FDA Events for Passage Bio (PASG)
This section highlights FDA-related milestones and regulatory updates for drugs developed by Passage Bio (PASG).
Over the past two years, Passage Bio has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
PBFT02. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
PBFT02 - FDA Regulatory Timeline and Events
PBFT02 is a drug developed by Passage Bio for the following indication: Dementia with granulin mutations (FTD-GRN).
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:
- PBFT02
- Announced Date:
- June 23, 2025
- Indication:
- Dementia with granulin mutations (FTD-GRN)
Announcement
Passage Bio, Inc. today reported updated data from the ongoing Phase 1/2 upliFT-D clinical trial evaluating PBFT02 for the treatment of frontotemporal dementia (FTD) with granulin (GRN) mutations and provided program updates and anticipated milestones.
AI Summary
Passage Bio, Inc. has shared promising updated data from its ongoing Phase 1/2 upliFT-D clinical trial that is evaluating PBFT02 for treating frontotemporal dementia (FTD) with GRN mutations. The results show that PBFT02 consistently raises CSF progranulin (PGRN) levels in a durable manner over 18 months and slows the rise of plasma neurofilament light (NfL) levels compared to natural disease progression. Notably, a lower Dose 2 (50% of Dose 1) significantly increased CSF PGRN, nearing healthy adult levels. The company is adjusting the trial protocol by introducing a short course of low dose prophylactic anticoagulation and modifying inclusion criteria to enroll patients at an earlier stage of disease. Passage Bio is on track to seek regulatory feedback on the design of its pivotal trial for FTD-GRN in the first half of 2026.
Read Announcement- Drug:
- PBFT02
- Announced Date:
- January 10, 2025
- Indication:
- Dementia with granulin mutations (FTD-GRN)
Announcement
Passage Bio, Inc. today reported updated data from the ongoing Phase 1/2 upliFT-D clinical trial evaluating PBFT02 for the treatment of frontotemporal dementia (FTD) with granulin (GRN) mutations and anticipated upcoming milestones.
AI Summary
Passage Bio, Inc. reported updated interim data from its ongoing Phase 1/2 upliFT-D clinical trial evaluating PBFT02 for treating frontotemporal dementia (FTD) with granulin (GRN) mutations. The trial demonstrated that Dose 1 of PBFT02 produced a durable increase in cerebrospinal fluid PGRN levels and early indications of reduced plasma NfL levels, a biomarker associated with disease progression, when compared to published natural history data.
The company is introducing Dose 2, which is 50% lower than Dose 1, to explore optimal dosing in FTD-GRN and FTD-C9orf72 patients and support its regulatory strategy. Passage Bio plans to report 12-month data from Dose 1 along with interim safety and biomarker data from Dose 2 in the second half of 2025, and it aims to seek regulatory feedback on the registrational trial design in the first half of 2026.
Read Announcement- Drug:
- PBFT02
- Announced Date:
- September 16, 2024
- Indication:
- Dementia with granulin mutations (FTD-GRN)
Announcement
Passage Bio, Inc present updated data from the ongoing global Phase 1/2 upliFT-D clinical trial evaluating PBFT02, an adeno-associated virus (AAV)-delivery gene therapy for the treatment of patients with frontotemporal dementia (FTD) with granulin (GRN) mutations, at the 14th International Conference on Frontotemporal Dementias (ISFTD2024).
AI Summary
Passage Bio, Inc. will present updated data at the 14th International Conference on Frontotemporal Dementias (ISFTD2024) from its ongoing global Phase 1/2 upliFT-D clinical trial. The study is evaluating PBFT02, an adeno-associated virus (AAV) gene therapy designed to treat patients with frontotemporal dementia (FTD) caused by granulin (GRN) mutations. In the trial’s first cohort, Dose 1 of PBFT02 resulted in a robust and durable increase in cerebrospinal fluid (CSF) progranulin levels that were maintained for up to 12 months after treatment. Additionally, after patients received a revised immunosuppression regimen, the therapy was well-tolerated with no serious adverse events observed. Juan Chavez, M.D., Vice President of Clinical Development, will present these important interim safety and biomarker findings during an oral session at the conference. This data underscores PBFT02’s potential as a promising one-time treatment for FTD-GRN patients.
