FDA fast-tracks oral treatment for brain tumors with IDH1 gene mutations
Safusidenib aims to slow cancer growth and address unmet patient needs
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Safusidenib, an experimental oral treatment for people with gliomas carrying IDH1 gene mutations, has received fast-track designation from the U.S. Food and Drug Administration (FDA), according to developer Nuvation Bio.
The designation aims to accelerate the development and review of new therapies for serious conditions with unmet medical needs. For Nuvation, it provides increased access to FDA guidance and the potential for a faster regulatory path to bring the drug to market.
“People with IDH1-mutant glioma urgently need additional treatment options,” David Hung, MD, founder, president, and CEO of Nuvation, said in a company press release. “We were eager to pursue Fast Track Designation for safusidenib to hopefully reach these patients on an expedited timeline.
“We look forward to working with the FDA as we advance our mission to provide an effective therapy to nearly every patient whose life is impacted by this disease,” Hung added.
How safusidenib targets brain tumors
Gliomas are cancers marked by the uncontrolled growth of glia, the brain cells that normally support nerve function. In the vast majority of these cases, mutations in the IDH1 gene, which encodes a protein of the same name, help to drive tumor growth.
Even though these patients typically have better survival rates than those with gliomas not associated with IDH1 mutations, “gliomas are not currently curable and prognosis worsens for those with high-risk features,” the release stated.
Safusidenib is an oral, brain-penetrating compound designed to selectively block mutant forms of the IDH1 protein, thereby slowing or reducing tumor growth.
Nuvation recently announced long-term data from a Japan-based Phase 2 clinical trial (NCT04458272) testing safusidenib in 27 adults with IDH1-mutant grade 2 glioma who had not yet undergone chemotherapy or radiotherapy.
Results showed that 79.1% of participants were free from disease progression after three years. In addition, 51.9% responded to the therapy, meaning their tumors decreased in size following treatment. One patient who initially responded has since experienced disease progression, and no new safety issues were reported, according to the company. These findings build on previously reported two-year data.
Clinical trials test safusidenib across glioma types
Nuvation is now running a Phase 3 trial called SIGMA (NCT05303519) to test safusidenib in certain people with IDH1-mutant astrocytoma or oligodendroglioma, two types of glioma. Study sites are recruiting in the U.S., Australia, and China.
The main part of the study is expected to include up to 300 people with either grade 2 or 3 IDH1-mutant astrocytoma with high-risk features, or grade 4 IDH1-mutant astrocytoma, who have already undergone standard treatments such as radiation.
These participants will be randomly assigned to receive either safusidenib or a placebo to evaluate the therapy’s effect on disease progression. If positive, data from this part of the trial will be used to support an application seeking safusidenib’s approval.
An additional group of 40 people with grade 3 IDH1-mutant oligodendroglioma who have undergone surgery but have not received chemotherapy or radiation is also expected to be enrolled. All of these participants will be treated with safusidenib, with the primary goal of evaluating response rates.
Nuvation also recently launched a Phase 2 trial (NCT07703436) to evaluate safusidenib in people with grade 2 or 3 IDH-mutant glioma. To be eligible, patients must have undergone surgery and experienced disease progression while on Voranigo (vorasidenib), an approved therapy for IDH-mutant glioma. All participants will receive safusidenib, with the main goal of tracking response rates. This study has not yet begun recruitment.
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