Ivacaftor: A Powerful CFTR Potentiator for Cystic Fibrosis Treatment
Discover the science behind Ivacaftor, a key player in CFTR modulator therapies and a beacon of hope for cystic fibrosis patients.
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CFTR Potentiator
Ivacaftor is a highly effective pharmaceutical chemical that acts as a potentiator for the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein. It is specifically designed to target mutations like G551D and F508del, significantly enhancing the function of the CFTR channel.
- Unlock improved lung function with Ivacaftor's targeted approach to cystic fibrosis treatment, addressing specific CFTR gene mutations.
- Explore the detailed Ivacaftor mechanism of action, which involves increasing the open probability of defective CFTR channels to restore chloride transport.
- Learn about the Ivacaftor oral bioavailability, making it a convenient and effective option for patients requiring consistent therapeutic levels.
- Understand the critical role of Ivacaftor in CFTR modulator therapies, offering new possibilities for managing cystic fibrosis symptoms and progression.
Key Advantages
Enhanced Chloride Secretion
Ivacaftor's primary advantage lies in its ability to significantly increase chloride secretion by holding the CFTR channel gate open, as evidenced in studies involving specific CFTR mutations like G551D.
Targeted Therapy for CF
As a leading CFTR potentiator, Ivacaftor offers a targeted therapy solution for cystic fibrosis patients, providing a crucial treatment option for those with specific genetic predispositions.
Improved Patient Outcomes
The use of Ivacaftor in treating cystic fibrosis has demonstrated improvements in lung function and overall quality of life, making it a cornerstone in modern CF management strategies.
Key Applications
Pharmaceutical Manufacturing
Produced with high purity (often exceeding 98% by HPLC), Ivacaftor serves as a vital active pharmaceutical ingredient (API) for therapeutic formulations.
Drug Discovery and Development
Ivacaftor is extensively used in research to understand CFTR protein function and to develop new therapeutic agents for cystic fibrosis and related conditions.
Clinical Research
The efficacy of Ivacaftor in various Ivacaftor F508del mutation and Ivacaftor G551D mutation patient groups is continually explored in clinical trials.
Biochemical Assays
Its role as a CFTR potentiator makes Ivacaftor an important tool in biochemical assays aimed at studying ion channel function and drug interactions.