EN-374 granted rare pediatric disease, orphan drug designations for chronic granulomatous disease | Image Credit: © Araki Illustrations - © Araki Illustrations - stock.adobe.com.

Both rare pediatric disease and orphan drug designations from the FDA have been granted to Ensoma, for the company's lead program, EN-374, for the treatment X-linked chronic granulomatous disease (X-CGD), according to a press release. Ensoma plans to submit an investigational new drug application with the federal agency in the first half of 2025.

CGD, a rare and severe genetic disease, impair the immune system, leaving patients susceptible to recurrent and severe bacterial and fungal infections. These infections can lead to chronic and life-threatening dysregulated inflammation and serious complications.

CGD affects approximately 1 in 100,000-200,000 live births, and the median life expectancy for individuals with the condition is around 45 years. Further X-CGD is the most common form of the condition, impacting 60%-70% of CGD patients. This form of the condition is caused by mutations in the CYBB gene that prevent white blood cells (neutrophils) from fighting infection.

EN-374 is designed to treat X-CGD via delivery of a functional CYBB gene directly to hematopoietic stem cell (HSCs) in vivo. It uses a promoter for selective expression in neutrophils, the primary cell type affected by X-CGD. Successful expression will restore patients’ ability to fight infection.

“These designations from the FDA highlight the significant medical need in chronic granulomatous disease,” said Drew Dietz, MD, MSCR, in a statement.

Dietz is the vice president of clinical development at Ensoma.

"Current treatments, including antibiotics, antifungals, interferon gamma and allogeneic stem cell transplantation, offer limited benefit and/or come with significant burdens. EN-374 represents the first in vivo HSC-directed therapy for X-CGD, building on a mechanism that has been validated ex vivo. Designed to function for any CYBB mutation, this approach offers the potential to improve upon transformative benefits of ex vivo gene therapies in a way that’s simpler, potentially safer and more accessible for patients. Our team is incredibly proud to be at the forefront of advancing this groundbreaking therapy. We remain on track to submit an IND in the first half of this year.”

About FDA designations

Rare pediatric disease designation is granted by the FDA to incentivize development of treatments for serious or life-threatening rare diseases that primarily affect children aged 18 years or younger and impact fewer than 200,000 people in the United States If a Biologics License Application for EN-374 is approved in the United States, Ensoma may be eligible to receive a Priority Review Voucher from the FDA.

Orphan drug designation is granted by the FDA to products that prevent, diagnose or treat a rare disease or condition affecting fewer than 200,000 people in the U.S. The designation affords Ensoma the potential for certain benefits, including up to 7 years market exclusivity after approval, tax credits for qualified clinical trials and exemption from certain FDA fees.

Reference:

Ensoma Receives Rare Pediatric Disease and Orphan Drug Designations for EN-374 for Treatment of Chronic Granulomatous Disease. Ensoma. Press release. February 13, 2025. Accessed February 13, 2025. https://www.businesswire.com/news/home/20250213170131/en/Ensoma-Receives-Rare-Pediatric-Disease-and-Orphan-Drug-Designations-for-EN-374-for-Treatment-of-Chronic-Granulomatous-Disease