The US Food and Drug Administration (FDA) has granted orphan drug designation to the investigational agent ATX-F8-117 for use in patients with hemophilia A.
The drug is designed to prevent the development of factor VIII (FVIII) inhibitors in patients receiving FVIII therapy or treat patients who already have FVIII inhibitors.
ATX-F8-117 received orphan designation from the European Medicines Agency in November 2014.
“These designations emphasize the need for an effective treatment for hemophilia A patients developing factor VIII inhibitors that occurs in approximately 30% of patients,” said Keith Martin, CEO of Apitope, the company developing ATX-F8-117.
“This results in poor clotting of the blood, leading to severe health issues. This orphan drug designation follows extensive preclinical evaluation, and we look forward to advancing a clinical development program for this important medical condition.”
ATX-F8-117 consists of 2 peptides derived from FVIII. Research conducted by Apitope investigators has shown that ATX-F8-117 induces T-cell tolerance toward FVIII in HLA-DRB1*1501 transgenic mice and decreases FVIII inhibitor formation in mice with FVIII neutralizing antibodies.
About orphan designation
The FDA grants orphan designation to encourage companies to develop therapies for diseases that affect fewer than 200,000 individuals in the US.
Orphan designation provides a company with research and development tax credits, an opportunity to obtain grant funding, exemption from FDA application fees, and other benefits.
If ATX-F8-117 is approved to treat patients with hemophilia A, orphan designation will provide Apitope with 7 years of marketing exclusivity in the US.
