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Fitusiran prophylaxis reduced bleeds by 61% in people with Hem A or B

July 21, 2022

Read the original press release here. A median annualized bleeding rate (ABR) of 0.0 was reported in the overall study population during fitusiran prophylaxis (80 mg monthly) Fitusiran is a novel, investigational subcutaneously administered small interference RNA therapy, in development for the prophylactic treatment of people with hemophilia A or B, with or without inhibitor […]

Once-Weekly Efanesoctocog Alfa: an update from Sanofi

July 21, 2022

Read the original press release here Investigational once-weekly efanesoctocog alfa prophylaxis met the primary efficacy endpoint providing clinically meaningful bleed protection for people with severe hemophilia A Results underscore the ability of efanesoctocog alfa to sustain normal to near-normal factor levels and the potential to transform prophylactic treatment, providing people with hemophilia A with higher […]

STAQ Pharma, Inc. Advises of Printing Error

June 27, 2022

STAQ Pharma, Inc. has advised the Hemophilia Alliance and the National Hemophilia Foundation of a typographical printing error on a few units of lot 21114255A Desmopressin Acetate 1.5 mg/mL. This does not involve any patient safety issues. The expiration date on a few of the vials shows 2021-06-28 (June 28, 2021) when the correct expiration […]

FDA Accepts CSL Behring鈥檚 Biologics License Application for Etranacogene Dezaparvovec for Priority Review 聽

June 2, 2022

HFA received the following announcement from CSL Behring. See full press release. FDA Accepts CSL Behring鈥檚 Biologics License Application for Etranacogene Dezaparvovec for Priority Review 聽 聽If approved, etranacogene dezaparvovec would be the first gene therapy option for people living with hemophilia B This milestone underscores CSL Behring鈥檚 promise to develop and deliver a truly […]

FDA Grants Breakthrough Therapy Designation for Hemophilia A

June 2, 2022

HFA received the following press release from Sanofi. FDA grants efanesoctocog alfa Breakthrough Therapy designation for hemophilia A Efanesoctocog alfa is the first factor VIII therapy to be awarded Breakthrough Therapy designation by the FDA Designation is based on XTEND-1 Phase 3 study data demonstrating a clinically meaningful prevention of bleeds and superiority in prevention […]

BioMarin Issues Clinical Program Update

June 2, 2022

HFA received this clinical program update from BioMarin. BioMarin Hemophilia A Clinical Development Program: An Update for the Hemophilia Community BioMarin is providing an update to the community regarding our ongoing gene therapy clinical trial program in hemophilia A. BioMarin’s investigational gene therapy for hemophilia A has not been approved for use; it is in […]

Bayer Discontinues Kogenate庐 FS in the United States

June 2, 2022

HFA received the following update from Bayer. For more than 25 years, Bayer has remained committed to supporting patients around the world by keeping a constant pulse on their evolving needs as well as those of their caregivers and healthcare providers (HCPs). Recognizing the growing shift in patient use toward more recent products, such as […]

Pfizer Reports First Quarter 2002 Update of Gene Therapy Trial

May 3, 2022

Read the original press release here: https://s28.q4cdn.com/781576035/files/doc_financials/2022/q1/Q1-2022-PFE-Earnings-Release.pdf During a recent quarterly stakeholder report, Pfizer announced that in March 2022 the US Food and Drug Administration lifted a clinical hold that had been placed on the Phase 3 AFFINE study in November 2021 following the observance of Factor VIII levels greater than 150% in some study […]

Freeline Announces First Patient Dosed in Hemophilia B Trial

March 11, 2022

Read original press release here. LONDON, March 09, 2022 (GLOBE NEWSWIRE) — Freeline TherapeuticsHoldings plc announced that the first patient was dosed in its Phase 1/2 B-LIEVE dose-confirmation clinical trial of FLT180a for the treatment of hemophilia B, a debilitating genetic bleeding disorder caused by a deficiency in the clotting factor IX protein. FLT180a uses […]

Sanofi and SOBI Announce Results of XTEND-1 Phase 3 Study

March 11, 2022

Read the original press pelease here. Efanesoctocog alfa met primary and key secondary endpoints in pivotal study in hemophilia A, demonstrating superiority to prior factor prophylaxis treatment Once-weekly efanesoctocog alfa met primary endpoint in phase 3 study, resulting in a clinically meaningful prevention of bleeding episodes (bleed protection) In the key secondary endpoint, efanesoctocog alfa […]

BioMarin provides update to ongoing Phase 3 hemophilia A gene therapy study

February 4, 2022

An update for the hemophilia community from BioMarin, regarding the ongoing Phase 3 BioMarin hemophilia A gene therapy study, and a serious adverse event deemed, by the Independent Data Safety Monitoring Committee, unrelated to the investigational gene therapy from the phase 1/2 study. View the original Clinical Development Program Update BioMarin鈥檚 investigational gene therapy for […]

FDA Approves Prophylactic Treatment with VONVENDI庐 [von Willebrand Factor (Recombinant)] for Adult Patients Living with Severe Type 3 von Willebrand Disease (VWD)

