HCell Inc. has received Orphan Drug Designation from the U.S. Food and Drug Administration for HC017AA. The treatment is meant for children with alopecia areata, a disease that causes hair loss. The Austin, Texas, biotech company announced the news on January 15, 2018.
Jae Hyun Lim, M.D., Ph.D., is the company’s co-founder and chief science officer. He called the designation a major milestone for the HC017AA program. He also said HCell is preparing a clinical study, since there is a large unmet need for children with this condition.
The FDA gives this status to drugs and biologics for rare diseases. It defines those as conditions that affect fewer than 200,000 people in the U.S. Before granting it, the Office of Orphan Products Development reviews requests from sponsors. If HC017AA is approved, HCell will be eligible for seven years of U.S. market exclusivity. It may also get tax credits for qualified clinical research costs and a waiver of filing fees under the Prescription Drug User Fee Act, subject to certain conditions.
Dr. Lim said the designation brings potential financial benefits. It also fits the company’s plan to seek faster approval pathways in the U.S.
HC017AA is being developed as a non-surgical topical injection. It aims to regrow hair with a proprietary blend of commercially sourced biologic material and the patient’s own tissue. HCell plans to start a human clinical study in the United States.
Alopecia areata is an autoimmune skin disease. When the immune system attacks hair follicles by mistake, hair falls out in patches. It often appears suddenly, within a few days, usually as coin-sized spots. In some cases it spreads to the whole body or to about half of the scalp, and it can come and go. There is no FDA-approved treatment and no known cure.
HCell is privately held and develops treatments for several forms of alopecia. Besides HC017AA, it is working on regulatory steps for HC0100, a treatment for androgenic alopecia in men and women. The company plans preclinical work on that product first, then a human study in the U.S.
