Global Cystic Fibrosis Market - 2022-2029

Market Overview

Cystic Fibrosis Market is estimated to reach at a CAGR of 13.2% during the forecast period (2022-2029).

Cystic fibrosis (CF) is a life-limiting autosomal recessive genetic disorder that causes severe damage to secretory glands of the body, like the lungs, pancreas, liver, and intestines. It is more common in the lungs. The disease is caused by the mutation of a gene that encodes a chloride-conducting transmembrane channel called the cystic fibrosis transmembrane conductance regulator (CFTR).

Market Dynamics

The major driving forces are the increasing prevalence, rising demand for pipeline drugs, increasing awareness among the healthcare fraternity about cystic fibrosis therapy, and favorable initiatives undertaken by nonprofit organizations.

Favorable initiatives taken by regional and national nonprofit organizations are expected to drive market growth.

One of the primary factors driving growth is the positive actions performed by regional and national nonprofit groups. These projects assist CF patients. These organizations aid in the research & development activities of these therapeutics and help improve the quality of life of these patients. For instance, the North American Cystic Fibrosis Conference (NACFC) provides a collaborative and educational forum for cystic fibrosis professionals to share advances in R&D related to CF therapeutics. Also, a five-year deal was announced on 12 September 2019, according to which the NHS Scotland has decided to provide £ 100,000 per year for cystic fibrosis drugs like Orkambi and Symkevi. Thus, favorable initiatives are driving the market.

Moreover, the key players are operating in the market, collaborating with nonprofit organizations to develop novel therapeutics for the treatment. Some of the drugs in clinical trials are Vertex has completed one Phase II study (NCT03912233) of their next-generation triple combination therapy, VX-121 + tezacaftor + VX-561 (deutivacaftor), in CF patients aged 18 and older with one F508del mutation and one minimal function CFTR mutation, or two F508del mutations. Thus, from the above statements, the market is expected to drive in the forecast period.

Restraint:

However, due to the limited information available, extensive research is essential as CF is a rare disease. The research and development cost for CF is very high, leading to the enormous prices of the end products. The high cost of drugs and treatment is one of the factors that hamper the growth of the cystic fibrosis market.

Industry Analysis

The cystic fibrosis market provides in-depth analysis of the market based on various industry factors such as unmet needs, pricing analysis, supply chain analysis, regulatory analysis etc.

Segment Analysis

CFTR modulators segment is expected to hold the largest market share in the cystic fibrosis market.

The CFTR modulators segment accounted for the largest market share in 2021. This is owing to the increased number of US FDA approvals of the drugs. For instance, Ivacaftor (KALYDECO), a CFTR modulator introduced by Vertex pharmaceuticals, was developed in conjunction with the Cystic fibrosis Foundation (CFF). Also, on June 21, 2019, the US FDA approved SYMDEKO (tezacaftor/ivacaftor and ivacaftor) of Vertex pharmaceuticals to treat Cystic fibrosis in children ages 6-11 years with specific mutations in the CFTR gene. Moreover, On Apr. 19, 2022, The Cystic Fibrosis Foundation invested $5 million in Sionna Therapeutics company. The company is focused exclusively on developing cystic fibrosis transmembrane conductance regulator (CFTR) modulators, a type of therapy that treats the underlying cause of CF. Sionna is developing a pipeline of potential modulators that could benefit people with the most common CF-causing mutation, F508del, and may eventually offer an alternative to currently approved CFTR modulators. The novel modulators target NBD1, the gene region where the F508del mutation occurs. No modulator currently on the market targets NBD1. Thus, from the above statements, the market segment is expected to hold the largest market share in the forecast period.

Geographical Analysis

North American region is expected to hold the largest market share in the global cystic fibrosis market.

North America accounted for the highest revenue share in 2021. The rising prevalence of cystic fibrosis and the presence of government initiatives are the factors to drive in the forecast period. For instance, according to the cystic fibrosis foundation (CFF), there are close to 40,000 children and adults living with cystic fibrosis in the United States (and an estimated 105,000 people have been diagnosed with CF across 94 countries), and CF can affect people of every racial and ethnic group. According to the CFF's national patient registry, the median age of a person with CF is currently 33.4 years. The average life expectancy of CF patients has increased significantly compared to the patients 30 years back. Thus, the increasing prevalence of CF has boosted North America's share in the global cystic fibrosis market.

Furthermore, Hopkins University and Medicine researchers developed a novel treatment for a pathogen in December 2020. Mycobacterium abscesses are a pathogen that is drug-resistant and harmful to persons with lung diseases or cystic fibrosis. Thus, from the above statements, the North American region is expected to hold the largest market share in the forecast period.

Competitive Landscape

Major key players in the cystic fibrosis market are Vertex Pharmaceuticals Inc., AbbVie Inc., F. Hoffmann-La Roche Ltd., Novartis AG, Teva Pharmaceutical Industries Ltd, Gilead Sciences, Allergan, AstraZeneca, Alcresta Therapeutics Inc., and Merck & Co. Inc.

Vertex Pharmaceuticals, Inc.:

Overview:

Vertex Pharmaceuticals, Inc. was founded by Joshua S. Boger in 1989 and is headquartered in Boston, MA. The company discovers, develops, manufactures and commercializes small molecule drugs for patients with serious diseases. It focuses on developing and commercializing therapies for the treatment of cystic fibrosis, infectious diseases including viral infections such as influenza and bacterial infections, autoimmune diseases such as rheumatoid arthritis, cancer, inflammatory bowel disease and neurological disorders including pain and multiple sclerosis.

Product Portfolio:

TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor): TRIKAFTA is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients aged 6 years and older who have at least one copy of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or another mutation that is responsive to treatment with TRIKAFTA.

The global cystic fibrosis market report would provide an access to an approx. 45+market data table, 40+figures and 180pages.