Fast track status to CF drug in US

April 4, 2020 0 By FM

Fast Track designation has been granted for MRT5005 by the US FDA for the treatment of cystic fibrosis (CF).  

MRT5005, the first mRNA therapeutic with delivery to the lung, is designed to address the underlying cause of CF, regardless of genetic mutation, by delivering mRNA encoding the fully functional cystic fibrosis transmembrane conductance regulator (CFTR) protein to cells in the lung through nebulisation. 

The phase 1/2 clinical trial of MRT5005 is currently ongoing.

The randomized, double-blind, placebo-controlled phase 1/2 clinical trial of MRT5005 will enroll at least 40 adult patients with CF who have two Class I and/or Class II mutations. The primary endpoint of the trial will be the safety and tolerability of single and multiple escalating doses of MRT5005 administered by nebulization, Translate Bio said.