Beqvez (fidanacogene elaparvovec-dzkt) / Roche, Pfizer - LARVOL DELTA

First Data from the American Thrombosis and Hemostasis Network Transcends Registry Hemophilia Gene Therapy Outcomes Arm (ISTH 2025) - P | "Background Adeno-associated viral vector (AAV)-mediated gene therapy for hemophilia B (HB) and hemophilia A (HA) is a reality for patients since conditional approval for marketing of etranacogene dezaparvovec-drlb and fidanacogene elaparvovec-dzkt for HB, and valoctocogene roxaparvovec-rvox for HA. Three participants required steroid treatment for vector-induced transaminitis. Table or Figure Upload"

Gene therapy • Cardiovascular • Dermatology • Fatigue • Gene Therapies • Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Pain • Rare Diseases • Thrombosis • Urticaria

Multi-Year Follow-Up of Fidanacogene Elaparvovec for Moderately Severe/Severe Hemophilia B (ISTH 2025) - P3 | "No treatment-related SAEs were reported beyond Yr 1. Table or Figure Upload"

Gene Therapies • Hemophilia • Hemophilia B • Oncology • Rare Diseases

A phase 2 dose-escalation substudy of fidanacogene elaparvovec in adults with hemophilia B (ISTH 2025) - "The most frequently reported TRAE (Table 1) was increased alanine aminotransferase (ALT); all increases were managed with increasing doses of CS administered orally (1 pt in cohort 2 also received mycophenolate mofetil post CS) and all AEs were resolved by the cutoff date. One pt (cohort 2; history of cranial hemorrhage) resumed preventative prophylaxis on Day 351 based on their FIX levels. Table or Figure Upload"

Clinical • P2 data • Gene Therapies • Hemophilia • Hemophilia B • Rare Diseases

Six-year outcomes following fidanacogene elaparvovec in adults with hemophilia B (ISTH 2025) - P2 | "No pts resumed FIX prophylaxis. Table or Figure Upload"

Clinical • Gene Therapies • Hemophilia • Hemophilia B • Oncology • Rare Diseases

Gene-based therapies for hemophilia. (PubMed, Res Pract Thromb Haemost) - "Clinical outcomes have shown promise through gene therapies like valoctocogene roxaparvovec for hemophilia A and etranacogene dezaparvovec and fidanacogene elaparvovec for hemophilia B. Each therapy has demonstrated efficacy in reducing bleeding rates and maintaining factor activity. Current trials are exploring new vectors, transgenes, and methods to overcome existing limitations. Gene therapy holds the potential to revolutionize hemophilia treatment, offering a path toward long-term management and improved quality of life for patients."

Journal • Gene Therapies • Genetic Disorders • Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Rare Diseases

Deconstructing FDA's Expedited Program Determinations: Analyzing how the Agency may Set the Bar for RMAT Designation (ASGCT 2025) - "Parexel explored the relationship between the early-phase data from press releases around the time of RMAT designation and pivotal data from FDA review memos for marketing applications for two cases of RMAT-designated gene therapies, VYJUVEK (beremagene geperpavec) and BEQVEZ (fidanacogene elaparvovec-dzkt), that were approved in 2023 and 2024. Conducting analyses, such as the one listed above, that contextualize the RMAT-designation supporting data within the totality of publicly disclosed information for that development program, can help inform developers in making decisions about the amount of evidence and timing for submission of RMAT designation requests. Disease Focus of Abstract:Other Other: Rare Disease"

Gene Therapies • Hematological Disorders • Hemophilia • Hemophilia B • Rare Diseases

Fidanacogene Elaparvovec for Hemophilia B - A Multiyear Follow-up Study. (PubMed, N Engl J Med) - P2 | "Fidanacogene elaparvovec was associated with no or only low-grade adverse effects over a period of 3 to 6 years. Efficacy was maintained in the long term at 5×1011 vg per kilogram, one of the lowest intravenous doses of AAV used for any indication. (Funded by Pfizer; ClinicalTrials.gov number, NCT03307980.)."

