{"id":11733,"date":"2021-02-08T15:10:15","date_gmt":"2021-02-08T09:40:15","guid":{"rendered":"https:\/\/www.delveinsight.com\/blog\/?p=11733"},"modified":"2025-05-06T15:59:51","modified_gmt":"2025-05-06T10:29:51","slug":"cystic-fibrosis-market-size","status":"publish","type":"post","link":"https:\/\/www.delveinsight.com\/blog\/cystic-fibrosis-market-size","title":{"rendered":"Cystic Fibrosis Market: A Rapidly Expanding Playing Field for Mid-Cap Pharma"},"content":{"rendered":"\n<p class=\"has-drop-cap\">Cystic Fibrosis is a rare inherited disorder that causes loss of lung function and damages the digestive system. It has a higher prevalence in the Caucasian population. For years there was no treatment available targeting the underlying cause, however, this changed in 2012 with the launch of <strong>Vertex Pharma\u2019s Kalydeco<\/strong>.<\/p>\n\n\n\n<p>Analysts at DelveInsight expect major changes in the Cystic Fibrosis market landscape in the foreseeable future. The major factors shaping the market outlook include prevalence, monopoly of a pharma company, diagnostics and screening methodologies, entry of other market players in the Cystic Fibrosis market domain, as well as restraints. <br><br>Some of the highlights from the ongoing transformation of the current market dynamics are as follows:<\/p>\n\n\n\n<ul class=\"wp-block-list\"><li><strong>Rapidly Growing Eligible Patient Pool<\/strong><\/li><\/ul>\n\n\n\n<p><strong>As recently as 2000\u2019s pediatric population represented, the survival of ~65% of the prevalent patient pool<\/strong>, has significantly increased with the advent of CFTR therapies. At the end of the forecast period, the share of adult patients is expected to double in number. This also increases the eligible patient pool for an ever-expanding list of available CFTR modulator therapies. <strong>It is estimated that ~30% of eligible patients are still unable to receive CFTR modulator therapies.<\/strong><\/p>\n\n\n\n<ul class=\"wp-block-list\"><li><strong>Domination of Vertex Pharma as a Market Leader<\/strong><\/li><\/ul>\n\n\n\n<p>Earlier only the symptomatic medications were available to treat patients with cystic fibrosis. They did little to correct the damage and improve the survival rates of patients. However, with the launch of <strong>Vertex pharma\u2019s Orkambi<\/strong> <strong>in Jan 2012<\/strong> for patients with G551D mutation who are at least 6 years old, the Cystic Fibrosis therapeutic market landscape transformed. Subsequently, Orkambi was approved in December 2014 for R117H and other gating mutations. Orkambi got approved in July 2015 for patients with F508Del who are at least 12 years old, and in September 2016, the drug received approval for patients who are at least 6 years old. <strong>Furthermore, in August 2017, <\/strong>another<strong> Vertex Pharma\u2019s product Kalydeco<\/strong> got approved for 23 residual functional mutations. The company did not stop here. <strong>Vertex continued to expand its Cystic Fibrosis drug arsenal with the approval of Symdeco and Trikafta in 2018 and 2019, respectively.<\/strong><\/p>\n\n\n\n<ul class=\"wp-block-list\"><li><strong>Promising Market Outlook<\/strong><\/li><\/ul>\n\n\n\n<p>Early screening and newly available therapies undoubtedly have led to an increase in the survival of patients and improved the pace of the market size growth<strong>.<\/strong> Increasing prevalence is a major contributing factor driving the growth of the market size. The total <strong><a href=\"https:\/\/www.delveinsight.com\/report-store\/cystic-fibrosis-epidemiology-forecast?utm_source=blog&amp;utm_medium=article&amp;utm_campaign=v\" target=\"_blank\" rel=\"noreferrer noopener\">Cystic Fibrosis cases<\/a><\/strong> in the 6MM (the US, and EU5 (the UK, Germany, France, Italy, and Spain)) are projected to increase from 69,400 cases in 2020 with a <strong>CAGR of 3.5%.<\/strong> The United States had the highest Cystic Fibrosis prevalent population in the 6 MM. Among the European countries, the UK followed by France had the highest patient burden. Whereas, Spain had the lowest number of cases.\u00a0 Additionally, the development of new therapies shall further drive the increase in the Cystic Fibrosis market. Talking about the market scenario, DelveInsight estimates that <strong>United States<\/strong> accounted for the highest market size, with <strong>80.9%<\/strong> of the market share of Cystic Fibrosis as compared to <strong>EU5<\/strong> <strong>(19.1%).<\/strong> The total market size of Cystic Fibrosis in 6MM was <strong>USD 5,141 Million <\/strong>in 2020.