FDA grants GS-248 orphan drug designation for systemic sclerosis
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The FDA has granted orphan drug designation to Gesynta Pharma’s GS-248, an oral small molecule, selective membrane-associated prostaglandin E synthase-1 (mPGES-1) inhibitor, for systemic sclerosis, according to a company press release.
"The orphan drug designation granted to our drug candidate GS-248 for the treatment of systemic sclerosis provides an opportunity for extended market exclusivity, which is a valuable complement to its strong patent protection,” Patric Stenberg, CEO of Gesynta Pharma, said in the release. “Strengthened by this positive news and our constructive dialogue with the FDA, we continue working towards the goal of being able to offer an effective and safe treatment for patients with systemic sclerosis."
GS-248 is currently undergoing phase 2 clinical trials to evaluate its capacity to normalize vascular blood flow and reduce pain in patients with Raynaud’s phenomenon secondary to SSc, the company added.
In one of those trials — a randomized, double-blind, placebo-controlled study — an estimated enrollment of 80 patients with SSc from four European countries will receive either GS-248 120 mg or placebo once daily. The study, designed to evaluate the efficacy of GS-248 vs. placebo in Raynaud's phenomenon among patients with SSc, will include an enrolment period, a treatment period and a follow-up period, with a total of five visits over approximately 10 weeks. The trial is expected to be completed in June.
According to the company, GS-248 demonstrates a “unique and promising” mechanism of action, which may prove useful in other inflammatory diseases as well.
Gesynta acquired rights to GS-248 from Orexo through an agreement signed in 2017. According to that agreement, Orexo will receive a tiered “double-digit” share of future revenue generated from GS-248 projects.
GS-248 is now the second agent in as many weeks to receive orphan drug status from the FDA for SSc. Biotherapeutics firm aTyr announced on April 13 that the FDA had granted an orphan drug designation for its immunomodulator efzofitimod (ATYR1923) in the treatment of SSc.
In their statement, aTyr said efzofitimod (ATYR1923) downregulates innate and adaptive immune responses in uncontrolled inflammatory disease states through selective modulation of neuropilin-2 (NRP2). The company added that it had established clinical proof-of-concept for efzofitimod in a phase 1b/2a study in patients with pulmonary sarcoidosis, a form of interstitial lung disease (ILD).
Reference:
Orexo´s partner Gesynta Pharma granted Orphan Drug Designation by the FDA for OX-MPI (GS-248) for the treatment of systemic sclerosis. https://orexo.com/news-room/press-releases-and-news/2022-04-27-orexo-s-partner-gesynta-pharma-granted-orphan-drug-designation-by-the-fda-for-ox-mpi-gs-248-for-the-treatment-of-systemic-sclerosis. April 27, 2022. Accessed April 27, 2022.
NIH U.S. National Library of Medicine. https://clinicaltrials.gov/ct2/show/NCT04744207. Accessed April 27, 2022.
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