Global CFTR Modulators Market Opportunity, Drug Sales and Clinical Trials Insight 2028 Report Insight:
The approval of first CF transmembrane conductance regulator (CFTR) modulator has changed the face of cystic fibrosis in the era of precision medicine. Cystic fibrosis is a rare, life-shortening genetic disease caused by mutations of the cystic fibrosis transmembrane conductance regulator – or CFTR – gene. These modulators are designed to correct the malfunctioning protein made by the CFTR gene. This drug class has grown considerably from 2012, up to four agents available in the global market. Kalydeco was the first approved CFTR modulatory drug which has shown robust response in the market by significantly increasing the median survival rates in patients. The complexity of the disease has surged the development of several combinational therapies which have the ability to better tackle the complex nature of the disease. To overcome this, Orkambi, Symdeko, and Trikafta have been approved which belong to the class of CFTR combinational drugs. Currently, the market is dominated by combinational drugs owing to their enhanced efficacy in the management of disease and ability to overcome limitations of monotherapy.
The encouraging response of CFTR modulators in the market has led to further research and development activities in this domain. Several promising compounds are currently being evaluated which can be proven to be alternate to VX-770 to overcome gating mutations. Vertex pharmaceutical is conducting clinical trial on VX-561 (also called deutivacaftor) which an analogue of VX-770 in which one of the tert-butyl groups was replaced by a per-deuterated one. The preclinical data has demonstrated that the drug has similar activity as VX-770; however it has enhanced stability suggesting it could be prescribed once daily only, contrary to VX-770, which must be administered every 12 hours. The company is also developing another corrector, VX-121, which has reached clinical phase 3. It is being assessed in combination with VX-661, VX-770, and/or VX-56.
Although CFTR modulators have shown to dominate the cystic fibrosis treatment market, but there are still several challenges which need to be overcome. The currently available CFTR modulators drugs are target the underlying cause of cystic fibrosis. However, some patients with rare CFTR mutations are still ineligible to receive currently marketed therapies. Therefore, the opportunity remains for developers to investigate CFTR modulators for less common mutations. In addition, resistance against these drugs is another challenge which needs to be overcome.
As per our analysis, the global CFTR modulator market is expected to surpass US$ 25 Billion by 2028. The increasing prevalence and increasing funding from several nonprofit organizations including, Cystic Fibrosis Foundation, Cystic Fibrosis Worldwide, South African Cystic Fibrosis Trust, and South African Cystic Fibrosis Association, will drive the market growth but also encourages the research and development activities in cystic fibrosis. These organizations are investing huge amount of funds to find various CFTR modulators for cystic fibrosis and to support the research and developmental activities by prominent players. Further, robust clinical pipeline of drugs which will gain approval during forecast period will also boost the growth of market.
Our report “Global CFTR Modulator market” enables the key stakeholders about the major trends, drivers, investments, and government initiatives toward the disease treatment. The report also provides comprehensive details of the existing pure-play companies and new players entering the market with key drugs in research and development. Moreover, the report provides insights about the major challenges that are going to hamper the market growth during the forecast period. Additionally, report provides information on the globally approved CFTR modulators along with their patent, price, dosage, and sales analysis till Q1 2022.