Orphan Drug Designation from the FDA for RaphaLX™

New Biotic received Orphan Drug Designation (ODD) from the USA FDA for RaphaLX™, a biotherapeutic for treatment of Amyotrophic Lateral Sclerosis (ALS).

Irvine, CA January 3, 2017. New Biotic, a privately held biotech company based in Irvine, CA, announced today that the U.S. Food and Drug Administration has granted Orphan Drug Designation to RaphaLX™ for the treatment of patients with amyotrophic lateral sclerosis (ALS). RaphaLX™ is a biotherapeutic to target and mitigate glutamate-mediated excitotoxicity and reactive oxidative stress (ROS) in the nervous and brain cells. 

ALS, sometimes called Lou Gehrig's disease, is a rapidly progressive, invariably fatal neurological disease that attacks the nerve cells (neurons) responsible for controlling voluntary muscles (muscle action we are able to control, such as those in the arms, legs, and face).

About 50% of those diagnosed with this disease die within 2 years, and the majority will not survive 5 years. Most end up in hospice creating a heavy financial and social burden exceeding a $ 1.4 mil per patient that is borne by insurance companies and the patient’s family. There is currently no cure for this condition and there are only two drugs approved by FDA to date:  Rilutek, which was first approved 22 years ago, only prolongs life by some 2-3 months and the recently approved Radicava, which does not prolong life but may provide relief in terms of slowing down some functional impairment of the disease.

Under the U.S. Orphan Drug Act, the FDA’s Office of Orphan Products Development provides special status and incentives to encourage the development of drugs for diseases affecting fewer than 200,000 people in the U.S. Orphan Drug Designation (ODD) conveys up to seven years of marketing exclusivity if the compound receives regulatory approval from the FDA and offers various development incentives, including tax credits related to clinical trial expenses, an exemption from the FDA-user fee and FDA assistance in clinical trial design. The granting of orphan designation does not alter the standard regulatory requirements, timing and process for obtaining marketing approval. Safety and effectiveness of a drug must be established through adequate and well-controlled studies.


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