Drugs / prademagene zamikeracel
Regulatory milestones approvals, filings & regulatory actions · 2 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed | US (FDA) | — | epidermolysis bullosa simplex 2d, generalized, intermediate or severe, autosomal recessive | 2023-09-26 | “the Company has submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) seeking approval of EB-101” abeonatherapeutics.com ↗ |
| Clinical hold | US (FDA) | — | epidermolysis bullosa simplex 2d, generalized, intermediate or severe, autosomal recessive | 2019-09-17 | “Submitted additional transport stability data for EB-101 in response to September 17 FDA Clinical Hold Letter regarding the planned Phase 3 VIITAL™ study.” abeonatherapeutics.com ↗ |
Trials 2
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 3 | NCT05725018 | Apr 2023 → Sep 2025 overdue | recessive dystrophic epidermolysis bullosa | Abeona Therapeutics, Inc | Active not recruiting | No outcome recorded |
| Phase 3 | NCT04227106 | Jan 2020 → Oct 2022 | recessive dystrophic epidermolysis bullosa | Abeona Therapeutics, Inc | Completed | Met primary |
News releases announcing trial results or a regulatory action · 33
| Date | Issuer | Release |
|---|---|---|
| 2026-08-04 | Abeona Therapeutics, Inc | Regulatory CMS Grants New Technology Add-On Payment (NTAP) Status for ZEVASKYN® abeonatherapeutics.com ↗ |
| 2025-10-30 | Abeona Therapeutics, Inc | Regulatory Abeona Therapeutics® Announces Permanent J-Code for ZEVASKYN® (prademagene zamikeracel) abeonatherapeutics.com ↗
The new J-code for ZEVASKYN, J3389 (Topical administration, prademagene zamikeracel, per treatment) becomes effective on January 1, 2026. |
| 2025-06-24 | Abeona Therapeutics, Inc | Results Abeona Therapeutics® Announces Publication in The Lancet of Phase 3 VIITAL™ Study Data in Recessive Dystrophic Epidermolysis Bullosa abeonatherapeutics.com ↗
This article, titled, “Prademagene Zamikeracel for Recessive Dystrophic Epidermolysis Bullosa Wounds (VIITAL): A Two-Centre, Randomised, Open-Label, Intrapatient-Controlled Phase 3 Trial,” representing the primary publication of the full VIITAL study data, is now available online and will be published in a future print issue of The Lancet . |
| 2025-04-29 | Abeona Therapeutics, Inc | Regulatory U.S. FDA Approves ZEVASKYN™ (prademagene zamikeracel), the First and Only Cell-Based Gene Therapy for Patients with Recessive Dystrophic Epidermolysis Bullosa (RDEB) abeonatherapeutics.com ↗
There is no cure for RDEB and ZEVASKYN is the only FDA-approved product to treat RDEB wounds with a single application. |
| 2024-11-12 | Abeona Therapeutics, Inc | Regulatory Abeona Therapeutics® Announces FDA Acceptance of BLA Resubmission of Pz-cel for the Treatment of Recessive Dystrophic Epidermolysis Bullosa abeonatherapeutics.com ↗
Abeona Therapeutics Inc. (Nasdaq: ABEO) today announced that the U.S. Food and Drug Administration (FDA) has accepted for review Abeona’s resubmission of its Biologics License Application (BLA) for prademagene zamikeracel (pz-cel), its investigational autologous cell-based gene therapy, as a potential new treatment for recessive dystrophic epidermolysis bullosa (RDEB). |
| 2024-10-29 | Abeona Therapeutics, Inc | Regulatory Abeona Therapeutics® Completes Pz-cel Biologics License Application Resubmission to U.S. Food and Drug Administration abeonatherapeutics.com ↗ |
| 2024-08-13 | Abeona Therapeutics, Inc | Regulatory Abeona Therapeutics® Announces Favorable Medicare Reimbursement Decisions for Pz-cel abeonatherapeutics.com ↗ |
| 2024-04-22 | Abeona Therapeutics, Inc | Regulatory Abeona Therapeutics Provides Regulatory Update on Pz-cel abeonatherapeutics.com ↗
The U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) in response to the Company’s Biologics License Application (BLA) for pz-cel for the treatment of patients with recessive dystrophic epidermolysis bullosa (RDEB). |
| 2024-03-18 | Abeona Therapeutics, Inc | Regulatory Abeona Therapeutics Reports Full Year 2023 Financial Results and Announces Completion of FDA Inspections abeonatherapeutics.com ↗
Abeona also announced today that the U.S. Food and Drug Administration (FDA) has completed a Pre-License Inspection (PLI) of its Cleveland, Ohio manufacturing facility related to the Company’s Biologics License Application (BLA) for pz-cel (prademagene zamikeracel) for recessive dystrophic epidermolysis bullosa (RDEB). |
