FDA Events for Aeterna Zentaris (AEZS)
This section highlights FDA-related milestones and regulatory updates for drugs developed by Aeterna Zentaris (AEZS).
Over the past two years, Aeterna Zentaris has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
AEZS-130-P02. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
AEZS-130-P02 - FDA Regulatory Timeline and Events
AEZS-130-P02 is a drug developed by Aeterna Zentaris for the following indication: Childhood-onset Growth Hormone Deficiency (CGHD).
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- AEZS-130-P02
- Announced Date:
- August 27, 2024
- Indication:
- Childhood-onset Growth Hormone Deficiency (CGHD)
Announcement
COSCIENS Biopharma announced the top-line results of its Phase 3 safety and efficacy study AEZS-130-P02 (the "DETECT-trial") evaluating macimorelin for the diagnosis of Childhood Onset Growth Hormone Deficiency ("CGHD").
AI Summary
COSCIENS Biopharma recently announced top‐line results from its Phase 3 DETECT-trial, which evaluated macimorelin as a diagnostic tool for Childhood Onset Growth Hormone Deficiency (CGHD). The study showed that macimorelin, at a dose of 1.0 mg/kg, triggered a strong release of growth hormone, confirming its ability to stimulate sufficient hormone production during testing. However, the trial did not meet its primary efficacy endpoint as defined by the study protocol. This outcome is thought to be influenced by the comparator tests—arginine and clonidine—which may have produced a high false positive rate, complicating the interpretation of macimorelin’s true performance. An independent expert panel adjudicated each case, and while macimorelin’s safety profile in children was excellent, further analysis of the data is needed to clarify these unexpected results and to determine the best path forward.
Read Announcement- Drug:
- AEZS-130-P02
- Announced Date:
- June 13, 2024
- Indication:
- Childhood-onset Growth Hormone Deficiency (CGHD)
Announcement
Aeterna Zentaris Inc. announced the clinical completion of its Phase 3 safety and efficacy study AEZS-130-P02 (the "DETECT-trial") evaluating macimorelin for the diagnosis of Childhood Onset Growth Hormone Deficiency ("CGHD").
AI Summary
Aeterna Zentaris Inc. announced the clinical completion of its Phase 3 DETECT-trial, which evaluated the safety and efficacy of macimorelin for diagnosing Childhood Onset Growth Hormone Deficiency (CGHD). The study, conducted in both Europe and North America, enrolled 100 pediatric subjects. It tested a single oral dose of macimorelin acetate (1.0 mg/kg) as a growth hormone stimulation test, with the final patient visit completed on June 13, 2024, in Europe.
This trial is the second and final study required under the Pediatric Investigation Plan agreed with the FDA and the European Medicines Agency (EMA). Top-line data and full study results are expected to be presented in Q3 2024. The successful completion of the DETECT-trial marks a major milestone for the company, as it aims to expand the approved use of macimorelin beyond adults to enhance diagnostics in pediatric endocrinology.
Read Announcement- Drug:
- AEZS-130-P02
- Announced Date:
- June 13, 2024
- Estimated Event Date Range:
- July 1, 2024 - September 30, 2024
- Target Action Date:
- Q3 2024
- Indication:
- Childhood-onset Growth Hormone Deficiency (CGHD)
Announcement
Aeterna Zentaris Inc. announced that Company expects to report top-line data and complete study results in Q3 2024
AI Summary
Aeterna Zentaris Inc. announced that it has successfully completed the final patient visit for its Phase 3 DETECT-trial in Europe. This trial evaluated the safety and efficacy of macimorelin for diagnosing Childhood Onset Growth Hormone Deficiency (CGHD). The study enrolled 100 subjects across Europe and North America, and its completion marks a significant milestone for the company as it broadens the potential use of macimorelin beyond its current approval for adults.
The company is on track to report top-line data and present the complete study results in the third quarter of 2024. This progress is eagerly anticipated by the company, as it underscores the potential for macimorelin to become a unique diagnostic tool in pediatric endocrinology, offering a promising option for diagnosing growth hormone deficiencies in children.
Read Announcement
Aeterna Zentaris FDA Events - Frequently Asked Questions
As of now, Aeterna Zentaris (AEZS) has not received any FDA approvals for its therapy in the last two years.
In the past two years, Aeterna Zentaris (AEZS) has reported FDA regulatory activity for AEZS-130-P02.
The most recent FDA-related event for Aeterna Zentaris occurred on August 27, 2024, involving AEZS-130-P02. The update was categorized as "Top-line results," with the company reporting: "COSCIENS Biopharma announced the top-line results of its Phase 3 safety and efficacy study AEZS-130-P02 (the "DETECT-trial") evaluating macimorelin for the diagnosis of Childhood Onset Growth Hormone Deficiency ("CGHD")."
Currently, Aeterna Zentaris has one therapy (AEZS-130-P02) targeting the following condition: Childhood-onset Growth Hormone Deficiency (CGHD).
More FDA Event Resources from MarketBeat
FDA Event Stage Terminology & Abbreviation Guide
- NDA: New Drug Application
- ANDA: Abbreviated New Drug Application
- sNDA: Supplemental New Drug Application
- BLA: Biologics License Application
- sBLA: Supplemental Biologics License Application
- FDA Approved: Approved by the FDA
- EMA: European Medicines Agency
- CE Mark: European Union Certification
- NMPA: China National Medical Products Administration
- MHLW: Japanese Ministry of Health
- FDA Meeting: Consultation with FDA
- Pre-IND: Pre-Investigational New Drug Meeting
- Breakthrough Therapy: Special FDA designation for promising therapies
- Fast Track: Accelerated FDA approval pathway
- Orphan Drug: Designation for rare disease treatments
- RPD: Rare Pediatric Disease Designation
- RMAT: Regenerative Medicine Advanced Therapy
- DSMB Review: Data Safety Monitoring Board Review
- IDMC Review: Independent Data Monitoring Committee
- MAA: MHRA Marketing Authorization Application
- RTF: Refusal to File (Rejected Application)
- 510(k): FDA Clearance for Medical Devices
- Rolling Submission: Staggered regulatory review process
This page (NASDAQ:AEZS) was last updated on 7/14/2025 by MarketBeat.com Staff
