Celtaxsys, Inc., a privately-owned, clinical stage drug discovery and development company focused on addressing serious inflammatory diseases, has just announced the U.S. Food and Drug Administration has granted the company’s experimental therapy CTX-4430 (oral leukotriene A4 hydrolase inhibitor) Orphan Drug Designation as a potential treatment for cystic fibrosis (CF). This new designation follows the orphan designation granted last year by the European Medicines Agency, and strengthens CTX-4430’s position as the most advanced immuno-modulator the company has to offer.
“We are pleased to have received orphan drug designation for CTX-4430 to treat the inflammatory component of cystic fibrosis, a disease that still results in unacceptable levels of morbidity and pre-mature mortality,” said Greg Duncan, President and Chief Executive Officer. “By down regulating over activated neutrophils and reducing neutrophil elastase in the lungs, we hope to preserve small airway cell structure, reduce lung clogging and to enhance CF patient lung function over time. Celtaxsys has now been granted orphan designation for CTX-4430 in both the US and the EU, geographies in which 90% of CF patients reside. We are currently scaling capital to progress CTX-4430 into a phase 2 trial later this year in CF for treatment of patients 18 years of age and older.”
The FDA Office of Orphan Products Development grants this particular designation to carefully-selected, groundbreaking biologics indicated for rare diseases that are estimated to affect less than 200,000 patients in the country. If a drug with Orphan Drug Designation is approved, it gives the product 7 years of U.S. marketing exclusivity post-FDA approval, and special financial incentives to fund its further development.
The orally administered CTX-4430 continues to progress through clinical trials, having completed a 96-subject Phase 1 clinical trial in August 2013 and a Phase 1B trial in adult cystic fibrosis patients in October 2014. The company plans to launch Phase 2 trials for CTX-4430 in 2015 for treating adult cystic fibrosis and moderately-severe acne.
In other cystic fibrosis developments, the Welsh Government is set to launch a year-long campaign, called “Time to Talk” that aims to ensure their employees are well-informed of the soon-to-be implemented shift to a soft opt-out system of organ donation, and what choices are available to them. Those who do not register will be considered opted in; participants must opt out if they do not want to be an organ donor. The government is calling on Wales-based private, public and voluntary employers to help promote the campaign beginning December 2015. The government’s next steps to fulfill the campaign’s main objectives are to provide information leaflets at employees’ desks, and put up informational posters in work environments.