Shire’s Hemophilia Gene Therapy Boosted by Orphan Drug Designation

The FDA has granted orphan drug designation to Shire’s hemophilia A gene therapy, giving it the boost it needs to catch up with others in the field.

Shire specializes in the development of drugs for rare diseases, including hemophilia A – a bleeding disorder caused by a lack of clotting factor VIII activity in the blood. The company’s gene therapy candidate, SHP654, will deliver a functional copy of factor VIII or IX that will allow the body to begin producing a constant level of the factor. This news makes Shire a competitor in a field expected to be worth $25B (€21B) by 2024, which has so far been led by Roche and Spark Therapeutics.

In July, Roche announced the results of a Phase III trial investigating its monoclonal antibody, emicizumab, during which it reduced the bleed rate by 87%. US biotech, Spark, reported preliminary Phase I/II results for its gene therapy approach in August. Its candidate, SPK-8011, reduced the annual bleeding rate of ten patients by 96%. UniQure has focused on hemophilia B but is working on a gene therapy for hemophilia A at the preclinical stage.

Image – Ramona Kaulitzki /

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