Delving into the Latest Updates on Lumacaftor with Synapse

Overview

Basic Info

Drug Type

Small molecule drug

Synonyms

Lumacaftor, Lumacaftor (USAN), Lumacaftor monotherapy

+ [3]

Target

CFTR

Mechanism

CFTR stimulants(Cystic fibrosis transmembrane conductance regulator stimulants)

Therapeutic Areas

Congenital DisordersEye DiseasesOther Diseases

Active Indication

Stargardt Disease

Inactive Indication

Cystic FibrosisLiver Injury

Originator Organization

Vertex Pharmaceuticals, Inc.

Active Organization

Johns Hopkins University School of Advanced International

Inactive Organization

Bayer AG

Vertex Pharmaceuticals, Inc.

Drug Highest PhasePreclinical

First Approval Date-

RegulationOrphan Drug (US)

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Structure/Sequence

Molecular FormulaC24H18F2N2O5

InChIKeyUFSKUSARDNFIRC-UHFFFAOYSA-N

CAS Registry936727-05-8

A Single-Dose, Bioequivalence and Food Effect Bioavailability Study in Healthy Volunteers Comparing the Commercial Lumacaftor 200 mg / Ivacaftor 125 mg Combination Film-Coated Tablet (Orkambi®) to the Lumacaftor 200 mg Film-Coated Tablet Formulation, and the Lumacaftor 200 mg Film-Coated Tablet Formulation in the Fasted to Fed State.

The objective of this study is to assess bioequivalence of lumacaftor from Lumacaftor 200 mg Film-Coated Tablet Formulation (Qanatpharma) versus the reference commercial product, Lumacaftor 200 mg /Ivacaftor 125 mg Combination Film-Coated Tablet (Orkambi®) in the fed state, and food-effect bioavailability of Lumacaftor 200 mg Film-Coated Tablet Formulation (Qanatpharma) in the fasted and fed state in healthy, non-smoking, male and non-pregnant female volunteers, 18 to 55 years of age, inclusive.

Start Date17 Nov 2023

Sponsor / Collaborator-

NCT04415268

/ WithdrawnNot ApplicableIIT

Combined Effect of CFTR Protein Modulator Drugs and Exercise on Pulmonary Function, Fitness, Sweat Test and Quality of Life in Children With Cystic Fibrosis

This study aims to assess the effects of programmed exercise combined with CFTR protein modulator drugs in the cardiorespiratory fitness, strength, functional capacity and agility in a group of young patients with Cystic Fibrosis.

Start Date09 Jun 2020

Sponsor / Collaborator

Universidad Europea de Madrid SAU

NCT03601637

/ CompletedPhase 3

A Phase 3, 2-part, Open-label Study to Evaluate the Safety and Pharmacokinetics of Lumacaftor/Ivacaftor in Subjects 1 to Less Than 2 Years of Age With Cystic Fibrosis, Homozygous for F508del

This study will evaluate the safety and pharmacokinetics (PK) of lumacaftor (LUM) and ivacaftor (IVA) in participants 1 to less than 2 years of age with cystic fibrosis (CF), homozygous for F508del (F/F).

Start Date07 Sep 2018

Sponsor / Collaborator

Vertex Pharmaceuticals, Inc.

100 Clinical Results associated with Lumacaftor

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100 Translational Medicine associated with Lumacaftor

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100 Patents (Medical) associated with Lumacaftor

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683

Literatures (Medical) associated with Lumacaftor

31 Dec 2024·JOURNAL OF MEDICAL ECONOMICS

Clinical, economic, and societal burden of cystic fibrosis and the impact of the CFTR modulator, lumacaftor/ivacaftor: an assessment using linked registry data in Sweden

Article

Author: Aldvén, Martina ; Banefelt, Jonas ; McGarry, Lisa J. ; Monestrol, Isabelle de ; Krantz, Christina ; Lindblad, Anders ; Gilljam, Marita

AIMS:

We aimed to describe the clinical, economic, and societal burdens of cystic fibrosis (CF) and impact of CF transmembrane conductance regulator modulator (CFTRm) treatment on people with CF, caregivers, and healthcare systems.

MATERIAL AND METHODS:

This retrospective study used linked real-world data from Swedish national population-based registries and the Swedish CF Quality Registry to assess clinical, economic, and societal burden and CFTR impact in CF. Records from people with CF and a ten-fold control population without CF matched by sex, birth year, and location were compared during 2019. Outcomes for a subset aged >6 years initiating lumacaftor/ivacaftor (LUM/IVA) in 2018 were compared 12 months pre- and post-treatment initiation.

