Eluforsen CFTR (mutant) expression inhibitor Treatment of cystic fibrosis

摘要

Cystic fibrosis (CF) is a rare genetic disease with a mortality rate of about 1-2% per year and the median survival is to the mid-30s. The most common signs of the disease include progressive damage to the respiratory system and chronic digestive system problems. The severity of the disorder varies among affected individuals. In the last few years there has been significant progress in new therapies for this disease, and the number of deaths from CF has been reduced. New therapies are focused to reverse mucus secretion and sodium hyperabsorption across airway surfaces and submucosal glands, which cause the clinical signs of the condition. Following this line of treatment, eluforsen, a novel experimental therapeutic drug, has shown promising results. This new compound is a single-stranded 33-mer RNA oligonucleotide which helps to synthesize a normal cystic fibrosis transmembrane conductance regulator (CFTR) protein. Preclinical and clinical studies have revealed eluforsen's ability to reverse the loss of CFTR function. Patients treated with this new compound have observed a reduction of clinical signs of the disease. Eluforsen is still in early (I-II) phases of development; however, clinical results are very promising and the Food and Drug Administration has granted the product fast track designation.