| 2023-11-27 | Abeona Therapeutics, Inc | Regulatory Abeona Therapeutics Announces FDA Accepts and Grants Priority Review for Pz-cel Biologics License Application (BLA) abeonatherapeutics.com ↗ |
| 2023-09-26 | Abeona Therapeutics, Inc | Regulatory Abeona Therapeutics Submits Biologics License Application to U.S. FDA Seeking Priority Review and Approval of EB-101 for the Treatment of Patients with Recessive Dystrophic Epidermolysis Bullosa abeonatherapeutics.com ↗ |
| 2023-08-30 | Abeona Therapeutics, Inc | Regulatory Abeona Therapeutics Announces Positive Pre-BLA Meeting with FDA for EB-101 and Plans for BLA Submission abeonatherapeutics.com ↗ |
| 2023-06-09 | Abeona Therapeutics, Inc | Regulatory Abeona Therapeutics Announces Regulatory Update on Biologics License Application (BLA) for EB-101 abeonatherapeutics.com ↗ |
| 2023-05-11 | Abeona Therapeutics, Inc | Results Abeona Therapeutics Announces Additional Phase 3 VIITAL™ Study Results for EB-101 Presented at the International Societies for Investigative Dermatology 2023 Meeting abeonatherapeutics.com ↗
Abeona Therapeutics Inc. (Nasdaq: ABEO) today announced an oral presentation of additional data from the Company's pivotal Phase 3 VIITAL™ study evaluating its investigational EB-101 for recessive dystrophic epidermolysis bullosa (RDEB). |
| 2022-11-03 | Abeona Therapeutics, Inc | Results Abeona Therapeutics Announces Positive Topline Results with Both Co-Primary Endpoints Met in Pivotal Phase 3 VIITAL™ Study of EB-101 abeonatherapeutics.com ↗
The VIITAL study met its two co-primary efficacy endpoints demonstrating statistically significant, clinically meaningful improvements in wound healing and pain reduction in large chronic RDEB wounds. |
| 2021-07-07 | Abeona Therapeutics, Inc | Results Abeona Therapeutics Announces Updated EB-101 Phase 1/2a Clinical Results in Recessive Dystrophic Epidermolysis Bullosa at the Society for Pediatric Dermatology 46th Annual Meeting abeonatherapeutics.com ↗
Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in gene and cell therapy, today announced updated Phase 1/2a clinical trial results up to six years following treatment with Abeona’s investigational EB-101 for recessive dystrophic epidermolysis bullosa (RDEB). |
| 2020-07-10 | Abeona Therapeutics, Inc | Results Abeona Therapeutics Announces Two Presentations Related to Its RDEB Clinical Program at the Society for Pediatric Dermatology 45th Annual Meeting abeonatherapeutics.com ↗
The data presented at SPD showed that EB-101 treatment of large, chronic wounds resulted in considerable and durable reduction in wound burden, which was associated with long-term pain relief for up to five years. |
| 2019-12-09 | Abeona Therapeutics, Inc | Regulatory Abeona Therapeutics Cleared to Initiate Pivotal Phase 3 Clinical Trial Evaluating EB-101 Gene Therapy for Recessive Dystrophic Epidermolysis Bullosa abeonatherapeutics.com ↗ |
| 2019-11-12 | Abeona Therapeutics, Inc | Results Abeona Therapeutics Reports Third Quarter 2019 Financial Results and Business Updates abeonatherapeutics.com ↗
Three years after treatment with EB-101, a majority of RDEB patients had sustained wound healing, with 80% (16/20) of wounds achieving ≥50% healing, and 70% (14/20) achieving ≥75% |
| 2019-11-01 | EyebioKorea, Inc. | Results 아이바이오코리아, "약효, 편의성, 가성비 3박자 갖춘 안구건조증, 황반변성 치료제 개발중" eyebiokorea.com ↗
임상 1 상에서 독성 문제가 없다는 것을 확인했다 |
| 2019-10-15 | Abeona Therapeutics, Inc | Results Abeona Therapeutics Announces Publication of Positive Long-Term Data from Phase 1/2a Clinical Trial Evaluating EB-101 Gene Therapy for Recessive Dystrophic Epidermolysis Bullosa abeonatherapeutics.com ↗ |
| 2019-09-23 | Abeona Therapeutics, Inc | Regulatory Abeona Therapeutics Provides Regulatory Update Ahead of Pivotal Phase 3 Clinical Trial for EB-101 in Recessive Dystrophic Epidermolysis Bullosa abeonatherapeutics.com ↗
the FDA will not provide approval for the Company to begin its planned Phase 3 clinical trial for EB-101 until it submits to the FDA additional data points on transport stability of EB-101 to clinical sites |
| 2018-05-17 | Abeona Therapeutics, Inc | Results Abeona Reports Update from EB-101 Gene Therapy in Epidermolysis Bullosa at 21st Annual ASGCT Meeting abeonatherapeutics.com ↗
