FDA Activity Recap: April 2025 Features Major Approval in RDEB, Multiple Breakthrough Therapy Designations, and More

Last month, April 2025, the CGTLive^® team was diligently tracking the FDA's activities related to the development of cell and gene therapies for the treatment of rare, complex, and otherwise challenging diseases and disorders.

The agency has continued to ramp up its activities around these therapies as more of them progress through the pipeline in tandem. Last month proved no different, with the FDA approving Abeona Therapeutics' prademagene zamikeracel (pz-cel; EB-101), an autologous gene-corrected epidermal sheet therapy, for use in recessive dystrophic epidermolysis bullosa (RDEB), and handing out breakthrough therapy designations to uniQure's Huntington disease gene therapy AMT-130 and BrainChild Bio's pediatric brain tumor chimeric antigen receptor T-cell (CAR-T) therapy CAR-TBCB-276. Our team has highlighted these, and several other important actions, below.

Click the read more buttons for more details and information about each update.

FDA Approves Abeona Therapeutics' Epidermolysis Bullosa Gene Therapy Pz-Cel

April 29, 2025 — The FDA has approved Abeona Therapeutics' prademagene zamikeracel (pz-cel; EB-101), an autologous gene-corrected epidermal sheet therapy, for the treatment of patients with RDEB. The therapy will be marketed under the name Zevaskyn.

“Today’s approval of Zevaskyn represents a pivotal moment in the treatment of RDEB, answering the call of people living with the clinical, economic, and human impact of this devastating disease,” Vish Seshadri, PhD, MBA, the chief executive officer of Abeona, said in a statement. “We have heard from the RDEB community that there is a persistent unmet need to reliably address RDEB wounds, especially those that are chronic and prone to infection. Through a single surgical application, Zevaskyn can now offer people with RDEB the opportunity for wound healing and pain reduction in even the most severe wounds, as evidenced by the results from our pivotal phase 3 study.”

The agency's decision was based on results from the phase 3 VIITAL clinical trial (NCT04227106) and a phase 1/2a clinical (NCT01263379), both of which are now completed. Results from VIITAL reported in November 2022 indicated that treatment with the therapy significantly improved wound healing and reduced pain in 11 participants with 43 wound pairs. Notably, VIITAL met its primary end points, with over 50% wound healing and a greater magnitude of pain reduction benefit at 6 months after treatment compared to baseline and control wounds reported.

UniQure's Huntington Disease Gene Therapy AMT-130 Snags FDA Breakthrough Therapy Designation

April 24, 2025 — uniQure's AMT-130, an adeno-associated virus vector-based gene therapy currently being evaluated for the treatment of Huntington disease in 2 phase 1/2 clinical trials (NCT04120493 and NCT05243017), has been granted breakthrough therapy designation by the FDA.

Notably, AMT-130 has previously been granted regenerative medicine advanced therapy, orphan drug, and fast track designations by the agency. UniQure noted that as of April 2025, 45 patients have been treated with AMT-130.

“Receiving breakthrough therapy designation underscores both the urgent need for effective treatments for Huntington disease and the encouraging interim data demonstrating that AMT-130 has the potential to slow disease progression,” Walid Abi-Saab, MD, the chief medical officer at uniQure, said in a statement. “It’s a powerful recognition of the promise of AMT-130 and the important progress we’ve made. We deeply value the FDA’s continued commitment to advancing innovative gene therapies for patients with critical unmet needs, and we look forward to working closely with the agency to bring AMT-130 to the Huntington disease patient community as quickly as possible.”

BrainChild Bio's Pediatric Brain Tumor CAR-T BCB-276 Reels in Breakthrough Therapy Designation

April 25, 2025 — BrainChild Bio's BCB-276, an investigational CAR-T therapy that targets B7-H3 and is being developed for the treatment of a type of pediatric brain tumor referred to as diffuse intrinsic pontine glioma (DIPG), has received breakthrough therapy designation from the FDA.

