Blue­print plots re­turn to FDA with new Ay­vak­it da­ta in rare con­di­tion — and the an­a­lysts cheer

Over a decade af­ter launch, Blue­print Med­i­cines nabbed the first ap­proval for their first drug ear­li­er this year. Now, as they move for­ward with a Roche-part­nered glob­al launch, they’re tout­ing da­ta that could push them in­to more pa­tients.

The Jeff Al­bers-led Cam­bridge biotech re­leased their full piv­otal da­ta for Ay­vak­it in pa­tients with ad­vanced sys­temic mas­to­cy­to­sis. In one 53-per­son study, they showed that 76% of pa­tients re­spond­ed to the drug, 36% had com­plete re­spons­es and that on av­er­age their re­spons­es last­ed for just over 3 years. A small­er, 32-pa­tient study had a 75% re­sponse rate and most were still re­spond­ing af­ter 10.4 months, the last fol­low-up.

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