Read Announcement- Drug:
- PBFT02
- Announced Date:
- July 16, 2024
- Indication:
- Dementia with granulin mutations (FTD-GRN)
Announcement
Passage Bio, Inc. announced that the company received positive feedback in its Type C meeting process with the U.S. Food and Drug Administration (FDA) on its proposal to evaluate PBFT02 for treating frontotemporal dementia (FTD) patients with mutations in the C9orf72 gene.
AI Summary
Passage Bio received positive feedback from the FDA during its Type C meeting regarding its plans to test PBFT02 for treating frontotemporal dementia (FTD) in patients with C9orf72 gene mutations. The agency supported the company's proposal to expand the existing upliFT-D Phase 1/2 trial—originally focused on FTD patients with progranulin (PGRN) deficiencies—to include a new group of FTD-C9orf72 patients. This support was based on compelling preclinical evidence and early safety and PGRN expression data from the current trial. The expansion could address a significant unmet need for the estimated 21,000 FTD-C9orf72 patients in the United States and Europe.
Passage Bio plans to submit the revised trial protocol to health authorities and ethics committees soon, with an anticipated start of dosing these patients in the first half of 2025.
Read Announcement- Drug:
- PBFT02
- Announced Date:
- July 16, 2024
- Estimated Event Date Range:
- January 1, 2025 - June 30, 2025
- Target Action Date:
- 2025-H1
- Indication:
- Dementia with granulin mutations (FTD-GRN)
Announcement
Passage Bio, Inc. announced that its has Plan to initiate dosing of FTD-C9orf72 patients in 1H 2025
AI Summary
Passage Bio, Inc. has announced plans to expand its current upliFT-D trial to include frontotemporal dementia (FTD) patients with mutations in the C9orf72 gene. The company received positive feedback from the FDA during its Type C meeting process, which supports the proposed clinical development plans for evaluating PBFT02 in this new patient group.
Passage Bio aims to address an unmet need in the FTD-C9orf72 community, where limited treatment options currently exist. With compelling preclinical evidence and promising early safety and progranulin data from the first cohort of FTD-GRN patients, the company is moving forward by amending the trial protocol. The revised protocol is set to be submitted to health authorities and ethics committees, with plans to start dosing FTD-C9orf72 patients in the first half of 2025.
Read Announcement
Passage Bio FDA Events - Frequently Asked Questions
As of now, Passage Bio (PASG) has not received any FDA approvals for its therapy in the last two years.
In the past two years, Passage Bio (PASG) has reported FDA regulatory activity for PBFT02.
The most recent FDA-related event for Passage Bio occurred on June 23, 2025, involving PBFT02. The update was categorized as "Updated data," with the company reporting: "Passage Bio, Inc. today reported updated data from the ongoing Phase 1/2 upliFT-D clinical trial evaluating PBFT02 for the treatment of frontotemporal dementia (FTD) with granulin (GRN) mutations and provided program updates and anticipated milestones."
Currently, Passage Bio has one therapy (PBFT02) targeting the following condition: Dementia with granulin mutations (FTD-GRN).
More FDA Event Resources from MarketBeat
FDA Event Stage Terminology & Abbreviation Guide
- NDA: New Drug Application
- ANDA: Abbreviated New Drug Application
- sNDA: Supplemental New Drug Application
- BLA: Biologics License Application
- sBLA: Supplemental Biologics License Application
- FDA Approved: Approved by the FDA
- EMA: European Medicines Agency
- CE Mark: European Union Certification
- NMPA: China National Medical Products Administration
- MHLW: Japanese Ministry of Health
- FDA Meeting: Consultation with FDA
- Pre-IND: Pre-Investigational New Drug Meeting
- Breakthrough Therapy: Special FDA designation for promising therapies
- Fast Track: Accelerated FDA approval pathway
- Orphan Drug: Designation for rare disease treatments
- RPD: Rare Pediatric Disease Designation
- RMAT: Regenerative Medicine Advanced Therapy
- DSMB Review: Data Safety Monitoring Board Review
- IDMC Review: Independent Data Monitoring Committee
- MAA: MHRA Marketing Authorization Application
- RTF: Refusal to File (Rejected Application)
- 510(k): FDA Clearance for Medical Devices
- Rolling Submission: Staggered regulatory review process
This page (NASDAQ:PASG) was last updated on 7/10/2025 by MarketBeat.com Staff