The FDA’s decision was supported by data indicating overall survival benefit for patients with brain tumors from the phase 1 BrainChild-03 clinical trial (NCT04185038), which is being carried out by Seattle Children’s, BrainChild’s academic partner. The company intends to conduct a pivotal phase 2 clinical trial for BCB-276 in order to support a biologics license application for the CAR-T product for children and young adults with DIPG. This study is expected to launch in the fourth quarter of this year. A Type B meeting held between BrainChild and the FDA in late 2024 brought the agency into alignment with the company regarding the plan for the trial.

“Breakthrough therapy designation gives us the possibility to accelerate the development path for BCB-276 as a CAR T-cell therapy that can potentially transform the treatment of DIPG,” Michael Jensen, MD, the founder and chief scientific officer of BrainChild Bio, said in a statement. “This designation is a major milestone for the children and families afflicted with these devastating brain tumors and represents a new paradigm for treating central nervous system brain tumors in children and adults, including a large number of patients suffering with glioblastomas and brain metastases.”

Allogene Therapeutics’ ALLO-329 Snags FDA Fast Track Designations for 3 Rheumatology Indications

April 7, 2025 — Allogene Therapeutics’ ALLO-329, an investigational allogeneic CAR-T therapy, has been granted fast track designation from the FDA for active refractory moderate-to-severe systemic lupus erythematous (SLE); active severe/refractory idiopathic inflammatory myopathy (IIM), specifically including dermatomyositis, immune mediated necrotizing myopathy, and antisynthetase syndrome; and active refractory diffuse systemic sclerosis (SSc).

Allogene Therapeutics is currently set to evaluate ALLO-329 for these 3 indications in a phase 1 basket study referred to as RESOLUTION (NCT identifier pending). The trial will also include patients with lupus nephritis. Allogene received clearance of an investigational new drug (IND) application from the FDA for RESOLUTION in January 2025, and expects that the trial will begin in the middle of this year. Notably, the study will include 2 lymphodepletion arms: one in which patients will receive cyclophosphamide alone for lymphodepletion and the other in which no lymphodepletion regimen will be used.

"Receiving these designations for ALLO-329 underscores the versatility and transformative promise of this next-generation allogeneic CAR T investigational product in redefining the autoimmune treatment landscape," Zachary Roberts, MD, PhD, the executive vice president of research and development and the chief medical officer at Allogene. "Leveraging our extensive expertise, we've developed this off-the-shelf CAR T specifically for autoimmune diseases, prioritizing both scalability and the reduction or elimination of lymphodepletion – a key barrier in this patient population."

Fate Therapeutics’ iPSC-derived CAR-T FT819 Nabs FDA RMAT Designation for Lupus

April 14, 2025 — Fate Therapeutics’ FT819, an investigational allogeneic induced pluripotent stem cell (iPSC)-derived CAR-T therapy, has garnered regenerative medicine advanced therapy (RMAT) designation from the FDA for active moderate to severe systemic lupus erythematosus (SLE), including lupus nephritis (LN).

FT819, the development of which is supported by a California Institute of Regenerative Medicine grant that provides $7.9 million in funding, is currently being evaluated in a multicenter phase 1 clinical trial (NCT06308978) for the aforementioned SLE indications. Notably, patients in the study are receiving FT819 after a conditioning regimen that consists of either bendamustine alone or cyclophosphamide alone. Dose expansion in up to 10 patients is currently underway at a dose of 360 million cells and a dose of 900 million cells is being assessed in dose escalation. Initial clinical safety and activity data from the trial informed the FDA’s decision to provide the RMAT designation. Fate expects that additional data from the study will be announced at medical conferences this year.

“RMAT designation recognizes the unique therapeutic potential of our off-the-shelf CAR T-cell therapy to address the unmet need of a wide range of lupus patients,” Bob Valamehr, PhD, MBA, the president and chief executive officer of Fate Therapeutics, said in a statement. “We believe our current development strategy for FT819, which is designed to provide CAR T-cell therapy on-demand in a cost-effective manner and alleviate patient burden associated with intense conditioning chemotherapy and extended hospitalization, may enable treatment in the community setting and access to patients in underserved areas. With this designation, we look forward to working closely with the FDA as we seek to accelerate development of FT819 to bring this unique treatment to patients across the continuum of care.”