Australian monoclonal antibody company, Immune System Therapeutics Limited (IST), has announced that its breakthrough antibody treatment for terminal blood cancer had received Orphan Drug Designation from US health regulators.
Orphan drug status is granted by the United States Food and Drug Administration (FDA) to drugs that treat rare diseases and to diseases that have few available treatments.
It also gives the drug maker marketing exclusivity if approved, during which time competing drug makers cannot enter with rival treatments.
In the United States, the effective period of exclusivity for orphan drugs is now 12 years with the potential for an accelerated review process by the FDA.
IST Chief Executive Officer, Mr Alan Liddle, said, “This is another positive step towards achieving our business goal of developing an effective treatment for patients suffering from multiple myeloma. We are making excellent progress towards putting in place a robust global licensing structure that will expedite the clinical and regulatory processes needed to commercialize this promising new medical treatment.”
The FDA granted orphan drug designation for MDX-1097 based on review of the application, which included clinical and preclinical data demonstrating a favorable efficacy and safety profile.
On 17 June, IST announced it would expand the Phase 2 clinical trial of MDX-1097 in Australia following encouraging responses from the first dosed patients and a positive review of safety and efficacy data by an independent Data Monitoring Committee.
The Orphan Drug Designation is the first awarded to IST for its portfolio of antibody platform technologies currently being developed to target and treat a range of other cancers and diseases with major unmet clinical needs.