KRYS - Krystal's gene therapy for cystic fibrosis gets orphan drug status in EU

• The European Commission (EC) granted orphan designation to Krystal Biotech's ( NASDAQ: KRYS ) gene therapy KB407 to treat cystic fibrosis (CF).
• KB407 is an investigational, redosable gene therapy aimed at correcting the underlying cause of cystic fibrosis by delivering two copies of the CFTR gene directly to the airway epithelial cells when delivered via a nebulizer, according to the company.
• CF is an inherited disorder which damages the lungs, digestive system and other organs. The disorder affects the cells producing mucus, sweat and digestive juices and makes them thick causing them to block tubes and and passageways.
• The orphan drug status in the EU is aimed at therapies for treating or preventing diseases which affect fewer than five in 10,000 people in the EU. The designation provides incentives, including 10 years' of market exclusivity for the drug, if approved.
• The U.S. Food and Drug Administration had also granted orphan drug designation to KB407 to treat patients with CF.

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