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Shire’s Hemophilia Gene Therapy Granted Orphan Drug Status by FDA

October 27, 2017
Hemophilia Gene Therapy Granted Orphan Drug Status

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Shire’s SHP654, an investigational factor VIII (FVIII) gene therapy for the treatment of hemophilia A. The therapy was formerly known as BAX 888 – Shire changed the name to SHP654 when it acquired Baxalta in June 2016.

SHP654 was developed in part using Shire’s proprietary bioengineered adeno-associated virus serotype 8 (rAAV8). rAAV8 acts as a delivery vehicle, or vector, to carry the genetic codes that prompt FVIII production. This approach, which is being tested in mice with hemophilia A, delivers the corrected FVIII gene specifically into liver cells where the protein is normally generated. If effective, the therapy prompts the sustained production of FVIII and enhanced protection from bleeds.

“This important orphan drug designation highlights Shire’s commitment to patients with rare diseases; and for hemophilia patients specifically our aim is to help them achieve zero bleeds,” says Paul Monahan, MD, Senior Medical Director, Gene Therapy, Shire. “We know that hemophilia care is not one-size-fits-all and that every patient is unique, which is why we continue to focus on optimizing personal outcomes for hemophilia patients by developing innovations to transform care.”

According to a Shire news release, the company also received FDA clearance for an investigational new drug (IND) application it submitted earlier this year to initiate a global multi-center study with SHP654 to evaluate the safety and optimal dose needed to boost FVIII activity to therapeutically significant levels. The IND filing was based on the results of pre-clinical and phase 1 studies demonstrating the potential utility of this candidate. The company anticipates that the SHP654 Phase 1/2 study will begin by the end of 2017.

Source: Shire news release dated October 27, 2017