The impact of FDA and EMA regulatory decision-making process on the access to CFTR modulators for the treatment of cystic fibrosis

A Phase 3 Open-Label Study of ELX/TEZ/IVA in Children 6 Through 11 Years of Age With CF and at Least One F508del Allele

(2021) Edith T. Zemanick et al.   AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE

Cystic fibrosis

(2021) Michal Shteinberg et al.   LANCET

Triple Therapy for Cystic Fibrosis Phe508del–Gating and –Residual Function Genotypes

(2021) Peter J. Barry et al.   NEW ENGLAND JOURNAL OF MEDICINE

‘Bespoke Gene Therapy Consortium’ sets out to enable gene therapies for ultra-rare diseases

(2021) Katie Kingwell   NATURE REVIEWS DRUG DISCOVERY

CFTR Modulators: The Changing Face of Cystic Fibrosis in the Era of Precision Medicine

(2020) Miquéias Lopes-Pacheco   Frontiers in Pharmacology

Decision Making Under Uncertainty: Comparing Regulatory and Health Technology Assessment Reviews of Medicines in the United States and Europe

(2020) Rick A. Vreman et al.   CLINICAL PHARMACOLOGY & THERAPEUTICS

Assessment of significant benefit for orphan medicinal products by European regulators may support subsequent relative effectiveness assessments by health technology assessment organizations

(2020) Rick A. Vreman et al.   DRUG DISCOVERY TODAY

Tezacaftor/ivacaftor in people with cystic fibrosis heterozygous for minimal function CFTR mutations

(2020) Anne Munck et al.   Journal of Cystic Fibrosis

Ivacaftor in Infants Aged 4 to

(2020) Jane C. Davies et al.   AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE

A phase 3, double-blind, parallel-group study to evaluate the efficacy and safety of tezacaftor in combination with ivacaftor in participants 6 through 11 years of age with cystic fibrosis homozygous for F508del or heterozygous for the F508del-CFTR mutation and a residual function mutation

(2020) Jane C. Davies et al.   Journal of Cystic Fibrosis

Rectal Organoids Enable Personalized Treatment of Cystic Fibrosis

(2019) Gitte Berkers et al.   Cell Reports

Safety, pharmacokinetics, and pharmacodynamics of lumacaftor and ivacaftor combination therapy in children aged 2–5 years with cystic fibrosis homozygous for F508del-CFTR: an open-label phase 3 study

(2019) John J McNamara et al.   Lancet Respiratory Medicine

Theranostics by testing CFTR modulators in patient-derived materials: The current status and a proposal for subjects with rare CFTR mutations

(2019) Margarida D. Amaral et al.   Journal of Cystic Fibrosis

The future of cystic fibrosis care: a global perspective

(2019) Scott C Bell et al.   Lancet Respiratory Medicine

Efficacy and safety of the elexacaftor plus tezacaftor plus ivacaftor combination regimen in people with cystic fibrosis homozygous for the F508del mutation: a double-blind, randomised, phase 3 trial

(2019) Harry G M Heijerman et al.   LANCET

Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele

(2019) Peter G. Middleton et al.   NEW ENGLAND JOURNAL OF MEDICINE

Expanding access to CFTR modulators for rare mutations: The utility of n-of-1 trials

(2019) Amalia S. Magaret et al.   Journal of Cystic Fibrosis

Outrageous prices of orphan drugs: a call for collaboration

(2018) Lucio Luzzatto et al.   LANCET

Data from the US and UK cystic fibrosis registries support disease modification by CFTR modulation with ivacaftor

(2018) Leona Bessonova et al.   THORAX

Ivacaftor treatment of cystic fibrosis in children aged 12 to

(2018) Margaret Rosenfeld et al.   Lancet Respiratory Medicine

A framework to rank genomic alterations as targets for cancer precision medicine: the ESMO Scale for Clinical Actionability of molecular Targets (ESCAT)

(2018) J Mateo et al.   ANNALS OF ONCOLOGY

Theratyping in cystic fibrosis

(2018) Kathryn J. Crawford et al.   CURRENT OPINION IN PULMONARY MEDICINE

VX-445–Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles

(2018) Dominic Keating et al.   NEW ENGLAND JOURNAL OF MEDICINE

FDA OKs first in vitro route to expanded approval

(2017) Katie Kingwell   NATURE REVIEWS DRUG DISCOVERY

Academic, Foundation, and Industry Collaboration in Finding New Therapies

(2017) Bonnie W. Ramsey et al.   NEW ENGLAND JOURNAL OF MEDICINE

Tezacaftor–Ivacaftor in Residual-Function Heterozygotes with Cystic Fibrosis

(2017) Steven M. Rowe et al.   NEW ENGLAND JOURNAL OF MEDICINE

Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del

(2017) Jennifer L. Taylor-Cousar et al.   NEW ENGLAND JOURNAL OF MEDICINE

Efficacy and safety of lumacaftor and ivacaftor in patients aged 6–11 years with cystic fibrosis homozygous for F508del-CFTR : a randomised, placebo-controlled phase 3 trial

(2017) Felix Ratjen et al.   Lancet Respiratory Medicine

Safety, pharmacokinetics, and pharmacodynamics of ivacaftor in patients aged 2–5 years with cystic fibrosis and a CFTR gating mutation (KIWI): an open-label, single-arm study

(2016) Jane C Davies et al.   Lancet Respiratory Medicine

Classification of CFTR mutation classes

(2016) Fernando Augusto Lima Marson et al.   Lancet Respiratory Medicine

Progress in therapies for cystic fibrosis

(2016) Kris De Boeck et al.   Lancet Respiratory Medicine

Lumacaftor–Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR

(2015) Claire E. Wainwright et al.   NEW ENGLAND JOURNAL OF MEDICINE

Efficacy and safety of ivacaftor in patients with cystic fibrosis who have an Arg117His-CFTR mutation: a double-blind, randomised controlled trial

(2015) Richard B Moss et al.   Lancet Respiratory Medicine

Efficacy and safety of ivacaftor in patients with cystic fibrosis and a non-G551D gating mutation

(2014) Kris De Boeck et al.   Journal of Cystic Fibrosis

Efficacy and Safety of Ivacaftor in Patients Aged 6 to 11 Years with Cystic Fibrosis with aG551DMutation

(2013) Jane C. Davies et al.   AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE

Assessment of clinical response to ivacaftor with lung clearance index in cystic fibrosis patients with a G551D- CFTR mutation and preserved spirometry: a randomised controlled trial

(2013) Jane Davies et al.   Lancet Respiratory Medicine

Ivacaftor in Subjects With Cystic Fibrosis Who Are Homozygous for the F508del-CFTR Mutation

(2012) Patrick A. Flume et al.   CHEST

Managing Cystic Fibrosis

(2011) Malena Cohen-Cymberknoh et al.   AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE

Evaluation of Oncology Drugs at the European Medicines Agency and US Food and Drug Administration: When Differences Have an Impact on Clinical Practice

(2011) Francesco Trotta et al.   JOURNAL OF CLINICAL ONCOLOGY

A CFTR Potentiator in Patients with Cystic Fibrosis and theG551DMutation

(2011) Bonnie W. Ramsey et al.   NEW ENGLAND JOURNAL OF MEDICINE

Personalized Oncology Through Integrative High-Throughput Sequencing: A Pilot Study

(2011) S. Roychowdhury et al.   Science Translational Medicine

Rescue of CF airway epithelial cell function in vitro by a CFTR potentiator, VX-770

(2009) F. Van Goor et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA