The Avicena Group has announced that the FDA has granted orphan drug designation to HD-02, the company's proprietary drug candidate for the treatment of Huntington's disease.
The company recently reported 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. Additional findings demonstrated that HD-02 reduced serum 8-hydroxy-2'-deoxyguanosine (serum 8OH2'dG) levels, which are markedly elevated in HD patients. Some researchers believe that this decrease in serum 8OH2'dG may suggest reduced oxidative injury in patients with Huntington's disease. 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.