The First China-Independently Developed CNS Orphan Drug Submitted for FDA Approval!

The world's first oral edaravone tablet is set to hit the market.

On December 23, Auzone Pharma submitted a New Drug Application (NDA) to the U.S. FDA for AUKONTALS (edaravone tablets), targeting the treatment of amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease. The drug received orphan drug designation (ODD) from the FDA in 2021.

Auzone Pharma stated that its edaravone tablets not only lead in innovative formulation design but also hold significant clinical potential to address the multifaceted challenges ALS patients face. It is poised to become the first CNS orphan drug developed by a Chinese company to enter the U.S. market, aiming to tap into the rare disease ALS market.

ALS: A Tough Barrier to Cross

ALS is a fatal neurodegenerative disease that progressively destroys motor neurons in the brain and spinal cord, leading to loss of voluntary muscle control. Though its prevalence is relatively low, the disease devastates patients' quality of life.

As global aging intensifies, the number of ALS patients worldwide is expected to grow, fueling demand for effective treatments. According to Stratistics MRC, the global ALS treatment market is projected to grow from $860 million in 2024 to $2.31 billion by 2030, with a compound annual growth rate of 12.6%.

Despite this, the market lacks convenient, safe, and affordable treatments, particularly oral medications, limiting broad access to treatment and improvements in patients' quality of life.

Edaravone's 2017 U.S. market approval provided a new option, but both its injectable form and the later-approved oral suspension faced challenges with patient adherence. Auzone Pharma's oral tablet formulation significantly improves patient compliance while offering superior drug stability and reduced treatment costs.

Achieving the "First" Title

Edaravone is currently the only neuroprotective agent with evidence-based medicine to back its efficacy. It has been shown to inhibit lipid peroxidation, vascular endothelial damage, reduce brain edema and tissue injury, and delay neuron death.

Why, then, has the development of an oral tablet lagged behind other formulations?

Auzone Pharma explained that edaravone's low water solubility and its status as a P-glycoprotein (Pgp) substrate result in extremely low oral bioavailability (3–5%), making oral formulations a challenge for over 20 years.

Using a proprietary solid dispersion technology, Auzone Pharma increased oral bioavailability by several dozen times. The formulation patent has been granted in China and the U.S., with European approval expected soon.

As the world's first oral edaravone tablet, AUKONTALS boasts clear differentiation. Compared to existing injectable and oral suspension forms, the tablet offers enhanced convenience and patient adherence. Additionally, its ability to be stored at room temperature significantly improves the medication experience, alleviating burdens for both patients and caregivers.

Fast-Tracking CNS Progress to Create a "Pipeline-in-a-Pill" Blockbuster

ALS represents Auzone Pharma's starting point for commercialization. AUKONTALS is expected to receive FDA approval and launch by 2025. Beyond ALS, the company is actively exploring broader clinical applications, including:

• Acute ischemic stroke (AIS): A pivotal clinical trial application is expected to be submitted to the FDA in January 2025, with a launch application planned for 2026.
• Alzheimer's disease (AD): An international Phase II clinical trial is set to begin in early 2025.
• Autism spectrum disorder (ASD): Edaravone tablets have received approval from China's CDE to conduct a Phase II clinical trial. AUKONTALS is among the few clinical-stage drugs targeting core symptoms of ASD, offering new hope to children and families affected by the condition.

Auzone Pharma's CEO stated: "In the coming years, we will continue focusing on CNS innovation, striving to maximize AUKONTALS's potential as a pipeline-in-a-pill while also establishing a robust CNS product portfolio. Our goal is to become a globally competitive and impactful biotech company in the CNS field."

While the rare disease market poses commercialization challenges compared to more mature sectors like oncology, Auzone Pharma is not going it alone.

In July 2024, the company partnered with Huadong Medicine, granting exclusive rights for the development, registration, production, and commercialization of edaravone tablets (TTYP01) for AIS in mainland China, Hong Kong, Macau, and Taiwan. Under the agreement, Auzone Pharma will receive an upfront payment of ￥100 million, milestone payments totaling ￥1.185 billion, and high double-digit royalties.

For retained overseas rights, Auzone Pharma is exploring partnerships with other pharmaceutical companies to accelerate clinical progress. Collaborating with leading research institutions and global pharmaceutical companies, Auzone aims to expedite AUKONTALS's worldwide rollout and maximize its value.

As the world's first oral edaravone tablet, AUKONTALS not only provides new hope for ALS patients but also represents a major milestone in Auzone's commercialization journey. Industry experts believe this innovative product could fill numerous gaps in ALS treatment and inspire greater enthusiasm and progress in CNS drug development.