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News on March 16, 2021 /bioon.
com" target="_blank">/ --Roche recently announced new exploratory 2-year long-term data for the second part of the SUNFISH study.
SUNFISH is a global placebo-controlled study that is evaluating the oral drug Evrysdi (risdiplam) for the treatment of type 2 or bedridden type 3 spinal muscular atrophy (SMA) patients aged 2-25 years.
Studies have shown that in the primary and secondary endpoint measurements, the improvement in motor function observed with Evrysdi treatment at 12 months continues to improve or maintain at 24 months.
According to the natural history of the disease, the motor function of patients with SMA type 2 and type 3 will decline over time without treatment.
These data will be announced at the 2021 Muscular Dystrophy Association (MDA) Virtual Clinical and Scientific bioon.
com/tags/%E4%BC%9A%E8%AE%AE/" target="_blank">Conference on March 15-18 .
bioon.com" target="_blank">/ --Roche recently announced new exploratory 2-year long-term data for the second part of the SUNFISH study.
SUNFISH is a global placebo-controlled study that is evaluating the oral drug Evrysdi (risdiplam) for the treatment of type 2 or bedridden type 3 spinal muscular atrophy (SMA) patients aged 2-25 years.
Studies have shown that in the primary and secondary endpoint measurements, the improvement in motor function observed with Evrysdi treatment at 12 months continues to improve or maintain at 24 months.
According to the natural history of the disease, the motor function of patients with SMA type 2 and type 3 will decline over time without treatment.
These data will be announced at the 2021 Muscular Dystrophy Association (MDA) Virtual Clinical and Scientific bioon.
com/tags/%E4%BC%9A%E8%AE%AE/" target="_blank">Conference on March 15-18 .
com" target="_blank">In the primary and secondary endpoint measurements, the improvement in motor function observed with Evrysdi treatment at 12 months continued to improve or was maintained at 24 months.
bioon.
com/tags/%E4%BC%9A%E8%AE%AE/" target="_blank">meeting
Evrysdi is a liquid preparation that can be administered orally or via a feeding tube at home, once a day.
The drug can be used to treat infants, children, adolescents, and adults with all types (type 1, type 2, and type 3) of SMA.
In August 2020, Evrysdi was the first to be approved in the United States.
So far, the drug has been approved in 7 countries (the United States, Chile, Brazil, Ukraine, South Korea, Georgia, Russia) and has submitted marketing applications in more than 30 countries, including China.
The drug can be used to treat infants, children, adolescents, and adults with all types (type 1, type 2, and type 3) of SMA.
In August 2020, Evrysdi was the first to be approved in the United States.
So far, the drug has been approved in 7 countries (the United States, Chile, Brazil, Ukraine, South Korea, Georgia, Russia) and has submitted marketing applications in more than 30 countries, including China.
It is worth mentioning that Evrysdi is the first oral therapy for SMA and the first SMA therapy that can be administered at home.
Evrysdi is a motor neuron survival gene 2 (SMN2) mRNA splicing modifier, which treats SMA by increasing the production of motor neuron survival protein (SMN).
SMN protein is distributed throughout the body and is essential for maintaining healthy motor neurons and exercise.
Evrysdi is the first oral therapy for SMA and the first SMA therapy that can be administered at home. Evrysdi is a motor neuron survival gene 2 (SMN2) mRNA splicing modifier, which treats SMA by increasing the production of motor neuron survival protein (SMN).
SMN protein is distributed throughout the body and is essential for maintaining healthy motor neurons and exercise.
In bioon.
com/course_video/lin-chuang-shi-yan-de-feng-xian-jian-kong239833.
html">clinical trials , Evrysdi has shown clinically significant improvements in motor function in SMA patients of different ages and disease severity (including type 1, type 2, and type 3).
Infants treated with Evrysdi can sit for at least 5 seconds without support, which is a common key movement milestone in the natural course of SMA disease.
In addition, compared with natural medical history, Evrysdi also improved survival without permanent ventilation.
bioon. com/course_video/lin-chuang-shi-yan-de-feng-xian-jian-kong239833.
html">clinical trials , Evrysdi has shown clinically significant improvements in motor function in SMA patients of different ages and disease severity (including type 1, type 2, and type 3).
