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According to a new research report by RNCOS entitled, “Global Cystic Fibrosis Therapeutics Market Outlook 2022”, there have been a lot of developments in the cystic fibrosis therapeutics industry in last few years. Owing to the growing population, the incidences of cystic fibrosis is proliferating, further driving the demand of cystic fibrosis therapeutics. As a result, expanding cystic fibrosis therapeutics demands has enabled companies to capitalize on the technological innovations for expanding their product portfolio accompanied by increased research work. A number of promising cystic fibrosis products with different modes of action are in development.
 
One of the most advanced is ataluren (Translarna; PTC Therapeutics), a small-molecule agent that putatively makes ribosomes less sensitive to stop-codons. Phase III clinical trials are currently in progress in Europe and in the US. However, in May 2014, ataluren received a positive opinion from the Committee for Medicinal Products for Human Use at the European Medicines Agency. Moreover, Arikace (Insmed Incorporated), a liposomal formulation of the antibiotic amikacin, has successfully filed an MAA within the European Union for the treatment of individuals with CF who had been colonized with P. aeruginosa.
 
Similarly, Vertex Pharmaceuticals, in collaboration with the CFF, initiated a Phase III trial for lumacaftor (also known as VX-809), both alone and in combination with Tezacaftor (VX-661) (in Phase III) for the ΔF508 CFTR mutation. In March 2012, the UK Cystic Fibrosis Gene Therapy Consortium initiated a Phase II trial of a gene therapy (GL67A/pGM169). The product is a combination of a cationic liposome (GL67A) and plasmid DNA encoding CFTR (pGM169).
 
Furthermore, cavosonstat/N91115 (Nivalis Therapeutics Inc), a small-molecule agent that has the potential to address a defect in CFTR, resulting from mutations in the CFTR gene, the underlying cause of CF), is in Phase II development.
 
In addition, NM-001 (Lynovex; NovaBiotics), a biologically active oral antibacterial–mucolytic peptide has completed Phase IIa development for cystic fibrosis. Also, orphan drug designation has been granted by the FDA (in the US) and COMP (in Europe) for Lynovex in the treatment of cystic fibrosis.
 
Another pipeline therapy is Sollpura (Anthera Pharmaceuticals), which is in Phase III trials for the treatment of exocrine pancreatic insufficiency. 
 
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