Blue­print Med­i­cines slips as it changes end­point for tri­al in aim to great­ly broad­en Ay­vak­it la­bel

Blue­print is forg­ing ahead on its slow crawl to broad­en its mar­ket for Ay­vak­it.

Hav­ing nabbed its fourth ap­proval for the drug in 2021 for ad­vanced sys­temic mas­to­cy­to­sis (SM) — a very rare dis­ease in which a type of white blood cells known as mast cells ac­cu­mu­late in the body — Blue­print is tar­get­ing the non-ad­vanced form of the dis­ease, which im­pacts the vast ma­jor­i­ty of SM pa­tients.

How­ev­er, for the sec­ond part of its Phase II tri­al in non-ad­vanced SM, Blue­print an­nounced that it will be swap­ping its pri­ma­ry end­point with a key sec­ondary end­point based on an FDA rec­om­men­da­tion. The tri­al’s pri­ma­ry end­point was orig­i­nal­ly the pro­por­tion of pa­tients with a ≥30% de­crease in to­tal symp­tom score, or TSS, but the new pri­ma­ry end­point will be­come the mean change in TSS.

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