Journal • Fibrosis • Hematological Disorders • Hemophilia • Hemophilia B • Hepatitis B • Hepatitis C • Hepatology • Human Immunodeficiency Virus • Immunology • Infectious Disease • Liver Cirrhosis • Oncology • Rare Diseases

Safety and Effectiveness of Giroctocogene Fitelparvovec or Fidanacogene Elaparvovec in Patients With Hemophilia A or B Respectively (clinicaltrials.gov) - P3 | N=263 | Recruiting | Sponsor: Pfizer | Trial completion date: Sep 2039 ➔ Feb 2040 | Trial primary completion date: Sep 2039 ➔ Feb 2040

Trial completion date • Trial primary completion date • Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Rare Diseases

Pfizer discontinues hemophilia treatment Beqvez, emptying its gene therapy portfolio (Fierce Pharma) - "The New York pharma is ending global development and commercialization of Beqvez less than a year after an FDA approval for the gene therapy to treat hemophilia B...Several reasons led to the discontinuation, including limited interest from patients and doctors toward hemophilia gene therapies to date, a Pfizer spokesperson told Fierce Pharma in a statement....No patients seem to have received commercial Beqvez since its FDA nod in April 2024. The Pfizer spokesperson said the company will communicate the news to patients and providers that are in the treatment qualification process, adding that the company remains committed to supporting those who received the med in any clinical trial."

Discontinued • Hemophilia B

Gene-ius at work: Hemophilia B treatment enters a new era. (PubMed, Am J Health Syst Pharm) - "For the first time in history, individuals with hemophilia B have access to potentially curative therapies through gene therapy. Both etranacogene dezaparvovec and fidanacogene elaparvovec offer significant efficacy, reducing the number of bleeding episodes and raising FIX concentrations with a single lifetime administration. While concerns remain regarding long-term safety and durability, these therapies represent a major advancement in reducing treatment burden and improving quality of life for patients. The future of hemophilia B management now holds the promise of greater independence from frequent prophylactic treatments."

Journal • Gene Therapies • Hematological Disorders • Hemophilia • Hemophilia B • Rare Diseases

Access to Fidanacogene Elaparvovec: Hemophilia B Gene Therapy Directed by a First-in-Class Companion Diagnostic (EAHAD 2025) - No abstract available

Companion diagnostic • Gene therapy • Gene Therapies • Hematological Disorders • Hemophilia • Hemophilia B • Rare Diseases

Impact of Age, Race, and Geographic Region on Efficacy Outcomes Following Gene Therapy for Hemophilia B With Fidanacogene Elaparvovec: Results From the Phase 3 BENEGENE-2 Trial (EAHAD 2025) - No abstract available

Clinical • Gene therapy • P3 data • Gene Therapies • Hematological Disorders • Hemophilia • Hemophilia B • Rare Diseases

Efficacy and Safety of Fidanacogene Elaparvovec by Hepatitis History: Results From the BENEGENE-2 Trial (EAHAD 2025) - No abstract available

Clinical • Hepatology • Inflammation

Efficacy and Safety of Fidanacogene Elaparvovec by BMI: Results From the BENEGENE-2 Trial (EAHAD 2025) - No abstract available

Clinical

In Vitro and In Vivo Potency Differences between AAVRh74var and AAV5 Vectors Encoding the Same High-Activity Human Factor IX Variant, FIX-R338L, Expression Cassette: Implications for Hemophilia B Gene Therapy (ASH 2024) - "The only other approved HB gene therapy (etranacogene dezaparvovec) utilizes the AAV5 capsid and is administered at a 40-fold higher dose (2×1013 gc/kg). Overall, the results demonstrate differences between the AAVRh74var and AAV5 capsids. This is consistent with fidanacogene elaparvovec (AAVRh74var) achieving clinically robust efficacy, despite a several-fold lower dose than AAV5-based gene therapies."

Gene therapy • Preclinical • Gene Therapies • Hematological Disorders • Hemophilia • Hemophilia B • Hepatocellular Cancer • Oncology • Rare Diseases • Solid Tumor

Fidanacogene Elaparvovec-dzkt for Treatment of Adults with Moderate to Severe Hemophilia B (ASH 2024) - No abstract available

Clinical • Hematological Disorders • Hemophilia • Hemophilia B • Rare Diseases

Use of Fidanacogene Elaparvovec, a Gene Therapy Vector, to Deliver a Stable, Fully Functional Human Factor IX Transgene for the Treatment of Hemophilia B: A Combined Analysis of Safety (ASH 2024) - P2, P3 | "Conclusions This combined analysis demonstrates the favorable safety profile of fidanacogene elaparvovec in the largest dataset with the longest follow-up for an HB gene therapy. LTFU will continue for up to 15 years."

