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Actionable news in ONCE: SPARK THERAPEUTICS Inc COMMON STOCK,

Spark Therapeutics Reports Second Quarter 2017 Financial Results and Recent Business Progress

Preliminary interim data for SPK-8011 Phase 1/2 dose-escalation clinical trial in hemophilia A show initial human proof-of-concept in three participants

In the two participants at the initial dose, stable factor VIII levels average greater than 12 percent with no spontaneous bleeds

Investigational LUXTURNATM (voretigene neparvovec) Prescription Drug User Fee Act (PDUFA) date set for
Jan. 12, 2018; granted rare pediatric disease designation by FDA

PHILADELPHIA, Aug. 02, 2017 (GLOBE NEWSWIRE) -- Spark Therapeutics (ONCE), a fully integrated gene therapy company dedicated to challenging the inevitability of genetic disease, announced today financial results for the second quarter of 2017 and recent business progress.

“This has been an unprecedented time at Spark Therapeutics, marked by the achievement of significant regulatory milestones for investigational LUXTURNA™ (voretigene neparvovec). The U.S. Food and Drug Administration (FDA) has accepted for filing the Biologics License Application (BLA) for LUXTURNA, which is the first BLA for a gene therapy for an inherited disease, and we have submitted the marketing authorization application (MAA) to the European Medicines Agency (EMA). Additionally, LUXTURNA has been designated by FDA as a drug for a rare pediatric disease...


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