FDA Grants Alopecia Areata Treatment Orphan Drug Designation


HCell announced today that HC017AA, a treatment for Alopecia Areata in pediatric patients, has been granted Orphan Drug Designation by the U.S. FDA.

HCell announced today that HC017AA, a treatment for Alopecia Areata in pediatric patients, has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA).

“The granting of Orphan Drug Designation by the U.S. FDA is a significant milestone for HCell in the HC017AA development program,” said Jae Hyun Lim M.D., Ph.D, Co-Founder and Chief Science Officer at HCell Inc. in a press release. “We are working diligently to prepare for the clinical study for this significant, yet unmet, medical need for Alopecia Areata in pediatric patients.”

Alopecia Areata is an autoimmune disease in which the immune system attacks hair follicles, causing hair to fall out in small, round patches near the scalp. Approximately 2% of the United States population will develop alopecia during their lifetime.

The condition is asymptomatic, with only 14% of patients experiencing a burning sensation in the affected area. Additionally, it is not uncommon for men who suffer from it to experience a loss of facial hair. While it is most common for people to have complete loss of scalp hair, often referred to as alopecia totalis, an extremely small percentage of patients suffer from alopecia univeralis, or a complete loss of hair on the entire body.

HC017AA is a non-surgical treatment for alopecia areata being developed as a topical injection by regenerating hair through a combination of biologic and autologous tissue. With the new designation, HCell Inc. is given 7 years of exclusive U.S. marketing of HC017AA, including tax credits for qualified clinical research costs, and a waiver of the Prescription Drug User Fee Act (PDUFA) filing fees. Through the Orphan Drug Designation, the FDA believes HC017AA offers promising research and data to cure alopecia areata in pediatric patients.

Patients with alopecia will experience many episodes of remission and recurrence throughout their lifetime. The cause of alopecia is unknown, however, researchers are led to believe that the development of the disease is genetic. Per the National Institute of Health (NIH), an estimated 20% of patients with alopecia have a family member who also suffers from the condition. Most patients manage hair loss with corticosteroid injection and topical therapies, and some use cosmetic treatments to hide bald patches. The number of diagnoses among patients with alopecia is growing and there is currently no FDA approved cure for the disease.

“ODD Status from the FDA’s recognition gives HCell Inc. a number of potential financial benefits and is aligned with our ongoing work seeking expedited pathways towards product approval in the United States,” said Dr. Lim.

HCell plans on initiating a human clinical study in the United States to conduct further research.

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