Vertex Announces UK MHRA Approval of ALYFTREK® (Deutivacaftor/Tezacaftor/Vanzacaftor), a Once-Daily Next-in-Class CFTR Modulator for the Treatment of Cystic Fibrosis
Vertex Pharmaceuticals (VRTX) has received UK MHRA approval for ALYFTREK®, a once-daily triple combination CFTR modulator therapy for cystic fibrosis treatment. The medication is approved for patients 6 years and older with at least one F508del mutation or another responsive mutation in the CFTR gene.
In head-to-head clinical trials, ALYFTREK demonstrated non-inferior ppFEV1 results and improved sweat chloride levels compared to existing treatments. This marks Vertex's fifth CFTR modulator regimen, offering a more flexible and less burdensome treatment option.
The company is currently working with NICE and NHS to ensure patient access in the UK. ALYFTREK received FDA approval in December 2024, with additional regulatory applications pending in Europe, Canada, Switzerland, Australia, and New Zealand.
Vertex Pharmaceuticals (VRTX) ha ricevuto l'approvazione della MHRA del Regno Unito per ALYFTREK®, una terapia a combinazione tripla di modulatori CFTR da assumere una volta al giorno per il trattamento della fibrosi cistica. Il farmaco è approvato per pazienti di età pari o superiore a 6 anni con almeno una mutazione F508del o un'altra mutazione responsiva nel gene CFTR.
Negli studi clinici comparativi, ALYFTREK ha dimostrato risultati di ppFEV1 non inferiori e miglioramenti nei livelli di cloruro nel sudore rispetto ai trattamenti esistenti. Questo segna il quinto regime di modulatori CFTR di Vertex, offrendo un'opzione terapeutica più flessibile e meno gravosa.
L'azienda sta attualmente collaborando con NICE e NHS per garantire l'accesso dei pazienti nel Regno Unito. ALYFTREK ha ricevuto l'approvazione della FDA a dicembre 2024, con ulteriori domande regolatorie in corso in Europa, Canada, Svizzera, Australia e Nuova Zelanda.
Vertex Pharmaceuticals (VRTX) ha recibido la aprobación de la MHRA del Reino Unido para ALYFTREK®, una terapia de combinación triple de moduladores CFTR que se toma una vez al día para el tratamiento de la fibrosis quística. El medicamento está aprobado para pacientes de 6 años o más con al menos una mutación F508del o otra mutación sensible en el gen CFTR.
En ensayos clínicos comparativos, ALYFTREK demostró resultados de ppFEV1 no inferiores y niveles de cloruro en sudor mejorados en comparación con los tratamientos existentes. Esto marca el quinto régimen de moduladores CFTR de Vertex, ofreciendo una opción de tratamiento más flexible y menos gravosa.
La compañía está trabajando actualmente con NICE y NHS para garantizar el acceso de los pacientes en el Reino Unido. ALYFTREK recibió la aprobación de la FDA en diciembre de 2024, con solicitudes regulatorias adicionales pendientes en Europa, Canadá, Suiza, Australia y Nueva Zelanda.
Vertex Pharmaceuticals (VRTX)는 ALYFTREK®에 대해 영국 MHRA의 승인을 받았습니다. 이는 낭포성 섬유증 치료를 위한 하루 한 번 복용하는 3중 조합 CFTR 조절제 치료제입니다. 이 약물은 6세 이상 환자에게 승인되었으며, 최소한 하나의 F508del 변이 또는 CFTR 유전자에서 반응하는 다른 변이를 가진 환자에게 사용됩니다.
직접 비교 임상 시험에서 ALYFTREK는 기존 치료제와 비교하여 ppFEV1 결과에서 비열등성을 보였고, 땀 염소 수치가 개선되었습니다. 이는 Vertex의 다섯 번째 CFTR 조절제 요법으로, 보다 유연하고 부담이 적은 치료 옵션을 제공합니다.
회사는 현재 영국에서 환자 접근성을 보장하기 위해 NICE 및 NHS와 협력하고 있습니다. ALYFTREK는 2024년 12월에 FDA 승인을 받았으며, 유럽, 캐나다, 스위스, 호주 및 뉴질랜드에서 추가 규제 신청이 진행 중입니다.
