Late-Stage Brain Cancer Drug Granted Orphan Medicinal Product Status


Orbus Therapeutics plans for Phase III Clinical Trial of eflornithine in patients suffering from recurrent anaplastic astrocytoma after achieving Orphan Medicinal Product status from European Medicines Agency (EMA).

Orbus logoEflornithine is a novel cytostatic agent being developed as a brain cancer drug to treat glioma. Earlier, eflornithine was granted the Orphan Drug Designation and Breakthrough Therapy Designation by the U.S. Food and Drug Administration (FDA) for the treatment of patients suffering from anaplastic glioma. Orbus Therapeutics is developing it for the treatment of patients suffering from a rare form of brain cancer- anaplastic astrocytoma which is a subtype of anaplastic glioma.

The largest subset of anaplastic glioma is the anaplastic astrocytoma and represents approximately 75 percent of AG patients. Roughly every year, about 2,500 new anaplastic astrocytoma patients diagnosed in the US.

types of brain tumor

Figure 1. Types of brain tumors (photo credit: Johns Hopkins Medicine). 

In the patients with newly diagnosed or recurrent anaplastic astrocytoma, eflornithine has shown an increased overall survival rate in a controlled environment and randomized single arm clinical studies. Now the company is planning to start a Phase III clinical trial to evaluate the safety and efficacy profile of eflornithine in anaplastic astrocytoma patients.

chemical structure of Eflornithin

Eflornithine, scientifically referred as α-difluoromethylornithine or DFMO is a drug that has been found to be effective in the treatment of excessive hair growth called facial hirsutism as well as in sleeping sickness. Eflornithine binds irreversibly to Ornithine decarboxylase (ODC) preventing the access of the active site by the natural substrate, ornithine. Due to this inhibition mechanism it is also referred as “suicide inhibitor”.  

Eflornithine pharmacology

Figure 2. Mechanism of action of eflornithine (photo credit: Fred Opperdoes).

It is one of the Model List of Essential Medicines prepared by WHO thus being an important medicine in our day to day lives.

The further success of eflornithine in Phase III will be big relief for patients suffering from recurrent anaplastic astrocytoma.

Featured image credit: Brain astrocytoma of a human © bond80 (Stock Photo ID: 94172768)

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