FDA Events for Benitec Biopharma (BNTC)
This section highlights FDA-related milestones and regulatory updates for drugs developed by Benitec Biopharma (BNTC).
Over the past two years, Benitec Biopharma has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
BB-301. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
BB-301 - FDA Regulatory Timeline and Events
BB-301 is a drug developed by Benitec Biopharma for the following indication: Oculopharyngeal Muscular Dystrophy (OPMD).
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:
- BB-301
- Announced Date:
- July 9, 2025
- Indication:
- Oculopharyngeal Muscular Dystrophy (OPMD)
Announcement
Benitec Biopharma Inc. announced the recommendation of the independent Data Safety Monitoring Board (DSMB) to continue enrollment of the Phase 1b/2a Clinical Treatment Study (NCT06185673) following completion of the comprehensive review of safety information for all six Subjects enrolled into Cohort 1.
AI Summary
Benitec Biopharma Inc. announced that the independent Data Safety Monitoring Board (DSMB) has recommended continuing enrollment in its Phase 1b/2a Clinical Treatment Study (NCT06185673). This decision came after a thorough review of safety data for all six subjects enrolled in Cohort 1. The DSMB meeting was held once the final subject completed the 28-day post-dosing visit, and the positive recommendation reflects a favorable safety profile for the low dose of BB-301. Following this update, the company expects the enrollment for Cohort 2 to begin in the fourth quarter of 2025. Benitec’s continued advancement in this gene therapy trial marks an important milestone, reinforcing the company’s efforts to develop effective treatments with a robust safety foundation for the OPMD patient community.
Read Announcement- Drug:
- BB-301
- Announced Date:
- March 19, 2025
- Indication:
- Oculopharyngeal Muscular Dystrophy (OPMD)
Announcement
Benitec Biopharma Inc. announces continued durable improvements in swallowing function and reductions in total dysphagic symptom burden following administration of the low-dose of BB-301 in the first three Subjects treated in the BB-301 Phase 1b/2a single-arm, open-label, sequential, dose-escalation cohort study (NCT06185673) in Oculopharyngeal Muscular Dystrophy (OPMD).
AI Summary
Benitec Biopharma Inc. announced positive interim results from its Phase 1b/2a study evaluating low-dose BB-301 in patients with Oculopharyngeal Muscular Dystrophy (OPMD). In the study, the first three subjects treated with BB-301 demonstrated durable improvements in swallowing function and significant reductions in dysphagic symptom burdens. Notably, Subject 1 showed reduced post-swallow residue at 12 months, while Subject 2, who suffered from ineffective swallowing, achieved nearly complete resolution of problematic sequential swallows and reached a clinically normal swallowing profile at 12 months. Similarly, Subject 3 achieved a normal profile just 3 months after treatment. These outcomes, assessed via videofluoroscopic swallowing studies and patient-reported measures, highlight the promising potential of BB-301 as a gene therapy intervention for OPMD, with detailed findings to be presented at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference.
Read Announcement- Drug:
- BB-301
- Announced Date:
- February 14, 2025
- Indication:
- Oculopharyngeal Muscular Dystrophy (OPMD)
Announcement
Benitec Biopharma Inc. announced the acceptance of a late-breaking oral abstract for the BB-301 Phase 1b/2a Clinical Treatment Study ongoing in Subjects diagnosed with Oculopharyngeal Muscular Dystrophy (OPMD) with moderate dysphagia. Interim clinical study updates for the first three Subjects will be discussed in an oral presentation at the Muscular Dystrophy Association Clinical and Scientific Conference on March 19, 2025 at 1:15 pm Central Time.
AI Summary
Benitec Biopharma Inc. recently announced that a late-breaking oral abstract for its BB-301 Phase 1b/2a Clinical Treatment Study has been accepted. The study focuses on patients diagnosed with Oculopharyngeal Muscular Dystrophy (OPMD) who are experiencing moderate dysphagia, a challenging complication that affects swallowing.
