Latest news with #SMN
Yahoo
6 days ago
- Business
- Yahoo
Roche's Evrysdi tablet approved by European Commission as first and only for Spinal Muscular Atrophy (SMA)
Simplified storage and administration of new tablet formulation may provide greater freedom and independence for people with SMA Evrysdi offers the same efficacy and safety demonstrated in available oral solution Evrysdi is the only non-invasive disease-modifying SMA treatment, with more than 18,000 people with SMA treated globally to date Basel, 04 June 2025 - Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today that the European Commission (EC) has approved a label extension for Evrysdi® (risdiplam) to include a new, room-temperature stable tablet for people living with spinal muscular atrophy (SMA). The 5mg tablet (approx. 6.5mm), which can either be swallowed whole or dispersed in water, can be taken with or without food and does not require refrigeration, when stored at room temperature. Administered at home, Evrysdi is the only non-invasive disease modifying treatment available for people living with SMA. 'The new Evrysdi tablet with its flexible administration represents progress toward more versatile SMA disease management," said Levi Garraway, M.D., Ph.D., Chief Medical Officer and Head of Global Product Development, Roche. 'With over 18,000 people treated to date, Evrysdi's proven efficacy, safety and convenience has significantly improved the course of disease for people living with SMA.' Evrysdi is designed to treat SMA by increasing and sustaining the production of SMN protein throughout the entire central nervous system (CNS) and in peripheral tissues. Together with this innovative mode of action, the new tablet formulation offers additional portability and convenience benefits for the thousands of people living with SMA, their families and caregivers. 'We welcome the development of new treatment formulations that have the potential to further simplify disease management and care for people living with SMA,' Nicole Gusset, Chief Executive Officer, SMA Europe commented. 'This is a disease requiring daily management, and it is paramount that people living with SMA, and those who care for them, are given options to optimise treatment administration.' The approval is based on data from a bioequivalence study (NCT04718181) evaluating the 5mg tablet formulation of Evrysdi, which can either be swallowed whole or dispersed in water. Results presented at SMA Europe's 4th Scientific International Congress in 2024 demonstrated that the tablet formulation and original oral solution provided bioequivalence to Evrysdi, meaning individuals taking the tablet can expect the same established efficacy and safety as the oral solution. The 5mg tablet formulation is suitable for people two years of age or older, who weigh 20kg (44 lbs) or more and are able to swallow without the use of a feeding tube. The original oral solution will remain available for those on other doses of Evrysdi and for those who may prefer the oral solution. 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 of motor neuron (SMN) protein deficiency. Evrysdi is administered daily at home or on the go, either in liquid form (by feeding tube or by mouth) or in the form of a tablet, which can either be swallowed whole or dispersed in water. Evrysdi is designed to treat SMA by increasing and sustaining the production of SMN protein in the CNS and peripheral tissues. SMN protein is found throughout the body and is critical for maintaining healthy motor neurons and core functions. 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 (FDA) 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 more than 100 countries, with more than 18,000 people with SMA treated globally. Evrysdi is currently being, or has been, evaluated in numerous global multicenter trials in people with SMA: FIREFISH (NCT02913482) – an open-label, two-part pivotal clinical trial in infants with Type 1 SMA. Infants were approximately 5.5 months of age (median) at the time of enrollment and of the 58 infants that completed the first year of treatment, 52 entered the open-label extension study. The study met its primary endpoint and has concluded after 5 years of follow up. 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 and has concluded after five years of follow up. 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 prior to receiving Evrysdi. The study has completed recruitment (n=174). RAINBOWFISH (NCT03779334) – an open-label, single-arm, multicenter study, investigating the efficacy, safety, pharmacokinetics, and pharmacodynamics of Evrysdi in babies (n=26), from birth to 6 weeks of age (at first dose) with genetically diagnosed SMA who are not yet presenting with symptoms. The study met its primary endpoint. MANATEE (NCT05115110) – a Phase II/III 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. HINALEA 1 (NCT05861986) and HINALEA 2 (NCT05861999) – Phase IV clinical studies to evaluate the effectiveness and safety of Evrysdi in patients under 2 years of age at enrollment, who received onasemnogene abeparvovec gene therapy either pre-symptomatically or post-symptomatically, following a genetically confirmed diagnosis of 5q–autosomal recessive SMA. The studies are currently recruiting. PUPFISH (NCT05808764) – a Phase II, open-label study to investigate the pharmacokinetics and safety of Evrysdi in babies with SMA who are under 20 days of age (at first dose). The study is currently recruiting. 