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news.arGentis Pharmaceuticals has reported that the FDA has granted orphan drug designation to the company's product candidate ARG201 for the treatment of diffuse systemic sclerosis, also known as systemic scleroderma, a debilitating and fatal autoimmune disease.
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Orphan drug designation entitles arGentis Pharmaceuticals to seven years of marketing exclusivity for ARG201(native type 1bovine collagen) upon regulatory approval, as well as the opportunity to apply for grant funding from the US government to defray costs of clinical trial expenses, tax credits for clinical research expenses and potential waiver of the FDA’s application user fee.
ARG201 has completed an NIH-funded, 168-patient, double-blind, placebo-controlled Phase II clinical trial that demonstrated a statistically and clinically significant improvement in Modified Rodnan Skin Scores (MRSS), a measure of the change in skin thickening and an FDA-mandated endpoint, at 15 months in late phase patients receiving the treatment versus the placebo group (p=0.006). ARG201 was also shown to be safe and well-tolerated.
Tom Davis, CEO of arGentis Pharmaceuticals, said: “This is a very important milestone in bringing ARG201 to tens of thousands of patients with diffuse systemic sclerosis. We are moving as rapidly as possible to make this a reality.”