Irish biotech Shire has received an orphan drug designation from the FDA for its gene therapy candidate SHP654 (BAX 888).
SHP654 is being developed to treat the bleeding disorder haemophilia A, which an estimated 14,000 people in the US suffer from.
Paul Monahan, senior medical director, gene therapy, Shire, said: “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.
“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 optimising personal outcomes for haemophilia patients by developing innovations to transform care.”
According to Shire, the goal of haemophilia 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.
The biotech is aiming to implement this in SHP654 with technology acquired from Chatham Therapeutics by using a AAV8 vector to deliver a codon-optimised, B-domain deleted factor VIII to a patient’s liver, which would then be produced to manage bleeds.
Shire is not without competition however as Roche won an FDA priority review back in August for emicizumab prophylaxis, a once-weekly subcutaneous treatment for patients with haemophilia A, and has ongoing trials with and without inhibitors to treat the same indication.
The FDA is expected to make a decision on the Swiss pharmaceutical firm’s