FDA Grants Orphan Drug Status to Gene Therapy Candidate SHP654 for Hemophilia A

The U.S. Food and Drug Administration (FDA) recently granted orphan drug status to SHP654 (BAX 888), an investigational factor VIII (FVIII) gene therapy candidate being developed by Shire for the treatment of hemophilia A.

Gene therapy allows the delivery of a functional copy of the defective gene. In hemophilia A, this is the gene for factor VIII. SHP654 is a gene therapy that uses an adeno-associated virus serotype 8 (AAV8) vector to deliver a modified FVIII gene specifically to a patient’s liver, where FVIII is produced.

“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,” Paul Monahan, MD, senior medical director of gene therapy at Shire, said in a press release.

“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,” he said.

In experiments with genetically engineered animal models of hemophilia A, Shire researchers found that SHP654 led to sustained production of factor VIII. It was also deemed safe and well tolerated.

The FDA’s orphan drug designation is meant to advance drug development for rare diseases that affect fewer than 200,000 people in the United States.

Shire was also granted FDA investigational new drug (IND) status for SHP654. Shire had submitted an IND application for SHP654 earlier this year so the company could initiate a Phase 1/2 global study to evaluate the safety and optimal dose needed to boost factor VIII levels and affect bleeding in hemophila A. The trial is expected to begin by the end of 2017.

The IND application was supported by the results of preclinical studies and a Phase 1 trial that were presented at the Congress of the International Society on Thrombosis and Haemostasis July 8-13, 2017, in Berlin, Germany.

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