FDA Approves Genentech’s Evrysdi Tablet as First and Only Tablet for Spinal Muscular Atrophy (SMA)
Genentech, a Roche Group member, announced FDA approval for Evrysdi® (risdiplam) tablet, the first and only tablet treatment for spinal muscular atrophy (SMA). The new 5 mg tablet, which can be swallowed whole or dispersed in water, provides the same efficacy and safety as the current oral solution and can be stored at room temperature.
The approval was based on a bioequivalence study confirming comparable exposure to risdiplam between the tablet and oral solution formulations. The tablet is suitable for patients 2 years or older weighing more than 44 lbs (20 kgs). Evrysdi, approved in over 100 countries, is the only non-invasive disease-modifying SMA treatment available.
The oral solution will remain available for patients on other doses or those who prefer it. The new tablet formulation aims to provide greater independence and convenience for SMA patients through simplified dose administration.
Genentech, un membro del Gruppo Roche, ha annunciato l'approvazione della FDA per Evrysdi® (risdiplam) in forma di compressa, il primo e unico trattamento in forma di compressa per l'atrofia muscolare spinale (SMA). La nuova compressa da 5 mg, che può essere ingerita intera o disciolta in acqua, offre la stessa efficacia e sicurezza della soluzione orale attuale e può essere conservata a temperatura ambiente.
L'approvazione si basa su uno studio di bioequivalenza che conferma un'esposizione comparabile al risdiplam tra la compressa e le formulazioni della soluzione orale. La compressa è adatta per pazienti di 2 anni o più che pesano più di 44 libbre (20 kg). Evrysdi, approvato in oltre 100 paesi, è l'unico trattamento non invasivo modificante la malattia per la SMA disponibile.
La soluzione orale rimarrà disponibile per i pazienti che assumono altre dosi o per coloro che la preferiscono. La nuova formulazione in compressa mira a fornire maggiore indipendenza e comodità per i pazienti affetti da SMA attraverso una somministrazione semplificata della dose.
Genentech, un miembro del Grupo Roche, anunció la aprobación de la FDA para Evrysdi® (risdiplam) en forma de tableta, el primer y único tratamiento en tableta para la atrofia muscular espinal (AME). La nueva tableta de 5 mg, que se puede tragar entera o disolver en agua, proporciona la misma eficacia y seguridad que la solución oral actual y se puede almacenar a temperatura ambiente.
La aprobación se basó en un estudio de bioequivalencia que confirma una exposición comparable al risdiplam entre la tableta y las formulaciones de solución oral. La tableta es adecuada para pacientes de 2 años o más que pesen más de 44 libras (20 kg). Evrysdi, aprobado en más de 100 países, es el único tratamiento no invasivo modificador de la enfermedad para la AME disponible.
La solución oral seguirá estando disponible para los pacientes que tomen otras dosis o para aquellos que la prefieran. La nueva formulación en tableta tiene como objetivo proporcionar mayor independencia y conveniencia para los pacientes con AME a través de una administración de dosis simplificada.
제넨텍(Genentech), 로슈 그룹의 일원, 은 Evrysdi® (리스디플람) 정제의 FDA 승인을 발표했습니다. 이는 척수 근육 위축증 (SMA)을 위한 첫 번째이자 유일한 정제 치료제입니다. 새로운 5mg 정제는 통째로 삼키거나 물에 녹일 수 있으며, 현재의 경구 용액과 동일한 효능과 안전성을 제공하며 실온에서 보관할 수 있습니다.
이번 승인은 정제와 경구 용액 제형 간의 리스디플람에 대한 유사한 노출을 확인한 생물학적 동등성 연구를 기반으로 했습니다. 이 정제는 44파운드(20kg) 이상의 체중을 가진 2세 이상의 환자에게 적합합니다. Evrysdi는 100개 이상의 국가에서 승인되었으며, SMA를 위한 유일한 비침습적 질병 변경 치료제입니다.
경구 용액은 다른 용량을 사용하는 환자나 이를 선호하는 환자를 위해 계속 제공될 것입니다. 새로운 정제 제형은 SMA 환자에게 간소화된 용량 투여를 통해 더 큰 독립성과 편리함을 제공하는 것을 목표로 합니다.
Genentech, membre du groupe Roche, a annoncé l'approbation par la FDA de Evrysdi® (risdiplam) sous forme de comprimé, le premier et unique traitement sous forme de comprimé pour l'atrophie musculaire spinale (AMS). Le nouveau comprimé de 5 mg, qui peut être avalé entier ou dissous dans l'eau, offre la même efficacité et sécurité que la solution orale actuelle et peut être conservé à température ambiante.
L'approbation a été basée sur une étude de bioéquivalence confirmant une exposition comparable au risdiplam entre les formulations de comprimés et de solution orale. Le comprimé est adapté aux patients âgés de 2 ans ou plus pesant plus de 44 livres (20 kg). Evrysdi, approuvé dans plus de 100 pays, est le seul traitement non invasif modifiant la maladie pour l'AMS disponible.
La solution orale restera disponible pour les patients sous d'autres doses ou ceux qui la préfèrent. La nouvelle formulation en comprimé vise à offrir plus d'indépendance et de commodité aux patients atteints d'AMS grâce à une administration simplifiée de la dose.
