The Avicena Group has announced today that the United States Food and Drug Administration (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 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. 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.
Orphan drug designation is designed to provide developers of orphan drug products with incentives including: eligibility for seven-year marketing exclusivity following drug approval; tax credits for clinical research; reduced NDA filing fees; grants for further clinical research and development; 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. This is due to the fact that those conditions classified as orphan drug diseases are serious or life-threatening and afflict less than 200,000 patients annually in the U.S.