FDA Events for Horizon Therapeutics Public (HZNP)
This section highlights FDA-related milestones and regulatory updates for drugs developed by Horizon Therapeutics Public (HZNP).
Over the past two years, Horizon Therapeutics Public has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
Bempikibart. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
Bempikibart - FDA Regulatory Timeline and Events
Bempikibart is a drug developed by Horizon Therapeutics Public for the following indication: For Severe Alopecia Areata.
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:
- Bempikibart
- Announced Date:
- April 30, 2025
- Indication:
- For Severe Alopecia Areata
Announcement
Q32 Bio Inc. announced that the United States Food and Drug Administration ("FDA") has granted Fast Track designation (FTD) to Q32 Bio's bempikibart (ADX-914) for the treatment of AA.
AI Summary
Q32 Bio Inc. recently announced that the FDA has granted Fast Track designation to its investigational drug bempikibart (ADX‑914) for treating alopecia areata (AA). This designation recognizes the serious nature of AA and the significant unmet medical need in treating the condition. Bempikibart, a fully human anti‑IL‑7Rα antibody, works by blocking IL‑7 and TSLP signaling to help re-regulate the immune system. Its potential as a novel and differentiated treatment option is backed by promising clinical data observed in the ongoing Phase 2 SIGNAL‑AA trial.
The Fast Track status is designed to speed up the development and review process by allowing more frequent interactions with the FDA, which could ultimately lead to faster approval. This milestone underscores the commitment of Q32 Bio to deliver a potentially transformative therapy for patients suffering from AA.
Read Announcement- Drug:
- Bempikibart
- Announced Date:
- April 16, 2025
- Estimated Event Date Range:
- January 1, 2026 - June 30, 2026
- Target Action Date:
- H1 2026
- Indication:
- For Severe Alopecia Areata
Announcement
Q32 Bio Inc. announced that SIGNAL-AA Part B topline data readout on-track for 1H'26 --
AI Summary
Q32 Bio Inc. announced that it is on track to report the SIGNAL-AA Part B topline data in the first half of 2026. This signal-phase trial is an important step in the clinical development of bempikibart, a fully human anti-IL-7Rα antibody designed to treat patients with alopecia areata. In Part B, approximately 20 patients with severe or very severe alopecia areata will be enrolled in an open-label study.
The dosing plan includes an initial loading regimen of 200mg given weekly for four weeks, followed by a maintenance dose of 200mg every other week over 32 weeks, for a total treatment period of 36 weeks, with a follow-up period extending to 52 weeks. The topline data readout is expected to provide insights into the efficacy and safety of bempikibart and help pave the way for future pivotal trials aimed at offering a new treatment option for patients.
Read Announcement- Drug:
- Bempikibart
- Announced Date:
- April 16, 2025
- Indication:
- For Severe Alopecia Areata
Announcement
Q32 Bio Inc. announced that the Company has dosed the first patients in both the Part A open-label extension (OLE) and Part B of the SIGNAL-AA Phase 2a clinical trial evaluating bempikibart in patients with alopecia areata (AA).
AI Summary
Q32 Bio Inc. announced a key milestone in the SIGNAL-AA Phase 2a clinical trial for its experimental drug, bempikibart, targeting alopecia areata (AA). The company has dosed the first patients in both the Part A open-label extension (OLE) and Part B portions of the study. The updated trial builds on earlier encouraging results from Part A, where patients demonstrated sustained responses and improvements in their Severity of Alopecia Tool (SALT) scores. In Part B, approximately 20 patients with severe AA will receive a treatment regimen that starts with a loading dose followed by a maintenance dose over 36 weeks, with follow-up continuing through 52 weeks. Q32 Bio believes that the favorable clinical activity and the well-tolerated safety profile of bempikibart could offer a new, differentiated treatment option for AA, potentially transforming care for patients if approved.
Read Announcement- Drug:
- Bempikibart
- Announced Date:
- March 11, 2025
- Indication:
- For Severe Alopecia Areata
Announcement
Q32 Bio Inc. provided recent corporate updates.
AI Summary
Q32 Bio Inc., a clinical-stage biotechnology company, recently provided corporate updates focused on its bempikibart program for treating alopecia areata (AA). At the 2025 American Academy of Dermatology Annual Meeting, the company presented late-breaking Phase 2a Part A data showing encouraging clinical activity. Patients experienced continued hair growth responses even months after completing treatment, and bempikibart demonstrated a strong safety profile with durable results.
Building on these positive results, Q32 Bio is set to advance bempikibart in two additional studies: an open-label extension (OLE) of the SIGNAL-AA trial and Phase 2a Part B. The company plans to dose patients in the first half of 2025, with topline data from Part B expected in the first half of 2026. Financially, Q32 Bio reported $78 million in cash and cash equivalents as of December 31, 2024, providing runway into the second half of 2026.
Read Announcement- Drug:
- Bempikibart
- Announced Date:
- March 8, 2025
- Indication:
- For Severe Alopecia Areata
Announcement
Q32 Bio Inc. announced additional results from Part A of its SIGNAL-AA Phase 2a clinical trial of bempikibart in patients with alopecia areata (AA) at the 2025 American Academy of Dermatology (AAD) Meeting in Orlando, FL.
AI Summary
Q32 Bio Inc. presented promising findings from Part A of its SIGNAL-AA Phase 2a trial for bempikibart at the 2025 American Academy of Dermatology Meeting. The trial focused on patients with severe alopecia areata and demonstrated significant improvements in hair loss, measured by a reduction in Severity of Alopecia Tool (SALT) scores. At week 24, patients treated with bempikibart experienced notable decreases in their SALT scores compared to the placebo group. Remarkably, many patients maintained or even deepened their hair regrowth after dosing ended at week 24, with continued positive results observed through week 36 and up to week 55 in some cases. The data further indicated that bempikibart is safe and well-tolerated, with supportive pharmacokinetic and receptor engagement results, emphasizing its potential as a long-lasting treatment option for alopecia areata.
Read Announcement
Horizon Therapeutics Public FDA Events - Frequently Asked Questions
As of now, Horizon Therapeutics Public (HZNP) has not received any FDA approvals for its therapy in the last two years.
In the past two years, Horizon Therapeutics Public (HZNP) has reported FDA regulatory activity for Bempikibart.
The most recent FDA-related event for Horizon Therapeutics Public occurred on April 30, 2025, involving Bempikibart. The update was categorized as "Data Presentation," with the company reporting: "Q32 Bio Inc. announced that the United States Food and Drug Administration ("FDA") has granted Fast Track designation (FTD) to Q32 Bio's bempikibart (ADX-914) for the treatment of AA."
Currently, Horizon Therapeutics Public has one therapy (Bempikibart) targeting the following condition: For Severe Alopecia Areata.
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:HZNP) was last updated on 7/14/2025 by MarketBeat.com Staff