RESULTS:

People with CF (n = 743) had >10 times more inpatient and outpatient specialist visits annually vs controls (n = 7406). Those aged >18 had an additional 77·7 (95% CI: 70·3, 85·1) days of work absence, at a societal cost of €11,563 (95% CI: 10,463, 12,662), while caregivers of those aged <18 missed an additional 6.1 (5.0, 7.2) workdays. With LUM/IVA treatment, people with CF (n = 100) had significantly increased lung function (mean change in ppFEV₁ [3·8 points; 95% CI: 1·1, 6·6]), on average 0·5 (95% CI: -0·8, -0·2) fewer pulmonary exacerbations and 45·2 (95% CI: 13·3, 77·2) fewer days of antibiotics. Days of work lost by caregivers of people with CF aged <18 decreased by 5·4 days (95% CI: 2·9, 7·9).

CONCLUSION:

CF is associated with a high clinical economic and societal burden in Sweden. Improvements in clinical status observed in people with CF treated with LUM/IVA were reflected in reduced caregiver and societal burden.

13 Dec 2024·ORGANIC LETTERS

Pd-Catalyzed C(sp²)–C(sp³) Suzuki Coupling and Synthesis of Lumacaftor Using Designed Monophosphine Ligands

Article

Author: Guo, Xu ; Jia, Fushun ; Zhang, Xiangdong ; Liu, Guodu

Novel monophosphine ligands L1 and L2 with a C-P ring were designed and synthesized for the efficient Pd-catalyzed C(sp²)-C(sp³) Suzuki coupling. With 0.5 mol % Pd₂dba₃ and 2 mol % L1, aryl halides coupled with alkylboronic acids to give the products in up to 99% yield. The aryl thianthrene salt was further applied for late-stage methylation in 97% yield. The active pharmaceutical ingredient lumacaftor was synthesized by aryl-alkyl and aryl-aryl Suzuki coupling reactions using L1 and L2.

01 Dec 2024·PEDIATRIC PULMONOLOGY

Effects on growth, weight and body composition after CFTR modulators in children with cystic fibrosis

Article

Author: Merino Sánchez‐Cañete, A. ; Garriga García, M. ; Tutau Gómez, C. ; Morales Tirado, A. ; Castro Millan, AM. ; Gascón Galindo, C. ; Sierra San Nicolás, S. ; Merino Sanz, N. ; Murray Hurtado, M. ; Tabares González, A. ; Gutiérrez Martínez, JR. ; Suárez González, M. ; Martín Fernández, C. ; Loverdos Eseverri, I. ; González Jiménez, D. ; Martín Rivada, A. ; Vicente Santamaría, S. ; Ortiz Pérez, P. ; Salcedo Lobato, E. ; Crehuá Gaudiza, E. ; Ramos Riesgo, T. ; García Romero, R. ; Torcuato Rubio, E. ; Muñoz Codoceo, MR. ; García Volpe, C. ; De la Mano Hernández, A. ; Del Brio Castillo, R. ; Álvarez Beltrán, M. ; Medina Martínez, M. ; López Rozas, M. ; Blitz Castro, E. ; López Cárdenes, CM.

Abstract:

Background:

Cystic Fibrosis (CF) is associated with compromised nutrition status, which is responsible for morbidity and mortality along with lung function decline. This study was designed to examine changes in anthropometric markers and body composition parameters by bioelectrical impedance analysis after CFTR modulator (CFTRm) treatment.

Methods:

We compared anthropometric parameters and body composition before and after 6 and 12 months of CFTRm treatment. Results are stratified into subgroups according to the modulator used with dual therapy with lumacaftor + ivacaftor or tezacaftor + ivacaftor (LUMA/TEZ + IVA) or triple therapy with elexacaftor + tezacaftor + ivacaftor (ELE + TEZ + IVA). Body composition data are available in patients treated with ELE + TEZ + IVA.

Results:

Two hundred and thirty‐four children (55.1% male) were recruited. The median age was 13.6 years (inter‐quartile range [IQR] 10.7–16.1). We can observe a statistically significant increase in the weight Z score and BMI Z score after CFTRm. In terms of changes in body composition, we observe a significant increase in fat mass (FM) expressed both in kilograms and as a percentage at 6 months (p < .05; Wilcoxon‐test), with no such differences found at 12 months. We also observe a statistically significant increase in fat‐free‐mass (FFM), expressed in kilograms at 6 and 12 months (p < .05; Wilcoxon‐test).

