This section highlights FDA-related milestones and regulatory updates for drugs developed by Abeona Therapeutics (ABEO).
Over the past two years, Abeona Therapeutics has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
ABO-102, PMN310, pz-cel, and ZEVASKYN. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
ABO-102 - FDA Regulatory Timeline and Events
ABO-102 is a drug developed by Abeona Therapeutics for the following indication: Sanfilippo syndrome type A (MPS IIIA).
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:
- ABO-102
- Announced Date:
- December 19, 2024
- Indication:
- Sanfilippo syndrome type A (MPS IIIA)
Announcement
Ultragenyx Pharmaceutical nnounced the submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA or the Agency) seeking accelerated approval for UX111 (ABO-102) AAV gene therapy as a treatment for patients with Sanfilippo syndrome type A (MPS IIIA).
AI Summary
Ultragenyx Pharmaceutical recently announced that it has submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for its new gene therapy, UX111 (also known as ABO‑102). This submission is aimed at securing accelerated approval for the treatment of patients with Sanfilippo syndrome type A (MPS IIIA), a rare and severe genetic disorder that affects cognitive and motor functions.
The company’s strategy with UX111 focuses on utilizing an AAV gene therapy platform to address the underlying genetic causes of the syndrome. By seeking accelerated approval, Ultragenyx hopes to significantly reduce the time to bring this vital treatment to patients in need. This submission underlines the company’s commitment to advancing innovative therapies for rare diseases and improving the quality of life for affected individuals.
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PMN310 - FDA Regulatory Timeline and Events
PMN310 is a drug developed by Abeona Therapeutics for the following indication: PMN310 is a novel monoclonal antibody.
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:
- PMN310
- Announced Date:
- February 25, 2025
- Indication:
- PMN310 is a novel monoclonal antibody
Announcement
ProMIS Neurosciences Inc. announced important progress in the PRECISE-AD Phase 1b clinical trial with the dosing of multiple patients with its lead drug candidate, PMN310, designed for the treatment of Alzheimer's disease (AD).
AI Summary
ProMIS Neurosciences Inc. announced key progress in the PRECISE-AD Phase 1b clinical trial for its lead drug candidate, PMN310, aimed at treating Alzheimer’s disease. The trial has begun dosing multiple patients to evaluate the safety, tolerability, and pharmacokinetics of PMN310. This milestone is important because PMN310 is designed to target toxic amyloid-beta oligomers, which are believed to contribute significantly to Alzheimer’s progression, while avoiding non-toxic amyloid forms like monomers and plaques. The selective mechanism of PMN310 could offer a safer and more effective treatment option compared to current therapies. Researchers and clinicians are optimistic that the clinical data from this trial will highlight PMN310’s potential to help patients and provide further insights into Alzheimer’s treatment, marking a promising step forward in addressing the unmet need for better Alzheimer’s therapeutics.
Read Announcement- Drug:
- PMN310
- Announced Date:
- January 10, 2025
- Indication:
- PMN310 is a novel monoclonal antibody
Announcement
ProMIS Neurosciences Inc. announced the initiation of its Phase 1b clinical trial (PRECISE-AD) evaluating its lead therapeutic candidate, PMN310, in Alzheimer's disease (AD).
AI Summary
ProMIS Neurosciences Inc. has recently launched its Phase 1b clinical trial, called PRECISE-AD, to evaluate its lead therapeutic candidate, PMN310, in the treatment of Alzheimer’s disease. This study is designed to test PMN310—a humanized IgG1 antibody that selectively targets toxic amyloid-beta oligomers, which are believed to drive AD pathology—while minimizing interactions with amyloid plaques. By focusing on oligomers, PMN310 could reduce the risk of side effects like amyloid-related imaging abnormalities (ARIA) that are common with other AD treatments.
The trial will enroll approximately 100 patients with mild cognitive impairment or early Alzheimer’s and will assess the drug’s safety, tolerability, and pharmacokinetics over a 12-month period. Researchers also plan to evaluate key biomarkers and clinical efficacy measures, with initial interim data expected in the first half of 2026.
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pz-cel - FDA Regulatory Timeline and Events
pz-cel is a drug developed by Abeona Therapeutics for the following indication: For the treatment of patients with recessive dystrophic epidermolysis bullosa.
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:
- pz-cel
- Announced Date:
- April 29, 2025
- Indication:
- For the treatment of patients with recessive dystrophic epidermolysis bullosa
Announcement
Abeona Therapeutics Inc. announced the U.S. Food and Drug Administration (FDA) has approved ZEVASKYN™ (pronounced as ‘ZEE-vah-skin') (prademagene zamikeracel) gene-modified cellular sheets, also known as pz-cel, as the first and only autologous cell-based gene therapy for the treatment of wounds in adult and pediatric patients with recessive dystrophic epidermolysis bullosa (RDEB), a serious and debilitating genetic skin disease.
