Date: 2016-10-18
Type of information: Granting of the orphan status in the EU
Product name: Resunab™
Compound: (6aR,10aR)-3-(1,1-dimethylheptyl)-6a,7,10,10a-tetrahydro-1-hydroxy-6,6-dimethyl-6H-Dibenzo(b,d)pyran-9-carboxylic acid
Therapeutic area: Rare diseases - Genetic diseases
Action mechanism: cannabinoid receptor agonist. Resunab™ is a novel synthetic oral drug that is a preferential agonist to the CB2 receptor expressed on activated immune cells. CB2 activation triggers endogenous pathways that resolve inflammation and halt fibrosis. Pre-clinical and Phase 1 studies have shown Resunab to have a favorable safety, tolerability and pharmacokinetic profile. It has also demonstrated promising potency in pre-clinical models of inflammation and fibrosis. Resunab™ triggers resolution of inflammation by increasing production of "Specialized Pro-resolving Lipid Mediators of Inflammation" and anti-inflammatory mediators, while reducing production of pro-inflammatory mediators and reducing the numbers of immune cells in affected tissues. Resunab™ has direct effects on fibroblasts to halt tissue scarring. In effect, Resunab triggers endogenous pathways to turn "off" chronic inflammation and fibrotic processes, without causing immunosuppression.
Company: Corbus Pharmaceuticals (USA - MA)
Disease: cystic fibrosis
Latest news: * On October 18, 2016, Corbus Pharmaceuticals announced that the European Commission has granted Orphan Designation in the European Union for the Company's novel synthetic oral endocannabinoid-mimetic drug, Resunab, for the treatment of cystic fibrosis. Corbus is currently testing Resunab in cystic fibrosis in the U.S. and Europe in a phase 2 , double-blinded, randomized, placebo-control trial that is supported in part by a $5 million development award from the Cystic Fibrosis Foundation Therapeutics, Inc. The study enrolled 89 adults who had CF and forced expiratory volume in 1 second (FEV1) percent predicted at least 40% predicted, without respect to their CFTR mutation, infecting pathogen, or baseline treatment. This Phase 2 trial will evaluate Resunab's safety, tolerability, and potential clinical benefit, as measured by FEV1 and Cystic Fibrosis Questionnaire-Revised Respiratory Symptom scale. The trial also will test the impact of Resunab on bacterial load in the lungs and biomarkers of inflammation in the sputum and blood. As previously reported, enrollment in the trial is complete, and the trial is on track to finish in 2016, with top-line safety and efficacy results anticipated early in 2017. * On October 13, 2015, the FDA has granted orphan drug designation for (6aR, 10aR)-3-(1’,1’-dimethylheptyl)-delta8-tetrahydro-cannabinol-9-carboxylic acid (Resunab™) for the treatment of cystic fibrosis. The FDA has also designated as a Fast Track development program to Corbus Pharmaceuticals' Resunab™ for the treatment of cystic fibrosis. The Company recently initiated an international, multi-center, phase 2, double-blinded, randomized, placebo-control clinical study with multiple doses of Resunab in cystic fibrosis supported by a $5 million development award from Cystic Fibrosis Foundation Therapeutics, Inc. The study will enroll approximately 70 adults with CF, irrespective of their CFTR mutation. Corbus Pharmaceuticals looks forward to reporting top-line safety and efficacy results this study at the end of 2016.
Patents:
Submission of marketing authorization application USA :
Submission of marketing authorization application UE:
Withdrawal of marketing authorization application USA:
Withdrawal of marketing authorization application UE:
US authorization:
UE authorization:
Favourable opinion UE:
Favourable opinion USA:
Orphan status USA: 2015-10-13
Orphan status UE: 2016-10-18
Pediatric exclusivit _USA:
Pediatric exclusivity UE: OTC status: Other news:
