HANOVER, Md.,—Processa Pharmaceuticals, Inc., a clinical stage biopharmaceutical company developing products to improve the survival and/or quality of life for patients who have a high unmet medical need, has announced today that the U.S. Food and Drug Administration (FDA) has granted orphan-drug designation to its leading clinical compound PCS499 for treatment of necrobiosis lipoidica (NL).
“We are very pleased that PCS499 has received Orphan Drug Designation from FDA for the treatment of NL. NL can have a major impact on the quality of life of patients and PCS499 will be the first treatment targeted to this condition. We plan to begin our clinical studies in 2018 and to accelerate the development of PCS499 in order to provide clinical benefit to NL patients as soon as possible,” said Dr. David Young, CEO Processa Pharmaceuticals, Inc.
According to Processa’s website, “NL is an idiopathic disease of the dermis, characterized by collagen degeneration, granuloma formation, fat deposition, and endothelial wall thickening. It is a chronic, disfiguring skin condition that develops more commonly in women than in men on the pretibial region of the lower extremities, but can also occur on other areas including the face, scalp, forearm, and trunk. NL usually presents initially as red papules that enlarge to form patches or plaques with an atrophic yellow center. Ulceration occurs in approximately 30% of NL patients, which can lead to more severe complications, such as deep tissue infections and osteonecrosis that can threaten life of the limb. Rare cases of squamous cell carcinoma in the NL region have also been reported.
“Although the pathogenesis of NL remains unclear, various potential pathophysiological mechanisms for development and progression of NL have been proposed. These include: autoimmune, inflammatory response; tissue hypoxia; enhanced platelet aggregation and coagulation; and collagen abnormalities, including fibrosis and increased collagen crosslinking.”
“No approved therapies exist for NL and current treatment approaches are based on empirical evidence. Potent topical steroids are often prescribed as first-line therapy, but there is little data to support their efficacy and use sometimes leads to skin atrophy and scarring,” Processa’s website notes. “Other treatments also have been reported, but results are inconsistent. These include: systemic and intralesional corticosteroids, topical calcineurin inhibitors, anti-platelet agents, various immunosuppressants (chloroquine, thalidomide, TNF-alpha blockers, etc.), and pentoxifylline.”
Approximately 74,000 - 185,000 people in the United States and 200,000 - 500,000 people worldwide are affected by NL. These numbers include non-diabetic patients and 0.3% of all diabetic patients. NL is a multi-faceted disorder affecting the skin and the tissue under the skin and occurs in women/men 20 - 60 years of age with the potential to last for months or years. There is currently no FDA approved treatment and no known biotech or pharma companies developing a drug for NL.
The FDA Office of Orphan Products Development grants Orphan Drug Designation to investigational drugs and biologics intended for the treatment of rare diseases that affect fewer than 200,000 people in the U.S. Orphan drug status is intended to facilitate drug development for rare diseases and may provide several benefits to drug developers, including seven years of market exclusivity upon regulatory product approval, exemptions from certain FDA application fees, and tax credits for qualified clinical trials costs.