Efzofitimod Scores FDA Fast Track Designation for SSc-ILD

The experimental therapy is designed to modulate the activity of the protein neuropilin-2

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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The U.S. Food and Drug Administration (FDA) has granted the experimental therapy efzofitimod fast track status as a potential treatment for interstitial lung disease (ILD) associated with systemic sclerosis (SSc).

The designation is given to speed up the development and review of therapies designed to treat serious health conditions. It allows efzofitimod’s developer, aTyr Pharma, to have more frequent meetings with the FDA during the development process.

“This Fast Track designation reflects the potential of efzofitimod to address a significant unmet need for patients with SSc- ILD, which is the leading cause of death in scleroderma patients,” Sanjay Shukla, MD, president and CEO of aTyr, said in a press release.

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The FDA granted efzofitimod orphan drug designation as a potential SSc-ILD treatment earlier this year.

“There is a need for more effective and safer therapies for fibrotic lung diseases, including sarcoidosis and ILD that results from scleroderma. We believe this designation further validates efzofitimod and greatly expands the market potential for this first-in-class therapeutic,” Shukla said.

Efzofitimod is designed to reduce inflammation and fibrosis (scarring) by modulating the activity of neuropilin-2 (NRP2), a protein involved in activating certain immune cells central to the disease processes in SSc-ILD.

The medication also received FDA fast track and orphan drug designations as a potential treatment for pulmonary sarcoidosis, a disease that’s also driven by increased lung inflammation.

Data from a Phase 1b/2a clinical trial (NCT03824392) that tested efzofitimod against a placebo in people with pulmonary sarcoidosis showed it to be generally well tolerated, and suggested that treatment boosted lung function and eased symptoms such as coughing and fatigue.

It also reduced levels of pro-inflammatory markers that are key to developing both pulmonary sarcoidosis and SSc-ILD, according to aTyr. These findings, in combination with preclinical research in SSc animal models, aided the FDA’s decision to give efzofitimod orphan drug designation for SSc-ILD.

aTyr is sponsoring a Phase 3 clinical trial, called EFZO-FIT (NCT05415137), to further assess efzofitimod’s effectiveness and safety profile for pulmonary sarcoidosis.