'Vorasidenib' receives Orphan Drug Designation in KOR

A targeted anticancer drug for brain tumors

The Ministry of Food and Drug Safety (MFDS) announced the Orphan Drug Designation (ODD) in October

Demonstrated 2.5-fold improvements in PFS through its Phase 3 trial

'Vorasidenib,' an anticancer drug targeting brain cancer, has been designated an orphan drug in South Korea.

 

The Ministry of Food and Drug Safety (MFDS) announced this on October 8th through the Orphan Drug Designation (ODD) notice.

 

Vorasidenib is an orally administered dual inhibitor of isocitrate dehydrogenase (IDH) 1/2 that selectively penetrates the blood-brain barrier.

 

The drug's efficacy for selective malignant brain tumors (neuroglioma) was demonstrated through a double-blind Phase 3 clinical trial led by a research team at the University of California, Los Angeles (UCLA).

 

The research team administered the targeted anticancer drug vorasidenib (40 mg single dose per day) or placebo to 331 patients with specified brain tumors (residual or recurrent Grade 2 glioma with IDH mutation) who have undergone brain tumor surgery as their only treatment.

 

Grade 2 gliomas with isocitrate dehydrogenase (IDH) mutation are malignant brain tumors that cause considerable disability in patients, leading to early death.

 

Gliomas tend to advance slowly but are fatal and affect people in their 30s.

 

Study participants were randomly assigned to vorasidenib-treated patients (study group) and placebo-treated patients (control group).

 

168 of these patients took vorasidenib, and 163 took the placebo.

 

Study results showed that the progression-free survival (PFS) for the vorasidenib-treated patient group was 27.7 months and that for the placebo-treated patient group was 11.1 months.

 

Vorasidenib delayed the progression of malignant tumors by 16.6 months and lowered the death risk to 39%.

 

Additionally, it improved the time until the next anticancer therapy (chemotherapy, radiotherapy), reducing the patient death risk to 26%.

 

The side effects experienced by the vorasidenib-treated patient group were 22.8%, whereas 13.5% in the placebo-treated patient group.

 

However, no significant side effects have been found.

 

The primary endpoint was imaging based PFS and the secondary endpoint was the time to the next anticancer intervention.

 

Of those study participants, 226 (about 68%) patients had a follow-up of 14.2 months (median value) and continued to take vorasidenib or placebo.

 

At 30 months (Sept.

 

2022), 72% of the vorasidenib-treated patient group was still taking the drug and had not experienced disease progression.

 

Meanwhile, France-based Servier Pharmaceuticals developed vorasidenib and is proceeding with the approval process in the U.S.

 

and Europe.