Changes: A turbulent year for FDA oversight

Characterizing 2025 from a regulatory perspective is uniquely challenging. Perhaps David Bowie said it best when he said, “I don’t know where I’m going from here, but I promise it won’t be boring.”

In 2025, the agency found itself in a state of sustained disruption, punctuated by staff cuts, resignations and abrupt leadership changes. In March, longtime CBER director Dr. Peter Marks resigned under reported pressure from the Department of Health and Human Services. Scott Steele, a senior CBER advisor, took over as interim leader until May, when Dr. Vinay Prasad — an outspoken critic of Marks — was appointed CBER director. A month later, FDA’s OTP director, Nicole Verdun, and deputy director, Rachael Anatol, were placed on administrative leave. In late July, Prasad resigned from CBER amid political controversy — only to return to his position weeks later.

In November, hundreds of biotech leaders signed a letter to FDA Commissioner Dr. Marty Makary, noting that, in a survey of industry CEOs, 82% of respondents said they were worried about the FDA’s ability to function.

It wasn’t all bad news though. In June, the FDA eliminated the REMS for currently approved BCMA- and CD19-directed autologous CAR-T cell therapies — a move that eased the burden on patients and health care systems. In September, CDER and CBER jointly proposed the Rare Disease Evidence Principles (RDEP) process to facilitate the approval of drugs to treat rare diseases with very small patient populations. CBER then issued three rare disease-focused draft guidances, emphasizing greater regulatory flexibility with respect to the development and post-approval studies of CGTs.

In November, in another win for rare disease therapies, the agency outlined a novel path to market entry, known as the plausible mechanism pathway, for bespoke, personalized therapies where randomized trials are not feasible.

All told, the cell and gene therapy sector walked away with five new approvals, and some key label expansions. While this falls slightly below the seven approvals recorded in both 2023 and 2024, it remains a meaningful achievement.

Here’s a rundown of 2025 FDA approvals:

Encelto (revakinagene taroretcel-lwey)
Neurotech Pharmaceuticals

Approval: March 2025
Indication: Macular telangiectasia type 2 (MacTel)

Encelto is the first and only FDA approved treatment for MacTel, a rare neurodegenerative disease of the retina in adults that causes progressive and irreversible vision loss. Encelto utilizes a surgically implanted encapsulated cell therapy technology designed to continually deliver therapeutic doses of ciliary neurotrophic factor to the retina to assist in slowing the progression of the disease

Zevaskyn (prademagene zamikeracel)
Abeona Therapeutics

Approval: April 2025
Indication: Recessive dystrophic epidermolysis bullosa (RDEB)

Zevaskyn is an autologous cell-based gene therapy for the treatment of wounds in adult and pediatric patients with RDEB, a serious and debilitating genetic skin disease. The therapy, administered through a one-time surgical procedure, is the only FDA-approved product to treat RDEB wounds with a single application.

Papzimeos (zopapogene imadenovec-drba)
Precigen

Approval: August 2025
Indication: Recurrent respiratory papillomatosis (RRP)

Papzimeos, a non-replicating adenoviral vector-based immunotherapy, is the first and only FDA-approved therapy for adults with RRP. The rare, debilitating and potentially life-threatening disease is caused by chronic HPV 6 or HPV 11 infection, which results in recurrent benign tumors in the respiratory tract. Papzimeos, administered via subcutaneous injection, offers a novel mechanism of action distinct from traditional treatments, which rely primarily on repeated surgical interventions.

Itvisma (onasemnogene abeparvovec-brve)
Novartis

Approval: November 2025
Indication: Children two years and older with spinal muscular atrophy (SMA)

Itvisma was approved for the treatment of children two years and older, teens and adults living with SMA with a confirmed mutation in the survival motor neuron 1 (SMN1) gene, making it the first and only gene replacement therapy available for a broad SMA population. Itvisma is an intrathecal formulation of Novartis’ Zolgensma, administered directly to the central nervous system via a single intrathecal injection independent of patient weight.

Waskyra (etuvetidigene autotemcel)
Fondazione Telethon

Approval: December 2025
Indication: Wiskott-Aldrich syndrome (WAS)

Waskyra is the first gene therapy for WAS and the first approved cell and gene therapy product from a nonprofit applicant. Waskyra is indicated for pediatric patients six months and older and adults with WAS who have a mutation in the WAS gene and for whom hematopoietic stem cell transplantation is appropriate and no suitable human leukocyte antigen-matched related stem cell donor is available. WAS is characterized by bleeding, eczema, recurrent infections, and increased susceptibility to autoimmunity and lymphoreticular malignancies. Waskyra consists of a single administration of autologous CD34+ hematopoietic stem and progenitor cells that have been transduced with a lentiviral vector encoding the WAS gene.

Label expansions

Vyjuvek (beremagenegeperpavec-svdt)
Krystal Biotech

Update: September 2025
Expanded indication: Dystrophic epidermolysis bullosa (DEB) patients from birth; Application flexibility

The label for Vyjuvek, a topical gene therapy, was expanded to include DEB patients from birth and to provide patients and caregivers the ability to apply Vyjuvek themselves. The herpes-simplex virus type 1 vector-based gene therapy was approved in May 2023 for the treatment of wounds in patients six months of age and older with the serious, rare skin disease with mutation(s) in the collagen type VII alpha 1 chain (COL7A1) gene. The original approval required that the therapy was administered by a health care professional, either in a health care setting or in the patient’s home. The label update also affords patients greater flexibility in managing wound dressings.

Breyanzi (liso-cel)
Bristol Myers Squibb

Update: December 2025
Expanded indication: Marginal zone lymphoma (MZL)

Breyanzi was approved for use in for adults with relapsed or refractory MZL, making it the only CAR-T cell therapy approved by the agency for five cancer types. First approved in the U.S. in 2021 for relapsed or refractory large B-cell lymphoma, Breyanzi is a CD19-directed CAR-T cell therapy with a 4-1BB costimulatory domain, which enhances the expansion and persistence of the CAR-T cells.

Omisirge (omidubicel-onlv)
Gamida Cell

Update: December 2025
Expanded indication: Severe aplastic anemia (SAA)

Omisirge, novel stem cell product from umbilical cord blood, was approved for patients six years and older with SAA following reduced intensity conditioning and for whom a compatible donor is not available, making it the first hematopoietic stem cell transplant therapy to treat patients with SAA, a rare, life-threatening hematologic disorder in which the bone marrow fails to produce sufficient blood cells. The therapy was first approved in 2023 for patients 12 years and older with hematologic malignancies who are planned for umbilical cord blood transplantation following myeloablative conditioning to reduce the time to neutrophil recovery and the incidence of infection.