Infants treated with Evrysdi can sit for at least 5 seconds without support, which is a common key movement milestone in the natural course of SMA disease.
In addition, compared with natural medical history, Evrysdi also improved survival without permanent ventilation.
com/course_video/lin-chuang-shi-yan-de-feng-xian-jian-kong239833.
html">Clinical Trials
Eugenio Mercuri, MD, Ph.
D.
, Department of Pediatric Neurology, Catholic University of Rome, Italy, the lead researcher of the SUNFISH study, said: “These results are based on the one-year results of the SUNFISH trial.
It is important to show that two years of treatment improve or stabilize motor function.
In addition, since no new safety signals have been discovered, these second-year results may support Evrysdi’s favorable earnings risk profile for a longer period of time.
”
D.
, Department of Pediatric Neurology, Catholic University of Rome, Italy, the lead researcher of the SUNFISH study, said: “These results are based on the one-year results of the SUNFISH trial.
It is important to show that two years of treatment improve or stabilize motor function.
In addition, since no new safety signals have been discovered, these second-year results may support Evrysdi’s favorable earnings risk profile for a longer period of time.
”
SMA boy (picture from: drpgx.
com)
com)
The patients in the second part of the SUNFISH study were between 2-25 years old.
They were treated with Evrysdi (n=120) or placebo and Evrysdi (n=60; patients in the placebo group received placebo for 12 months.
Then received Evrysdi treatment for 12 months).
This study evaluated some exploratory 24-month endpoints and provided important insights into motor function and its impact on daily life.
They were treated with Evrysdi (n=120) or placebo and Evrysdi (n=60; patients in the placebo group received placebo for 12 months.
Then received Evrysdi treatment for 12 months).
This study evaluated some exploratory 24-month endpoints and provided important insights into motor function and its impact on daily life.
The survey results show that Evrysdi: (1) According to the measurement of the motor function measurement 32 scale (MFM-32), it maintains the improvement of motor function during 12-24 months.
(2) As measured by the Modified Upper Limb Module (RULM) and Hammersmith Motor Function Scale Extended Version (HFMSE), motor function increased during 12-24 months.
(3) Using MFM-32, RULM, and HFMSE measurements, patients who started taking Evrysdi treatment 12 months after receiving placebo showed stable motor function.
(4) During 12-24 months, according to the SMAIS upper limb module measurement reported by the caregiver, the total score increased from the baseline, and the SMAIS score reported by the patient was stable.
(2) As measured by the Modified Upper Limb Module (RULM) and Hammersmith Motor Function Scale Extended Version (HFMSE), motor function increased during 12-24 months.
(3) Using MFM-32, RULM, and HFMSE measurements, patients who started taking Evrysdi treatment 12 months after receiving placebo showed stable motor function.
(4) During 12-24 months, according to the SMAIS upper limb module measurement reported by the caregiver, the total score increased from the baseline, and the SMAIS score reported by the patient was stable.
Compared with the first year, there were fewer serious adverse events, high-level adverse events, and treatment-related adverse events observed in the two treatment groups in the second year.
During 12-24 months, the most common adverse events observed in the Evrysdi group and the placebo+Evrysdi group were upper respiratory tract infection (15.
8% and 10%, respectively), nasopharyngitis (21.
7% and 16.
7%, respectively), Fever (13.
3% and 10%, respectively), headache (10% and 16.
7%, respectively), diarrhea (7.
5% and 10%, respectively), vomiting (11.
7% and 13.
3%, respectively), and cough (10%, respectively) And 8.
3%).
The most common serious adverse events were pneumonia (6.
7% and 0%, respectively) and influenza (0.
8% and 0%, respectively).
During 12-24 months, the most common adverse events observed in the Evrysdi group and the placebo+Evrysdi group were upper respiratory tract infection (15.
8% and 10%, respectively), nasopharyngitis (21.
7% and 16.
7%, respectively), Fever (13.
3% and 10%, respectively), headache (10% and 16.
7%, respectively), diarrhea (7.
5% and 10%, respectively), vomiting (11.
7% and 13.
3%, respectively), and cough (10%, respectively) And 8.