The oral abstract will discuss interim clinical updates based on data from the first three subjects treated with BB-301. Researchers will present these findings at the Muscular Dystrophy Association Clinical and Scientific Conference on March 19, 2025, at 1:15 pm Central Time. This presentation highlights the potential of the “Silence and Replace” gene therapy platform to provide durable and meaningful improvements in swallowing function, offering new hope for those affected by OPMD.
Read Announcement- Drug:
- BB-301
- Announced Date:
- October 12, 2024
- Indication:
- Oculopharyngeal Muscular Dystrophy (OPMD)
Announcement
Benitec Biopharma Inc. announces continued durable improvements in swallowing following administration of the low-dose of BB-301 in the study's first two subjects treated in the BB-301 Phase 1b/2a single-arm, open-label, sequential, dose-escalation cohort study (NCT06185673) in Oculopharyngeal Muscular Dystrophy (OPMD).
AI Summary
Benitec Biopharma Inc. announced promising interim results from its BB-301 Phase 1b/2a study in patients with Oculopharyngeal Muscular Dystrophy (OPMD). The low-dose BB-301 treatment produced lasting improvements in swallowing for the first two subjects. Subject 1 showed significant improvement at 9 months post-dose, while Subject 2 achieved similar clinical benefits at 6 months. In fact, Subject 2’s Sydney Swallow Questionnaire score reached a level representative of clinically normal swallowing. These improvements were measured by both subjective assessments and videofluoroscopic studies that showed reduced pharyngeal residue and fewer abnormal swallowing patterns. The encouraging results support the potential of BB-301 as an innovative gene therapy approach to treat dysphagia in OPMD. Further details and upcoming study data will be discussed during a conference call on October 14 at 8:30 am EDT.
Read Announcement- Drug:
- BB-301
- Announced Date:
- September 18, 2024
- Indication:
- Oculopharyngeal Muscular Dystrophy (OPMD)
Announcement
Benitec Biopharma Inc. announced that a Late Breaking Abstract on BB-301 was accepted for oral presentation at the 29th Annual Congress of the World Muscle Society, taking place in Prague, Czech Republic from October 8-12, 2024.
AI Summary
Benitec Biopharma Inc. announced that its Late Breaking Abstract on BB-301 has been accepted for oral presentation at the 29th Annual Congress of the World Muscle Society in Prague, Czech Republic. The event is scheduled from October 8-12, 2024, and the abstract will be presented on October 12, 2024.
The presentation, entitled "Interim Clinical Data Summary: A Phase 1b/2a Open-label, Dose Escalation Study to Evaluate the Safety and Clinical Activity of Intramuscular Doses of an AAV9-based gene therapy (BB-301) Administered to Subjects with Oculopharyngeal Muscular Dystrophy (OPMD) with Dysphagia," will be led by Professor Milan R. Amin from New York University. This study focuses on BB-301’s potential as a gene therapy for OPMD using a unique "Silence and Replace" approach. More detailed findings will be released at a later date.
Read Announcement- Drug:
- BB-301
- Announced Date:
- July 15, 2024
- Indication:
- Oculopharyngeal Muscular Dystrophy (OPMD)
Announcement
Benitec Biopharma Inc. announces continued durable improvements in the radiographic assessments of swallowing efficiency and the subject-reported outcome instrument as assessed at the 180-day timepoint following the administration of the low-dose of BB-301 to the study's first subject (Subject 1) treated in the BB-301 Phase 1b/2a single-arm, open-label, sequential, dose-escalation cohort study (NCT06185673) in Oculopharyngeal Muscular Dystrophy (OPMD).
AI Summary
Benitec Biopharma Inc. has reported promising interim clinical results for its investigational gene therapy BB-301 in a Phase 1b/2a study for Oculopharyngeal Muscular Dystrophy (OPMD). At the 180-day assessment, Subject 1 who received a low dose of BB-301 showed sustained improvements in swallowing function. Radiographic evaluations demonstrated that the post-dose average Total Pharyngeal Residue—measuring the amount of food or liquid remaining in the throat after the first swallow—was significantly lower than pre-dose levels. For three of four food types, these values were the lowest seen in a nine-month pre-dose period, while the fourth was similar to the lowest observed pre-dose. Additionally, subject-reported outcomes via the Sydney Swallow Questionnaire continued to show meaningful reductions in dysphagic symptoms, indicating enhanced swallowing ability over time.
