FDA Decisions to Look For in 1H 2026

As 2026 kicks off, CGTLive® is taking a look ahead at some of the most noteworthy cell and gene therapy products that are certain or likely to have major FDA decisions coming in the first half of the new year. Several therapies already have Prescription Drug User Fee Act (PDUFA) dates on the calendar, and others have recently had biologics license applications (BLAs) submitted to the FDA, or are otherwise nearing a decision from the agency.

Click the “read further” links for past coverage of these therapies.

Tabelecleucel

Indication: EBV-positive posttransplant lymphoproliferative disease (EBV+ PTLD)

PDUFA: January 10, 2026.

FDA Accepts Atara Biotherapeutics’ Resubmission of BLA for Tabelecleucel

July 31, 2025 — The FDA has accepted Atara Biotherapeutics' resubmission of a BLA for allogeneic Epstein-Barr virus (EBV)–specific T-cell immunotherapy tabelecleucel (tab-cel; Ebvallo), which is intended to treat patients 2 years or older with EBV+ PTLD who have received at least 1 prior therapy.

Notably, the BLA resubmission was accepted with priority review and the PDUFA target action date has been set for January 10, 2026. Atara pointed out that as of now there are no therapies approved by the FDA for the aforementioned treatment setting.

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Clemidsogene lanparvovec

Indication: Mucopolysaccharidosis type 2 (MPSII, also known as Hunter syndrome)

PDUFA: February 8, 2026.

Patients Treated With REGENXBIO's MPS II Gene Therapy RGX-121 Sustain 82% Median Reduction in CSF HS D2S6 Levels Through 1 Year

September 10, 2025 — According to a new data update from REGENXBIO's pivotal phase 1/2/3 CAMPSIITE clinical trial (NCT03566043) evaluating clemidsogene lanparvovec (RGX-121), an investigational adeno-associated virus (AAV) vector-based gene therapy for mucopolysaccharidosis type 2 (MPSII, also known as Hunter syndrome), 13 patients treated in the pivotal phase have shown an 82% reduction in heparan sulfate (HS) D2S6 levels in the cerebrospinal fluid (CSF) through 1 year posttreatment. The company originally reported the updated data at the International Congress of Inborn Errors of Metabolism (ICIEM), held in Kyoto, Japan, from September 2 to 6, 2025, and subsequently summarized key points in a press release.

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Deramiocel

Indication: Duchenne muscular dystrophy (DMD) cardiomyopathy

PDUFA: TBD.

In Response to FDA’s Release of Full CRL for Deramiocel, Capricor Publishes Full Response to the Agency

September 9, 2025 — Following the FDA’s public release of the full complete response letter (CRL) that it issued to Capricor Therapeutics for the company’s BLA for deramiocel, an investigational allogeneic cardiosphere-derived cell therapy intended for the treatment of DMD cardiomyopathy, Capricor has responded by publishing its full response to the agency.

Capricor stated that it was not notified in advance by the FDA that the latter would be publishing the CRL publicly on its website. Furthermore, the FDA did not publish Capricor’s response to the CRL, which, according to the company, provides clarifications on the feedback received and insight on its proposed plans to address remaining issues. As such, Capricor published its response to the CRL on its own website.

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DTX401

Indication: Glycogen storage disease type Ia (GSDIa).

PDUFA: TBD.

FDA Accepts Atara Biotherapeutics’ Resubmission of BLA for Tabelecleucel

January 5, 2026 — Ultragenyx has completed its submission of a rolling BLA to the FDA for DTX401, an investigational AAV serotype 8 (AAV8) vector gene therapy expressing the human G6PC gene that is intended to treat Glycogen Storage Disease Type Ia (GSDIa).

The BLA is supported by 96-week data from the randomized, placebo-controlled phase 3 GlucoGene clinical trial (NCT05139316). Ultragenyx noted that the nonclinical and clinical modules of the BLA were submitted to the FDA in August 2025; it has now also completed submission of the chemistry, manufacturing, and controls (CMC) module of the BLA, thus finishing the BLA package.

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Marnetegragene autotemcel

Indication: leukocyte adhesion deficiency type 1 (LAD-I)

PDUFA: March 28, 2026.

BLA for Rocket’s LAD-I Gene Therapy Under Review by FDA Again

October 14, 2025 — The FDA has accepted Rocket Pharmaceuticals’ resubmission of a BLA for marnetegragene autotemcel (RP-L201, to be marketed as Kresladi), a genetically modified autologous hematopoietic stem cell therapy intended to treat LAD-I.

The FDA has set the PDUFA action date for the BLA at March 28, 2026. Data from a global phase 1/2 clinical trial (NCT03812263) that showed a 100% overall survival rate in all enrolled patients for at least 1 year post treatment, and through the entire duration of follow-up, support the BLA. According to Rocket, this trial met all of its primary and secondary end points, with the incidence of significant infections having decreased substantially in comparison with levels before treatment and observed indications of skin lesion improvement and restored wound-healing capability. With regard to safety, Rocket noted that Kresladi was “well tolerated” and that there were no serious adverse events deemed related to treatment reported in the now completed study.

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Orca-T

Indication: various hematologic malignancies

PDUFA: April 6, 2026.

Orca Bio’s BLA for Hematologic Malignancy Cell Therapy Orca-T Accepted by FDA With Priority Review

October 7, 2025 — Orca Bio’s BLA for Orca-T, an investigational allogeneic cell therapy product intended to treat various hematologic malignancies, including acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), and myelodysplastic syndromes (MDS), has been accepted by the FDA with priority review.

The PDUFA target action date for the BLA has been set for April 6, 2026. The BLA is supported by data from an open-label, randomized phase 3 clinical trial (Precision-T; NCT05316701). The pivotal, multicenter Precision-T study is evaluating Orca-T against conventional allogeneic hematopoietic stem cell transplant (allo-HSCT) for the treatment of AML, ALL, and MDS. Notably, patients treated with Orca-T showed a statistically significant improvement in survival free of moderate to severe chronic graft-vs-host disease (GVHD) compared with patients treated with allo-HSCT, and as such, Precision-T met its primary end point.

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