Vertex Pharmaceuticals (VRTX) a reçu l'approbation de la MHRA du Royaume-Uni pour ALYFTREK®, une thérapie à combinaison triple de modulateurs CFTR à prendre une fois par jour pour le traitement de la fibrose kystique. Le médicament est approuvé pour les patients âgés de 6 ans et plus présentant au moins une mutation F508del ou une autre mutation réactive dans le gène CFTR.
Dans des essais cliniques comparatifs, ALYFTREK a montré des résultats de ppFEV1 non inférieurs et des niveaux de chlorure dans la sueur améliorés par rapport aux traitements existants. Cela marque le cinquième régime de modulateurs CFTR de Vertex, offrant une option de traitement plus flexible et moins contraignante.
L'entreprise travaille actuellement avec NICE et NHS pour garantir l'accès des patients au Royaume-Uni. ALYFTREK a reçu l'approbation de la FDA en décembre 2024, avec d'autres demandes réglementaires en attente en Europe, au Canada, en Suisse, en Australie et en Nouvelle-Zélande.
Vertex Pharmaceuticals (VRTX) hat die Genehmigung der MHRA im Vereinigten Königreich für ALYFTREK® erhalten, eine einmal täglich einzunehmende dreifache Kombinationstherapie mit CFTR-Modulatoren zur Behandlung von zystischer Fibrose. Das Medikament ist für Patienten ab 6 Jahren mit mindestens einer F508del-Mutation oder einer anderen reaktionsfähigen Mutation im CFTR-Gen zugelassen.
In direkten klinischen Studien zeigte ALYFTREK nicht unterlegene ppFEV1-Ergebnisse und verbesserte Schweißchloridwerte im Vergleich zu bestehenden Behandlungen. Dies markiert das fünfte CFTR-Modulator-Regime von Vertex und bietet eine flexiblere und weniger belastende Behandlungsoption.
Das Unternehmen arbeitet derzeit mit NICE und NHS zusammen, um den Zugang der Patienten im Vereinigten Königreich sicherzustellen. ALYFTREK erhielt im Dezember 2024 die FDA-Zulassung, während weitere regulatorische Anträge in Europa, Kanada, der Schweiz, Australien und Neuseeland ausstehen.
- UK MHRA approval expands market access for new CF treatment
- Clinical trials showed improved sweat chloride levels vs existing treatments
- Once-daily dosing offers better treatment convenience
- Broader mutation coverage than previous CFTR therapies
- Pending regulatory approvals in other key markets create uncertainty
- Access depends on ongoing negotiations with NICE and NHS
Insights
Vertex's UK approval for ALYFTREK (deutivacaftor/tezacaftor/vanzacaftor) represents a significant commercial and clinical advancement in cystic fibrosis treatment. This marks Vertex's fifth CFTR modulator, expanding their dominant CF franchise into new territory with several key advantages:
The approval includes patients with mutations not previously addressable with existing therapies, effectively broadening the treatable population. Clinical data demonstrated non-inferiority on lung function compared to Vertex's existing triple combination, while showing superior results in reducing sweat chloride to near-carrier levels – a biomarker that correlates with long-term disease outcomes.
The once-daily dosing regimen improves on the twice-daily schedule of current therapies, addressing treatment burden issues that impact adherence. Professor Horsley's commentary that patients achieved carrier-level sweat chloride suggests potential for reduced long-term CF complications, which could significantly impact treatment duration and patient outcomes.
This UK approval follows December 2024 FDA authorization, establishing a consistent global regulatory pathway with applications pending in other major markets. The approval strengthens Vertex's market position, though commercial impact depends on pricing negotiations with NHS England and NICE – typically stringent assessors of specialized medicine value.
For Vertex, whose business model centers on maintaining CF leadership through continuous innovation, this approval represents both a defensive strategy to protect their franchise and an opportunity to improve patient outcomes while potentially extending treatment duration.
- Deutivacaftor/tezacaftor/vanzacaftor approved for people with cystic fibrosis 6 years and older with at least one responsive mutation in the CFTR gene, including additional mutations not previously approved with other CFTR modulator therapies -
- In head-to-head clinical trials, deutivacaftor/tezacaftor/vanzacaftor was non-inferior on ppFEV1 and further decreased sweat chloride compared to ivacaftor/tezacaftor/elexacaftor in combination with ivacaftor -
“For more than 20 years we have been focused on discovering medicines that treat the underlying cause of the disease with the goal of helping people live longer and better lives. The approval of ALYFTREK, our fifth CFTR modulator regimen, represents another significant milestone in that journey for people with CF in the UK,” said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex.
