Certa’s FT011 granted orphan drug status in Europe for SSc

Designation grants developer 7 years market exclusivity if therapy is approved
by Patricia Inácio, PhD | July 23, 2024

The European Medicines Agency (EMA) has granted orphan drug status to FT011, an oral therapy candidate that Certa Therapeutics is developing for systemic sclerosis (SSc).

The U.S. Food and Drug Administration (FDA) awarded the therapy a similar status last year, followed by fast track designation this year.

Orphan drug status is given by both agencies to medicines designed for the treatment of serious or life-threatening rare diseases — those affecting fewer than five in 10,000 people in the European Union or fewer than 200,000 individuals in the U.S. Among its benefits is a period of marketing exclusivity should the therapy be approved; that period is seven years in the U.S. and 10 years in Europe.

“With limited treatment options available for patients with SSc, the EMA orphan drug designation and FDA orphan drug and fast track designations reflect the potential for FT011 to address a critical need for people living with this debilitating and life-threatening condition,” Darren Kelly, PhD, founder and CEO of Certa, said in a company press release.
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“These are significant validations for the company and highlight the unmet need for novel medicines to reduce inflammation and scarring in chronic fibrotic diseases, which are often progressive and can have a poor prognosis,” Kelly added.

Orphan drug designation follows award of similar status in US last year
SSc, also known as scleroderma, is an autoimmune disease marked by a self-reactive immune system that causes fibrosis, or scarring, of the skin. It also may affect several internal organs.

FT011 is a first-in-class experimental therapy that blocks the G protein-coupled receptor 68 (GPR68), a driver of tissue scarring. GPR68 is silent under normal conditions, but becomes activated in response to injury.

“GPR68 inhibition modulates the biological pathways causing inflammation and fibrosis to the skin in patients with SSc,” Kelly said.

In the lab, the therapy demonstrated efficacy in studies done using animal models of inflammatory and scarring disease, according to Certa.

Then, a Phase 2 clinical trial (NCT04647890) randomly assigned 30 patients with diffuse cutaneous SSc to one of two daily doses (200 or 400 mg) of FT011 or a placebo. This was given on top of standard-of-care treatment for about three months.

The results showed that 60% of patients treated with the higher, 400 mg dose, as well as 20% of those who received 200 mg had clinically meaningful improvements. That percentage was lower, at 10%, among the participants given the placebo.

FT011 also reduced skin thickness and improved lung function, as measured by forced vital capacity, which assesses the total amount of air a person can exhale after a deep breath. Both patients and physicians tended to favor FT011 over the placebo.
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The company now is planning a Phase 2b study to confirm the benefits of FT011. Additionally, it is developing biomarkers and gene signatures to identify which patients are more likely to respond to the treatment.