Clinical • Gene therapy • Anemia • Fibrosis • Gene Therapies • Genetic Disorders • Hematological Disorders • Hemophilia • Hemophilia B • Hepatocellular Cancer • Hepatology • Immunology • Infectious Disease • Nephrology • Oncology • Polycystic Kidney Disease • Rare Diseases • Renal Disease

A Review of Etranacogene Dezaparvovec and Fidanacogene Elaparvovec for the Treatment of Hemophilia B (ASHP 2024) - No abstract available

Review • Hematological Disorders • Hemophilia • Hemophilia B • Rare Diseases

Gene Therapy for Hemophilia. (PubMed, Blood Adv) - "Evolution Long-term clinical studies have provided insight into the efficacy, safety, and durability of AAV-mediated gene therapies for hemophilia A and B. Gene therapies have now been approved for hemophilia A (valoctocogene roxaparvovec) and hemophilia B (etranacogene dezaparvovec; fidanacogene elaparvovec) in select regions. Application The approved gene therapies are associated with a strong reduction in bleeding tendency and increased FVIII or FIX activity levels, without the need for additional FVIII or FIX replacement therapies, in the vast majority of patients. Future Steps Improvements in AAV technology, protein expression, and immunogenicity may render AAV-mediated gene therapies more efficient, longer lasting and safer for people with hemophilia A or B."

Gene therapy • Journal • Gene Therapies • Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Rare Diseases

Gene Therapy with Fidanacogene Elaparvovec in Adults with Hemophilia B. (PubMed, N Engl J Med) - P3 | "Fidanacogene elaparvovec was superior to prophylaxis for the treatment of participants with hemophilia B, leading to reduced bleeding and stable factor IX expression. (Funded by Pfizer; BENEGENE-2 ClinicalTrials.gov number, NCT03861273.)."

Gene therapy • Journal • Gene Therapies • Hematological Disorders • Hemophilia • Hemophilia B • Rare Diseases

Safety and Effectiveness of Giroctocogene Fitelparvovec or Fidanacogene Elaparvovec in Patients With Hemophilia A or B Respectively (clinicaltrials.gov) - P3 | N=263 | Recruiting | Sponsor: Pfizer | Trial completion date: Apr 2038 ➔ Sep 2039 | Trial primary completion date: Apr 2038 ➔ Sep 2039

Trial completion date • Trial primary completion date • Hematological Disorders • Hemophilia • Rare Diseases

Beqvez - another gene therapy for hemophilia B. (PubMed, Med Lett Drugs Ther) - No abstract available

Gene therapy • Journal • Gene Therapies • Hematological Disorders • Hemophilia • Rare Diseases

Efficacy and safety of fidanacogene elaparvovec in adults with moderately severe or severe hemophilia B: updated results from the phase 3 BENEGENE-2 gene therapy trial (ISTH 2024) - P3 | "Forty-five participants (median [range] age, 29 [18–62] years) were dosed with fidanacogene elaparvovec 5e11 vg/kg. As of August 2023, 44 participants completed ≥15 months follow-up (up to 4.0 years); the 45th participant had >12 months follow-up; we previously reported on 41 with ≥15 months of follow-up). Total ABR was reduced by 71% from Week 12 to Month 15 post-gene therapy vs prophylaxis (mean ABR, 1.3 vs 4.4; P< 0.0001; Table 1)."

Clinical • Gene therapy • P3 data • Gene Therapies • Hematological Disorders • Hemophilia • Rare Diseases

Field Study and Correlative Studies of Factor IX Variant FIX-R338L in Participants Treated with Fidanacogene Elaparvovec. (PubMed, Thromb Haemost) - P2 | " These results demonstrate FIX-R338L activity can be measured with OS and CS assays in clinical laboratories and provide insight into assay variability when measuring FIX with endogenously produced FIX-R338L. The findings may help establish best practices for measuring FIX-R338L activity (Clinicaltrials.gov identifier: NCT02484092)."

Journal • Gene Therapies • Hematological Disorders • Hemophilia • Rare Diseases

U.S. FDA Approves Pfizer’s BEQVEZ (fidanacogene elaparvovec-dzkt), a One-Time Gene Therapy for Adults with Hemophilia B (Pfizer Press Release) - "Pfizer Inc...announced today that the U.S. Food and Drug Administration (FDA) has approved BEQVEZ (fidanacogene elaparvovec-dzkt) for the treatment of adults with moderate to severe hemophilia B who currently use factor IX (FIX) prophylaxis therapy, or have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes, and do not have neutralizing antibodies to adeno-associated virus serotype Rh74var (AAVRh74var) capsid as detected by an FDA-approved test. BEQVEZ is a one-time treatment that is designed to enable people living with hemophilia B to produce FIX themselves rather than the current standard of care, which requires regular intravenous infusions of FIX that are often administered multiple times a week or multiple times a month."

BLA • Gene Therapies • Genetic Disorders • Hemophilia