Hemophilia B Treatment SPK-9001 Wins Access to EMA’s PRIority MEdicines Program

Hemophilia B Treatment SPK-9001 Wins Access to EMA’s PRIority MEdicines Program

The European Medicines Agency (EMA) will include SPK-9001 — an investigational drug for patients living with hemophilia B —  in Priority Medicines (PRIME), a voluntary European regulatory initiative to support medicines that target an unmet medical need.

PRIME aims to optimize development plans and speed up evaluation so that these priority medicines can quickly reach patients within the 28-member European Union. The announcement was made by SPK-9001’s developers, Spark Therapeutics and Pfizer,

“We are pleased that investigational SPK-9001 has been granted access to the PRIME program, which potentially will help accelerate the delivery of this potential gene therapy to European patients living with hemophilia B,” Spark CEO Jeffrey D. Marrazzo said in a  press release. “We appreciate the EMA’s dedication to moving treatments forward for patients who are underserved with current options, and we look forward to our continued collaboration on SPK-9001.”

SPK-9001 is a new and bioengineered adeno-associated virus (AAV) capsid, or a virus’ protein shell. It carries a high-activity human factor IX variant that is codon-optimized, meaning it generates the ideal DNA sequence for protein production. The drug is being investigated in an ongoing Phase 1/2 trial in the United States.

The study (NCT02484092), is an open-label, dose-escalation clinical trial evaluating SPK-9001 in 15 patients with hemophilia B, a genetic bleeding disorder resulting in the lack of ability to produce blood-clotting factor IX (FIX).

Patients with hemophilia B suffer repeated bleeding events, which can cause chronic joint disease and sometimes death due to the blood’s inability to clot efficiently.

The trial’s primary endpoint is the number of patients experiencing drug-related adverse events after one year of treatment. This will be assessed by physical exam, vital signs, standard clinical lab tests, and with the Bethesda assay for FIX inhibitor. The secondary endpoint is the change from baseline (measured in the beginning of the study) in circulating FIX activity (IU/dL or % normal) after one year of treatment with SPK-9001.

The U.S. Food and Drug Administration (FDA) granted breakthrough therapy designation to SPK-9001, the lead investigational candidate in the companies’ SPK-FIX program, in July 2016. The same drug received FDA’s orphan drug designation in September 2015.

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Daniela holds a PhD in Clinical Psychology from The University of Edinburgh, United Kingdom, a MSc in Health Psychology and a BSc in Clinical Psychology. Her work has been focused on vulnerability to psychopathology and early identification and intervention in psychosis.

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