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’s Kalydeco.

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.

Some of the highlights from the ongoing transformation of the current market dynamics are as follows:

  • Rapidly Growing Eligible Patient Pool

As recently as 2000’s pediatric population represented, the survival of ~65% of the prevalent patient pool, 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. It is estimated that ~30% of eligible patients are still unable to receive CFTR modulator therapies.

  • Domination of Vertex Pharma as a Market Leader

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 Vertex pharma’s Orkambi in Jan 2012 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. Furthermore, in August 2017, another Vertex Pharma’s product Kalydeco got approved for 23 residual functional mutations. The company did not stop here. Vertex continued to expand its Cystic Fibrosis drug arsenal with the approval of Symdeco and Trikafta in 2018 and 2019, respectively.

  • Promising Market Outlook

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. Increasing prevalence is a major contributing factor driving the growth of the market size. The total Cystic Fibrosis cases 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 CAGR of 3.5%. 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.  Additionally, the development of new therapies shall further drive the increase in the Cystic Fibrosis market. Talking about the market scenario, DelveInsight estimates that United States accounted for the highest market size, with 80.9% of the market share of Cystic Fibrosis as compared to EU5 (19.1%). The total market size of Cystic Fibrosis in 6MM was USD 5,141 Million in 2020.

  • Key Challenges for Vertex Pharma

Several drugs−currently in developmental stages−offer promising alternatives to treat Cystic Fibrosis. Proteostasis Therapeutics has PTI-428, PTI-801, and PTI-808 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. Vertex Pharmaceuticals is also looking forward to expanding its portfolio, with next-generation correcter VX-121 in combination with VX-561 and tezacaftor in an ongoing Phase 2 clinical trial. MRT5005 (Translate Bio Inc) 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, eluforsen by ProQR Therapeutics is a novel RNA-based oligonucleotide targeting patients with F508del mutation. Other Cystic Fibrosis pipeline therapies that are in development include Arrowhead Pharmaceuticals’ ARO-ENaC, Eloxx Pharmaceuticals’ ELX-02, Ionis Pharmaceuticals’ IONIS-ENaC-2.5, and Verona Pharma’s RPL 554.

  • Market Leaders in the Making

With a cornucopia of drugs in the pipeline, the Cystic Fibrosis market size is projected to increase. Among the emerging therapies, the most promising ones include the triple combination of PTI-428, PTI-801, PTI-808 offered by Proteostasis Therapeutics, the triple combination of VX-121, VX-561, and Tezacaftor by Vertex Pharmaceuticals, MRT 5005 by Translate Bio and Eluforsen by ProQr. Their combined market share in the USA is projected at USD 8,868 million in 2030.

  • Key Hurdles in the Sight

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 US $15,571. As cystic fibrosis is one of the rare diseases, which has been extensively researched and well-funded; therefore, many players—Vertex Pharmaceuticals, Novartis, and Pharmaxis—in 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.