Shire, a global biotechnology leader in rare diseases, announced the FDA awarded Orphan Drug Designation to Shire’s gene therapy candidate SHP654 (also designated as BAX 888), an investigational factor VIII (FVIII) gene therapy for the treatment of hemophilia A. The regulatory agency also granted Shire investigational new drug (IND) status for SHP654.

Orphan Drug Designation is intended to advance drug development for rare diseases. The FDA provides Orphan Drug Designation to drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S. Hemophilia A is the most common type of hemophilia, a bleeding disorder that causes longer-than-normal bleeding due to a lack of clotting factor VIII (FVIII) activity in the blood. It is estimated that approximately 14,000 people in the U.S. suffer from hemophilia A.

“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,” said Paul Monahan, M.D., 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.”

Gene therapy is the delivery of a functional copy of the defective gene. For hemophilia, this is the gene for factor VIII or factor IX so the body can produce its own factor instead of relying on exogenous factor administration. The goal of hemophilia gene therapy is to provide a constant level of factor expression over several years and eliminate the peaks and troughs associated with factor replacement therapy. Shire is studying SHP654 as a potential treatment for hemophilia A patients through the delivery of a long-term, constant level of factor VIII expression.

Shire also received FDA clearance for the 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 factor VIII activity levels and affect hemophilic bleeding. The company anticipates that the SHP654 phase I/II study will begin by the end of 2017.