FDA approves Roche’s Evrysdi tablet as first and only tablet for Spinal Muscular Atrophy (SMA)
Roche (RHHBY) has received FDA approval for a new tablet formulation of Evrysdi (risdiplam), the only non-invasive disease-modifying treatment for Spinal Muscular Atrophy (SMA). The 5 mg tablet, approved for patients two years and older weighing over 20 kg, can be swallowed whole or dispersed in water and stored at room temperature.
A bioequivalence study demonstrated that the tablet provides comparable exposure to risdiplam as the original oral solution, ensuring the same efficacy and safety profile. The oral solution will remain available for patients on other doses or those who prefer it. The new tablet formulation is expected to be available in the coming weeks.
Evrysdi is currently approved in over 100 countries, offering simplified dose administration that may provide greater freedom and independence for SMA patients in their daily activities such as working, traveling, and education.
Roche (RHHBY) ha ricevuto l'approvazione della FDA per una nuova formulazione in compresse di Evrysdi (risdiplam), l'unico trattamento modificante la malattia non invasivo per l'Atrofia Muscolare Spinale (SMA). La compressa da 5 mg, approvata per pazienti di età pari o superiore a due anni e con un peso superiore a 20 kg, può essere ingerita intera o dispersa in acqua e conservata a temperatura ambiente.
Uno studio di bioequivalenza ha dimostrato che la compressa offre un'esposizione comparabile a risdiplam rispetto alla soluzione orale originale, garantendo lo stesso profilo di efficacia e sicurezza. La soluzione orale rimarrà disponibile per i pazienti che assumono altre dosi o che la preferiscono. Si prevede che la nuova formulazione in compresse sarà disponibile nelle prossime settimane.
Evrysdi è attualmente approvato in oltre 100 paesi, offrendo una somministrazione semplificata della dose che può fornire maggiore libertà e indipendenza ai pazienti SMA nelle loro attività quotidiane come lavorare, viaggiare e studiare.
Roche (RHHBY) ha recibido la aprobación de la FDA para una nueva formulación en tabletas de Evrysdi (risdiplam), el único tratamiento no invasivo modificador de la enfermedad para la Atrofia Muscular Espinal (AME). La tableta de 5 mg, aprobada para pacientes de dos años o más que pesen más de 20 kg, puede ser tragada entera o disuelta en agua y almacenada a temperatura ambiente.
Un estudio de bioequivalencia demostró que la tableta proporciona una exposición comparable a risdiplam en comparación con la solución oral original, asegurando el mismo perfil de eficacia y seguridad. La solución oral seguirá estando disponible para los pacientes que necesiten otras dosis o que la prefieran. Se espera que la nueva formulación en tabletas esté disponible en las próximas semanas.
Evrysdi está actualmente aprobado en más de 100 países, ofreciendo una administración de dosis simplificada que puede proporcionar mayor libertad e independencia a los pacientes con AME en sus actividades diarias como trabajar, viajar y estudiar.
로슈 (RHHBY)는 FDA로부터 Evrysdi (리시플람)의 새로운 정제 제형에 대한 승인을 받았습니다. 이는 척수 근육 위축증(SMA)을 위한 유일한 비침습적 질병 수정 치료제입니다. 5mg 정제는 2세 이상의 환자 중 체중이 20kg 이상인 경우에 승인되었으며, 통째로 삼키거나 물에 녹여 실온에서 보관할 수 있습니다.
생물학적 동등성 연구에 따르면, 이 정제는 원래의 경구 용액과 비교할 때 리시플람에 대한 노출이 유사하여 동일한 효능과 안전성 프로필을 보장합니다. 경구 용액은 다른 용량을 복용하는 환자나 이를 선호하는 환자를 위해 계속 제공될 것입니다. 새로운 정제 제형은 향후 몇 주 내에 제공될 것으로 예상됩니다.
Evrysdi는 현재 100개국 이상에서 승인되어 있으며, SMA 환자들이 일상 활동(예: 일하기, 여행하기, 교육받기)을 하는 데 있어 더 큰 자유와 독립성을 제공할 수 있는 간소화된 용량 관리 방법을 제공합니다.
Roche (RHHBY) a obtenu l'approbation de la FDA pour une nouvelle formulation de comprimés de Evrysdi (risdiplam), le seul traitement non invasif modifiant la maladie pour l'Atrophie Musculaire Spinale (AMS). Le comprimé de 5 mg, approuvé pour les patients de deux ans et plus pesant plus de 20 kg, peut être avalé entier ou dissous dans l'eau et conservé à température ambiante.
Une étude de bioéquivalence a démontré que le comprimé offre une exposition comparable à celle du risdiplam par rapport à la solution orale originale, garantissant le même profil d'efficacité et de sécurité. La solution orale restera disponible pour les patients ayant d'autres doses ou ceux qui la préfèrent. La nouvelle formulation de comprimés devrait être disponible dans les prochaines semaines.
