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DNA helix
SPK-9001, an investigational therapy intended to treat hemophilia B, has been granted access to the European Medicines Agency’s (EMA) PRIority MEdicines (PRIME) program.
The goal of the EMA’s PRIME program is to accelerate the development of therapies that may offer a major advantage over existing treatments or benefit patients with no treatment options.
Through PRIME, the EMA offers early and enhanced support to developers in order to optimize development plans and speed regulatory evaluations to potentially bring therapies to patients more quickly.
To be accepted for PRIME, a therapy must demonstrate the potential to benefit patients with unmet medical need through early clinical or nonclinical data.
About SPK-9001
SPK-9001 is a bio-engineered adeno-associated virus capsid expressing a codon-optimized, high-activity human factor IX variant enabling endogenous production of factor IX. The therapy is being developed by Spark Therapeutics and Pfizer Inc.
SPK-9001 is under investigation in a phase 1/2 trial. Results from this trial were presented at the 2016 ASH Annual Meeting.
The presentation included data on 9 patients who received a single intravenous infusion of SPK-9001 at 5 x 1011 vg/kg. The patients were followed for 52 weeks, with a long-term follow-up of 14 years.
Before their SPK-9001 infusion, the patients required anywhere from 0 to 159 factor IX infusions each year. Three patients had annualized bleeding rates (ABRs) of 0, and the remaining 6 patients had ABRs of 1, 2, 4, 10, 12, and 49.
After SPK-9001, 8 patients were able to stop receiving factor IX infusions and achieved an ABR of 0. One patient had an ABR of 2 and required on-demand factor IX therapy. (All patients stopped receiving factor IX prophylaxis).
The researchers said there were no unexpected vector or procedure-related adverse events, and none of the patients developed factor IX alloinhibitory antibodies.
