Acer Therapeutics’ asset ACER-001 is a potential game-changer in the treatment of Urea Cycle Disorders (UCDs) and Maple Syrup Urine Disease (MSUD). ACER-001 is a taste-masked sodium phenylbutyrate drug that has been developed for the treatment of these rare genetic disorders.
Acer Therapeutics has submitted a New Drug Application (NDA) to the US Food and Drug Administration (FDA) for the approval of ACER-001 and is awaiting the FDA’s decision. The ACER-001 has already been approved in Europe as a treatment for MSUD, and the FDA is expected to make a decision later this year.
The drug has already been tested in multiple clinical trials and has shown positive results in terms of efficacy and safety. Acer Therapeutics is also looking to use the 505(b)(2) regulatory pathway to seek FDA approval for ACER-001, which could make it easier to gain approval from the FDA.
In addition, Acer Therapeutics has taken several steps to ensure ACER-001 is approved by the FDA. The company has held a pre-NDA meeting with the FDA to discuss the development and regulatory requirements of ACER-001. Acer Therapeutics also conducted a bioequivalence study to ensure the drug can be safely and effectively used in patients with UCDs and MSUD. The company has also submitted additional data to the FDA for review.
The potential approval of ACER-001 by the FDA could be a major breakthrough for the treatment of rare genetic disorders such as UCDs and MSUD. It would provide an effective treatment option for patients suffering from these disorders, and could help to improve their quality of life.
Furthermore, the approval of ACER-001 could also provide a significant revenue stream for Acer Therapeutics, which would allow the company to further develop and expand its portfolio of treatments.