Cystic Fibrosis Requires More Curative Therapies

Drug developers have a long road of opportunities to address unmet needs in the cystic fibrosis therapeutics market.

Drug developers have a long road of opportunities to address unmet needs in cystic fibrosis (CF) therapeutics market as per GlobalData, a leading data and analytics company.

Cystic fibrosis is a progressive genetic disease that damages multiple organs, including the lungs and pancreas with an average life expectancy of 47 years. The mutations in the CFTR gene that lead to insufficient flow of salt and water in and out of cells are responsible for causing the disease.

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‘Drug developers have a long road of opportunities to address unmet therapeutic needs like fewer intolerances, fewer daily doses, and least side effects in the drug market of cystic fibrosis (CF). This may enhance the psychological, social, and educational support available to CF patients.’

This results in a build-up of thick, sticky mucus that leads to chronic lung infections and severe lung disease. The treatment paradigm for CF has been transforming over the past decade with disease-modifying cystic fibrosis transmembrane conductance regulator (CFTR) modulators. This improves the quality of life and longevity of affected patients.

Drug Market of Cystic Fibrosis

Unlike the rapid growth of these different CFTR drugs in 2012, other drugs having symptomatic relief in CF such as antibiotics, mucolytics, and pancreatic enzyme replacement therapies do not bring a big difference to the market.

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“As there are very few promising pipeline agents in late-stage development for CF, numerous opportunities will remain for drug developers to address these unmet needs in the next decade and beyond. Opportunity remains for developers to investigate CFTR modulators for less common mutations. There are currently only two CFTR modulators in Phase II clinical trials, both being developed by AbbVie*,” says Mandana Emamzadeh, Ph.D., Pharma Analyst at GlobalData.

However, the efficacy of these new drugs is still unclear. Apart from CFTSs, other classes of drugs for the disease include antibiotics, and mucolytics (dornase alfa and mannitol).

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Thus new innovative approaches to target CF along with fewer intolerances, fewer daily doses, quick administration, low maintenance dose, and least side effects are always invited in its drug market over the next decade. This may enhance the psychological, social, and educational support available to CF patients.

Source-Medindia

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