Orphan Designation from the EMA for RaphaLX™

New Biotic, Inc., received Orphan Designation from the European Medicines Agency (EMA) for RaphaLX™, a biotherapeutic for treatment of Amyotrophic Lateral Sclerosis (ALS).

Irvine, CA, December 20, 2019. New Biotic, Inc., a biotech company based in Irvine, CA, announced the European Medicines Agency (EMA) has granted orphan designation to RaphaLX™ for the treatment of amyotrophic lateral sclerosis (ALS). RaphaLX™ is a microbiome-based biotherapeutic that targets glutamate-mediated excitotoxicity in brain cells.

ALS, sometimes called Lou Gehrig's disease, is a rapidly progressive and fatal neurological disease that attacks the nerve cells (neurons) responsible for controlling voluntary muscles. The majority of those diagnosed with ALS die within 3-5 years.

The European Commission grants orphan designation to medicines intended for the treatment, prevention or diagnosis of a disease that is life-threatening affecting no more than 5 in 10,000 in the EU. Orphan designation allows for protocol assistance, streamlined regulatory review processes and registration in addition to 10 years of market exclusivity in the EU upon regulatory approval.

To be considered for orphan designation in the EU, there must be plausible evidence of a drug candidate’s efficacy and of its potential to offer significant clinical benefit compared to already-approved treatments. New Biotic, Inc., based its submission on data from results of the RaphaLX™ pilot study in which patients demonstrated promising results in the study's primary and secondary endpoints along with the development of a potentially new biomarker to measure disease progression. The EMA orphan designation application was submitted through the company's European partner, MDC RegAffairs GmbH.

RaphaLX™ previously received orphan drug designation from the U.S. Food and Drug Administration (FDA) for the treatment of ALS in 2017.