KalVista Pharmaceuticals brings to Chiesi Group the product Ekterly, the first FDA-approved oral drug for acute treatment of swelling attacks from the rare disease hereditary angioedema. Ekterly’s tablet formulation offers a dosing edge compared to injectable HAE medications.

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Chiesi Global Rare Diseases is committing up to $115 million to begin a collaboration on Arbor Biotechnologies’ in vivo gene-editing therapy for primary hyperoxaluria type 1, an inherited liver disorder. It’s the rare disease company’s first foray into genetic medicines.

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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.

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A drug developed by Protalix BioTherapeutics and Chiesi Group is now FDA approved for treating Fabry disease, a rare inherited metabolic disorder. The drug, Elfabrio, is an enzyme replacement therapy.

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