FDA Orphan Drug Designation Granted to Hemophilia Gene Therapy; Clinical Studies to Begin Soon

Mathew Shanley

The U.S. Food and Drug Administration (FDA) has granted Shire with orphan drug designation for its gene therapy candidate SHP654, intended to treat hemophilia A. The drug includes technology acquired from Chatham Therapeutics, LLC, a spin-out of Asklepios Biopharmaceutical, Inc.

Gene therapy is predicated around the delivery of a functional copy of a defective gene. For hemophilia A specifically, the gene in question is FVIII. SHP654 is being studied as a potential treatment for patients with the condition via the delivery of a long-term, constant level of the coagulating factor VIII. The gene therapy candidate was a developed molecule from Baxter/Baxalta (where it was designated BAX 888). When Shire acquired Baxalta in June 2016, the molecule was renamed to SHP654.

Hemophilia A is a genetic bleeding disorder and the most common type of hemophilia, affecting approximately 14,000 people in the United States. It is typically the result of patients not having enough  factor VIII to combine factors IXa and X in the clotting cascade. Most treatments for hemophilia A consist of factor replacement therapy, but an estimated one-third of patients with hemophilia A develop inhibitors to standard factor VIII replacement therapies, limiting treatment options.

SHP654 uses an adeno-associated virus stereotype (AAV8) vector to deliver a codon-optimized, B-domain deleted FVIII (BD-VIII) specifically to a patient’s liver. There, FVIII would be manufactured and used to regulate bleeds. The primary goal of the potential therapy is to provide a constant level of factor expression over several years and eliminate the inconsistency often associated with factor replacement therapy.

“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, M.D., Shire’s Senior Medical Director of Gene Therapy 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.”

In addition to the orphan drug designation, the FDA also awarded Shire an investigational new drug (IND) status for the potential therapy earlier this year. A phase 1/2 study is expected to begin in the final quarter of 2017.

For more information from the FDA, including applications, designations and approvals, follow Rare Disease Report on Facebook, Twitter and LinkedIn.

Original image courtesy of WikiMedia Commons.
Printer Printing...