US FDA approves sodium phenylbutyrate to treat urea cycle disorders


eMediNexus    25 December 2022

The US Food and Drug Administration (FDA) have authorized a medication made by Acer Therapeutics Inc. to treat people who have urea cycle disorders (UCDs), a subset of hereditary disease.


According to the permission letter, which was released on Thursday the medicine, sodium phenylbutyrate, might be used as an adjuvant therapy to the standard of care for patients with UCDs. UCD is a hereditary disorder which results in an accumulation of poisonous ammonia in the blood due to a lack of certain enzymes. 


After receiving the FDA′s so-called comprehensive response letter in June, the company resubmitted its marketing application for the drug in July.


(Source:  https://health.economictimes.indiatimes.com/news/pharma/us-fda-approves-acer-therapeutics-genetic-disorder-drug/96472324)

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