FDA Grants FDA Fast Track Designation for aTyr’s Efzofitimod to Treat SSc-ILD

The U.S. Food and Drug Administration granted Fast Track designation to aTyr Pharma for its lead therapeutic candidate, efzofitimod, for the treatment of systemic sclerosis-associated interstitial lung disease.

Photo: Sanjay Shukla, president and CEO of aTyr

Systemic sclerosis is a chronic, progressive, autoimmune disease characterized by inflammation and fibrosis of connective tissues throughout the body, including the skin and other internal organs. Systemic sclerosis-associated interstitial lung disease, or SSc-ILD occurs in the lungs. It is estimated that approximately 100,000 people in the U.S. are affected by SSc and 55-65 percent may develop ILD. SSc-ILD causes inflammation in the lungs and, if left untreated, can result in scarring or fibrosis that causes permanent loss of lung function. ILD is the primary cause of death in patients with SSc. Current treatment options for SSc-ILD are limited and mainly focus on slowing disease progression and are associated with significant toxicity.

Efzofitimod is a first-in-class immunomodulator that downregulates innate and adaptive immune responses in uncontrolled inflammatory disease states via selective modulation of neuropilin-2 (NRP2). Clinical proof-of-concept was recently established for efzofitimod in a phase 1b/2a study in patients with pulmonary sarcoidosis, a major form of ILD, and the company is currently investigating efzofitimod in patients with pulmonary sarcoidosis in a global phase 3 study.

“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,” said Sanjay Shukla, president and CEO of aTyr. “As highlighted in the ILD sessions at the recent European Respiratory Society International Congress, it is clear that 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.”

Efzofitimod has been shown to reduce lung and skin fibrosis in animal models of SSc and idiopathic pulmonary fibrosis, where it matched or outperformed known anti-fibrotic agents, including nintedanib and pirfenidone. The pathology of SSc-ILD is driven by the same immune cells that are central to pulmonary sarcoidosis pathology, and NRP2 is upregulated on these cells. Efzofitimod has also been shown to reduce key pro-inflammatory markers that are central to this pathology in a clinical study in patients with pulmonary sarcoidosis. Based on these findings, efzofitimod previously received U.S. FDA orphan drug designation for SSc.

The FDA’s Fast Track designation helps facilitate development and expedite the review of drugs to treat serious or life-threatening diseases with unmet need. Fast Track designation provides certain benefits, including more frequent interactions with the FDA throughout the development program, as well as eligibility for accelerated approval, priority review and rolling review.

Author: Rare Daily Staff