Chiesi wins FDA nod for rare skin disease gel

Dec. 20, 2023

Chiesi Global Rare Diseases, a Boston-based unit of Italian pharma company Chiesi Group, announced the U.S. FDA has approved its topical gel for the treatment of patients 6 months and older with junctional epidermolysis bullosa (JEB) and dystrophic epidermolysis bullosa (DEB).

Branded Filsuvez, the gel is the first approved treatment for wounds associated with JEB, a rare, moderate-to-severe form of EB with blisters beginning in infancy. 

Known colloquially as ‘butterfly disease,’ EB is a debilitating inherited skin disease that causes a person’s skin to be so fragile it can be injured just from touch. The disorder affects infants, children and adults and recurrent blistering and chronic wounds can be intensely painful.

Chiesi picked up the drug earlier this year through its $1.25 billion acquisition of Amryt Pharma, a Dublin-based biopharma focusing on treatments for rare and orphan diseases. 

Filsuvez, which is made from birch tree bark extract, is administered at home, allowing for integration into existing treatment routines. The gel is applied topically to the wound at each dressing change.

While it's the first drug approved specifically for the JEB type of EB, it's the second drug approved for DEB this year. In May, the FDA approved Krystal Biotech's redosable gene therapy, Vyjuvek, for the treatment of DEB. While Vyjuvek's administration doesn't require as much specialized expertise as traditional gene therapies, it still must be administered by a health care professional, either in a health care setting or in the patient's home.