Breakthrough Therapy designation for Sanbexin sublingual tablets granted by the United States Food and Drug Administration

NANJING, China, Sept. 5, 2024 /PRNewswire/ -- On September 2, 2024, Simcere Pharmaceuticals Group Ltd. (2096.HK) announced that Sanbexin Sublingual Tablets (Edaravone and Dexborneol sublingual tablets), an innovative drug for stroke, has been granted the Breakthrough Therapy designation by the U.S. Food and Drug Administration (FDA) for the treatment of Acute Ischemic Stroke (AIS). Sanbexin sublingual tablets is the first innovative drug in the world to be designated with the Breakthrough Therapy by the FDA for the treatment of stroke.

The Breakthrough Therapy designation stems from the provisions of the "Food and Drug Administration Safety and Innovation Act". It is designed to expedite the development and regulatory review of drugs for treating serious diseases and addressing significant unmet medical needs. The designation will be beneficial for the drugs to obtain the FDA's guidance in the clinical development, accelerate the overseas clinical development process, and is also expected to significantly shorten the time for marketing review through the priority review designation.

The Breakthrough Therapy designation is based on the significant improvement in efficacy metrics demonstrated by the sublingual tablets in a previous clinical study. The data from a multicenter, randomized, double-blind, parallel, placebo-controlled Phase III clinical study in China for the treatment of AIS demonstrated that Sanbexin sublingual tablets significantly improved neurological recovery and independent living ability in patients with AIS compared to placebo, meeting the expected efficacy endpoints with a favorable safety profile. JAMA Neurology published the study's key findings online.

The designation will be beneficial for Simcere to obtain the FDA's guidance in the clinical development of Sanbexin sublingual tablets, accelerate the overseas clinical development process, and is also expected to significantly shorten the time for marketing review through the priority review designation.

According to a recent article in The Lancet, stroke is the second leading cause of death and disability worldwide, with 12 million new cases and 6.6 million deaths each year. Among them, acute ischemic stroke is the most common type of stroke, accounting for about 70% of all strokes, and constituting a heavy disease burden for all countries around the world. The efficacy of acute ischemic stroke treatment is highly time-dependent. Receiving treatment as early as possible will help patients to improve their disease outcome and avoid disability.

Sanbexin sublingual tablets is a brain cytoprotective agent composed of edaravone and dexborneol, two active ingredients with synergistic anti-oxidant and anti-inflammatory effects, which can significantly reduce brain cell injury or impairment caused by AIS. Such unique sublingual formulation can quickly disintegrate once in contact with the saliva once place under the tongue and can be absorbed into the blood through the sublingual venous plexus, which is expected to increase the flexibility of stroke treatment. Sequential therapy consisting of the marketed Sanbexin concentrated solution for Injection. These two formulae enable patients to receive a complete course of treatment in and outside of the hospital.

On June 28, 2023, the new drug application (NDA) for Sanbexin sublingual tablets in China is accepted by the National Medical Products Administration. The first indication is for the improvement of neurological symptoms, daily activities, and functional impairment due to AIS. Phase I clinical trials for Sanbexin sublingual tablets on healthy volunteers have been completed in the United States.