Shire Submits Application To US FDA For Gene Therapy For Hemophilia

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LONDON (Alliance News) - Biotechnology firm Shire PLC said on Thursday it has submitted an application to a US regulator for a gene therapy drug that treats hemophilia.

The FTSE 250 company filed an investigational new drug application to the US Food & Drug Administration for candidate SHP654, also designated as BAX 888, a gene therapy for the treatment of hemophilia A.

Candidate SHP654 aims to protect hemophilia A patients against bleeds through the delivery of a "long-term, constant level of factor expression".

The company said the application, which was based on the results of pre-clinical and phase one studies, to the FDA represented the "latest step forward for Shire's gene therapy program, which shows promise for both hemophilia A and B populations".

Hemophilia A, the most common type of hemophilia, is a rare bleeding disorder that causes longer-than-normal bleeding due to lack of clotting in the blood.

The severity of hemophilia A is determined by the amount of factor VIII in the blood, with more severity associated with lower amounts of factor, according to the World Federation of Hemophilia.

Shire said that more than half of patients with hemophilia A have the severe form of the condition and about 25-30% of individuals with severe hemophilia A develop inhibitors, which occurs when a person with hemophilia has an immune response to treatment with clotting factor concentrates.

"Shire is leveraging decades of scientific leadership in hemophilia to advance research in gene therapy for this community," said Paul Monahan, senior medical director of gene therapy at Shire.

"Drawing from our rich heritage, Shire is well equipped to sustainably support the development of gene therapies that aim to advance current standards of care and minimize the burden of this disease. SHP654 uses a proprietary technology platform designed to produce sustained levels of factor similar to the natural mechanisms of the body. Our goal with gene therapy for hemophilia is to uphold the highest standards for safety and efficacy."

Shares in Shire were up 0.3% to 4,347.00 pence on Thursday.

By Maryam Cockar; [email protected]; @MaryamCockar

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