Chiesi Group’s Filsuvez is the second drug to win FDA approval for epidermolysis bullosa and the first for junctional EB, a more severe form of the rare skin disease. The birch tree bark-derived drug is from Chiesi’s $1.25 billion Amryt Pharma acquisition.
Krystal Biotech’s Vyjuvek is approved to treat recessive and dominant forms of dystrophic epidermolysis bullosa (DEB), a rare genetic skin disorder. The redosable gene therapy, applied to the skin, delivers a gene that gets cells to produce collagen that DEB patients lack.
The future looks bright for the integrated specialty pharmacy model – for health systems, providers, and most importantly, for patients.
Telaria is developing a gene therapy for the rare skin disorder recessive dystrophic epidermolysis bullosa. The company was formed by Replay, a gene therapy biotech that forms subsidiaries that leverage its platform technologies for writing and delivering genetic medicines.
North Carolina startup Scioderm announced today it will kick off a Phase 3 clinical trial for its lead product, Zorblisa. Its lead compound helps speed up wound closure in patients with Epidermolysis Bullosa – a very painful skin condition among children that stems from extreme skin fragility, and can lead to terrible blistering and itching. Though considered a rare disease […]