Benitec Biopharma (BNTC) FDA Approvals

Benitec Biopharma's Drug in the FDA Approval Process

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.

Results - April 28,2026

Phase 1b/2a

• Drug: BB-301
• Announced Date: April 28, 2026
• Indication: Oculopharyngeal Muscular Dystrophy (OPMD)

Benitec Biopharma Inc. announced the acceptance of the interim clinical results from the ongoing BB-301 Phase 1b/2a first-in-human study for Oculopharyngeal Muscular Dystrophy (OPMD) with moderate dysphagia for oral presentation at the 2026 American Society of Gene and Cell Therapy Annual Meeting (ASGCT).

Benitec Biopharma announced that interim clinical results from its first‑in‑human BB‑301 Phase 1b/2a study for oculopharyngeal muscular dystrophy (OPMD) with moderate dysphagia were accepted for oral presentation at the 2026 American Society of Gene and Cell Therapy (ASGCT) Annual Meeting. The company will present early safety and efficacy findings from the ongoing trial to the gene therapy research community.

The interim dataset includes 12‑month post‑treatment follow‑up results for the first four Cohort 1 completers, 24‑month post‑treatment follow‑up for the initial Cohort 1 patient, and interim clinical outcomes for the first Cohort 2 patient. These timepoints give an early view of durability and safety as the trial progresses through dose cohorts.

BB‑301 is currently the only clinical‑stage therapeutic specifically in development to treat dysphagia in patients diagnosed with OPMD, making these interim results particularly important for patients and researchers focused on this rare swallowing disorder.

Results - March 9,2026

Phase 1b/2a

• Drug: BB-301
• Announced Date: March 9, 2026
• Indication: Oculopharyngeal Muscular Dystrophy (OPMD)

Benitec Biopharma Inc. announced promising interim clinical results from the BB-301 Phase 1b/2a first-in-human study (NCT06185673) evaluating low dose and high dose BB-301 treatment for Oculopharyngeal Muscular Dystrophy (OPMD) with moderate dysphagia.

Benitec Biopharma announced promising interim results from the BB-301 Phase 1b/2a first-in-human study (NCT06185673) testing low and high doses of BB-301 for Oculopharyngeal Muscular Dystrophy (OPMD) patients with moderate dysphagia. Patients treated with both low and high doses showed significant improvements in throat closure, throat emptying, and overall dysphagia symptom burden, indicating meaningful clinical benefit on swallowing function.

Low-dose BB-301 produced highly durable effects, with clinical and radiographic improvements continuing to deepen up to two years after treatment. The first high-dose patient (Patient B) showed an unusually strong early dose-response, suggesting BB-301 may have disease‑modifying potential for OPMD-related dysphagia. The open-label, intramuscular dose‑escalation study will present these interim findings at the 2026 MDA Clinical & Scientific Conference. BB-301 is currently the only clinical‑stage therapy specifically developed to treat dysphagia in OPMD.

Late-breaking abstract - February 23,2026

Phase 1b/2a

• Drug: BB-301
• Announced Date: February 23, 2026
• Indication: Oculopharyngeal Muscular Dystrophy (OPMD)

Benitec Biopharma Inc. announced the acceptance of a late-breaking abstract for the BB-301 Phase 1b/2a Clinical Treatment Study ongoing in Oculopharyngeal Muscular Dystrophy Patients (OPMD) with moderate dysphagia. Interim clinical study results for Patients enrolled into Cohort 1 and Cohort 2 will be discussed in a poster presentation at the Muscular Dystrophy Association Clinical and Scientific Conference, being held in Orlando, Florida on March 9, 2026.

Benitec Biopharma announced that a late‑breaking abstract from its BB‑301 Phase 1b/2a treatment study in patients with oculopharyngeal muscular dystrophy (OPMD) and moderate dysphagia has been accepted for a poster presentation. The presentation will take place at the Muscular Dystrophy Association Clinical & Scientific Conference in Orlando, Florida on March 9, 2026. The study is ongoing, and the poster will share interim clinical findings from early cohorts.

The poster will report 12‑month follow‑up results for the first four Cohort 1 completers, 24‑month clinical results for the first Cohort 1 patient, and interim results for the first Cohort 2 patient. These data offer early information on safety and clinical effect over one to two years in the first treated participants and will be discussed at the conference to inform the research community about the BB‑301 program’s progress.

Clinical Study - January 11,2026

Phase 1b/2a

• Drug: BB-301
• Announced Date: January 11, 2026
• Indication: Oculopharyngeal Muscular Dystrophy (OPMD)

Benitec Biopharma Inc. announced that the first patient treated in Cohort 1 of the BB-301 Phase 1b/2a clinical study (NCT06185673) evaluating BB-301 for the treatment of dysphagia in oculopharyngeal muscular dystrophy (OPMD) has completed the 24-month post-treatment assessment.

