FDA Grants Orphan Status to Liquid Glioma Treatment SH-110

The U.S. Food and Drug Administration (FDA) granted orphan drug designation to SH-110, a liquid medication designed to treat glioma, a rare form of brain cancer, in patients who have trouble swallowing pills.

“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,” Sharon Cunningham, CEO of Shorla, said in the news release. “SH-110 is a proprietary product that offers them a safer and more convenient formulation to treat this rare brain cancer.”

SH-110 is expected to improve treatment flexibility and access for patients with glioma, which affects about 13,000 adults and 2,000 children in the U.S. annually, according to the National Brain Tumor Society.

While SH-110 offers broader dosing and administration options to support compliance and coverage, its most significant benefit may be for patients who have difficulty swallowing, known as dysphagia, as per the release.

These individuals often depend on specialized compounding pharmacies or must modify their own capsules to take their medication. SH-110 will be part of an expanding catalog of patient-friendly cancer therapies developed by Shorla and recently approved by regulators, according to Shorla.

“This is a difference-maker in the lives of those who suffer from glioma,” Orlaith Ryan, chief technical officer and co-founder of Shorla, said in the news release. “SH-110 complements our other Shorla treatments that are designed to be easier for patients to use and for caregivers and providers to administer.”

SH-110 marks the third oral liquid in Shorla’s portfolio of accessible cancer therapies and reflects the company’s continued focus on developing treatments that improve both safety and usability.

Orphan drug designation is status granted by the FDA to drugs intended to treat rare diseases or conditions that affect fewer than 200,000 people in the United States, according to the FDA. It provides incentives such as tax credits, user fee waivers, and seven years of market exclusivity upon approval.

More Liquid Suspension Treatment FDA Approvals

Alongside SH-110, Shorla offers two other liquid suspension treatments: Jylamvo, an oral methotrexate for acute lymphoblastic leukemia (ALL) and other cancers, and Imkeldi, a liquid version of imatinib used in ALL, chronic myeloid leukemia, gastrointestinal stromal tumors and additional indications.

Jylamvo was initially approved by the FDA in November 2022 for select uses in adults with ALL, cutaneous T-cell lymphoma, and relapsed or refractory non-Hodgkin lymphoma. The approval was expanded in October 2024 to support treatment of children with ALL as well.

Jylamvo is an orange-flavored liquid form of methotrexate designed to remove the need for pill splitting, crushing, or compounding into a liquid. According to the National Cancer Institute, methotrexate may work by blocking cells from using folic acid to make DNA, which can help destroy cancer cells and reduce immune activity.

Jylamvo also comes in a formulation that remains stable at room temperature for 90 days, so it does not require cold chain storage before dispensing.

“This approval follows Jylamvo’s successful use in adult patients and represents a critical step forward in addressing the unmet need of pediatric care in oncology and autoimmune diseases,” Cunningham said in a previous news release from Shorla Oncology. “We are pleased to offer a convenient, patient-friendly alternative for both adult and pediatric patients in the U.S., as we continue to develop innovative solutions for those with limited treatment options.”

Imkeldi gained FDA approval in November 2024.

References

1. “Shorla Oncology announces FDA orphan drug designation for SH-110 to treat rare brain cancer using oral liquid,” Shorla Oncology. Accessed July 16, 2025.
2. “Shorla Oncology announces US Food and Drug Administration expanded approval of JYLAMVO (methotrexate), an oncology and autoimmune drug for pediatric indications,” by Shorla Oncology. Accessed July 16, 2025.
3. “Shorla Oncology announces FDA approval of Imkeldi (imatinib) oral solution, an oral liquid for treatment of certain leukemias and other cancers,” by Shorla Oncology. Accessed July 16, 2025.

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