Conclusion:

Weight status improved and changes in body composition occurred in children after CFTRm therapy, including an increase of fat mass. Further studies are needed to confirm these changes in body composition and their impact on disease progression.

100 Deals associated with Lumacaftor

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External Link

KEGG	Wiki	ATC	Drug Bank
D10134	Lumacaftor	R07AX30	DB09280

R&D Status

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Cystic Fibrosis	Phase 3	CZ	Vertex Pharmaceuticals, Inc.	01 May 2013
Cystic Fibrosis	Phase 3	IE	Vertex Pharmaceuticals, Inc.	01 May 2013
Cystic Fibrosis	Phase 3	IT	Vertex Pharmaceuticals, Inc.	01 May 2013
Liver Injury	Phase 1	CZ	Vertex Pharmaceuticals, Inc.	01 Jun 2013
Liver Injury	Phase 1	SK	Vertex Pharmaceuticals, Inc.	01 Jun 2013
Stargardt Disease	Preclinical	US	Johns Hopkins University School of Advanced International	01 Jul 2018

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Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


ESGCT2023	Not Applicable	Cystic Fibrosis	-	VX809 (Normal Human Bronchial Epithelial (NHBE) cells)	hgxwegyurc(kgoysjgozf) = approximately 2 to 2.5 times uixvemsdrp (kvrnpdzhhw )	-	24 Oct 2023
				VX809 (Cystic Fibrosis Bronchial Epithelial (CFBE) cells)
NCT03601637 (CTgov)	Phase 3	Cystic Fibrosis	61	IVA+LUM (Part B: LUM/IVA)	znvnvylbap(iwtqirgdwf) = oxxknnpmvu fcykjzfwxh (dakfbfkovz, gavvpozmyc - trdmlosohf) View more	-	06 Jan 2023
				IVA+LUM (Part A: LUM/IVA)	czmzwcatbz(ibvnzgfktr) = inlmhsjhfd ytgnnkhsio (iccyiffpay, xwgflkgijz - tsicppmfaq) View more
NCT02514473 (CTgov)	Phase 3	Cystic Fibrosis	206	Placebo (Placebo)	jwckhaztsc(npufppofjm) = sggrkgyelm hvjjyzzrmu (qxwdwaezpv, yranbgkkoe - srgvmhxakc) View more	-	23 Oct 2017
				VX-770+VX-809 (LUM/IVA)	jwckhaztsc(npufppofjm) = zumosjpwph hvjjyzzrmu (qxwdwaezpv, krqmwikhak - nftgvnmnmx) View more
NCT02514473 (Pubmed \| Lancet Respir Med) Manual	Phase 3	Cystic Fibrosis F508del	204	lumacaftor+ ivacaftor	axzbsxoonn(iynwuznofs): difference = -1.09 (95% CI, -1.43 to -0.75), P-Value = <0.0001 View more	Positive	01 Jul 2017
				Placebo
NCT01897233 (Pubmed \| Pediatrics \| Am J Respir Crit Care Med) Manual	Phase 3	Cystic Fibrosis F508del mutation	58	Lumacaftor/Ivacaftor	dmoqogtlsn(nxzlsidkza) = bqgkufsxsv abnbuvexon (qkrkjehqci ) View more	Positive	01 Apr 2017
NCT01225211 (Pubmed)	Phase 2	Cystic Fibrosis	126	Lumacaftor/Ivacaftor	kgjxjvjdvo(dkddddeavz) = yettctulvb bbqeafbmqn (njdsieabzm ) View more	-	01 Feb 2017
				Placebo	kgjxjvjdvo(dkddddeavz) = cuxodtlflm bbqeafbmqn (njdsieabzm ) View more
NCT00865904 (CTgov)	Phase 2	Cystic Fibrosis	93	Placebo (Placebo)	xtcglzphno(uebzvydiry) = ingrbkxzba ctktrgpkex (rmgsqerjgt, zszkqquuhw - orfnxdxroc) View more	-	28 Aug 2015
				VX-809 (VX-809, 25 mg)	xtcglzphno(uebzvydiry) = aupslwemsd ctktrgpkex (rmgsqerjgt, yzqupmgyhq - fpveeuahwo) View more
NCT01225211 (Pubmed \| Lancet Respir Med)	Phase 2	Cystic Fibrosis	312	Lumacaftor 200 mg + Ivacaftor 150 mg	pjxgtgzslw(hihkzbopve) = hsgtlrxgbu zrwfvollpl (pighbioytu )	-	01 Jul 2014