The Food and Drug Administration (FDA) has reportedly accepted a New Drug Application seeking accelerated approval for dordaviprone (ONC201) for the treatment of patients with recurrent H3 K27M-mutant diffuse glioma.
The application has been granted Priority Review and has been assigned a Prescription Drug User Fee Act target action date of Aug. 18, according to a news release issued by dordaviprone’s manufacturer, biopharmaceutical company Chimerix.
“This significant milestone brings us one step closer to our goal of accelerating access to the first medicine specific to patients diagnosed with recurrent H3 K27M-mutant diffuse glioma," said Mike Andriole, Chief Executive Officer of Chimerix, in a statement in the news release. "Patients with this form of high-grade glioma face a very difficult prognosis with few treatment options beyond palliative care. Our team is working expeditiously with FDA to facilitate their review as we simultaneously prepare for a potential commercial launch in order to ensure rapid availability to patients in need."
Glossary:
New Drug Application: according to the FDA, this is the vehicle through which drug sponsors formally propose that the FDA approve a new pharmaceutical for sale and marketing in the United States.
Priority Review: a designation that, according to the FDA, will direct overall attention and resources to the evaluation of applications for drugs that, if approved, would be significant improvements in the safety or effectiveness of the treatment, diagnosis or prevention of serious conditions when compared to standard applications.
Prescription Drug User Fee Act target action date: the deadline for the FDA to approve a new drug.
Fast Track Designation: a process designed to facilitate the development, and expedite the review of drugs to treat serious conditions and fill an unmet medical need, according to the FDA.
Orphan Drug Designation: a designation, according to the FDA, for a drug or biological product to prevent, diagnose or treat a rare disease or condition, qualifying sponsors for incentives such as tax credits for clinical trials, exemption from user fees and potential seven years of market exclusivity.
When Chimerix announced the submission of its New Drug Application for accelerated approval to the FDA for dordaviprone for patients with recurrent H3 K27M-mutant diffuse glioma in December 2024, Andriole stated in a news release that the drug could have a potential commercial launch in the United States in 2025.
Dordaviprone, Chimerix noted, has already received Rare Pediatric Disease Designation for H3 K27M-mutant glioma and has applied for a Rare Pediatric Disease Priority Review Voucher in the New Drug Application submission.
Additionally, dordaviprone has been granted both Fast-Track Designation and Orphan Drug Designation in the United States.
In a 2024 article on the phase 3 ACTION study of dordaviprone in patients with newly diagnosed H3 K27M-mutant diffuse glioma published in Neuro-Oncology, researchers noted that H3 K27M-mutant diffuse glioma primarily affects children and young adults. The disease is associated with a poor prognosis, with no effective systemic therapy currently available.
Dordaviprone, researchers noted, has demonstrated efficacy in patients with recurrent disease.
The drug is described in the press release as a novel, first-in-class small molecule imipridone that selectively targets mitochondrial protease ClpP and dopamine receptor D2.
According to the National Cancer Institute, dordaviprone is currently being evaluated in seven clinical trials as a monotherapy and in combination with other drugs for the treatment of diseases including colorectal cancer, meningioma, endometrial cancer, myelodysplastic syndrome, peritoneal cancer and gliomas.
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