FDA Grants Orphan Drug Status to Cudetaxestat

Cudetaxestat, a potential anti-scarring medication, has been granted orphan drug status by the U.S. Food and Drug Administration (FDA) for the treatment of scleroderma.

“There are limited options and high medical need for patients suffering from systemic sclerosis [SSc, or scleroderma],” Daven Mody, vice president of regulatory affairs with Blade Therapeutics, the company developing cudetaxestat, said in a press release.

“We look forward to continuing our clinical development efforts for cudetaxestat in lung fibrosis [scarring] and potentially exploring other areas of high medical need such as SSc,” Mody said.

Orphan drug status provides companies with incentives to support the development of medicines that might treat rare conditions, defined as those affecting fewer than 200,000 people in the U.S. This is the second orphan drug designation for cudetaxestat — a first was granted in February for the potential treatment of idiopathic pulmonary fibrosis (IPF).

Also known as BLD-0409, Cudetaxestat is an oral, small molecule that works by inhibiting autotaxin, an enzyme involved in the production of a lipid (fat) signaling molecule, LPA, in a reversible manner.

Autotaxin has been shown to promote fibrosis (scarring), and its excessive activity and levels have been observed in various fibrotic diseases in response to cell or tissue damage. High levels of LPA lead to activation of myofibroblasts, the cells responsible for the synthesis and buildup of scar tissue.

According to Blade, cudetaxestat has shown anti-fibrotic activity supporting its potential for the treatment of lung and liver scarring. Data from Phase 1 studies indicate that the therapy is well-tolerated and has a positive safety profile.

The company is launching a Phase 1 clinical trial to assess the effects of cudetaxestat on the way in which two medications used for IPF treatment — Ofev (nintedanib) and Esbriet (pirfenidone) — move into, through, and out of the body, a process called pharmacokinetics.  That trial (NCT04939467) is now enrolling up to 86  healthy volunteers; contact information can be found here.

It is expected to be completed in the first quarter of 2022.

According to Blade, the trial’s results will help define the design of a Phase 2 study planned for 2022 to evaluate the safety and efficacy of cudetaxestat in people with IPF.

Research involving rats has shown that cudetaxestat does not interact with Ofev when the two treatments were administered at the same time.