Read Announcement- Drug:
- BB-301
- Announced Date:
- April 18, 2024
- Indication:
- Oculopharyngeal Muscular Dystrophy (OPMD)
Announcement
Benitec Biopharma Inc. announces positive interim clinical data from the 90-day timepoint following the administration of BB-301 to the study's first subject (Subject 1) treated in the BB-301 Phase 1b/2a single-arm, open-label, sequential, dose-escalation cohort study (NCT06185673) in Oculopharyngeal Muscular Dystrophy (OPMD).
AI Summary
Benitec Biopharma Inc. announced positive interim clinical data at the 90-day mark from its Phase 1b/2a study testing the experimental gene therapy BB-301 in a patient with Oculopharyngeal Muscular Dystrophy (OPMD). Subject 1, the first patient treated, showed significant improvements in swallowing function compared to baseline measurements taken during the natural history observational period. The study used videofluoroscopic swallowing studies (VFSS) and the Sydney Swallow Questionnaire to measure these outcomes. Notably, the patient demonstrated improved pharyngeal muscle function and better swallowing efficiency with various food textures, including thin liquids, solid foods, and thicker consistencies.
These early clinical results are encouraging, as they suggest that BB-301 may offer a new approach to addressing the dysphagia and muscle weakness associated with OPMD. Further patient treatment and additional data are anticipated as the study progresses.
Read Announcement- Drug:
- BB-301
- Announced Date:
- April 9, 2024
- Estimated Event Date Range:
- April 18, 2024 - April 18, 2024
- Target Action Date:
- April 18, 2024
- Indication:
- Oculopharyngeal Muscular Dystrophy (OPMD)
Announcement
Benitec Biopharma Inc. announced it will host a virtual R&D Day, on Thursday, April 18, 2024 from 9:00 AM to 11:00 AM ET. To register, click here.
AI Summary
Benitec Biopharma, a clinical-stage biotechnology company based in Hayward, California, announced that it will host a virtual R&D Day on Thursday, April 18, 2024 from 9:00 AM to 11:00 AM ET. This online event is part of the company’s ongoing efforts in advancing its gene therapy research using the “Silence and Replace” DNA-directed RNA interference platform.
The R&D Day will feature presentations from two key opinion leaders discussing aspects of oculopharyngeal muscular dystrophy (OPMD), including its clinical symptoms, disease progression, and current treatment approaches. The event will also highlight the company’s clinical development program for its investigational gene therapy agent BB-301, currently in a Phase 1b/2a trial for treating OPMD-derived dysphagia. Those interested in learning more and engaging in the live Q&A session are encouraged to register using the provided link.
Read Announcement
Benitec Biopharma FDA Events - Frequently Asked Questions
As of now, Benitec Biopharma (BNTC) has not received any FDA approvals for its therapy in the last two years.
In the past two years, Benitec Biopharma (BNTC) has reported FDA regulatory activity for BB-301.
The most recent FDA-related event for Benitec Biopharma occurred on July 9, 2025, involving BB-301. The update was categorized as "Enrollment Update," with the company reporting: "Benitec Biopharma Inc. announced the recommendation of the independent Data Safety Monitoring Board (DSMB) to continue enrollment of the Phase 1b/2a Clinical Treatment Study (NCT06185673) following completion of the comprehensive review of safety information for all six Subjects enrolled into Cohort 1."
Currently, Benitec Biopharma has one therapy (BB-301) targeting the following condition: Oculopharyngeal Muscular Dystrophy (OPMD).
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:BNTC) was last updated on 7/14/2025 by MarketBeat.com Staff
