Date: 2015-06-01
Type of information: Granting of the orphan status in the EU
Product name: ATX-F8-117 - combination of H-Lys-Lys-Gly-Pro-Arg-Cys(SH)-Leu-Thr-Arg-Tyr-Tyr-Ser-Ser-Phe-Val-Asn-Met-Glu-Gly-Lys-Lys-OH and H-Lys-Lys-Gly-Asp-Asn-Ile-Met-Val-Thr-Phe-Arg-Asn-Gln-Ala-Ser-Arg-Pro-Tyr-Gly-Lys-Lys-OH
Compound: combination of H-Lys-Lys-Gly-Pro-Arg-Cys(SH)-Leu-Thr-Arg-Tyr-Tyr-Ser-Ser-Phe-Val-Asn-Met-Glu-Gly-Lys-Lys-OH and H-Lys-Lys-Gly-Asp-Asn-Ile-Met-Val-Thr-Phe-Arg-Asn-Gln-Ala-Ser-Arg-Pro-Tyr-Gly-Lys-Lys-OH
Therapeutic area: Rare diseases - Genetic diseases - Hematological diseases
Action mechanism: peptide. ATX-F8-117, combines two peptides derived from Factor VIII.
Company: Apitope International (UK)
Disease: hemophilia A
Latest news: * On June 1st, 2015, the FDA has granted orphan drug designation for immunologically active synthetic peptides (ATX-F8-117) for the treatment of hemophilia A. * On November 25, 2014, Apitope announced that the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) has granted orphan medicinal product designation to ATX-F8-117 for the treatment of haemophilia A. Apitope has, through its patented discovery platform, completed the research work to confirm that the two peptides in ATX-F8-117, derived from Factor VIII, have the potential to treat and prevent inhibitor development in haemophilia A patients treated with Factor VIII. ATX-F8-117 is currently in preclinical development for the treatment of Factor VIII inhibitors which develop in approximately 30 percent of patients and results in poor clotting leading to severe health issues for patients. * On 7-9 October 2014, the Committee for Orphan Medicinal Products (COMP) has recommended the granting of an orphan designation for a combination of H-Lys-Lys-Gly-Pro-Arg-Cys(SH)-Leu-Thr-Arg-Tyr-Tyr-Ser-Ser-Phe-Val-Asn-Met-Glu-Gly-Lys-Lys-OH and H-Lys-Lys-Gly-Asp-Asn-Ile-Met-Val-Thr-Phe-Arg-Asn-Gln-Ala-Ser-Arg-Pro-Tyr-Gly-Lys-Lys-OH for treatment of haemophilia A.
Patents:
Submission of marketing authorization application USA :
Submission of marketing authorization application UE:
Withdrawal of marketing authorization application USA:
Withdrawal of marketing authorization application UE:
US authorization:
UE authorization:
Favourable opinion UE:
Favourable opinion USA:
Orphan status USA: 2015-06-01
Orphan status UE: 2014-11-19
Pediatric exclusivit _USA:
Pediatric exclusivity UE: OTC status: Other news:
