Axsome and Aytu's Positive Day with the FDA

FDA_Sarah Silbiger/Getty Images

Courtesy of Sarah Silbiger/Getty Images

Amid government crackdowns on pharmaceutical companies and increasingly strict federal guidelines, two companies received positive feedback today on their treatments for depression and Vascular Ehlers-Danlos Syndrome (VEDS), respectively. 

New York-based Axsome Therapeutics received some long-awaited good news from the U.S. Food and Drug Administration today. The regulator proposed some postmarketing requirements and commitments regarding Axsome’s New Drug Application (NDA) for its AXS-05 drug - an indication that marketing may occur sometime in the future.  

Axsome has been working on AXS-05, a treatment for major depressive disorder, for years. Originally, the drug was created to treat other Central Nervous System (CNS) disorders. It first received FDA Breakthrough Therapy designation as a treatment for Alzheimer’s disease agitation.

AXS-05 is an uncompetitive N-methyl-D-aspartate receptor agonist that increases the bioavailability of dextromethorphan, which means it works differently than most other oral medications on the market, that treats major depressive disorder. Currently, the drug is covered by more than 100 issued U.S. and international patents. The drug has also received FDA Breakthrough designation and was granted Priority Review by the FDA to treat major depressive disorder.

The company filed for an NDA for AXS-05 last summer, but by late August, the FDA notified Axsome that the review would not be completed by its original target date of August 22, 2021. The news was made even more frustrating because the FDA did not request any additional information.

Now, recent communications indicate that a decision may be close. Axsome, in a regulatory filing, said that the FDA had asked for more materials, and Axsome has agreed to the terms. The company expects regulatory action as early as Q2 of this year.

The good news skyrocketed Axsome’s shares by nearly 27% in premarket trading Tuesday. The depression treatment market is expected to hit $16.06 billion in the next five years. Since depression is the fourth leading cause of disability across the globe, any medicinal relief garners significant attention on the stock market.

Another recipient of good news from the FDA is Colorado-based Aytu BioScience. Aytu, a pharmaceutical company that develops novel therapeutics, announced that the FDA granted Fast Track designation to AR101, a protein kinase C (PKC) β inhibitor, as a treatment for patients with VEDS.

With the designation, Aytu plans to enroll 260 VEDS patients in its PREVEnt Trial. Patients will be treated either with the current standard of care or with AR101.

The drug had already received Orphan Drug designation from the FDA, and it received the same designation in Europe from the European Medicines Agency.

"Receipt of Fast Track designation enables more frequent interaction with the FDA throughout the AR101 development process, along with a shorter review timeframe. With this important designation now in hand, we are focused on getting the operational elements of the pivotal PREVEnt study of AR101 in place such that we can initiate it as quickly as possible, with plans to begin patient dosing by late 2022 or early 2023," said Josh Disbrow, CEO at Aytu.

Though the initial beneficiaries of these announcements are Aytu and Axsome, the real winner will be the patients. Drugs that receive Orphan Drug or Breakthrough Therapy designations treat serious diseases of high unmet need, and these designations offer more frequent communication between the company and the FDA. Hopefully, the news from the FDA regarding these drugs means the companies are one step closer to providing viable treatment options to patients.

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