Krystal Biotech rockets on positive results with Vyjuvek

US gene therapy company Krystal Biotech (Nasdaq: KRYS) saw its shares skyrocket more than 120% to $88.24 by close of trading yesterday, after it announced positive top-line results from the pivotal GEM-3 trial of investigational beremagene geperpavec (B-VEC), now known as Vyjuvek, for the treatment of dystrophic epidermolysis bullosa (dystrophic EB).

The primary endpoint of the trial evaluated complete wound healing of topical Vyjuvek compared to placebo at six-month timepoints and met statistical significance. Vyjuvek is the first non-invasive, topical and redosable gene therapy in development, and the only genetically corrective approach to treat dystrophic EB that has successfully completed a double blinded Phase III trial.

Ireland-based Amryt Pharma (AIM: AMYT) filed a New Drug Application (NDA) for its Oleogel-S10, trade named Filsuvez, for the treatment of the cutaneous manifestations of junctional and dystrophic bullosa earlier this year, but last week the Food and Drug Administration extended the review period for the NDA by three months to February 28, 2022