Gene therapy for cystic fibrosis receives EC orphan drug designation

Child with cystic fibrosis on ventilator

The European Commission has granted orphan designation for Krystal Biotech’s KB407 for the treatment of cystic fibrosis (CF). 

KB407 is an investigational, redosable gene therapy designed to correct the underlying cause of cystic fibrosis by delivering two copies of the CFTR gene directly to the airway epithelial cells when delivered via a nebuliser.  

By inducing expression of full length, normal CFTR protein in the lung, treatment with KB407 has potential to restore ion and water flow into and out of lung cells to correct the lung manifestations of the disease in patients regardless of their underlying genetic mutation. 

“We are pleased that the European Commission has granted orphan drug designation to KB407, which we believe is an important step toward advancing this option to treat patients with CF,” said Suma Krishnan, President of Research and Development at Krystal Biotech. 

The US Food and Drug Administration has also granted Orphan Drug designation for KB407 to treat patients with CF.

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