FDA Awards Orphan Drug Status To Several Biotech Products

A handful of new drugs and therapeutics recently received orphan drug status from the Food and Drug Administration, or FDA. It qualified their sponsors for various development incentives.

First on the list is ADS-5102, Adamas Pharmaceuticals' product candidate in the treatment of levodopa-induced dyskinesia associated with Parkinson's disease — a condition that currently has no FDA-approved treatment options. Adamas Pharmaceuticals is already conducting multiple phase III trials evaluating the effectiveness of ADS-5102 for this indication.

According to company CEO Gregory Went, "Adamas is committed to bringing new treatments to patients suffering with movement disorders related to neurological disorders. With positive data from our Phase II/III study, a pivotal Phase III program well underway, and 10 issued US patents, we are pleased with our ongoing momentum."

Similarly, Nascent Biotech, Inc. (OTC:NBIO) has also been granted orphan drug designation from the Office of Orphan Products Development of the FDA for its Pritumumab product, which is used in the treatment of brain cancer. There are approximately 8,000 new cases of brain cancer annually, but viable treatment options are few.

Pritumumab targets an abnormal variant of a normal intracellular protein expressed on the outside of cancer cells. It has already undergone Phase I and Phase II clinical trials, demonstrating an increase in survival rate from three percent to 29 percent. Nascent Biotech's Chief Science Officer Dr Mark Glassy remarked, "We anticipate that by modifying the dosing of the previous Japanese human clinical trials during upcoming domestic human clinical trials, we have the potential to markedly improve response rates." Company shares jumped from 1.15 to 1.24 upon the announcement.

Prolong Pharmaceutical also received orphan drug status for Sanguinate, an experimental biologic for the treatment of sickle cell disease. It is the only biological product currently under clinical development for sickle cell disease's co-morbidities, like sickle cell crisis, leg ulcers, acute chest syndrome and stroke. With its orphan drug status, Sanguinate will be awarded a period of marketing exclusivity upon FDA approval, among other incentives and benefits. The drug is currently undergoing Phase II clinical trial to establish efficacy.

FDA also awarded orphan drug designation to Reata Pharmaceuticals' bardoxolone methyl, which is used to treat pulmonary arterial hypertension, a life-threatening disease that involves pulmonary vasoconstriction, vascular remodeling and right ventricular hypetrophy. Preclinical trials showed that bardoxolone methyl contained antioxidant, anti-inflammatory and bioenergetics properties, which could give patients improved tolerance. The drug is currently undergoing Phase II clinical trial to evaluate its safety, tolerability, and efficacy.

More than 350 million people all over the world suffer from rare diseases, which are defined as diseases that affect fewer than 200,000 persons combined in a particular rare disease group. Unfortunately, 95 percent of these rare diseases do not have a single approved drug treatment from the FDA.

The Orphan Drug Act of 1983 sought to change that reality by supporting the development and commercialisation of drugs that treat rare diseases. Though it does not indicate that a certain therapeutic is safe or effective, orphan drug designation qualifies the sponsor for certain benefits from the federal government, including tax incentives, enhanced patent protection and marketing rights, clinical research subsidies and government support for research and development.

To contact the writer, email: v.hernandez@ibtimes.com.au