Gesynta Pharma’s GS-248 Wins FDA Orphan Drug Designation

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by Patricia Inácio, PhD |

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The U.S. Food and Drug Administration (FDA) has granted orphan drug status to Gesynta Pharma’s GS-248, its oral therapy for people with systemic sclerosis (scleroderma).

Orphan drug status aims to encourage therapies for rare diseases through benefits such as seven years of market exclusivity and exemption from FDA fees.

“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 a press 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,” Stenberg said.

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Gesynta submitted an investigational new drug (IND) application last year to conduct clinical trials in the U.S.

GS-248, initially developed by Orexo, blocks a protein called microsomal prostaglandin E synthase-1 (mPGES-1), known for its role in boosting inflammation in scleroderma and other conditions.

mPGES-1 promotes the release of prostaglandin E2 (PGE2), an inflammatory protein. By blocking mPGES-1, GS-248 is believed to reduce inflammation. It’s also been reported to widen blood vessels and improve blood flow.

This may be particularly helpful for people with scleroderma who develop Raynaud’s phenomenon, a painful condition marked by small blood vessels that supply blood to the fingers and toes narrowing.

In a Phase 1 trial (NCT04036227) in Sweden, GS-248 given once per day for 10 days to healthy volunteers was found safe and tolerable. Moreover, it blocked mPGES-1, led to a reduction in PGE2 release, and promoted vasodilation.

In a Phase 2 trial (NCT04744207) underway in Belgium, the Netherlands, Poland, and the U.K., up to 80 scleroderma patients with Raynaud’s will be assigned randomly to GS-248 (120 mg) or a placebo. Each will be given daily for four weeks.

The trial’s main aims include assessing the safety and effectiveness of GS-248 in diminishing Raynaud’s phenomenon. Additional goals include assessing its effect on blood flow.

Enrollment is active and more information is available here. Top-line data are expected this year.

“We are very pleased with the progress Gesynta Pharma has made with our former OX-MPI (GS-248) project. Obtaining orphan drug status in the U.S., is a true milestone to ensure the pharmaceutical reaches the patients suffering from systemic sclerosis as soon as possible,” Nikolaj Sørensen, Orexo’s president and CEO, said.

“We are looking forward to see the result of the ongoing phase 2 trial later this year. With a successful outcome we are confident Gesynta Pharma, with their experienced and competent management team, are well positioned to develop OX-MPI (GS-248) to a commercial success,” he said.

Orexo is entitled to receive double digit shares on any future revenues from GS248.