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 RocheFounded 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. For over 125 years, sustainability has been an integral part of Roche's business. As a science-driven company, our greatest contribution to society is developing innovative medicines and diagnostics that help people live healthier lives. Roche is committed to the Science Based Targets initiative and the Sustainable Markets Initiative to achieve net zero by 2045. 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 All trademarks used or mentioned in this release are protected by law. Roche Global Media RelationsPhone: +41 61 688 8888 / e-mail: Hans Trees, PhDPhone: +41 79 407 72 58 Sileia UrechPhone: +41 79 935 81 48 Nathalie AltermattPhone: +41 79 771 05 25 Lorena CorfasPhone: +41 79 568 24 95 Simon GoldsboroughPhone: +44 797 32 72 915 Karsten KleinePhone: +41 79 461 86 83 Nina MählitzPhone: +41 79 327 54 74 Kirti PandeyPhone: +49 172 6367262 Yvette PetillonPhone: +41 79 961 92 50 Dr Rebekka SchnellPhone: +41 79 205 27 03 Attachment Media Release Evrysdi Tablet EC Approval EnglishError in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
7 days ago
- Business
- Yahoo
Roche's Evrysdi tablet approved by European Commission as first and only for Spinal Muscular Atrophy (SMA)
Simplified storage and administration of new tablet formulation may provide greater freedom and independence for people with SMA Evrysdi offers the same efficacy and safety demonstrated in available oral solution Evrysdi is the only non-invasive disease-modifying SMA treatment, with more than 18,000 people with SMA treated globally to date Basel, 04 June 2025 - Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today that the European Commission (EC) has approved a label extension for Evrysdi® (risdiplam) to include a new, room-temperature stable tablet for people living with spinal muscular atrophy (SMA). The 5mg tablet (approx. 6.5mm), which can either be swallowed whole or dispersed in water, can be taken with or without food and does not require refrigeration, when stored at room temperature. Administered at home, Evrysdi is the only non-invasive disease modifying treatment available for people living with SMA. 'The new Evrysdi tablet with its flexible administration represents progress toward more versatile SMA disease management," said Levi Garraway, M.D., Ph.D., Chief Medical Officer and Head of Global Product Development, Roche. 'With over 18,000 people treated to date, Evrysdi's proven efficacy, safety and convenience has significantly improved the course of disease for people living with SMA.' Evrysdi is designed to treat SMA by increasing and sustaining the production of SMN protein throughout the entire central nervous system (CNS) and in peripheral tissues. Together with this innovative mode of action, the new tablet formulation offers additional portability and convenience benefits for the thousands of people living with SMA, their families and caregivers. 'We welcome the development of new treatment formulations that have the potential to further simplify disease management and care for people living with SMA,' Nicole Gusset, Chief Executive Officer, SMA Europe commented. 'This is a disease requiring daily management, and it is paramount that people living with SMA, and those who care for them, are given options to optimise treatment administration.' The approval is based on data from a bioequivalence study (NCT04718181) evaluating the 5mg tablet formulation of Evrysdi, which can either be swallowed whole or dispersed in water. Results presented at SMA Europe's 4th Scientific International Congress in 2024 demonstrated that the tablet formulation and original oral solution provided bioequivalence to Evrysdi, meaning individuals taking the tablet can expect the same established efficacy and safety as the oral solution. The 5mg tablet formulation is suitable for people two years of age or older, who weigh 20kg (44 lbs) or more and are able to swallow without the use of a feeding tube. The original oral solution will remain available for those on other doses of Evrysdi and for those who may prefer the oral solution. 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 of motor neuron (SMN) protein deficiency. Evrysdi is administered daily at home or on the go, either in liquid form (by feeding tube or by mouth) or in the form of a tablet, which can either be swallowed whole or dispersed in water. Evrysdi is designed to treat SMA by increasing and sustaining the production of SMN protein in the CNS and peripheral tissues. SMN protein is found throughout the body and is critical for maintaining healthy motor neurons and core functions. 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 (FDA) 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 more than 100 countries, with more than 18,000 people with SMA treated globally. Evrysdi is currently being, or has been, evaluated in numerous global multicenter trials in people with SMA: FIREFISH (NCT02913482) – an open-label, two-part pivotal clinical trial in infants with Type 1 SMA. Infants were approximately 5.5 months of age (median) at the time of enrollment and of the 58 infants that completed the first year of treatment, 52 entered the open-label extension study. The study met its primary endpoint and has concluded after 5 years of follow up. 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 and has concluded after five years of follow up. 