Opsumit(macitentan), an orally available endothelin receptor antagonist, resulted from a tailored drug discovery process in Actelion’s laboratories.
Tracleer (bosentan), an orally available endothelin receptor antagonist, was the first oral treatment approved for PAH.
Uptravi (selexipag), originally discovered and synthesized by Nippon Shinyaku, is the only approved oral, selective IP receptor agonist targeting the prostacyclin pathway in PAH.
Veletri (epoprostenol for injection), an intravenous prostacyclin, is stable at room temperature (77°F/25°C) for up to 24 hours, removing the need for patients to carry ice packs.
Valchlor (mechlorethamine) 0.016% gel is applied topically once daily to affected areas of the skin. Valchlor is currently only available in the US and is approved for the topical treatment of Stage IA and IB mycosis fungoides-type cutaneous T-cell lymphoma in patients who have received prior skin-directed therapy.
Zavesca (miglustat) available as oral capsules, is a glucosylceramide synthase inhibitor indicated as monotherapy for the treatment of adult patients with mild to moderate type I Gaucher disease for whom enzyme replacement therapy is not a therapeutic option.
In the European Union, Zavesca is also indicated for the treatment of progressive neurological manifestations in adult patients and paediatric patients with Niemann-Pick type C (NP-C) disease, a very rare, invariably progressive and eventually fatal neurodegenerative genetic disorder affecting both children and adults.