FDA Grants Orphan Drug Designation to SH-110 for Oral Suspension in Glioma

SH-110 in Glioma

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The FDA has granted orphan drug designation to SH-110 for the treatment of patients with glioma, enabling access to a liquid formulation for those who have difficulty swallowing.

“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, chief executive officer of Shorla Oncology, stated in a news release. “SH-110 is a proprietary product that offers them a safer and more convenient formulation to treat this rare brain cancer.”

Gliomas represent a heterogeneous group of tumors originating from neuroglial progenitor cells in the brain or spinal cord that are associated with significant morbidity and mortality.2 With respect to mortality, recent data from the United States have shown an overall age-standardized mortality rate of 4.33 deaths per 100,000 population. Moreover, data indicate that health-related quality of life is the primary driver of treatment decisions for up to 81% of patients, with 81% and 79% of patients with low- and high-grade glioma, respectively, favoring quality of life over survival with respect to treatment decisions.

SH-110, now the third oral liquid in Shorla Oncology’s portfolio, is a palatable oral suspension developed for the treatment of patients with glioma who suffer from dysphagia or have difficulty swallowing.1

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

In addition to SH-110, Shorla markets 2 other liquid suspension products: an oral liquid methotrexate (Jylamvo) for the treatment of patients with acute lymphoblastic leukemia (ALL) and other indications, and an oral liquid imatinib (Imkeldi) for the treatment of patients with ALL, chronic myeloid leukemia, gastrointestinal stromal tumors, and other indications.3,4

Oral liquid methotrexate received its first oncologic indications from the FDA in November 2022 for use as part of maintenance therapy in adults with ALL, as single-agent or combination therapy in adults with cutaneous T-cell lymphoma, and as part of combination chemotherapy in adults with relapsed or refractory non-Hodgkin lymphoma.5 In October 2024, the FDA announced it had expanded the agent’s approval to include the treatment of patients with ALL.6

The oral solution of imatinib received FDA approval in November 2024.7

To hear from Kate Gasparini, PharmD, BCOP, BCPPS, of Memorial Sloan Kettering Cancer Center, on the challenges of administering oral therapies without a liquid formulation, click here.

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 16, 2025. https://www.businesswire.com/news/home/20250715632403/en/Shorla-Oncology-Announces-FDA-Orphan-Drug-Designation-for-SH-110-to-Treat-Rare-Brain-Cancer-Using-Oral-Liquid
2. Pöhlmann J, Weller M, Marcellusi A, et al. High costs, low quality of life, reduced survival, and room for improving treatment: an analysis of burden and unmet needs in glioma. Front Oncol. Published online March 20, 2024. doi:10.3389/fonc.2024.1368606
3. Jylamvo. Prescribing information. Shorla Oncology; 2024. Accessed July 16, 2025. https://shorlaoncology.com/pdf/Jylamvo_Final_Label.pdf
4. Imkeldi. Prescribing information. Shorla Oncology; 2024. Accessed July 16, 2025. https://www.accessdata.fda.gov/drugsatfda_docs/label/2024/219097s000lbl.pdf
5. Jylamvo (methotrexate) – new drug approval. Optum Rx. 2022. Accessed July 16, 2025. https://professionals.optumrx.com/content/dam/optum3/professional-optumrx/news/rxnews/drug-approvals/drugapproval_jylamvo_2022-1206.pdf
6. Shorla Oncology announces US Food and Drug Administration (FDA) expanded approval of JYLAMVO (methotrexate), an oncology and autoimmune drug for pediatric indications. News release. Shorla Oncology. October 29, 2024. Accessed July 16, 2025. https://www.businesswire.com/news/home/20241029627376/en/
7. Shorla Oncology announces FDA approval of Imkeldi (imatinib) oral solution, an oral liquid for the treatment of certain forms of leukemia and other cancers. News release. Shorla Oncology. November 25, 2024. Accessed July 16, 2025. https://www.businesswire.com/news/home/20241125044117/en