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 prior to receiving Evrysdi. The study has completed recruitment (n=174). RAINBOWFISH (NCT03779334) – an open-label, single-arm, multicenter study, investigating the efficacy, safety, pharmacokinetics, and pharmacodynamics of Evrysdi in babies (n=26), from birth to 6 weeks of age (at first dose) with genetically diagnosed SMA who are not yet presenting with symptoms. The study met its primary endpoint. MANATEE (NCT05115110) – a Phase II/III 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. HINALEA 1 (NCT05861986) and HINALEA 2 (NCT05861999) – Phase IV clinical studies to evaluate the effectiveness and safety of Evrysdi in patients under 2 years of age at enrollment, who received onasemnogene abeparvovec gene therapy either pre-symptomatically or post-symptomatically, following a genetically confirmed diagnosis of 5q–autosomal recessive SMA. The studies are currently recruiting. PUPFISH (NCT05808764) – a Phase II, open-label study to investigate the pharmacokinetics and safety of Evrysdi in babies with SMA who are under 20 days of age (at first dose). The study is currently recruiting. 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 RocheFounded 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. For over 125 years, sustainability has been an integral part of Roche's business. As a science-driven company, our greatest contribution to society is developing innovative medicines and diagnostics that help people live healthier lives. Roche is committed to the Science Based Targets initiative and the Sustainable Markets Initiative to achieve net zero by 2045. 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 All trademarks used or mentioned in this release are protected by law. Roche Global Media RelationsPhone: +41 61 688 8888 / e-mail: Hans Trees, PhDPhone: +41 79 407 72 58 Sileia UrechPhone: +41 79 935 81 48 Nathalie AltermattPhone: +41 79 771 05 25 Lorena CorfasPhone: +41 79 568 24 95 Simon GoldsboroughPhone: +44 797 32 72 915 Karsten KleinePhone: +41 79 461 86 83 Nina MählitzPhone: +41 79 327 54 74 Kirti PandeyPhone: +49 172 6367262 Yvette PetillonPhone: +41 79 961 92 50 Dr Rebekka SchnellPhone: +41 79 205 27 03 Attachment Media Release Evrysdi Tablet EC Approval EnglishError in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
The Star
08-05-2025
- Business
- The Star
‘Ramli will lead Penang effectively'
(From left) Chow, Mohd Zarif and Penang infrastructure, transport and digital committee chairman Zairil Khir Johari at the MRT Corp Aidilfitri open house in Komtar, Penang. — ZHAFARAN NASIB/The Star PENANG is confident that new Governor Tun Ramli Ngah Talib will lead the state effectively, given his experience in Perak. Penang Chief Minister Chow Kon Yeow said Ramli was an experienced leader and administrator who had served the country for many decades. 'We believe he will be able to contribute to the state's continued development,' he said. Born on March 16, 1941, in Pasir Salak, Perak, Ramli demonstrated his leadership credentials as the eighth Perak mentri besar, a position he held for 17 years from 1982 to 1999, making him the longest-serving chief executive of the state. His political journey began in 1978 when he was elected Kampung Gajah assemblyman and then appointed a state executive councillor. After his tenure in the state administration, Ramli went on to the federal level when he was elected as Pasir Salak MP in 2004, Bernama reported. In his first term in Parliament, he was entrusted to chair the Public Accounts Committee (PAC), a key parliamentary oversight body, before being appointed Speaker of the Dewan Rakyat, a post he held until 2008. Ramli's leadership within Umno was equally significant, having chaired the Perak Umno liaison committee for 16 years (1984 to 2000) and being member of the party supreme council from 1980 to 2006. Chow said with the appointment officially completed in accordance with Article One of the State Constitution, the state secretary's office (SUK) will now handle several matters, including the swearing-in ceremony which will take place on May 1 at Dewan Sri Pinang. 'Invitations will be issued by the SUK office, and other events will be announced in due course to welcome the new head of state,' he said. On April 24, the former Dewan Rakyat Speaker was appointed the ninth Yang di-Pertua Negri of Penang, effective May 1. He received the instrument of appointment from His Majesty Sultan Ibrahim, King of Malaysia, during a presentation ceremony at Istana Negara. At the ceremony, Ramli, 84, was also conferred the Seri Maharaja Mangku Negara (SMN) award which carries the title Tun. Ramli's appointment was made in accordance with Article 1 of the Penang Constitution for a term of four years, until April 30, 2029. The ceremony was also attended by Prime Minister Datuk Seri Anwar Ibrahim and Chow. An appreciation ceremony was held on April 28 at The Light to honour Tun Ahmad Fuzi Abdul Razak for his service as the Yang di-Pertua Negri for one term. Earlier, Chow attended the Mass Rapid Transit (MRT) Corp Sdn Bhd's Hari Raya open house at The Top in Komtar. MRT Corp Sdn Bhd chief executive officer Datuk Mohd Zarif Hashim hosted over 400 guests at the event where they were treated to ketupat, rendang, nasi tomato, satay, cendol and others. 'Those who attended were stakeholders, VIP guests, private and public sectors and the contractors,' he said.
