GS-248 for Systemic Sclerosis Earns Orphan Drug Designation

Earlier this month, I wrote about how efzitimod, a therapy for systemic sclerosis, received Orphan Drug designation from the FDA. Now, another therapy – this one from Gesynta Pharma (“Gesynta”) – has also been granted this designation for the same indication. According to Healio, GS-248 received Orphan Drug designation in late April 2022 for patients with systemic sclerosis.

Five years ago, Gesynta acquired the rights to GS-248 from Orexo. Now, the therapy has the potential to change the treatment landscape for systemic sclerosis. Scleroderma News Today explains that GS-248, taken orally:

Blocks a protein called microsomal prostaglandin E synthase-1 (mPGES-1), known for its role in boosting inflammation in scleroderma [by promoting] the release of prostaglandin E2 (PGE2), an inflammatory protein. By blocking mPGES-1, GS-248 is believed to reduce inflammation, widen blood vessels and improve blood flow.

Currently, researchers are evaluating the safety, efficacy, and tolerability of GS-248 in a series of two clinical trials. At least one of these trials should be completed by the end of summer 2022.

What is Systemic Sclerosis?

Also referred to as systemic scleroderma, systemic sclerosis is an autoimmune disorder characterized by fibrosis in the skin and organs. In systemic sclerosis, the body mistakenly attacks healthy tissue. The body generates too much collagen, which causes thickened skin and scarring. While some individuals have localized sclerosis (thickening in only one area of the body), systemic sclerosis can also affect the organs. Systemic sclerosis is 4x more likely to affect females than males. Symptoms, which typically manifest in middle age, can include:

  • Raynaud’s phenomenon
  • Swollen fingers and hands
  • Heartburn
  • Dry mouth
  • Unintended weight loss
  • Hardened or thickened skin
  • Painful calcium deposits under the skin
  • High blood pressure
  • Shortness of breath
  • Ulcerating sores on the fingers
  • Gastrointestinal distress
  • Difficulty swallowing
  • Joint pain
  • Organ damage and/or organ failure
Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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