European CHMP grants orphan drug status to Orbus’ eflornithine brain cancer drug

Eflornithine is a novel cytostatic agent, which is being developed to treat patients with recurrent anaplastic astrocytoma, a rare form of central nervous system cancer.

The drug irreversibly inhibits ornithine decarboxylase, a crucial enzyme in mammalian polyamine biosynthesis that is up-regulated in several types of cancer.

Eflornithine already secured orphan drug designation and breakthrough therapy designation in the US to treat anaplastic glioma.

In controlled, randomized and single arm clinical trials, the drug demonstrated an increase in overall survival of patients with newly diagnosed or recurrent anaplastic astrocytoma.

Orbus aims to start a phase 3 clinical trial to assess the safety and efficacy of eflornithine in patients with recurrent anaplastic astrocytoma.

Orbus Therapeutics co-founder and CEO Bob Myers said: "Eflornithine now has orphan designation in Europe and the United States, along with breakthrough therapy designation in the United States.

"Given these important regulatory designations and our previously announced Series A financing, we are looking forward to initiating our Phase 3 clinical trial this year."

Anaplastic astrocytoma is the largest subset of anaplastic glioma, and represents about 75% of AG patients.

There are around 2,500 new anaplastic astrocytoma cases diagnosed in the US per year.

Image: Micrograph of an anaplastic astrocytoma. Photo: courtesy of Nephron.