impact on
preserving precious muscle function and improving the daily lives
of people with SMA.”The CHMP decision is based on the RAINBOWFISH
interim analysis (n=18), which included 6 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 is currently approved in the EU for the treatment
of patients aged two months or older.6Roche 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=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.
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 Medicines Agency.
Evrysdi. Available at
https://www.ema.europa.eu/en/medicines/human/summaries-opinion/evrysdi-0.
Accessed July 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] Dangouloff T, et al.
Clinical Evidence Supporting Early Treatment Of Patients With
Spinal Muscular Atrophy: Current Perspectives. Ther Clin Risk
Manag. 2019;15:1153-1161.[5] Kong L, et al. Impaired prenatal motor
axon development necessitates early therapeutic intervention in
severe SMA. Sci Transl Med. 2021;13(578):eabb6871.[6] Evrysdi
(risdiplam). Summary of Product Characteristics. Available at
https://www.ema.europa.eu/en/documents/product-information/evrysdi-epar-product-information_en.pdf.
Accessed July 2023.Roche Group Media Relations
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