aTyr Pharma Receives FDA Fast Track Designation for Efzofitimod (ATYR1923) for Treatment of Pulmonary Sarcoidosis
aTyr Pharma, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted the company Fast Track designation for its lead therapeutic candidate, efzofitimod (ATYR1923), for the treatment of pulmonary sarcoidosis, a major form of interstitial lung disease.
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, and the company is currently investigating efzofitimod in a global pivotal phase 3 study called EFZO-FIT™. Efzofitimod previously received FDA orphan drug designation for the treatment of sarcoidosis.
“The Fast Track designation for efzofitimod underscores the significant need for a new therapy that provides clinically meaningful outcomes for patients living with pulmonary sarcoidosis,” said Sanjay S. Shukla, M.D., M.S., President and CEO of aTyr. “Fast Track designation reinforces the potential of this novel immunomodulator to be a transformative, disease modifying therapy and address a major unmet need for the sarcoidosis community. We are currently investigating efzofitimod in a global pivotal Phase 3 study called EFZO-FIT™ and we look forward to the opportunity to work closely with the FDA to potentially expedite the delivery of a new treatment to patients in need.”
“The designation of Fast Track for efzofitimod is important news for the many sarcoidosis patients who remain on prednisone for controlling their disease,” said Robert P. Baughman, M.D., Emeritus Profess of Medicine at the University of Cincinnati. “The last drugs approved by the FDA for sarcoidosis were prednisone and other glucocorticoids in the 1950s and most sarcoidosis patients with chronic disease remain on prednisone, with or without other agents which have not been approved by the FDA.”
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.
Sarcoidosis is an immune-mediated disease characterized by the formulation of granulomas, clumps of inflammatory cells, in one or more organs of the body, predominantly in the lungs. Almost 200,000 Americans live with pulmonary sarcoidosis and the prognosis ranges from benign and self-limiting to chronic, debilitating disease, with 1 in 5 cases resulting in scarring, or fibrosis, of the lung, which causes permanent loss of lung function and in many cases death. Current treatment options include corticosteroids and other immunosuppressive therapies, which have limited efficacy and are associated with serious side-effects that many patients cannot tolerate long-term.