3%).
The most common serious adverse events were pneumonia (6.
7% and 0%, respectively) and influenza (0.
8% and 0%, respectively).
Levi Garraway, MD, Roche’s Chief Medical Officer and Head of Global Product Development, said: “These encouraging results confirm that the efficacy and safety of Evrysdi in patients with type 2 and type 3 SMA can be maintained over time.
Therefore, these findings further Highlights the potential long-term benefits of this pioneering drug for patients with SMA disease of different ages and severity.
"
Levi Garraway, MD, Roche’s Chief Medical Officer and Head of Global Product Development, said: “These encouraging results confirm that the efficacy and safety of Evrysdi in patients with type 2 and type 3 SMA can be maintained over time. Therefore, these findings further Highlights the potential long-term benefits of this pioneering drug for patients with SMA disease of different ages and severity.
"
Therefore, these findings further Highlights the potential long-term benefits of this pioneering drug for patients with SMA disease of different ages and severity.
"
The chemical structure of risdiplam (picture source: medchemexpress.
cn)
cn)
Evrysdi is an oral liquid.
Its active pharmaceutical ingredient, risdiplam, is a survival motor neuron gene 2 (SMN2) splicing modifier designed to continuously increase and maintain the level of SMN protein in the central nervous system and peripheral tissues.
More and more clinical evidences show that SMA is a multi-system disease, and the loss of SMA protein may affect many tissues and cells outside the central nervous system.
After oral administration, risdiplam presents a systemic distribution, continuously increasing the level of SMN protein in the central nervous system and peripheral tissues, and has been shown to improve the motor function of patients with type 1, type 2, and type 3 SMA.
Its active pharmaceutical ingredient, risdiplam, is a survival motor neuron gene 2 (SMN2) splicing modifier designed to continuously increase and maintain the level of SMN protein in the central nervous system and peripheral tissues.
More and more clinical evidences show that SMA is a multi-system disease, and the loss of SMA protein may affect many tissues and cells outside the central nervous system.
After oral administration, risdiplam presents a systemic distribution, continuously increasing the level of SMN protein in the central nervous system and peripheral tissues, and has been shown to improve the motor function of patients with type 1, type 2, and type 3 SMA.
As part of the collaboration with the SMA Foundation and PTC Therapeutics, Genentech led the clinical development of Evrysdi.
As part of a large-scale, extensive and robust clinical trial project in the SMA field, Evrysdi is conducting research in more than 450 people.
The project covers infants from 2 months old to 60-year-olds who have different symptoms and motor functions, such as scoliosis or joint contractures, as well as patients who have previously received other SMA therapies.
The drug's bioon.
com/course_video/lin-chuang-shi-yan-de-feng-xian-jian-kong239833.
html">clinical trial population is intended to represent a broad, real-world population of SMA disease, with the goal of ensuring that all suitable patients can receive treatment.
bioon. As part of a large-scale, extensive and robust clinical trial project in the SMA field, Evrysdi is conducting research in more than 450 people.
The project covers infants from 2 months old to 60-year-olds who have different symptoms and motor functions, such as scoliosis or joint contractures, as well as patients who have previously received other SMA therapies.
The drug's bioon.
com/course_video/lin-chuang-shi-yan-de-feng-xian-jian-kong239833.
html">clinical trial population is intended to represent a broad, real-world population of SMA disease, with the goal of ensuring that all suitable patients can receive treatment.
com/course_video/lin-chuang-shi-yan-de-feng-xian-jian-kong239833.
html">Clinical Trials
Currently, Roche is conducting 4 global multi-center clinical studies (SUNFISH[NCT02908685], FIREFISH[NCT02913482], JEWELFISH[NCT03032172], RAINBOWFISH[NCT03779334]) to evaluate all types of Evrysdi treatment (type 1, type 2, type 3) The efficacy and safety of SMA and presymptomatic SMA in neonates.
Spinraza: The world's first SMA treatment drug, approved in China
Spinraza: The world's first SMA treatment drug, approved in ChinaSMA is a motor neuron disease that can cause muscle weakness and atrophy.
The disease is an autosomal recessive genetic disease caused by genetic defects.
