Cystic Fibrosis | Niche & Rare Disease Landscape & Forecast | US/EU5 | 2021

Cystic fibrosis (CF) is a genetic disease caused by any one of the more than 2,000 mutations identified in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. CFTR mutations lead to aberrant chloride transport in a variety of tissues, most notably in the lungs and pancreas; as a result, patients can develop pancreatic damage, which affects their metabolism and nutrient absorption. These patients can also develop chronic respiratory problems, such as persistent lung infection, stemming from the accumulation of thick, viscous mucus in the lungs. Respiratory failure is the leading cause of death in CF.

Historically, therapeutic options for CF had been limited to symptomatic therapies. However, disease-modifying therapies (DMTs) have become a reality for a subset of CF patients with the market entry of Vertex’s Kalydeco (ivacaftor), Orkambi, and Symdeko, as well as the recent approval of Vertex’s Trikafta, a three-drug combination therapy. Pipeline agents are poised to compete in this lucrative market by seeking to improve the performance of marketed DMTs or expand DMTs’ reach in the CF population.

QUESTIONS ANSWERED

How will the size of the CF population change through 2030? What percentage of the CF population receives drug treatment?

How is CF currently managed in the United States and EU5? What are the most pressing unmet clinical needs according to CF experts?

Which emerging therapies do CF experts consider most promising? If approved, how would emerging therapies influence the management of CF patients? What commercial impact will they have on the CF market?

CONTENT HIGHLIGHTS

Geographies: United States and EU5.

Primary research: Six country-specific interviews with thought-leading pulmonologists supported by survey data collected for this study

Epidemiology: Diagnosed prevalent and drug-treated cases of CF by country, diagnosed prevalent cases of CF by age, most frequent CFTR mutations by country.

Forecast: Drug-level sales and patient share of key CF therapies in 2030

Emerging therapies: Phase III/PR: 1 drug; Phase II: 2 drugs; coverage of select preclinical and Phase I products.

PRODUCT DESCRIPTION

Niche & Rare Disease Landscape & Forecast provides comprehensive market intelligence with world-class epidemiology, keen insight into current treatment paradigms, in-depth pipeline assessments, and drug forecasts supported by detailed primary and secondary research