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European Commission Approves Expanded Label for KAFTRIO® in Combination With Ivacaftor for People With Cystic Fibrosis

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Vertex Pharmaceuticals (VRTX) has received European Commission approval for an expanded label of KAFTRIO® in combination with ivacaftor for treating cystic fibrosis (CF). The expansion covers patients aged 2 years and older with at least one non-class I mutation in the CFTR gene.

This regulatory approval makes approximately 4,000 additional people in the European Union eligible for this treatment that addresses the underlying cause of their disease. Several countries including Austria, Denmark, Ireland, Norway, Sweden, and Germany will provide immediate access to eligible patients through existing reimbursement agreements.

Vertex Pharmaceuticals (VRTX) ha ricevuto l'approvazione della Commissione Europea per un'etichetta ampliata di KAFTRIO® in combinazione con ivacaftor per il trattamento della fibrosi cistica (FC). L'ampliamento riguarda i pazienti di età pari o superiore a 2 anni con almeno una mutazione non di tipo I nel gene CFTR.

Questa approvazione normativa rende circa 4.000 persone aggiuntive nell'Unione Europea idonee a questo trattamento che affronta la causa sottostante della loro malattia. Diversi paesi, tra cui Austria, Danimarca, Irlanda, Norvegia, Svezia e Germania, forniranno accesso immediato ai pazienti idonei tramite accordi di rimborso esistenti.

Vertex Pharmaceuticals (VRTX) ha recibido la aprobación de la Comisión Europea para una etiqueta ampliada de KAFTRIO® en combinación con ivacaftor para el tratamiento de la fibrosis quística (FQ). La expansión cubre a pacientes de 2 años o más con al menos una mutación no de tipo I en el gen CFTR.

Esta aprobación regulatoria hace que aproximadamente 4,000 personas adicionales en la Unión Europea sean elegibles para este tratamiento que aborda la causa subyacente de su enfermedad. Varios países, incluidos Austria, Dinamarca, Irlanda, Noruega, Suecia y Alemania, proporcionarán acceso inmediato a los pacientes elegibles a través de acuerdos de reembolso existentes.

Vertex Pharmaceuticals (VRTX)는 유럽연합 집행위원회로부터 KAFTRIO®와 ivacaftor의 조합에 대한 확장 라벨 승인을 받았습니다. 이 확장은 CFTR 유전자에서 최소 하나의 비 클래스 I 변이를 가진 2세 이상의 환자를 포함합니다.

이 규제 승인은 약 4,000명의 추가 환자가 질병의 근본 원인을 다루는 이 치료를 받을 수 있도록 유럽연합에서 자격을 부여합니다. 오스트리아, 덴마크, 아일랜드, 노르웨이, 스웨덴, 독일 등 여러 나라에서는 기존의 환급 계약을 통해 자격 있는 환자에게 즉각적인 접근을 제공할 것입니다.

Vertex Pharmaceuticals (VRTX) a reçu l'approbation de la Commission européenne pour une étiquette élargie de KAFTRIO® en combinaison avec l'ivacaftor pour le traitement de la fibrose kystique (FK). L'élargissement concerne les patients âgés de 2 ans et plus présentant au moins une mutation non de type I dans le gène CFTR.

Cette approbation réglementaire rend environ 4 000 personnes supplémentaires dans l'Union européenne éligibles à ce traitement qui s'attaque à la cause sous-jacente de leur maladie. Plusieurs pays, dont l'Autriche, le Danemark, l'Irlande, la Norvège, la Suède et l'Allemagne, fourniront un accès immédiat aux patients éligibles par le biais d'accords de remboursement existants.

Vertex Pharmaceuticals (VRTX) hat die Genehmigung der Europäischen Kommission für ein erweitertes Label von KAFTRIO® in Kombination mit Ivacaftor zur Behandlung von Mukoviszidose (CF) erhalten. Die Erweiterung umfasst Patienten ab 2 Jahren mit mindestens einer nicht-Klasse-I-Mutation im CFTR-Gen.

Diese regulatorische Genehmigung macht etwa 4.000 zusätzliche Personen in der Europäischen Union für diese Behandlung, die die zugrunde liegende Ursache ihrer Erkrankung anspricht, berechtigt. Mehrere Länder, darunter Österreich, Dänemark, Irland, Norwegen, Schweden und Deutschland, werden den berechtigten Patienten über bestehende Erstattungsvereinbarungen sofortigen Zugang gewähren.

