Shire’s haemophilia A gene therapy wins Orphan status

Shire’s gene therapy candidate SHP654, an investigational factor VIII gene therapy for the treatment of haemophilia A, has been awarded Orphan Drug Designation by US regulators.

The move qualifies Shire for access to various incentives designed to accelerate development of drugs for rare diseases, including tax credits for qualified clinical testing.

Haemophilia A is the most common type of haemophilia, a bleeding disorder that causes longer-than-normal bleeding due to a lack of clotting factor VIII activity in the blood. It is thought that around 14,000 people in the US have the condition.

In patients with haemophilia A, Factor VIII, a clotting protein, is either defective or missing. The goal of 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.

"This important Orphan Drug Designation highlights Shire’s commitment to patients with rare diseases; and for haemophilia patients specifically our aim is to help them achieve zero bleeds," noted Paul Monahan, senior medical director, Gene Therapy, at Shire.

"We know that haemophilia 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 haemophilia patients by developing innovations to transform care."

Shire also won FDA clearance 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 haemophilic bleeding. The Phase I/II study is expected to begin by the end this year.