AI Summary
Abeona Therapeutics Inc. announced that the FDA has approved ZEVASKYN™ (prademagene zamikeracel), marking a breakthrough as the first and only autologous cell-based gene therapy for treating wounds in both adult and pediatric patients with recessive dystrophic epidermolysis bullosa (RDEB). RDEB is a serious genetic skin disease with no cure, and patients suffer from chronic, painful wounds that are difficult to heal. ZEVASKYN works by using a patient’s own genetically modified skin cells to produce functional type VII collagen, which is essential for skin integrity and wound healing. The approval was supported by the pivotal Phase 3 VIITAL™ study, which demonstrated significant wound healing and pain reduction after a single treatment. ZEVASKYN is expected to be available at Qualified Treatment Centers starting in the third quarter of 2025.
Read Announcement- Drug:
- pz-cel
- Announced Date:
- March 20, 2025
- Indication:
- For the treatment of patients with recessive dystrophic epidermolysis bullosa
Announcement
Abeona Therapeutics Inc. announced progress in the ongoing review with the U.S. Food and Drug Administration (FDA) regarding prademagene zamikeracel (pz-cel) and plans for the U.S. commercial launch of pz-cel for recessive dystrophic epidermolysis bullosa (RDEB), if approved.
AI Summary
Abeona Therapeutics Inc. recently announced notable progress in its FDA review of prademagene zamikeracel (pz-cel), an investigational gene therapy for recessive dystrophic epidermolysis bullosa (RDEB). The FDA is currently evaluating Abeona’s Biologics License Application (BLA) for pz-cel, with a Prescription Drug User Fee Act (PDUFA) target action date set for April 29, 2025. As discussions continue regarding post-marketing requirements and the draft label, Abeona is actively preparing for a potential U.S. commercial launch. The company has outlined plans to treat the first patient in the third quarter of 2025 if approval is granted. In parallel, efforts are underway to onboard specialized treatment centers across the country, engage with payers to ensure patient access, and strengthen its manufacturing and supply chain capacity to support the launch.
Read Announcement- Drug:
- pz-cel
- Announced Date:
- August 13, 2024
- Indication:
- For the treatment of patients with recessive dystrophic epidermolysis bullosa
Announcement
Abeona Therapeutics Inc. announced that the Centers for Medicare and Medicaid Services (CMS) has granted a product-specific procedure code ICD-10-PCS (International Classification of Diseases, 10th Revision, Procedure Coding System) for prademagene zamikeracel (pz-cel), Abeona's investigational autologous cell-based gene therapy currently in development for recessive dystrophic epidermolysis bullosa (RDEB).
AI Summary
Abeona Therapeutics Inc. announced that the Centers for Medicare and Medicaid Services (CMS) has granted a product-specific ICD-10-PCS procedure code for its investigational gene therapy, prademagene zamikeracel (pz-cel). This code is designed for the treatment of recessive dystrophic epidermolysis bullosa (RDEB) and will be effective starting October 1, 2024, provided that pz-cel receives U.S. marketing approval. The new coding system is expected to simplify inpatient hospital billing, documentation, and analysis by offering an efficient and accurate way to track the use of the therapy. In addition, CMS has assigned Medicare reimbursement for pz-cel to one of the highest inpatient payment groups for cell and gene therapies, reinforcing the breakthrough nature of this treatment. This move supports Abeona’s efforts to ensure streamlined access and proper reimbursement for RDEB patients once the therapy is approved.
Read Announcement- Drug:
- pz-cel
- Announced Date:
- August 12, 2024
- Estimated Event Date Range:
- July 1, 2024 - December 31, 2024
- Target Action Date:
- H2 2024
- Indication:
- For the treatment of patients with recessive dystrophic epidermolysis bullosa
Announcement
Abeona Therapeutics Inc. announced that BLA resubmission remains on track for 2H 2024
AI Summary
Abeona Therapeutics Inc. announced that its resubmission of the Biologics License Application (BLA) for prademagene zamikeracel (pz-cel) remains on track for the second half of 2024. The company has made significant progress in addressing the Chemistry, Manufacturing, and Controls (CMC) issues raised by the FDA in its Complete Response Letter. Nearly all required CMC data has been generated, and Abeona is in the final stages of validating the remaining sterility and identity assays based on recent FDA feedback.
This progress was discussed during a recent Type A meeting with the FDA, where preliminary alignment was achieved for the planned resubmission. Abeona’s efforts aim to prepare the application for resubmission later this year, potentially advancing pz-cel as a new treatment option for patients with recessive dystrophic epidermolysis bullosa.
Read Announcement- Drug:
- pz-cel
- Announced Date:
- August 12, 2024
- Indication:
- For the treatment of patients with recessive dystrophic epidermolysis bullosa
Announcement
Abeona Therapeutics Inc. announced that it has completed a Type A meeting with the U.S. Food and Drug Administration (FDA) to discuss Abeona's forthcoming resubmission of its Biologics License Application (BLA) for prademagene zamikeracel (pz-cel), its investigational first-in-class, autologous cell-based gene therapy currently in development for recessive dystrophic epidermolysis bullosa (RDEB).
