ADC Reviews

FDA Grants Fast Track Designation to Efzofitimod for the Treatment of Pulmonary Sarcoidosis

pulmonary sarcoidosis

On August 11, aTyr Pharma announced that the FDA has granted Fast Track designation to Efzofitimod (ATYR1923) for the treatment of pulmonary sarcoidosis. This year, Efzofitimod has been granted two orphan drug designations by the FDA.

Sarcoidosis is an immune-mediated disease characterized by the formation of granulomas, clumps of inflammatory cells, in one or more organs of the body, predominantly invading the lung parenchyma. Nearly 200,000 Americans suffer from pulmonary nodules, with prognosis ranging from benign and self-limiting to chronic and debilitating, with 1 in 5 cases of lung scarring or fibrosis resulting in permanent loss of lung function even death. Existing treatment options include corticosteroids and other immunosuppressive therapies, however these have limited efficacy and are associated with severe side effects.

Efzofitimod is a first-in-class immunomodulatory agent composed of the immunomodulatory domain of histidyl-tRNA synthetase fused to the FC segment of a human antibody. Efzofitimod downregulates innate and adaptive immune responses in inflammatory disease states by selectively modulating neuropilin-2 (NRP2).

In a randomized, double-blind, placebo-controlled Phase Ib/IIa study, clinical proof-of-concept of Efzofitimod in patients with pulmonary nodules was established. The primary endpoint of the study was the safety and tolerability of multiple escalating doses of intravenous efzofitimod. The results demonstrated a favorable safety and tolerability profile for Efzofitimod compared to placebo, with dose-response and benefit trends, including steroid reduction and improved lung function, across key efficacy endpoints and across multiple analyzed populations. In addition, dose-dependent reductions in inflammatory biomarkers also confirmed the anti-inflammatory effects of Efzofitimod.

Currently, the product is in a Phase III EFZO-FIT study. aTyr Pharm is also conducting a Phase II clinical trial in patients with novel coronavirus infection.

In January 2020, aTyr Pharm and Kyorin Pharmaceutical entered into a collaboration on the development and commercialization of Efzofitimod in Japan. Under the agreement, aTyr is eligible to receive up to $175 million in total payments, including an upfront payment of $8 million and milestone payments of $167 million.

“Efzofitimod’s Fast Track designation underscores the huge need for new therapies in the pulmonary sarcoidosis field that provide patients with tangible clinical benefit,” said Sanjay S. Shukla, President and CEO of aTyr. “At the same time, Fast Track designation also Acknowledging the potential of this novel immunomodulator, Efzofitimod has the potential to be a transformative therapy to address an unmet clinical need in the field of pulmonary nodules. We look forward to the opportunity to work closely with the FDA to expedite the delivery of new treatments to patients in need. “