Evrysdi est actuellement approuvé dans plus de 100 pays, offrant une administration de dose simplifiée qui peut offrir une plus grande liberté et indépendance aux patients atteints d'AMS dans leurs activités quotidiennes telles que travailler, voyager et s'instruire.
Roche (RHHBY) hat die FDA-Zulassung für eine neue Tablettenformulierung von Evrysdi (Risdiplam) erhalten, der einzigen nicht-invasiven krankheitsmodifizierenden Therapie für Spinale Muskelatrophie (SMA). Die 5 mg Tablette, die für Patienten ab zwei Jahren mit einem Gewicht von über 20 kg zugelassen ist, kann ganz geschluckt oder in Wasser aufgelöst und bei Raumtemperatur gelagert werden.
Eine Bioäquivalenzstudie hat gezeigt, dass die Tablette eine vergleichbare Exposition gegenüber Risdiplam bietet wie die ursprüngliche orale Lösung, wodurch das gleiche Wirksamkeits- und Sicherheitsprofil gewährleistet ist. Die orale Lösung bleibt für Patienten, die andere Dosen benötigen oder sie bevorzugen, weiterhin verfügbar. Die neue Tablettenformulierung wird voraussichtlich in den kommenden Wochen erhältlich sein.
Evrysdi ist derzeit in über 100 Ländern zugelassen und bietet eine vereinfachte Dosierungsadministration, die SMA-Patienten mehr Freiheit und Unabhängigkeit in ihren täglichen Aktivitäten wie Arbeiten, Reisen und Bildung ermöglichen kann.
- FDA approval for new tablet formulation expands product line
- Room temperature storage capability improves convenience
- Wide market presence with approval in over 100 countries
- Demonstrated bioequivalence maintains established efficacy profile
- to patients weighing over 20 kg (44 lbs)
- Original oral solution still required for certain dosing needs
- Evrysdi is the only non-invasive disease-modifying SMA treatment and is approved in over 100 countries
- Evrysdi tablet can be stored at room temperature and offers the same demonstrated efficacy and safety as the currently available oral solution
- New tablet formulation may provide greater freedom and independence for people with SMA thanks to simplified dose administration
Basel, 12 February 2025 - Roche (SIX: RO, ROG; OTCQX: RHHBY), announced today that the U.S. Food and Drug Administration (FDA) has approved a New Drug Application (NDA) for an Evrysdi® (risdiplam) tablet for people living with spinal muscular atrophy (SMA). Evrysdi is the only non-invasive disease-modifying treatment for SMA. The 5 mg Evrysdi tablet can either be swallowed whole or dispersed in water.
"Evrysdi has robust potential to modify the SMA disease trajectory, and has already been used to treat thousands of patients to date. This approval marks another significant step forward," said Levi Garraway, M.D., Ph.D., Chief Medical Officer and Head of Global Product Development, Roche. "The Evrysdi tablet combines established efficacy with convenience, providing an additional flexible option for SMA management."
The approval of the Evrysdi tablet was based on the results of a bioequivalence study, which demonstrated that the 5 mg tablet, whether swallowed whole or dispersed in non-chlorinated drinking water (e.g., filtered water), and original oral solution provide comparable exposure to risdiplam. This means patients who take the tablet can expect the same established efficacy and safety as the Evrysdi oral solution. The Evrysdi oral solution will remain available for those on other doses of Evrysdi and for those who may prefer the oral solution.
"We cannot underestimate the value that comes with simplifying treatment administration and disease management for people who are living with SMA or those caring for them,” said Kenneth Hobby, President of Cure SMA. "This new room temperature stable formulation option offers an additional choice that may more conveniently fit into daily living activities such as working, traveling, and education."
As part of the label extension, the Evrysdi prescribing information has been updated to include guidance on tablet administration and storage.
The new tablet, expected to be available in the coming weeks, is suitable for people two years of age or older who weigh more than 20 kgs (44 lbs).
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) pre-mRNA 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 (by mouth only, swallowed whole or dispersed in water).
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 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 16,000 people with SMA treated globally.
Evrysdi is currently being, or has been, evaluated in numerous global multicentre trials in people with SMA:
- FIREFISH (NCT02913482) – an open-label, two-part pivotal clinical trial in infants with Type 1 SMA. Infants were approximately five and a half months of age (median) at the time of enrolment 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 five years of follow up.
- SUNFISH (NCT02908685) – a two-part, double-blind, placebo-controlled pivotal study in people aged two-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 six 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, 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 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 two to ten 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 active.
- HINALEA 1 (NCT05861986) and HINALEA 2 (NCT05861999) – Phase IV clinical studies to evaluate the effectiveness and safety of Evrysdi in patients under two years of age at enrolment, 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 SMA
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 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 Neuroscience
Neuroscience 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.
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