“The deutivacaftor/tezacaftor/vanzacaftor Phase 3 trial results showed that it is possible to further improve CFTR protein function with this once-a-day, more flexible and less burdensome regimen,” said Professor Alex Horsley, Professor of Respiratory Medicine at the University of
Vertex is working with the National Institute for Health and Care Excellence (NICE) and the NHS to ensure eligible patients can access this new therapy, which treats the underlying cause of CF, as soon as possible.
This medicine was approved for patients ages 6 years and older by the
About Cystic Fibrosis
Cystic fibrosis (CF) is a rare, life-shortening genetic disease affecting more than 94,000 people in
Today Vertex CF medicines are treating over 68,000 people with CF across more than 60 countries on six continents. This represents 2/3 of the diagnosed people with CF eligible for CFTR modulator therapy.
Diagnosis of CF is often made by genetic testing and is confirmed by testing sweat chloride (SwCl), which measures CFTR protein dysfunction. The diagnostic threshold for CF is SwCl ≥60 mmol/L, while levels between 30-59 indicate CF is possible and more testing may be needed to make the diagnosis of CF. A SwCl level of <30 mmol/L is seen in people who carry one copy of a CFTR gene mutation but do not have any manifestation of disease (carriers). Higher levels of SwCl are associated with more severe disease. Restoring CFTR function leads to lower levels of SwCl. SwCl levels below 60 mmol/L are associated with improved outcomes such as better and more stable lung function, fewer pulmonary exacerbations, better quality of life and improved survival. Restoring SwCl levels below 30 mmol/L has long been the ultimate treatment goal for Vertex, as levels below 30 mmol/L are considered normal and are typical of CF carriers who do not have disease.
About ALYFTREK® (deutivacaftor/tezacaftor/vanzacaftor)
In people with CF, mutations in the CFTR gene lead to decreased quantity and/or function of the CFTR protein channel at the cell surface. Vanzacaftor and tezacaftor are designed to increase the amount of CFTR protein at the cell surface by facilitating the processing and trafficking of the CFTR protein. Deutivacaftor is a potentiator designed to increase the channel open probability of the CFTR protein delivered to the cell surface to improve the flow of salt and water across the cell membrane.
ALYFTREK® (deutivacaftor/tezacaftor/vanzacaftor) is approved in the
For complete product information, please see the Summary of Product Characteristics that can be found on https://products.mhra.gov.uk/.
About Vertex
Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases and conditions. The company has approved therapies for cystic fibrosis, sickle cell disease, transfusion-dependent beta thalassemia and acute pain, and it continues to advance clinical and research programs in these areas. Vertex also has a robust clinical pipeline of investigational therapies across a range of modalities in other serious diseases where it has deep insight into causal human biology, including neuropathic pain, APOL1-mediated kidney disease, IgA nephropathy, primary membranous nephropathy, autosomal dominant polycystic kidney disease, type 1 diabetes and myotonic dystrophy type 1.
Vertex was founded in 1989 and has its global headquarters in
Vertex Special Note Regarding Forward-Looking Statements
This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, the statements by Carmen Bozic, M.D., and Professor Alex Horsley, in this press release, and statements regarding expectations for and the anticipated benefits of ALYFTREK, Vertex’s work with NICE and the NHS to ensure eligible patients can access this new therapy as soon as possible, and the status of the Marketing Authorization Applications for ALYFTREK with global regulatory authorities. While we believe the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that data from the company's development programs may not support registration or further development of its compounds due to safety, efficacy, and other reasons, and other risks listed under the heading “Risk Factors” in Vertex's most recent annual report and subsequent quarterly reports filed with the Securities and Exchange Commission at www.sec.gov and available through the company's website at www.vrtx.com. You should not place undue reliance on these statements. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.
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FAQ
What are the key benefits of Vertex's ALYFTREK compared to existing CF treatments?
Which patient population is eligible for ALYFTREK treatment in the UK?
When did VRTX receive FDA approval for ALYFTREK?
In which countries is Vertex seeking regulatory approval for ALYFTREK?
