Shire gets FDA orphan drug designation for hemophilia A treatment
The US Food and Drug Administration has awarded orphan drug designation - which is intended to advance drug development for rare diseases - and investigational new drug status to Shire's gene therapy candidate SHP654 for the treatment of haemophilia A.
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Paul Monahan, senior medical director of Gene Therapy at Shire, said: "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."
“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.”
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 US. 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 activity in the blood. It is estimated that around 14,000 people in the US suffer from it.
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