Positive
  • Expanded market access to approximately 4,000 new eligible patients in EU
  • Immediate access in six European countries through existing reimbursement agreements
  • Label expansion to younger age group (2 years and older)
  • Broader mutation coverage including ultra-rare mutations
Negative
  • Pending reimbursement negotiations needed in remaining EU countries before full access

-Approximately 4,000 people living with CF in the European Union are newly eligible for a medicine that treats the underlying cause of their disease for the first time-

LONDON--(BUSINESS WIRE)-- Vertex Pharmaceuticals (Nasdaq: VRTX) today announced that the European Commission has granted regulatory approval for a label expansion of KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor for the treatment of people with cystic fibrosis (CF). With this approval, the indication has been expanded to include all patients ages 2 years and older who have at least one non-class I mutation in the cystic fibrosis conductance regulator (CFTR) gene.

“We have been working for years to bring treatment options to all people with cystic fibrosis, including those with ultra rare mutations,” said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer, Vertex. “We are pleased that the European Commission has broadened the KAFTRIO indication to include all CF patients 2 years and older who have at least one non-class I mutation, ensuring that even more people living with CF can benefit from this transformative medicine.”

As a result of existing KAFTRIO reimbursement agreements in Austria, Denmark, Ireland, Norway and Sweden, and provisions for access in health care systems such as Germany, eligible patients in these countries will have access to the expanded indication of the therapy shortly.

Vertex will continue to work collaboratively with reimbursement authorities across the European Union to ensure access for all eligible patients, as quickly as possible.

About Cystic Fibrosis

Cystic fibrosis (CF) is a rare, life-shortening genetic disease affecting more than 94,000 people in North America, Europe and Australia. CF is a progressive, multi-organ disease that affects the lungs, liver, pancreas, GI tract, sinuses, sweat glands and reproductive tract. CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF, and these mutations can be identified by a genetic test. While there are many different types of CFTR mutations that can cause the disease, the vast majority of people with CF have at least one F508del mutation. CFTR mutations lead to CF by causing CFTR protein to be defective or by leading to a shortage or absence of CFTR protein at the cell surface. The defective function and/or absence of CFTR protein results in poor flow of salt and water into and out of the cells in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus, chronic lung infections and progressive lung damage that eventually leads to death for many patients. The median age of death is in the 30s, but with treatment, projected survival is improving.

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 KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in Combination With Ivacaftor

In people with certain types of mutations in the CFTR gene, the CFTR protein is not processed or folded normally within the cell, and this can prevent the CFTR protein from reaching the cell surface and functioning properly. KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in combination with ivacaftor is an oral medicine designed to increase the quantity and function of the CFTR protein at the cell surface. Elexacaftor and tezacaftor work together to increase the amount of mature protein at the cell surface by binding to different sites on the CFTR protein. Ivacaftor, which is known as a CFTR potentiator, is designed to facilitate the ability of CFTR proteins to transport salt and water across the cell membrane. The combined actions of ivacaftor, tezacaftor and elexacaftor help hydrate and clear mucus from the airways.

KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in combination with ivacaftor is approved in the European Union for the treatment of cystic fibrosis (CF) in patients aged 2 years and above who have at least one non-class I mutation in the cystic fibrosis conductance regulator (CFTR) gene.

For complete product information, please see the Summary of Product Characteristics that can be found on www.ema.europa.eu.

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 Boston, with international headquarters in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia, Latin America and the Middle East. Vertex is consistently recognized as one of the industry's top places to work, including 15 consecutive years on Science magazine's Top Employers list and one of Fortune’s 100 Best Companies to Work For.

Special Note Regarding Forward-Looking Statements

This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, statements made by Carmen Bozic, M.D., in this press release, statements regarding the potential benefits of KAFTRIO in combination with ivacaftor, expectations regarding the eligible patient population, and expectations for patient access to KAFTRIO. While Vertex believes 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 factors that could cause actual events or results to differ materially from those indicated by such forward-looking statements. Those risks and uncertainties include, among other things, that patients may not have access to KAFTRIO on the anticipated timeline, and other risks listed under the heading “Risk Factors” in Vertex's annual report and in subsequent filings filed with the Securities and Exchange Commission and available through the company's website at www.vrtx.com and www.sec.gov. 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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Source: Vertex Pharmaceuticals

FAQ

How many new patients will be eligible for KAFTRIO treatment after Vertex's EU label expansion?

Approximately 4,000 additional people living with cystic fibrosis in the European Union will become eligible for KAFTRIO treatment.

What age groups are covered in VRTX's new KAFTRIO approval in Europe?

The expanded label covers patients aged 2 years and older who have at least one non-class I mutation in the CFTR gene.

Which European countries will provide immediate access to expanded KAFTRIO treatment?

Austria, Denmark, Ireland, Norway, Sweden, and Germany will provide immediate access through existing reimbursement agreements.

What is the significance of VRTX's KAFTRIO label expansion in Europe?

The expansion allows treatment of the underlying cause of cystic fibrosis in more patients, particularly those with ultra rare mutations.
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