CFTR Modulator therapies are being widely accepted by pharmaceutical and biotechnology companies, academic institutes and contract research organizations for various applications.

These modulators are designed to correct the function of the defective protein made by the cystic fibrosis gene. Moreover, CFTR modulators have brought a personalized approach to correct the basic defect in the cystic fibrosis disease using gene class specific therapy. By grouping CFTR mutations into various classes, specific approaches can be developed that address the underlying molecular defect on an individual patient level and may prove useful in other CFTR-related diseases, such as pancreatitis in patients with mild CFTR variants. Also, this approach serves as a paradigm for the treatment of other genetic diseases.

Besides, advanced technologies have yielded a shift in research development towards therapies that target the underlying cause of the disease by enhancing CFTR function. Substantial efforts are being put by players to develop novel technology-based products. For instance, at present, Vertex dominates the entire CFTR modulator market share with its two marketed CFTR modulators, Kalydeco and Orkambi.

In addition, there are a plethora of CFTR modulators currently under different clinical development phase, thereby fueling the growth of the cystic fibrosis therapeutics market. Two types of disease-modifying CFTR modulators i.e. Potentiator molecules and Corrector molecules are the primary area of focus for therapies under development. More than 30 compounds have undergone preclinical investigation to determine their suitability for CFTR modulation. Promising candidates, such as, Ataluren, VX-661 and other potential CFTR modulator compounds are under investigation. In coming years, it is expected that all patients with cystic fibrosis will have a CFTR modulator medication or combination that corrects the underlying defect of their particular disease.

RNCOS is working on a market research report named “Global Cystic Fibrosis Therapeutics Market Outlook 2022” which will provide detailed information about CFTR modulators and other therapeutics for cystic fibrosis. This report is expected to be published in July 2017.

For more information on this report and Table of Contents; please e-mail us at [email protected]

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