Increasing prevalence of cystic fibrosis being witnessed mostly in Europe and the U.S. due to lack of awareness among people towards genetic diseases. In addition, some other factors contributing to the increasing occurrence of cystic fibrosis disease include smoking during pregnancy, hereditary disorder, and premature mortality of infants. The therapeutic pipeline of this particular disease is expected to grow in the future on account of major pharmaceutical companies betting involved in research & development of personalized medicines for the treatment of cystic fibrosis. In addition, various awareness programs and seminar on Cystic fibrosis are also being organized by the government, so as to make the people aware about the disease, its effects, and the possible treatments. Cystic fibrosis is the genetic disorder, which mainly affects the lungs. It is caused due to mutation in CFTR (cystic fibrosis transmembrane conductance regulator) gene. According to Center for Disease Control and Prevention, more than 30,000 people in the U.S. are infected with cystic fibrosis, while ratio of the same in newborn babies in Europe was recorded to be around one out of every 2,000 newborn babies.
Cystic fibrosis is an autosomal recessive disorder, which affects the digestive, respiratory & the reproductive system of the body. Cystic fibrosis involves the production of thick mucus in the lungs and leads to fatal infection. Mucus, a slippery material used to protect respiratory as well as digestive system, becomes abnormally thick in case of cystic fibrosis and causes damage to the lungs. The patients suffering from cystic fibrosis usually face the problem of indigestion. Major symptoms associated with cystic fibrosis or abdominal distention are loss of appetite, difficulty in breathing, gastrointestinal bleeding, persistent coughing, weight loss, bulky stool, excessive sweating, etc.
There are many drugs under clinical trial process which are being developed to be used in the treatment of cystic fibrosis. PBI-4050 is a drug being developed by Prometic Biosciences Inc. PBI-4050 shows the efficacy to treat cystic fibrosis &idiopathic pulmonary fibrosis. It is presently under Phase II clinical trials&is administered orally. PBI-4050 is a small molecule that targets extracellular matrix protein & inhibits their activity. QR-010 is a drug developed by ProQR therapeutics, in collaboration with the European Commission. It is presentlyunder Phase II clinical trials&is also administered orally. QR-010 modulates the CFTR (cystic fibrosis transmembrane conductance regulator) gene. PTI-428 is a drug developed by Proteostasis Therapeutics and is in Phase I clinical trials.PTI-428 drug is administered orally. PTI-428 modulates the CFTR (cystic fibrosis transmembrane conductance regulator) gene. RPL554 is a drug developed by Verona Pharma and is under Phase II clinical trials, currently. RPL554 inhibits the type 3 and type 4 cyclic nucleotide phosphodiestrase & also stimulates the CFTR (cystic fibrosis transmembrane conductance regulator) gene. All the drugs include (PBI-4050, QR-010, PTI-428 and RPL554) have the efficacy to treat Cystic Fibrosis.
Some of the companies having a pipeline of Crohn’s disease therapeutic include Galapagos NV, Prometic Biosciences, ProQR therapeutics, Verona pharmaceuticals, Proteostasis therapeutics, Pfizer Inc., Amgen Inc., Novartis AG, Gilead sciences etc.