The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to an orally available therapeutic currently under development by clinical stage biotechnology company Celtaxsys for the treatment of cystic fibrosis (CF). The drug is a once daily, oral leukotriene A4 hydrolase inhibitor called CTX-4430, which has already received the same designation in the European Union.
The Orphan Drug Designation granted by the FDA’s Office of Orphan Products Development classifies Celtaxsys’ novel drug as a potential treatment for a rare condition that strikes less than 200,000 people in the United States. The status will allow the Company to have marketing exclusivity in the country for 7 years after approval of the product, and provides financial incentives to assist in the product’s further research and development.
“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 the president and CEO of Celtaxsys, Greg Duncan, in a press release. “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,” added Duncan about CTX-4430, which has already been evaluated in a Phase 1 clinical trial with 96 participants and a Phase 1b trial in patients with CF last October.
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The company aims to offer a therapeutic option that can reduce the burden of patients who suffer from this genetic, chronic disease that causes abnormal mucus buildup in the lungs and digestive tract, resulting in a number of life-threatening and debilitating health conditions.
In other FDA-related CF news, the Food and Drug Administration recently granted approval to the eRapid Nebulizer System, the first electronic nebulizer that is specially designed to effectively administer Pulmozyme (dornase alfa), a widely prescribed mucus thinner for the treatment of CF. This breakthrough advancement in CF drug delivery enables the decrease of treatment time for CF patients taking Pulmozyme from 6-8 minutes a day to just 2-3 minutes, making it a preferred drug delivery system among adult and pediatric CF patients compared to traditional nebulizers.