Specialist development pharmaceutical company AMZELL B.V. and the FDA have reached an agreement on the protocol for the Phase 3 trial of its infantile epileptic disease therapy under an FDA Special Protocol Assessment (SPA). AMZELL is ready to initiate the trial and hopes to begin patient recruitment later this week.
AMZ002 is a sterile injectable hormone that would offer a purified synthetic polypeptide as alternative to existing FDA-approved treatment for epileptic seizures. The >95% purity of the synthetic hormone allows high and reproducible product manufacturing quality. AMZ002 was developed as a low viscosity liquid formulation to facilitate the treatment administration and minimise medication errors. The Phase 3 trial will compare AMZ002 to another active treatment in a superiority study.
Infantile epileptic disease can cause global neurodevelopmental delay, significant intellectual disability, and currently has limited treatment options and a poor prognosis. In most cases, the initial age of onset is between 3 and 12 months of age, and over 90% of cases begin before 12 months of age. Infantile epileptic disease is an orphan indication that affects fewer than 200,000 patients in the United States each year. The lack of treatment options for this devastating condition means that there is a considerable unmet need.
Dario N. Carrara PhD, Chief Executive Officer commented: “We are delighted to have agreed with the FDA on our AMZ002 Phase 3 trial protocol. Our purified, synthetic injectable should provide a treatment option for epileptic seizures, suited to best meet the needs of a highly vulnerable patient population, and we look forward to initiating the trial in the coming months.”
AMZ002 is the latest product to reach Phase 3 from AMZELL, which specialises in developing well-characterized active substances either through to proof-of-concept or registration for sale to commercial partners. AMZELL’s innovative platform drug delivery technologies provide effective drug delivery, good efficacy, and state of the art safety and compliance.
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