It can cause damage to the muscles around the patient’s body.
The patient’s main manifestation is systemic muscle atrophy and weakness.
The body gradually loses various motor functions, even breathing and swallowing.
SMA is the number one bioon.
com/course_video/zhong-guo-ren-qun-ying-yang-he-yi-chuan-yin416058.
html">genetic disease killer in infants under 2 years of age .
The disease is a relatively common "rare disease" with a prevalence of 1:6000-1:10000 in newborns.
According to related reports, the current number of SMA patients in China is about 3-5 million.
bioon. The disease is an autosomal recessive genetic disease caused by genetic defects.
It can cause damage to the muscles around the patient’s body.
The patient’s main manifestation is systemic muscle atrophy and weakness.
The body gradually loses various motor functions, even breathing and swallowing.
SMA is the number one bioon.
com/course_video/zhong-guo-ren-qun-ying-yang-he-yi-chuan-yin416058.
html">genetic disease killer in infants under 2 years of age .
The disease is a relatively common "rare disease" with a prevalence of 1:6000-1:10000 in newborns.
According to related reports, the current number of SMA patients in China is about 3-5 million.
com/course_video/zhong-guo-ren-qun-ying-yang-he-yi-chuan-yin416058.
html">Genetic
In December 2016, Spinraza (nusinersen), a drug developed by Biogen and its partner Ionis, was approved and became the world's first drug for the treatment of SMA.
The drug is an antisense oligonucleotide (ASO).
It is administered by intrathecal injection and delivers the drug directly to the cerebrospinal fluid (CSF) around the spinal cord.
It changes the splicing of SMN2 pre-messenger RNA (pre-mRNA) and increases Production of fully functional SMN protein.
In SMA patients, insufficient levels of SMN protein lead to degeneration of spinal cord motor neurons.
In clinical studies, Spinraza treatment significantly improved the motor function of SMA patients.
The drug is an antisense oligonucleotide (ASO).
It is administered by intrathecal injection and delivers the drug directly to the cerebrospinal fluid (CSF) around the spinal cord.
It changes the splicing of SMN2 pre-messenger RNA (pre-mRNA) and increases Production of fully functional SMN protein.
In SMA patients, insufficient levels of SMN protein lead to degeneration of spinal cord motor neurons.
In clinical studies, Spinraza treatment significantly improved the motor function of SMA patients.
In May 2019, the gene therapy Zolgensma (onasemnogene abeparvovec) from bioon.
com/tags/%E8%AF%BA%E5%8D%8E/">Novartis was approved, becoming the world's first gene therapy to treat SMA.
The drug prevents the progression of the disease by continuously expressing SMN protein after a single, one-time intravenous infusion, can solve the underlying cause of SMA, and is expected to improve the quality of life of patients in the long term.
bioon. com/tags/%E8%AF%BA%E5%8D%8E/">Novartis was approved, becoming the world's first gene therapy to treat SMA.
The drug prevents the progression of the disease by continuously expressing SMN protein after a single, one-time intravenous infusion, can solve the underlying cause of SMA, and is expected to improve the quality of life of patients in the long term.
com/tags/%E8%AF%BA%E5%8D%8E/">Novartis
In the Chinese market, Spinraza was approved at the end of February 2019 for the treatment of patients with 5q spinal muscular atrophy (5q-SMA).
This approval makes Spinraza the first drug to treat SMA in the Chinese market.
5q-SMA is the most common type of SMA, accounting for about 95% of all SMA cases.
This type of SMA is caused by a mutation in the SMN1 (survival motor neuron protein 1) gene on chromosome 5, hence the name 5q-SMA .
()
This approval makes Spinraza the first drug to treat SMA in the Chinese market.
5q-SMA is the most common type of SMA, accounting for about 95% of all SMA cases.
This type of SMA is caused by a mutation in the SMN1 (survival motor neuron protein 1) gene on chromosome 5, hence the name 5q-SMA .
()
Original Source: New 2-Year Data Show Genentech's Evrysdi (risdiplam) Continues to Demonstrate Improvement or Maintenance of Motor Function in People Aged 2-25 With Type 2 or Type 3 Spinal Muscular Atrophy (SMA)