Genentech, ein Mitglied der Roche-Gruppe, gab die FDA-Zulassung für Evrysdi® (Risdiplam) in Tablettenform bekannt, die erste und einzige Tablettenbehandlung für spinale Muskelatrophie (SMA). Die neue 5-mg-Tablette, die ganz geschluckt oder in Wasser aufgelöst werden kann, bietet die gleiche Wirksamkeit und Sicherheit wie die derzeitige orale Lösung und kann bei Raumtemperatur gelagert werden.
Die Zulassung basierte auf einer Bioäquivalenzstudie, die eine vergleichbare Exposition gegenüber Risdiplam zwischen der Tabletten- und der oralen Lösungsformulierung bestätigte. Die Tablette ist für Patienten ab 2 Jahren geeignet, die mehr als 44 Pfund (20 kg) wiegen. Evrysdi, das in über 100 Ländern zugelassen ist, ist die einzige nicht-invasive krankheitsmodifizierende Behandlung für SMA, die verfügbar ist.
Die orale Lösung bleibt für Patienten, die andere Dosen einnehmen oder diese bevorzugen, weiterhin verfügbar. Die neue Tablettenformulierung zielt darauf ab, SMA-Patienten durch vereinfachte Dosierungsanwendung mehr Unabhängigkeit und Komfort zu bieten.
- First and only tablet formulation approved for SMA treatment
- Room temperature storage capability improves convenience
- Approved in over 100 countries, indicating strong market presence
- Maintains same efficacy and safety profile as oral solution
- Provides additional flexible treatment option for patients
- to patients weighing more than 44 lbs (20 kgs)
- Only suitable for patients 2 years or older
– 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 –
“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., Genentech’s chief medical officer and head of Global Product Development. “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 2 years of age or older who weigh more than 44 lbs (20 kgs).
Genentech 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, as demonstrated in animal models. 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
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 5 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 to 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 active.
- 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 SMA
SMA is a severe, progressive neuromuscular disease that can be fatal. It affects approximately one in 10,000 babies and is among the leading genetic causes 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.
What is Evrysdi?
Evrysdi is a prescription medicine used to treat spinal muscular atrophy (SMA) in children and adults.
Important Safety Information
-
Before taking Evrysdi, tell your healthcare provider about all of your medical conditions, including if you:
- are pregnant or plan to become pregnant, as Evrysdi may harm your unborn baby. Ask your healthcare provider for advice before taking this medicine
-
are a woman who can become pregnant:
- Before you start your treatment with Evrysdi, your healthcare provider may test you for pregnancy
- Talk to your healthcare provider about birth control methods that may be right for you. Use birth control while on treatment and for at least 1 month after stopping Evrysdi
- Pregnancy Registry. There is a pregnancy registry for women who take Evrysdi during pregnancy. The purpose of this registry is to collect information about the health of the pregnant woman and her baby. If you are pregnant or become pregnant while receiving Evrysdi, tell your healthcare provider right away. Talk to your healthcare provider about registering with the Evrysdi Pregnancy Registry. Your healthcare provider can enroll you in this registry or you can enroll by calling 1-833-760-1098 or visiting http://www.evrysdipregnancyregistry.com
- are an adult male. Evrysdi may affect a man’s ability to have children (fertility). Ask a healthcare provider for advice before taking this medicine
- are breastfeeding or plan to breastfeed. It is not known if Evrysdi passes into breast milk and may harm your baby
- Tell your healthcare provider about all the medicines you take
- If you were prescribed Evrysdi for Oral Solution, you should receive Evrysdi from the pharmacy as a liquid. If the medicine in the bottle is a powder, do not use it. Contact your pharmacist for a replacement
- Avoid getting Evrysdi on your skin or in your eyes. If Evrysdi gets on your skin, wash the area with soap and water. If Evrysdi gets in your eyes, rinse your eyes with water
-
The most common side effects of Evrysdi include:
-
For later-onset SMA:
- fever
- diarrhea
- rash
-
For infantile-onset SMA:
- fever
- diarrhea
- rash
- runny nose, sneezing, and sore throat (upper respiratory infection)
- lung infection (lower respiratory infection)
- constipation
- vomiting
- cough
-
For later-onset SMA:
These are not all of the possible side effects of Evrysdi. For more information on the risk and benefits profile of Evrysdi, ask your healthcare provider or pharmacist.
You may report side effects to the FDA at 1-800-FDA-1088 or http://www.fda.gov/medwatch. You may also report side effects to Genentech at 1-888-835-2555.
Please see full Prescribing Information for additional Important Safety Information.
For more information, go to https://www.evrysdi.com/.
About Genentech in Neuroscience
Neuroscience is a major focus of research and development at Genentech. Our goal is to pursue groundbreaking science to develop new treatments that help improve the lives of people with chronic and potentially devastating diseases.
Genentech and Roche are 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 Genentech
Founded more than 40 years ago, Genentech is a leading biotechnology company that discovers, develops, manufactures and commercializes medicines to treat patients with serious and life-threatening medical conditions. The company, a member of the Roche Group, has headquarters in
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FAQ
When will the new Evrysdi tablet (RHHBY) be available for SMA patients?
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Will the Evrysdi oral solution (RHHBY) still be available after tablet launch?
What storage advantages does the new Evrysdi tablet (RHHBY) offer?
