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AMZ002 is a highly purified synthetic polypeptide hormone developed as a sterile injectable alternative to current FDA-approved treatments for epileptic seizures, specifically targeting infantile spasms (also known as West syndrome). The drug is designed to mimic the effects of natural adrenocorticotropic hormone (ACTH) and offers an alternative to existing ACTH-based therapies. AMZ002 has received both Rare Pediatric Disease and Orphan Drug designations in the United States. It was originally in-licensed from Ferring by Amzell, which holds global rights except in India where Ferring retains them. The product has completed Phase 1 trials and entered Phase 3 clinical development for infantile spasms but was later withdrawn or discontinued at this stage.
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