Benitec reported that the first patient treated in Cohort 1 of the BB-301 Phase 1b/2a study for dysphagia in oculopharyngeal muscular dystrophy (OPMD) has completed the 24‑month post‑treatment visit and continued to show strong, disease‑modifying benefit. Videofluoroscopic swallowing studies showed durable improvement in pharyngeal constrictor function (PhAMPC) with a 27% gain maintained from month 12 to month 24. Post‑swallow residue measures deepened: NRRSv improved from 35% at month 12 to 60% at month 24, and total pharyngeal residue improved from 32% to 39%. Patient‑reported swallowing symptoms (Sydney Swallow Questionnaire) improved 64% at month 12 and 78% at month 24.

Benitec also said Patient 4 remained a strong responder at month 12, and the first four Cohort 1 patients completed the 12‑month statistical follow‑up. All four met prespecified criteria and were classified as formal Responders, showing durable clinical benefit after BB‑301 treatment.

Interim Results - November 3,2025

Phase 1b/2a

• Drug: BB-301
• Announced Date: November 3, 2025
• Indication: Oculopharyngeal Muscular Dystrophy (OPMD)

Benitec Biopharma Inc. , today provides positive interim clinical results for the BB-301 Phase 1b/2a Clinical Trial.

Benitec Biopharma Inc. today announced positive interim results from its BB-301 Phase 1b/2a clinical trial in patients with oculopharyngeal muscular dystrophy (OPMD) and dysphagia. All six participants in Cohort 1 met the formal response criteria, achieving a 100% response rate after receiving a single treatment of BB-301.

After administration, patients experienced significant, lasting improvements in dysphagic symptom burden, reduced post-swallow residue, faster consumption of fixed liquid volumes, and better pharyngeal closure during swallowing. Follow-up periods ranged from three to twelve months, demonstrating durable benefits across multiple clinical and functional swallowing assessments.

Based on these encouraging data, the U.S. Food and Drug Administration granted Fast Track designation for BB-301, and the therapy also holds Orphan Drug Designation from both the FDA and the European Medicines Agency. Benitec plans to meet with the FDA in 2026 to confirm the design of the pivotal BB-301 study.

Provided Update - November 2,2025

Phase 1b/2a

• Drug: BB-301
• Announced Date: November 2, 2025
• Indication: Oculopharyngeal Muscular Dystrophy (OPMD)

Benitec Biopharma Inc. announced that it will provide an update on the six treated patients from Cohort 1 of its Phase 1b/2a Clinical Study of BB-301 being investigated for the treatment of Oculopharyngeal Muscular Dystrophy (OPMD).

Benitec Biopharma Inc., a clinical-stage gene therapy company, announced it will provide an update on six patients from Cohort 1 of its Phase 1b/2a study of BB-301 for Oculopharyngeal Muscular Dystrophy (OPMD). This trial uses Benitec’s proprietary “Silence and Replace” DNA-directed RNA interference (ddRNAi) platform to both silence disease-causing genes and deliver healthy replacement genes in a single treatment.

In Cohort 1, all six patients have received BB-301 and are being monitored for safety, tolerability, and early signs of efficacy. The data will help assess whether BB-301 can improve muscle strength and function in individuals with OPMD, a genetic disorder that weakens muscles involved in swallowing and eyelid movement.

The company will host a webcast on November 3, 2025, at 8:00 am EST to share these results. Interested parties can register online to join the live presentation and a Q&A session with Benitec’s leadership team.

Enrollment Update - July 9,2025

Phase 1b/2a

• Drug: BB-301
• Announced Date: July 9, 2025
• Indication: Oculopharyngeal Muscular Dystrophy (OPMD)

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.

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.

Provided Update - March 19,2025

• Drug: BB-301
• Announced Date: March 19, 2025
• Indication: Oculopharyngeal Muscular Dystrophy (OPMD)

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).

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.

Abstract - February 14,2025

Phase 1b/2a

• Drug: BB-301
• Announced Date: February 14, 2025
• Indication: Oculopharyngeal Muscular Dystrophy (OPMD)

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.

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.

Provided Update - October 12,2024

Phase 1b/2a

• Drug: BB-301
• Announced Date: October 12, 2024
• Indication: Oculopharyngeal Muscular Dystrophy (OPMD)

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).

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.

Late-Breaking Data - September 18,2024

• Drug: BB-301
• Announced Date: September 18, 2024
• Indication: Oculopharyngeal Muscular Dystrophy (OPMD)

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.

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.

Provided Update - July 15,2024

Phase 1b/2a

• Drug: BB-301
• Announced Date: July 15, 2024
• Indication: Oculopharyngeal Muscular Dystrophy (OPMD)

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).

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.