AI Summary
Abeona Therapeutics recently completed a Type A meeting with the U.S. FDA to discuss its upcoming resubmission of the Biologics License Application (BLA) for prademagene zamikeracel (pz-cel). Pz-cel is an investigational, first-in-class, autologous cell-based gene therapy aimed at treating recessive dystrophic epidermolysis bullosa (RDEB). During the meeting on August 8, 2024, the company presented data and reports addressing nearly all the Chemistry Manufacturing and Controls requirements noted in the previous review. The FDA provided preliminary alignment on Abeona’s resubmission plan, while two remaining issues—related to sterility and identity assay validations—are still being resolved under the agency’s guidance.
Abeona expects to resubmit its BLA in the second half of 2024, with an anticipated FDA action date roughly six months after submission. This progress is a key step toward potentially bringing a new treatment option to RDEB patients.
Read Announcement- Drug:
- pz-cel
- Announced Date:
- April 22, 2024
- Indication:
- For the treatment of patients with recessive dystrophic epidermolysis bullosa
Announcement
- Abeona Therapeutics Inc. announced a regulatory update for prademagene zamikeracel (pz-cel).
AI Summary
Abeona Therapeutics recently announced that the FDA issued a Complete Response Letter (CRL) for its Biologics License Application (BLA) for prademagene zamikeracel (pz-cel), a treatment for recessive dystrophic epidermolysis bullosa (RDEB). The CRL was based solely on the need for additional Chemistry Manufacturing and Controls (CMC) information, particularly regarding the validation of certain manufacturing and release testing methods. The FDA noted that the proposed timeline for submitting the updated CMC data would not allow enough time for a review before the May 25, 2024 PDUFA date. Importantly, there were no deficiencies identified in the clinical efficacy or safety data, and no additional clinical trials were requested. Abeona is committed to addressing the CMC requirements and expects to complete and resubmit the necessary updates in the third quarter of 2024.
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ZEVASKYN - FDA Regulatory Timeline and Events
ZEVASKYN is a drug developed by Abeona Therapeutics for the following indication: To Treat Wounds in Painful Skin Disorder.
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:
- ZEVASKYN
- Announced Date:
- June 24, 2025
- Indication:
- To Treat Wounds in Painful Skin Disorder
Announcement
Abeona Therapeutics Inc. announced that The Lancet has published results from the pivotal Phase 3 VIITAL study (NCT04227106) evaluating the efficacy and safety of ZEVASKYN (prademagene zamikeracel) gene-modified cellular sheets, also known as pz-cel, for the treatment of wounds in adult and pediatric patients with recessive dystrophic epidermolysis bullosa (RDEB).
AI Summary
Abeona Therapeutics Inc. announced that The Lancet has published the full results from its pivotal Phase 3 VIITAL study (NCT04227106) on ZEVASKYN™ (prademagene zamikeracel). This study evaluated the effectiveness and safety of these gene-modified cellular sheets, also called pz-cel, for treating wounds in both adult and pediatric patients with recessive dystrophic epidermolysis bullosa (RDEB). The research showed that a single treatment led to significant wound healing and pain reduction in large, chronic RDEB wounds, with a favorable safety profile.
The publication in The Lancet, a leading global medical journal, highlights the progress of this first and only autologous cell-based gene therapy approved by the FDA for RDEB wounds. These promising results support the potential of ZEVASKYN to improve quality of life for patients suffering from this lifelong, debilitating skin condition.
Read Announcement- Drug:
- ZEVASKYN
- Announced Date:
- May 14, 2025
- Indication:
- To Treat Wounds in Painful Skin Disorder
Announcement
Abeona Therapeutics Inc announced that Lurie Children's is now activated as the first Qualified Treatment Center (QTC) for ZEVASKYN (prademagene zamikeracel) gene-modified cellular sheets
AI Summary
Abeona Therapeutics Inc. announced that Ann & Robert H. Lurie Children’s Hospital of Chicago is now activated as the first Qualified Treatment Center (QTC) for ZEVASKYN (prademagene zamikeracel) gene-modified cellular sheets. This activation allows the hospital to begin identifying patients for ZEVASKYN treatment, a breakthrough gene therapy specifically designed to treat wounds in patients with recessive dystrophic epidermolysis bullosa (RDEB), a rare and severe skin disorder.
The therapy works by grafting gene-corrected cellular sheets onto challenging wounds, potentially offering long-term healing, pain reduction, and a lower risk of infections. Abeona is also supporting eligible patients and their families throughout the treatment process with its Abeona Assist™ comprehensive patient services program. This collaboration highlights a strong commitment to providing new treatment options for those affected by RDEB.
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