Del Mar Pharmaceuticals Receives European Orphan Drug Designation for VAL-083 for the Treatment of Glioma

Jan 07, 2013, 08:30 ET from Del Mar Pharmaceuticals

VANCOUVER, British Columbia, Jan. 7, 2013 /PRNewswire/ -- Del Mar Pharma today announced that the European Committee for Orphan Medicinal Products (COMP) has recommended the designation of VAL-083 as an orphan medicinal product for the treatment of glioma. The COMP recommendation will be ratified by the European Commission in accordance with Article 5(8) of Regulation (EC) No. 141/2000. 

Glioma is a type of tumor that starts in the brain or spine and includes glioblastoma multiforme (GBM), the most common and aggressive form of brain cancer. Del Mar Pharma is currently conducting Phase I/II clinical trial of VAL-083 in patients with recurrent GBM in the United States. In November 2012, Del Mar Pharma presented interim clinical data from this clinical trial at the annual meeting of the Society for NeuroOncology supporting that VAL-083 is safe and well tolerated. An overall response rate of 28.5%, where tumor growth had stabilized or regressed, has been observed at doses investigated to date.

To qualify for orphan designation in Europe, a medicine must meet a number of criteria:

  • the new medicine must be intended for the treatment, prevention or diagnosis of a disease that is life-threatening or chronically debilitating;
  • the prevalence of the disease in the EU most not be more than five (5) of every 10,000 people;
  • the new medicine must be of significant potential benefit to those affected by the disease.

"Orphan designation for VAL-083 as a potential treatment for glioma in the EU provides additional validation of our VAL-083 program and further extends our competitive positioning for the program", stated Jeffrey Bacha President & CEO of Del Mar Pharma.

Del Mar received orphan designation from the United States Food and Drug Administration in February 2012. "Obtaining orphan drug designation provides incentives for development and certain competitive advantages," added Mr. Bacha.

Orphan drugs generally follow the same regulatory development path as any other pharmaceutical product. However, incentives such as scientific advice and reduction or waiver of registration fees and access to specialized grant funding may be available to support and accelerate development of orphan drug candidates. In addition, Del Mar Pharma may sell VAL-083 as a treatment for glioma without competition for seven years in the US and for ten years in the EU following market approval, in respect of a medicinal product containing a similar active substance for the same indication.

VAL-083 represents a 'first in class' small-molecule chemotherapeutic. VAL-083 has been assessed in multiple NCI-sponsored clinical studies in various cancers including lung, brain, cervical, ovarian tumors and leukemia. Published pre-clinical and clinical data suggest that VAL-083 may be active against a range of tumor types, including GBM. VAL-083 is approved as a cancer chemotherapeutic in China for the treatment of chronic myelogenous leukemia and lung cancer.

About Del Mar Pharma

Del Mar Pharmaceuticals was founded in 2010 to develop and commercialize proven cancer therapies in new orphan drug indications where patients are failing modern targeted or biologic treatments. The Company's lead asset, VAL-083, benefits from extensive clinical research sponsored by the US National Cancer Institute, and is currently approved as a cancer in China. Published pre-clinical and clinical data suggest that VAL-083 may be active against a range of tumor types via a novel mechanism of action.

For further information, please visit
or contact Jeffrey A. Bacha, President & CEO  +1 (604) 629-5989

SOURCE Del Mar Pharmaceuticals