Advanced therapy medicinal product (ATMP)
Development and Regulatory status
Phase III Clinical Trials
Jun 19 · Spark Therapeutics initiated an observational phase III, six-month run-in study (NCT03432520) with an estimated primary completion date of December 2022 
Sep 18 · Has breakthrough therapy and orphan drug status in US .
Single-dose recombinant adeno-associated viral (rAAV) vector composed of a bio-engineered capsid with liver specific enhanced tropism and a codon-optimized expression cassette that encodes the SQ-FVIII variant of a B-domain-deleted (BDD) human F8 gene.
It affects 1:4,000 to 1:5,000 live male births worldwide .
Trial or other data
Jan 20 · No UK trial sites
Dec 18 · Data on the first participants in the ongoing Phase 1/2 clinical trial in hemophilia A found that the 5x1011 vg/kg and 1x1012 vg/kg dose cohorts had dramatic reductions in bleeds and infusions as well as stable FVIII activity with up to 78 weeks of follow-up .
Dec 16 · PI/II trial evaluating safety and efficacy of SPK 8011 in male patients with haemophilia A starts (NCT03003533). The open label study plans to enrol 30 male patients in the US, Australia and Canada. Primary outcome measures are number of study-related adverse events, including clinically significant abnormal laboratory values & changes from baseline in FVIII activity levels after a single outpatient administration of SPK-8011 after 52 weeks. Collection of these data is due to complete Aug 19 .