The results demonstrate robust and durable clinical effects achieved throughout various timepoints post-administration. |
| 2018-01-29 | Abeona Therapeutics, Inc | Regulatory Abeona Receives FDA Regenerative Medicine Advanced Therapy Designation for EB-101 Gene Therapy in Epidermolysis Bullosa abeonatherapeutics.com ↗ |
| 2017-08-29 | Abeona Therapeutics, Inc | Regulatory Abeona Therapeutics Receives FDA Breakthrough Therapy Designation for EB-101 Autologous Cell Therapy in Epidermolysis Bullosa abeonatherapeutics.com ↗
Abeona Therapeutics Inc. (Nasdaq:ABEO), a leading clinical-stage biopharmaceutical company focused on developing novel gene therapies for life-threatening rare diseases, announced today that the U.S. Food and Drug Administration has granted Breakthrough Therapy designation status to the Company’s EB-101 gene therapy program for patients with Recessive Dystrophic Epidermolysis Bullosa (RDEB). |
| 2017-05-30 | Abeona Therapeutics, Inc | Regulatory Abeona Therapeutics Receives Rare Pediatric Disease Designation for EB-101 Gene Therapy Product for Patients with Epidermolysis Bullosa abeonatherapeutics.com ↗ |
| 2017-05-25 | Abeona Therapeutics, Inc | Regulatory Abeona Therapeutics Receives FDA Orphan Drug Designation for EB-101 Gene Therapy Product for Patients with Epidermolysis Bullosa abeonatherapeutics.com ↗ |
| 2017-05-02 | Abeona Therapeutics, Inc | Results Abeona Therapeutics Provides Update on EB-101 Gene Therapy for Severe Form of Epidermolysis Bullosa from the Society for Investigative Dermatology Conference abeonatherapeutics.com ↗
EB-101 Demonstrates Significant Wound Healing (defined as greater than 50% healed) in 100% of Treated Wounds (36/36) at 3 Months |
| 2017-04-25 | Abeona Therapeutics, Inc | Results Abeona Therapeutics Announces EB-101 Clinical Update to be Presented at the Society for Investigative Dermatology Annual Meeting abeonatherapeutics.com ↗ |
| 2017-03-08 | Abeona Therapeutics, Inc | Regulatory Abeona Therapeutics Receives Orphan Drug Designation in the European Union for EB-101 Gene Therapy Clinical Trial for Epidermolysis Bullosa abeonatherapeutics.com ↗
The orphan designation also provides 10 years of market exclusivity in the European Union from similar medicines for similar indications, an important value driver for Abeona as we expand our clinical programs in Europe. |
| 2016-11-02 | Abeona Therapeutics, Inc | Results Abeona Therapeutics Announces JAMA Publication of Positive Phase 1 Study Results for EB-101 Gene Therapy Clinical Trial for Epidermolysis Bullosa abeonatherapeutics.com ↗ |
| 2016-09-08 | Abeona Therapeutics, Inc | Results Abeona Therapeutics Enrolls 5th Patient in Phase 1/2 Gene Therapy Clinical Trial for Epidermolysis Bullosa abeonatherapeutics.com ↗
EB-101 (gene-corrected skin grafts) has demonstrated promising clinical efficacy in patients for the treatment of recessive dystrophic epidermolysis bullosa (RDEB) Phase 1/2 clinical trial (n=5 subjects) enrolled with data indicating significant improvements in wound healing and gene expression 12 months post-grafting (n=4) |
| 2016-08-09 | Abeona Therapeutics, Inc | Results Abeona Therapeutics, EB Research Partnership and EB Medical Research Foundation to Collaborate on Epidermolysis Bullosa (EB) Treatments abeonatherapeutics.com ↗ |
All press releases naming this drug 45 releases
Evidence & citations 7 cited values
Every value below carries the sentence it was read from. 4 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | prademagene zamikeracel | “Prademagene zamikeracel is an autologous COL7A1 gene-modified cellular sheet that is sutured onto to a large, chronic RDEB wound.” PMID 40570869 ↗ Jun 2025 |
| Known as | EB-101 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT05725018 ↗ |
| Known as | Prademagen zamikeracel | ChEMBL registry synonym — accepted as the source's own label CHEMBL4594312 ↗ |
| Action | Restore | “gene-corrected keratinocyte sheets with C7 expression (EB-101)” NCT05725018 ↗ |
| Modality | Cell therapy | “Prademagene zamikeracel is an autologous COL7A1 gene-modified cellular sheet” PMID 40570869 ↗ Jun 2025 |
| Route | Other | “one-time surgical application of prademagene zamikeracel” PMID 40570869 ↗ Jun 2025 |
| Target | COL7A1 | “transduced with a recombinant retrovirus containing a full-length COL7A1 expression cassette for C7” NCT04227106 ↗ |