FDA Grants Orphan Drug Designation to DSP-0390 for Brain Cancer

The FDA has granted an orphan drug designation to DSP-0390 for the treatment of brain cancer.

The FDA has granted an orphan drug designation to DSP-0390 for the treatment of brain cancer, according to an announcement from Sumitomo Pharma Oncology.1

DSP-0390 is an investigational emopamil-binding protein (EBP) inhibitor, an endoplasmic reticulum membrane protein involved in cholesterol biosynthesis. Cytotoxicity can be induced more selectively against hyperproliferative glioblastoma multiforme (GBM) cells by inhibiting de novo cholesterol synthesis.2

“[DSP-0390] mediates de novo cholesterol synthesis for cell membrane structure and signaling, enabling aberrant growth of tumors,” Jatin J. Shah, MD, chief medical officer of Sumitomo Pharma Oncology, stated in a press release. “Notably, in patients with GBM, a form of high-grade glioma, increased de novo cholesterol synthesis is correlated with poor survival, and preclinical evidence has shown that DSP-0390 has cytotoxic activity against GBM cell lines.”

DSP-0390 demonstrated significant antitumor activity in orthotopic xenograft models of human GBM.2 Investigators are currently assessing the agent for patients with recurrent, high-grade glioma in a phase 1 trial (NCT05023551).

The first-in-human trial is enrolling patients who are at least 18 years old with a Karnofsky performance status of at least 70%, and adequate renal, hepatic, and hematologic function. Investigators will administer DSP-0390 once daily.

Patients will be excluded from the trial if they have multifocal disease; leptomeningeal metastasis or extracranial metastasis; abnormal electrocardiograms; or significant cardiovascular disease. Prior therapy with bevacizumab (Avastin) or other VEGF treatments is not permitted within 3 months prior to first study dose.3

The dose-escalation portion of the trial will enroll 21 to 30 patients with WHO grade III or IV malignant glioma who progressed after at least 1 line of prior therapy. Investigators will use this portion of the trial to identify the maximum tolerated dose (MTD) or recommended dose for expansion.

Safety–including treatment-emergent adverse effects (AEs), serious AEs, and dose-limiting toxicities–is a primary end point along with establishing MTD. Secondary end points include pharmacokinetics and preliminary antitumor activity.

The dose-expansion portion will include 20 to 40 patients with WHO grade IV GBM and measurable disease who progressed after primary therapy. The end points here are change in baseline tumor activity and safety of the recommended phase 2 dose.

The trial is currently enrolling in the United States and Japan.

“This designation for DSP-0390 underscores the profound need for novel brain cancer treatment options,” Patricia S. Andrews, chief executive officer and global head of Oncology at Sumitomo Pharma Oncology, stated in a press release. “We are excited to contribute to advancing critical research in brain cancer.”


  1. Sumitomo Pharma Oncology receives orphan drug designation for DSP-0390, an investigational emopamil-binding protein (EBP) inhibitor for the treatment of brain cancer. News release. Sumitomo Pharma Oncology. July 18, 2022. Accessed July 21, 2022. https://prn.to/3zls0Gm
  2. Reardon DA, Narita Y, Arakawa Y, et al. DSP-0390, an oral emopamil binding protein (EBP) inhibitor, in patients with recurrent high-grade glioma: A first-in-human, phase 1 study. J Clin Oncol. 2022;40(suppl 16):TPS2077. doi:10.1200/JCO.2022.40.16_suppl.TPS2077
  3. Study of DSP-0390 in patients with recurrent high-grade glioma. ClinicalTrials.gov. Updated April 19, 2022. Accessed July 21, 2022. https://clinicaltrials.gov/ct2/show/NCT05023551