NBM-BMX Earns FDA Orphan Drug Status for Uveal Melanoma

The FDA has granted orphan drug designation (ODD) to NBM-BMX, an investigational histone deacetylase 8 (HDAC8)-selective inhibitor, for the treatment of uveal melanoma.¹

NBM-BMX is a small-molecule inhibitor designed to modulate tumor DNA repair and survival pathways, specifically focusing on the mechanisms driving metastatic behavior in BAP1-mutant uveal melanocytes.² The agent has demonstrated promising preliminary efficacy in preclinical studies.

Last month, NBM-BMX also received fast track designation (FTD) from the FDA for the treatment of metastatic uveal melanoma.³ This latest designation from the FDA further supports the forthcoming clinical development of NBM-BMX through user fee exemptions, tax credits, and a potential 7 years of market exclusivity upon approval.⁴ These benefits are provided to NBM-BMX in recognition of the agent’s potential in uveal melanoma, a rare malignancy with a high likelihood of metastasis.⁵

“Receiving both [FTD] and now [ODD] underscores NBM-BMX’s promise in addressing a high unmet medical need for patients with uveal melanoma,” said John Soong, MD, CEO of NovelWise Pharmaceutical Corporation, in a news release.¹ “These regulatory milestones will accelerate the development pathway for NBM-BMX and support our mission of delivering innovative therapy options to patients suffering from rare and aggressive cancers.”

Clinical Investigations of NBM-BMX

In July 2025, Novelwise, the sponsor, announced the FDA’s clearance of the phase 1b/2 NBM-BMX-004 trial (NCT07136181) of NBM-BMX in uveal melanoma.⁶ The trial will evaluate the safety, efficacy, and pharmacokinetics of NBM-BMX as monotherapy in patients with metastatic uveal melanoma, with primary end points of dose-limiting toxicity incidence and maximum tolerated dose in phase 1b and objective response rate in phase 2.^2,6

While recruitment has not yet started, the sponsor has announced the completion of 3 site initiation visits, indicating progression toward enrollment. The trial intends to enroll 36 adult patients with histologically or cytologically confirmed metastatic uveal melanoma.

NBM-BMX will be administered as a 100-mg oral capsule twice daily on an empty stomach along with at least 180 mL of water for 28-day cycles until disease progression or intolerable toxicity. Patients must fast for at least 2 hours following each dose.

The study has an estimated completion slated for May 2029.

Additionally, the ongoing phase 1b/2 NBM-BMX-003 trial (NCT06012695) is investigating NBM-BMX in combination with temozolomide in glioblastoma, which is expected to set the precedent for NBM-BMX’s development in uveal melanoma.

An Expanded Access Program for NBM-BMX will also be available for eligible patients who do not have another satisfactory alternative option.

REFERENCES

1. FDA grants orphan drug designation to NBM-BMX for uveal melanoma. News release. NovelWise Pharmaceutical Corporation. November 23, 2025. Accessed November 24, 2025. https://tinyurl.com/4kjt7crb

2. Evaluation of the safety, efficacy, and pharmacokinetics of NBM-BMX in patients with metastatic uveal melanoma (NBM-BMX-UM). ClinicalTrials.gov. Updated October 24, 2025. Accessed November 24, 2025. https://clinicaltrials.gov/study/NCT07136181

3. NovelWise Pharmaceutical receives FDA fast track designation for NBM-BMX in metastatic uveal melanoma. News release. NovelWise Pharmaceutical Corporation. October 14, 2025. Accessed November 24, 2025. https://tinyurl.com/3kad8sfa

4. Designating an orphan product: drugs and biological products. US Food & Drug Administration. Updated August 12, 2024. Accessed November 24, 2025. https://tinyurl.com/5n77pu2w

5. Gelmi MC, Jager MJ. Uveal melanoma: Current evidence on prognosis, treatment and potential developments. Asia Pac J Ophthalmol. 2024;13(2):100060-100060. doi:10.1016/j.apjo.2024.100060

6. Novelwise Pharmaceuticals announces FDA “may proceed” clearance for Phase Ib/II trial of NBM-BMX in metastatic uveal melanoma. News release. NovelWise Pharmaceutical Corporation. July 30, 2025. Accessed November 24, 2025. https://tinyurl.com/2hrmcb8c