The Star
08-05-2025
- Politics
- The Star
Ramli sworn in as the ninth governor of Penang
GEORGE TOWN: Tun Ramli Ngah Talib took his oath of office as the ninth Yang di-Pertua Negri Pulau Pinang during the swearing-in ceremony at Dewan Sri Pinang here. Ramli, 84, succeeded Tun Ahmad Fuzi Abdul Razak, who had served as the eighth Penang governor since 2021. The new governor took his oath yesterday, witnessed by Penang High Court judge Datuk Rozana Ali Yusoff and State Secretary Datuk Zulkifli Long. Ramli arrived at Dewan Sri Pinang at 8.30am yesterday, accompanied by his wife, Toh Puan Raja Noora Ashikin Raja Abdullah. He then inspected the guard of honour mounted by the Second Battalion of the Malay Regiment, comprising three officers and 103 soldiers led by Mejar Mohd Salleh Jamal, before entering the ceremonial hall to take his oath of office and to sign the official document, accompanied by a 17-gun salute. Ramli was also conferred the state award Darjah Utama Pangkuan Negeri (DUPN), which carries the title of Datuk Seri Utama, presented by Chief Minister Chow Kon Yeow. Also present at the swearing-in ceremony were Deputy Chief Minister I Datuk Mohamad Abdul Hamid, Deputy Chief Minister II Jagdeep Singh Deo and other dignitaries. In his congratulations and loyalty speech, Chow described Ramli's appointment as a great honour for Penangites. 'He has an outstanding record of service, including having led the state of Perak Darul Ridzuan as Mentri Besar for nearly 17 years and contributing his dedicated service at the federal level as the Dewan Rakyat Speaker for more than three years,' he said. Royal appointment: Ramli and his wife Raja Noora Ashikin at the swearing-in ceremony in Penang. — Bernama Chow said Ramli's character and leadership reflect the stature of a true statesman and a distinguished and respected figure in the annals of the nation's governance. He said he is confident that under Ramli's leadership, Penang will continue to shine and be guided by new inspiration and spirit towards a more glorious, prosperous and harmonious future. Ramli received his letter of appointment from His Majesty Sultan Ibrahim, the King of Malaysia, in a ceremony at Istana Negara on April 24. At the ceremony, he was also conferred the award of Seri Maharaja Mangku Negara (SMN), which carries the title of Tun. Ramli will serve as the Penang Governor for a term of four years, until April 30, 2029. Born on March 16, 1941, in Pasir Salak, Perak, he was also the Deputy Transport Minister from 2000 to 2004.
Malay Mail
01-05-2025
- Politics
- Malay Mail
Ramli Ngah Talib sworn in as the ninth Yang Dipertua Negeri of Penang
GEORGE TOWN, May 1 — Tun Ramli Ngah Talib was today sworn in as the ninth Yang Dipertua Negeri of Penang in a full ceremonial event at Dewan Sri Pinang here, taking over from Tun Ahmad Fuzi Abdul Razak. Ramli, 84, took his oath of office and signed the official document, witnessed by Penang High Court Judge Datuk Rozana Ali Yusoff and State Secretary Datuk Zulkifli Long, accompanied by a 17-gun salute. He was also conferred the state award Darjah Utama Pangkuan Negeri (DUPN), which carries the title Datuk Seri Utama, presented by Chief Minister Chow Kon Yeow during the ceremony. Earlier, Ramli inspected a guard of honour mounted by the Second Battalion of the Royal Malay Regiment, consisting of three officers and 103 personnel, led by Major Mohd Solleh Jamal. Meanwhile, in delivering a congratulatory and loyalty message to the new Governor, Chow described Ramli's appointment as a great honour for the people of the state. He said Ramli's character and leadership reflect the stature of a true statesman and a highly respected figure in the nation's history. 'I am confident that under Tun's leadership and wisdom, Penang will continue to shine, guided by fresh inspiration and spirit towards a brighter, prosperous and harmonious future. 'Together with members of the state executive council, state assemblymen, members of Parliament, leaders at all levels and the people of Penang, we reaffirm our unwavering loyalty to Tun's leadership,' he said. On April 24, Ramli received his letter of appointment as the ninth Yang Dipertua Negeri of Penang, effective May 1, 2025, from His Majesty Sultan Ibrahim, King of Malaysia, in a ceremony at Istana Negara. At the same ceremony, he was also conferred the federal award of Seri Maharaja Mangku Negara (SMN), which carries the title Tun. He succeeds Ahmad Fuzi, 76, who had held the position since May 1, 2021, and was the fourth Yang Dipertua Negeri of Penang who was not originally from the state. Ramli previously held several key positions at both federal and state levels, including serving as the eighth Menteri Besar of Perak for 17 years from 1982 to 1999, Deputy Minister of Transport from 2000 to 2004, and Dewan Rakyat speaker from 2004 to 2008. — Bernama
