According to a story from Financial Buzz, the pharmaceutical company Proteostasis Therapeutics recently announced that the company’s experimental treatment PTI-428 was granted Orphan Drug Designation by the FDA. PTI-428 is in development for the treatment of cystic fibrosis.
Cystic fibrosis is a genetic disorder that primarily affects the function of the lungs. People with cystic fibrosis produce a heavy amount of thick, viscous mucus that can become a breeding ground for infectious bacteria, and people with the disorder a much more vulnerable to lung infections when compared to the general population. As a result, people with the condition often have to take antibiotics for much of their lives. Other symptoms of the disorder include progressively worsening breathing problems, poor growth, sinus infections, clubbing of the digits, infertility in men, and fatty stool. Cystic fibrosis has a major impact on lifespan, with many patients dying their 40s or 50s. Double lung transplantation is often a last resort for people whose lung function has severely declined. To learn more about cystic fibrosis, click here.
PTI-428 works as a transmembrance conductance regulator. The drug candidate is intended to treat cystic fibrosis in patients that have a homozygous F508del mutation that is found in the CFTR gene. The therapy may also be used to supplement another modulator, but can also be used a component of Proteostasis Therapeutic’s proprietary combination drug regimen that includes three different therapies, including PTI-801 and 808. This combination may be effective because 428 has demonstrated an ability to increase the concentration of CFTR protein, and this effect could be enhanced with correctors (801) and potentiators (808).
