FT011 granted orphan drug designation from FDA

If drug is approved, only Certa will be able to market it for 7 years

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to FT011, an experimental oral medication that Certa Therapeutics is developing as a treatment for systemic sclerosis.

The FDA gives this designation to investigational treatments for rare diseases, those affecting fewer than 200,000 people in the U.S. The designation comes with certain incentives, most notably a guarantee that, if FT011 is ultimately approved by the FDA, only Certa will be allowed to market the drug, without competition from generics or other formulations, for a period of seven years.

“We are very pleased that the FDA has granted Orphan Drug Designation to FT011 which we believe highlights the urgent need for innovation and new therapeutic options for scleroderma patients,” Darren Kelly, PhD, CEO and founder of Certa, said in a company press release.

Kelly added that the designation “represents an important milestone in the development of FT011, which has the potential to establish first-in-class clinical benefits by precisely targeting the root cause of fibrosis and offer treatment across multiple organs within these patients.”

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Scleroderma, also called systemic sclerosis or SSc, is marked by excessive fibrosis (scarring) that affects the skin and potentially internal organs. FT011 is designed to block the activity of a protein receptor that’s involved in driving fibrosis, but hasn’t been targeted by other medications, according to Certa.

Clinical trial results

The experimental therapy has shown promising efficacy in multiple laboratory models of scleroderma, and has shown the ability to decrease the activity of genetic markers of fibrosis, according to the company.

Earlier this year, Certa announced results from a Phase 2 clinical trial (NCT04647890) that enrolled 30 adults with diffuse cutaneous SSc. Participants were randomly divided into three groups: two groups received FT011, at doses of either 200 or 400 mg per day, on top of standard-of-care treatments, for about three months. The third group was given a placebo.

The study’s goal was to assess the safety and pharmacological properties of FT011, and results were generally positive with no serious safety issues reported and no side effects leading patients to stop the drug. Findings also indicated that 60% of patients given the higher dose of FT011 saw clinically meaningful improvements, compared with 20% of those given the lower dose and 10% of patients given placebo.