What is Palovarotene?
Palovarotene is a drug studied for chronic obstructive pulmonary disease (COPD), but that showed no positive effects in clinical trials.
How can Palovarotene benefit people living with EXT1/EXT2-CDG?
Defects in the EXT1 and EXT2 genes cause multiple osteochondromatosis (MO), or EXT1/EXT2-CDG, an autosomal dominant disease that causes multiple benign bone tumors that are covered by cartilage (osteochondromas). They grow often on the growing end of the long bones of the legs, arms, and fingers. Palovarotene stopped the growth of these benign bone tumours in a mouse model of the disease. Hence, palovarotene was repurposed for the treatment of EXT1/EXT2-CDG.
A randomized, double-blind, placebo-controlled phase 2 clinical trial (ClinicalTrials.gov NCT03442985) was conducted between 2018 and 2020. The trial was terminated early to analyze the accumulated data and evaluate the efficacy, safety and future of palovarotene as treatment for this CDG. After the evaluation of the results of this clinical trial, the pharmaceutical company Ipsen, was granted authorization to initiate a new trial to evaluate the effect of palovarotene in patients over 14 years of age (ClinicalTrials.gov NCT05027802).
Publications in CDG are constantly growing. We have selected articles of special interest for you, see below. Not all are open access, thus if you wish to access the full information, please contact us at https://worldcdg.org/contact