FDA Grants Necrobiosis Lipoidica Orphan Drug Designation


The rare condition affects the skin and can lead to ulceration and scarring, however, there are currently no FDA approved treatments for necrobiosis lipoidica.

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The US Food and Drug Administration (FDA) has granted Orphan Drug Designation to Processa Pharmaceuticals, Inc. for the company’s clinical compound PCS499 for the treatment of Necrobiosis lipoidica (NL). Processa Pharmaceuticals intends to begin clinical studies of PCS499 this year.

"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,” said David Young, PharmD, MS, CEO of Processa Pharmaceuticals, Inc. in a statement.

Necrobiosis lipoidica is a disorder affecting the skin and the tissue beneath the skin that is often, but not exclusively, associated with diabetes. The condition can lead to ulceration and scarring. It occurs in women and men from 20 to 60 years of age and has the potential to last for months or even years.

The condition affects about 74,000 to 185,000 patients in the United States and 200,000 to 500,000 people around the globe. These numbers include 0.3% of patients with diabetes as well as non-diabetic patients.

Currently, there is no FDA approved treatment for patients with necrobiosis lipoidica and no other known drugs for NL in development. The Orphan Drug designation may be granted to drugs and biologics intended for rare diseases that affect fewer than 200,000 people in the United States. The designation grants access to 7 years of market exclusivity upon regulatory approval, certain exemptions from FDA application fees, and tax credits for clinical trial costs that qualify.

“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 Young.

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