Axsome’s AXS-02 drug secures US FDA’s Fast Track designation

CRPS is a condition characterised by severe pain in a limb, accompanied by autonomic, sensory, motor and trophic changes. In few patients, the chronic pain can lead to significant and sometimes permanent disability.

AXS-02 had already received Orphan Drug Designation earlier from the FDA, and Orphan Medicinal Product Designation from the EMA, for the treatment of CRPS.

It is an oral, non-opioid, potentially first-in-class pain therapeutic being developed by Axsome for the treatment of chronic pain caused by CRPS. Currently no drug is in the approval stage to treat this orphan condition in either the US or the EU.

While FDA’s Fast Track designation program is designed to aid in the development and expedite the review of drugs that are intended to treat serious or life-threatening conditions, Orphan Drug Designation is granted to novel drugs intended to treat rare diseases, specifically affecting fewer than 200,000 people in the U.S.

Fast Track designation enables greater access to, and more frequent communication with, the FDA throughout the entire drug development and review process, to get important new drugs to patients faster.

It also provides the opportunity to submit sections of a New Drug Application (NDA) on a rolling basis, where the FDA may review portions of the NDA as they are received instead of waiting for the entire NDA submission. In addition, Fast Track designated products are eligible for Priority Review at the time of NDA submission.

Similarly, the Orphan designation may entitle Axsome to a period of seven years of marketing exclusivity in the US upon FDA approval and also confers special incentives to Axsome including tax credits towards the cost of clinical trials and a waiver of the company’s obligation to pay the FDA application user fees for the product as required by the Prescription Drug User Fee Act.

The EMA’s Orphan Medicinal Product Designation is designed to encourage the development of drugs that may provide significant benefit to patients suffering from rare diseases identified as life-threatening or chronically debilitating. Designation provides ten years of potential market exclusivity in the EU if the product candidate is approved and the orphan designation is maintained.

Axsome M.D. CEO Herriot Tabuteau said: "We are pleased that the FDA has granted Fast Track status for AXS-02 for the treatment of pain associated with CRPS, as it demonstrates their recognition of this condition as a significant unmet medical need.

"Our product’s Fast Track designation status, in addition to Orphan Drug designations from both the FDA and EMA [European Medicines Agency], provide significant regulatory confirmation as we work to bring our therapy to those suffering from this serious condition."