HOUSTON, TX – The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Diakonos’ dendritic cell vaccine DOC1021 for malignant glioma.
If the therapy is approved, the US-based immuno-oncology company is eligible for a potential seven years of market exclusivity in newly diagnosed or refractory glioblastoma multiforme, according to a 2 January press release. The orphan drug status also provides tax credits for US-based clinical trials.
In the same press release, Diakonos stated it had completed enrolment in a Phase I trial investigating DOC1021.
The single-arm, first-in-human study (NCT04552886) is evaluating the safety of the personalised vaccine administered to patients with glioblastoma who have completed standard-of-care treatments including chemotherapy and radiation therapy. Progression-free survival and overall survival will be evaluated as secondary outcomes.
Diakonos has administered four dose levels of its dendritic cell vaccine to a total of 16 patients in the trial. The company reported a good safety profile of DOC1021, with no attributable serious adverse events.
Highlighting efficacy signals, Diakonos added that 13 of the patients remain alive. The first patient was enrolled in October 2021. Around 5% of patients with glioblastoma multiforme currently survive beyond five years, while the average survival time is 12-18 months.
Diakonos has previously received a fast track designation from the FDA for the vaccine in October 2023. This designation speeds up the development and review of drugs showing early clinical promise for severe or life-threatening conditions.
Diakonos’ chief regulatory officer Ian Bellayr said: “Receiving the orphan drug designation from the FDA is a valuable addition to our existing fast track designation as it indicates that the cell therapy may be more broadly applicable to other oncological indications.”
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Jay Hartenbach