Cancer, Scleroderma Treatments Granted Orphan Drug Designation


The FDA has granted orphan drug designation to a potential first-in-class therapy for scleroderma and a PD-1 inhibitor for small cell lung cancer.

The FDA granted orphan drug designation (ODD) to two treatments — one for small cell lung cancer (SCLC) and one for systemic sclerosis.

Shanghai, China-based Shanghai Henlius Biotech said in a news release that the FDA granted the orphan drug status to Hansizhuang (serplulimab) for small cell lung cancer.

The pharma maker plans to file its NDA in China and MAA in the EU in 2022, making Hansizhuang potentially the world’s first PD-1 inhibitor for the first-line treatment of SCLC, the company said.

Henlius has undertaken multiple trials to investigate therapy options for both limited stage and extended stage small cell lung cancer.

Its global multi-center phase 3 study of Hansizhuang for the first-line treatment of ES-SCLC compared the efficacy and safety of Hansizhuang with placebo when combined with chemotherapy (carboplatin-etoposide) in previously untreated patients with SCLC.

This study included 585 patients in 128 sites in China, Turkey, Poland, and other countries. In December 2021, ASTRUM-005 met the primary study endpoint of the overall survival, reducing the risk of death by 38% of the overall population (41% in the Asian subgroup).

“Based on the excellent readouts, the company intends to file NDA for the indication shortly,” Henlius said.

Meanwhile San Diego, Calif.-based aTyr Pharma snagged orphan drug designation for its lead therapeutic candidate, efzofitimod, to treat systemic sclerosis, which is also known as scleroderma.

Efzofitimod is a potential first-in-class immunomodulator that downregulates innate and adaptive immune responses in uncontrolled inflammatory disease states via selective modulation of neuropilin-2 (NRP2), aTyr said in a news release.

Clinical proof-of-concept was recently established for efzofitimod in a phase 1b/2a study in patients with pulmonary sarcoidosis, a major form of interstitial lung disease (ILD).

Efzofitimod has been shown to reduce lung and skin fibrosis in animal models of systemic sclerosisand idiopathic pulmonary fibrosis, where it matched or outperformed known anti-fibrotic agents, including nintedanib and pirfenidone, the company said.

Sanjay S. Shukla, M.D.

Sanjay S. Shukla, M.D.

“The data we have presented in animal models along with the positive findings reported from our recent phase 1b/2a study in pulmonary sarcoidosis patients suggest that efzofitimod has the potential to be a new treatment option that resolves inflammation and subsequent fibrosis,” said Sanjay S. Shukla, M.D., 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.

About100,000 people in the United States are affected by systemic sclerosis and 55% to 65% may develop ILD, which causes inflammation in the lungs and, if left untreated, can result in scarring that causes permanent loss of lung function. ILD is the primary cause of death in patients with systemic sclerosis.

“Current treatment options for SSc-ILD are limited, mainly focused on slowing disease progression and are associated with significant toxicity,” aTyr said.

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