Orphan drug designation is designed to aid the development of treatments for serious or life-threatening diseases that afflict less than 200,000 US patients a year. Special incentives are given including, among other things, eligibility for seven-year marketing exclusivity following drug approval, tax credits for clinical research and assistance with the review of clinical trial protocols.
Additionally, orphan drug designation typically results in expedited FDA marketing review times as compared to other drugs.
Avicena recently announced the publication of positive phase I/II data for HD-02 in the journal Neurology. The findings showed that the drug was safe and well-tolerated by patients at a dose of eight grams/day, while resulting in elevated serum and brain levels of creatine.
The results also demonstrated that HD-02 reduced serum 8-hydroxy-2′-deoxyguanosine (serum 8OH2’dG) levels, which are markedly elevated in Huntington’s disease (HD) patients. Some researchers believe that this decrease in serum 8OH2’dG may suggest reduced oxidative injury in patients with HD.
The study’s investigators intend to use the findings from this trial to design late-stage studies of HD-02 aimed at examining the drugs’ ability to slow or halt the progression of Huntington’s disease.