<br><\/p>\n\n\n\n<ul class=\"wp-block-list\"><li><strong>Key Challenges for Vertex Pharma<\/strong><\/li><\/ul>\n\n\n\n<p>Several drugs\u2212currently in developmental stages\u2212offer promising alternatives to treat Cystic Fibrosis<strong>. Proteostasis Therapeutics<\/strong> has <strong>PTI-428, PTI-801, and PTI-808<\/strong> triplets in development with MAA submission planned in 2021. PTI-428, the first CFTR amplifier in development, has also received a breakthrough therapy designation by the FDA. <strong>Vertex Pharmaceuticals<\/strong> is also looking forward to expanding its portfolio, with next-generation correcter <strong>VX-121<\/strong> in combination with <strong>VX-561<\/strong> and tezacaftor in an ongoing Phase 2 clinical trial. <strong>MRT5005 (Translate Bio Inc)<\/strong> is the first mRNA product being developed for the treatment of CF by using mRNA encoding CFTR protein to the lung epithelial cells through nebulization. Meanwhile, <strong>eluforsen<\/strong> by <strong>ProQR Therapeutics<\/strong> is a novel RNA-based oligonucleotide targeting patients with F508del mutation. Other <strong><a href=\"https:\/\/www.delveinsight.com\/report-store\/cystic-fibrosis-pipeline-insight?utm_source=blog&amp;utm_medium=article&amp;utm_campaign=v\" target=\"_blank\" rel=\"noreferrer noopener\">Cystic Fibrosis pipeline therapies<\/a><\/strong> that are in development include <strong>Arrowhead Pharmaceuticals\u2019 ARO-ENaC, Eloxx Pharmaceuticals\u2019 ELX-02, Ionis Pharmaceuticals\u2019 IONIS-ENaC-2.5, and Verona Pharma\u2019s RPL 554<\/strong>.<\/p>\n\n\n\n<ul class=\"wp-block-list\"><li><strong>Market Leaders in the Making<\/strong><\/li><\/ul>\n\n\n\n<p>With a cornucopia of drugs in the pipeline, the <strong><a href=\"https:\/\/www.delveinsight.com\/report-store\/cystic-fibrosis-market?utm_source=blog&amp;utm_medium=article&amp;utm_campaign=v\" target=\"_blank\" rel=\"noreferrer noopener\">Cystic Fibrosis market size<\/a><\/strong> is projected to increase. Among the emerging therapies, the most promising ones include the triple combination of P<strong>TI-428, PTI-801, PTI-808<\/strong> offered by <strong>Proteostasis Therapeutics<\/strong>, the triple combination of <strong>VX-121, VX-561, and Tezacaftor<\/strong> by <strong>Vertex Pharmaceuticals<\/strong>, <strong>MRT 5005<\/strong> by <strong>Translate Bio<\/strong> and <strong>Eluforsen<\/strong> by <strong>ProQr<\/strong>. Their combined market share in the USA is projected at <strong>USD 8,868 million<\/strong> in 2030.<\/p>\n\n\n\n<ul class=\"wp-block-list\"><li><strong>Key Hurdles in the Sight<\/strong><\/li><\/ul>\n\n\n\n<p>Several factors are responsible for the projected rise in the market size. The prevalence is increasing due to increased survival aided by new drugs. Moreover, several promising emerging therapies will have better efficiency. However, several challenges need to be addressed to realize the full potential. Cost is an important factor as the mean annual healthcare expenditures have been estimated to be close to <strong>US $15,571. <\/strong>As cystic fibrosis is one of the rare diseases, which has been extensively researched and well-funded; therefore, many players\u2014Vertex Pharmaceuticals, Novartis, and Pharmaxis\u2014in the market are vying to find the cure for the disease. However, loyalty to Vertex Pharma portfolio products will affect the market share of new entrants.<\/p>\n","protected":false},"excerpt":{"rendered":"<p>Cystic Fibrosis is a rare inherited disorder that causes loss of lung function and damages the digestive system. It has a higher prevalence in the Caucasian population. For years there was no treatment available targeting the underlying cause, however, this changed in 2012 with the launch of Vertex Pharma\u2019s Kalydeco. Analysts at DelveInsight expect major [&hellip;]<\/p>\n","protected":false},"author":14,"featured_media":11734,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"_editorskit_title_hidden":false,"_editorskit_reading_time":0,"_editorskit_is_block_options_detached":false,"_editorskit_block_options_position":"{}","advgb_blocks_editor_width":"","advgb_blocks_columns_visual_guide":"","footnotes":""},"categories":[17],"tags":[16883,16882,16881,1395],"industry":[17225],"therapeutic_areas":[17243],"class_list":["post-11733","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-articles","tag-cystic-fibrosis-market","tag-cystic-fibrosis-treatment","tag-cystic-fibrosis-treatment-market","tag-vertex-pharmaceutical","industry-pharmaceutical","therapeutic_areas-respiratory-diseases"],"acf":[],"yoast_head":"<!-- This site is optimized with the Yoast SEO Premium plugin v25.8 (Yoast SEO 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