FDA Grants Orphan Drug Designation to SH-110, a Liquid Glioma Therapy

• The FDA has granted SH-110 orphan drug designation (ODD) for the treatment of patients with glioma.
• SH-110 is an oral suspension that provides a liquid form of treatment for patients with glioma who have difficulty swallowing.
• FDA orphan drug designation highlights its role in treating a rare, underserved population.

The FDA has granted ODD to SH-110, an oral liquid formulation developed to treat glioma.¹ SH-110 is designed specifically to benefit patients who experience dysphagia, a common and often debilitating condition in glioma, by providing a safe, palatable alternative to capsules or compounded therapies.

Each year in the US, approximately 13,000 adults and 2000 children are diagnosed with glioma, according to the National Brain Tumor Society. These patients often face unique treatment challenges due to the location of the tumor and associated neurologic impairments, including difficulty swallowing. For these individuals, treatment has historically required capsule-based agents that may need to be split, crushed, or compounded in specialty pharmacies, often under suboptimal conditions.

ODD is granted by the FDA to therapies intended for the treatment, diagnosis, or prevention of rare diseases affecting fewer than 200,000 people in the US. The designation provides incentives including tax credits for clinical trials, user fee waivers, and market exclusivity for 7 years upon approval.

“For too long, many patients with glioma and their pharmacists and caregivers had no other choice but to break open capsules and expose themselves to unnecessary hazards,” said Sharon Cunningham, chief executive officer of Shorla, in a press release. “SH-110 is a proprietary product that offers them a safer and more convenient formulation to treat this rare brain cancer.”

The development of SH-110 is part of Shorla’s broader mission to make oncology treatment more accessible and patient-centric. This approach has been seen with the company’s recently FDA-approved Tepylute (SH-105), a ready-to-dilute intravenous formulation of an older agent used for breast and ovarian adenocarcinoma.²

Originally approved in the 1950s, the legacy product was available only as a lyophilized powder that required complicated reconstitution and carried risks for medical staff handling a hazardous drug. Tepylute’s modern formulation streamlines preparation and administration, providing improved safety for both clinicians and patients.

While Tepylute is currently indicated for breast and ovarian cancer, it is also under formulation and manufacturing development as a potential treatment for brain metastases in patients with breast cancer. Commercial availability is projected for the third quarter of 2026.

REFERENCES:

1. Shorla Oncology announces FDA orphan drug designation for SH-110 to treat rare brain cancer using oral liquid. News release. Shorla Oncology. July 15, 2025. Accessed July 15, 2025. https://tinyurl.com/msz5em2b

2. Shorla Oncology announces FDA approval for TEPYLUTE, a novel formulation to treat breast and ovarian cancer. News release. Shorla Oncology. June 28, 2024. Accessed July 15, 2025. https://bit.ly/45Rdtko