When given in combination with ivacaftor as the fixed dose combination product Orkambi®, lumacaftor is indicated for the treatment of cystic fibrosis (CF) in patients age 6 years and older, who are homozygous for the F508del mutation in the CF transmembrane conductance regulatory (CFTR) gene. Lumacaftor is a CFTR corrector (ivacaftor is a potentiator) that improves CF symptoms and underlying disease pathology by aiding the conformational stability of F508-mutated CFTR, resulting in increased processing and trafficking of mature protein to the cell surface, where it exhibits improved chloride channel function compared to uncorrected F508del-CFTR. More specifically, lumacaftor acts directly with CFTR and/or CFT-associated proteins as a proteinfolding chaperone, preventing misfolding of CFTR ion channels and consequent destruction during processing in the endoplasmic reticulum. The restoration of chloride secretion has been shown to improve mucocilliary clearance; in individuals with CF, this is expected to alleviate mucus plugging, infection, and inflammation that leads to irreversible structural changes in the lungs, and eventually respiratory failure.
Affygility Solutions has an occupational exposure limit (OEL) and control band assignment for this active pharmaceutical ingredient (API). This monograph also contains the acceptable daily exposure (ADE) value.
OEL Fastrac monographs are a cost-effective and convenient way to meet the requirements for PDEs (ADEs) contained in the EMA Guideline on setting health based exposure limits for use in risk identification in the manufacture of different medicinal products in shared facilities (EMA/CHMP/CVMP/SWP/169430/2012), and to obtain the following information:
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Affygility Solutions’s OEL Fastrac service is an user-friendly system that enables us to make confident decisions and focus on moving the compound safely through our facility. We can quickly access the database to determine if a new drug is within the OEL Fastrac library, and if so, immediately download a detailed OEL banding report. The OEL Fastrac system has been a helpful tool in our overall EH&S program.