03|26|2020 Orphan Technologies Receives Rare Pediatric Disease Designation from FDA for OT-58 to Treat Cystathionine B-synthase Deficiency Homocystinuria
LEXINGTON, Mass.–(BUSINESS WIRE)–Orphan Technologies, a company dedicated to helping patients control their homocysteine levels, today announced that OT-58 for the treatment of cystathionine beta synthase deficiency homocystinuria has been designated a rare pediatric disease (RPD) by the US Food and Drug Administration (FDA).
03|02|2020 Orphan Technologies Publishes Study Showing Prevalence of Classical Homocystinuria Substantially Higher than Previously Published Estimates
LEXINGTON, Mass.–(BUSINESS WIRE)–Orphan Technologies, a company dedicated to helping patients control their homocysteine levels, published study showing that 12,113 patients in the United States are diagnosed with classical homocystinuria (HCU) using strict ICD-10 diagnostic criteria in the U.S. This prevalence rate is substantially higher than previously published estimates but in-line with genetic modeling estimates.
10|25|2019 Data from Orphan Technologies’ prospective Natural History Study in classical homocystinuria demonstrates that current treatments do not prevent severe disease outcomes
Orphan Technologies presented 3 Posters at SSIEM 2019 demonstrating:
- Current standard of care does not adequately control homocysteine levels in patients with classical homocystinuria
- Patients receiving care at centers of excellence still experience ocular deficits, inflammation, and metabolic and liver dysfunction
- Many diagnosed and treated patients have severe cognitive impairment and increased skeletal fragility despite young age
02|06|2019 Orphan Technologies Announces First Patients Treated in Phase 1/2 Trial of OT-58 in Homocystinuria
- Rare metabolic disorder causes severe cardiovascular, skeletal, neurologic, and ophthalmic consequences with no adequate treatments
12|07|2018 Orphan Technologies Presents Data Indicating that the Prevalence of Homocystinuria is Substantially Higher than Previously Estimated
- Data presented at ISPOR Europe 2018 demonstrates that the prevalence of homocystinuria (HCU) is greater than that of phenylketonuria (PKU)
- Study demonstrates that newborn screening fails to capture the vast majority of homocystinuria cases
02|20|2013 Orphan Technologies to Develop New Treatment for Life-Threatening Metabolic Disorder from the University of Colorado
Licensing and collaboration agreements enable development of enzyme replacement therapy for Homocystinuria, a rare metabolic disorder