WS13.6 GLPG1837 in subjects with cystic fibrosis (CF) and the G551D mutation: results from a phase II study (SAPHIRA1)

Davies, Jane C.; Bell, S.C.; Koningsbruggen-Rietschel, Silke van; McKone, E.F.; MacGregor, Gordon; Dřevı́nek, Pavel; Conrath, Katja; Kock, Herman de; Gesson, Charlotte; Kanters, D.; Steen, Olivier Van de

doi:10.1016/s1569-1993(17)30236-9

Cited by 4 publications

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“…This is currently incompletely understood and may depend on the duration of therapy pre-withdrawal. Washout certainly could carry adverse clinical consequences, a drop in FEV 1 already being apparent after a 7-day washout from ivacaftor in Galapagos' potentiator GLPG1837 study [7]. Head-to-head comparisons of drugs from different sponsors are unlikely to be feasible, particularly if (as seems likely) the onus falls upon the newer company to purchase and pay for blinding of the prior product.…”

Section: Optimal Future Trial Designmentioning

confidence: 99%

Speeding up access to new drugs for CF: Considerations for clinical trial design and delivery

Davies

Dřevı́nek

Elborn

et al. 2019

Journal of Cystic Fibrosis

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The last decade has witnessed developments in the CF drug pipeline which are both exciting and unprecedented, bringing with them previously unconsidered challenges. The Task Force group was brought together to consider these challenges and possible strategies to address them. Over the last 18 months, we have discussed internally and gathered views from a broad range of individuals representing patient organisations, clinical and research teams, the pharmaceutical industry and regulatory agencies. In this and the accompanying article, we discuss two main areas of focus: i) optimising trial design and delivery for speed and efficiency; ii) drug development for patients with rare CFTR mutations. We propose some strategies to tackle the challenges ahead and highlight areas where further thought is needed. We see this as the start of a process rather than the end and hope herewith to engage the wider community in seeking solutions to improved treatments for all patients with CF.

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Section: Optimal Future Trial Designmentioning

confidence: 99%

Speeding up access to new drugs for CF: Considerations for clinical trial design and delivery

Davies

Dřevı́nek

Elborn

et al. 2019

Journal of Cystic Fibrosis

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“…The potentiator GLPG1837 was tested in adults with an FEV 1% predicted of !40% and at least one G551D mutation or one S1251N mutation. 42,43 QBW251 has shown promise as a new potentiator in both patients with chronic obstructive pulmonary disease (COPD) and patients with CF with residual function mutations. 44,45 However, phase 3 clinical trials were not done with these compounds as they are being tested in a trajectory toward triple-combination therapy for subjects with F508del mutations.…”

Section: Triple Combination Therapymentioning

confidence: 99%

Treating the Underlying Cystic Fibrosis Transmembrane Conductance Regulator Defect in Patients with Cystic Fibrosis

Cuyx

Boeck

2019

Semin Respir Crit Care Med

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Detailed knowledge of how mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene disturb the trafficking or function of the CFTR protein and the use of high-throughput drug screens have allowed novel therapeutic strategies for cystic fibrosis (CF). The main goal of treatment is slowly but surely shifting from symptomatic management to targeting the underlying CFTR defect to halt disease progression and even to prevent occurrence of CF complications. CFTR potentiators for patients with class III mutations, mutation R117H (and in United States also for patients with specific residual function mutations) and the combination of a CFTR modulator plus a potentiator for patients homozygous for F508del, are the two classes of modulators that are in use in the clinic. Approval of these therapeutics has progressively expanded to include both younger patients and a wider range of CFTR mutations. For a significant proportion of patients with CF, current treatment is however still insufficient or unavailable.This review provides an overview of the clinical trial results and the real-life efficacy data of approved CFTR modulators. In addition, we discuss the entire pipeline of CFTR modulators: novel potentiators and correctors, amplifiers, stabilizers, and read-through agents. Furthermore, we discuss other strategies to improve CFTR function like nonsense-mediated decay inhibitors, modified transfer ribonucleic acids, antisense oligonucleotides, and genetic therapies.CFTR modulators are already changing the face of CF and the pipeline of new therapies continues to be exciting.

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Potentiators and Correctors in Paediatric Cystic Fibrosis Patients: A Narrative Review

et al. 2018

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WS13.6 GLPG1837 in subjects with cystic fibrosis (CF) and the G551D mutation: results from a phase II study (SAPHIRA1)

Cited by 4 publications

References 0 publications

Speeding up access to new drugs for CF: Considerations for clinical trial design and delivery

Speeding up access to new drugs for CF: Considerations for clinical trial design and delivery

Treating the Underlying Cystic Fibrosis Transmembrane Conductance Regulator Defect in Patients with Cystic Fibrosis

Potentiators and Correctors in Paediatric Cystic Fibrosis Patients: A Narrative Review

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