Lumacaftor/Ivacaftor longer trial on Heterozygous F508del Cystic Fibrosis Patients
At the end of November, the results of a clinical trial of Lumacaftor/Ivacaftor, also known as Orkambi, in CF patients with one F508Del mutation were published in the Annals of the American Thoracic Society. A past trial for Orkambi in this type of CF patient (heterozygous F508del) lasted 28 days …
Drug Development Pipeline for Cystic Fibrosis treatment
The CFF has a interesting and easy to use Drug Development Pipeline webpage which shows the stages of medication that is being developed for Cystic Fibrosis treatment. The stage of development of each medication is clearly indicated and the medications are divided into categories of therapeutic approach. The categories are: Restore CFTR …
Matching Volunteers to Clinical Trials by App
It takes on average 17 years for clinical breakthroughs in the laboratory to reach patients. A big part of the delay is finding enough patients to take part in clinical trials. Only 5 to 10 percent of eligible adults enroll—and of those who show initial interest, only 5 percent …
In the News: Inhaled Levofloxacin & QIDP Designation
Raptor Pharmaceutical’s MP-376 (Inhaled Levofloxacin) granted QIDP Status by the FDA on March 16, 2016. What is Qualified Infectious Disease Product (QIDP) Designation? QIDP status gives a drug priority review by the FDA, eligibility for Fast Track designation, and an extension of marketing exclusivity. This incentivizes pharmaceutical companies to devote …
We Lobby
A Brief History of Our Lobbying Efforts in 2015 In April, 2015, we sent this letter to the FDA Committee: Sent: Thursday, April 02, 2015 12:17 PM To: PADAC Subject: on line patient community at cysticfibrosis.com Dear Dr. Hong, We run an on line patient community at cysticfibrosis.com. Several …