The Cystic Fibrosis Foundation is pursuing more treatment options for people with cystic fibrosis, entering into a licensing agreement with AbbVie to develop a compound into a modulator therapy, known as a potentiator. Potentiators help the CFTR protein function better at the cell surface.
AbbVie is investigating combinations of other compounds for the development of another CFTR modulator. This could lead to additional treatment options for people with CF. On Monday, the U.S. Food and Drug Administration approved the triple-combination modulator, elexacaftor/tezacaftor/ivacaftor (Trikafta™) for people with CF ages 12 and older who have at least one F508del mutation.
The approval of Trikafta means that more than 90 percent of people with CF could eventually have a highly effective treatment for the underlying cause of their disease. However, not all people with CF will receive the same benefit from this new therapy and other modulators, and some may experience side effects or drug-drug interactions.
William Skach, M.D., senior vice president of research affairs for the Foundation, said he is excited about the potential to identify new therapeutic options for people with CF.
“Modulator therapies represent the most transformative treatment advance in the history of CF. With this collaboration, we are excited about the potential to identify new therapeutic options for people with CF and to continue our relentless pursuit of treatments for the underlying cause of CF for every person with the disease.”
Despite extraordinary progress in helping people with CF live longer and healthier lives, there is still critical work to be done to help all people living with this disease. Learn more about the potential treatments the Foundation is exploring for people with nonsense and other rare mutations that do not respond to modulators.
For more information on this announcement, please see the AbbVie release.