Theranexus, a clinical-stage biopharmaceutical company, announced today that it’s most advanced drug candidate, THN102, has received an Orphan Drug Designation (ODD) from the US Food and Drug Administration (FDA) for the treatment of narcolepsy.
ODD may be granted to drugs that are intended to improve the quality of life of patients affected by rare diseases and conditions that affect less than 200,000 people in the United States. This designation grants Theranexus some interesting incentives: especially, 7 years of exclusive marketing rights in the United States upon FDA approval, as well as an exemption from certain registration fees.
“We are very happy that this first exchange with the FDA has been fruitful. This first step attests to our desire to secure regulatory development of THN102, both in Europe and in the United States,” says Franck Mouthon, Theranexus’ CEO, in a release.
Françoise Brunner, Theranexus’ chief medical officer, says, “Obtaining orphan drug status from the FDA for THN102 reaffirms the significant need for a safer and more effective drug to improve the treatment of narcolepsy patients.”
As a drug candidate, the positive results of THN102 during a proof-of-concept study in healthy, sleep-deprived volunteers were recently presented in partnership with the French Armed Forces Biomedical Research Institute (IRBA), during the latest congress held by the European Sleep Research Society (ESRS 2016). Theranexus has initiated a Phase II study in patients with narcolepsy with the intent of demonstrating the superior efficacy of THN102 as compared to the reference treatment. Patient recruitment for this study should begin shortly.