February 2, 2022

Original Source: Takeda Press Release January 31, 2022 VONVENDI [von Willebrand Factor (Recombinant)] is the First and Only Treatment Approved for Routine Prophylaxis to Reduce the Frequency of Bleeding Episodes in Adults Living with Severe Type 3 VWD Receiving On-Demand Therapy VWD is the Most Common Inherited Bleeding Disorder, Affecting up to Three Million (or […]

Freeline Presents Long-Term Follow-Up Data from Phase 1/2 B-AMAZE Trial in Hemophilia B at the 2021 ASH Annual Meeting and Announces Early Initiation of Phase 1/2 B-LIEVE Dose-Confirmation Trial

December 20, 2021

B-AMAZE long-term follow up data demonstrate sustained FIX expression up to 3.5 years; potential for expression levels in the normal range with relatively low vector doses B-LIEVE trial to confirm dose and immune management for planned Phase聽3 pivotal trial launched one quarter ahead of latest guidance LONDON, Dec. 13, 2021 (GLOBE NEWSWIRE) — Freeline Therapeutics […]

Pfizer and Sangamo Announce Updated Phase 1/2 Results Showing Sustained Bleeding Control in Highest Dose Cohort Through Two Years Following Hemophilia A Gene Therapy

December 20, 2021

NEW YORK & BRISBANE, Calif.–(BUSINESS WIRE)–Pfizer Inc.聽and Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines company, today announced updated follow-up data from the Phase 1/2 Alta study of giroctocogene fitelparvovec, an investigational gene therapy for patients with moderately severe to severe hemophilia A. The Alta study data, in patients with severe hemophilia A, are being […]

Sigilon Therapeutics Announces Strategic Reprioritization

December 14, 2021

CAMBRIDGE, Mass., Dec. 13, 2021 (GLOBE NEWSWIRE) — Sigilon Therapeutics, Inc. (NASDAQ: SGTX), a biotechnology company that seeks to develop functional cures for chronic diseases through its Shielded Living TherapeuticsTM platform, today announced a strategic reprioritization to enable the Company to focus on MPS-1 and diabetes. 鈥淭here have been key learnings in our Phase 1/2 […]

uniQure and CSL Behring Announce Primary Endpoint Achieved in HOPE-B Pivotal Trial of Etranacogene Dezaparvovec Gene Therapy in Patients with Hemophilia B

December 13, 2021

Press release source: uniQure and CSL Behring via Globe Newswire Largest gene therapy study in hemophilia B achieved primary endpoint of non-inferiority in annualized bleeding rate after stable Factor IX (FIX) expression, assessed at 18 months following a single dose of etranacogene dezaparvovec Etranacogene dezaparvovec also achieved secondary endpoint demonstrating statistical superiority in reduction of […]

Sigilon Therapeutics Announces Update on SIG-001 Phase 1/2 Study in Hemophilia

November 29, 2021

Source:聽Sigilon press release Sigilon Therapeutics, Inc., a biotechnology company that seeks to develop functional cures for chronic diseases through its Shielded Living Therapeutics™ platform, reported that fibrosed spheres were observed during a retrieval procedure in a patient in its Phase 1/2 study of SIG-001 in severe or moderately severe hemophilia A. The SIG-001 trial had […]

Pfizer Giroctocogene Fitelparvovec (Hemophilia A Gene Therapy) AFFINE Phase 3 Study Update

November 5, 2021

The following is a news release from Pfizer. Read the full statement here (view PDF): We would like to share a recent update to our gene therapy clinical program for Hemophilia A (C3731003; AFFINE study) evaluating giroctocogene fitelparvovec, that we are developing with Sangamo. Following the observation of factor VIII (FVIII) levels greater than 150% […]

Recall: Monoject Flush Prefilled Saline Syringes by Aligned Medical Solutions Recalled Due to Potential for Plunger to Draw Back After Air Has Been Expelled and Reintroduce Air Back into Syringe

October 28, 2021

Monoject Flush Prefilled Saline Syringes by Aligned Medical Solutions: Recall – Due to Potential for Plunger to Draw Back After Air Has Been Expelled and Reintroduce Air Back into Syringe Read the full press release from the FDA Issue: Aligned Medical Solutions is recalling Cardinal Health鈥檚 Monoject Flush Prefilled Saline Syringes placed into 9,378 kits. […]

Astellas Reports Fourth Death In Orphan Disease Gene Therapy Clinical Trial

September 14, 2021

The following is a news release from Astellas Pharma Inc. Read the release in full here. Astellas Pharma Inc. (TSE: 4503,聽President and CEO:聽Kenji Yasukawa, Ph.D., “Astellas”)聽reports聽that聽the ASPIRO clinical trial鈥痯articipant, who鈥痙eveloped a serious adverse event as described on聽September 1, passed away聽on September聽9. The cause of death is still pending. Astellas聽is gathering the relevant clinical information and […]

Important Update Regarding Availability of Stimate

September 9, 2021

Important Update Regarding Availability of Stimate Source: CSL Press release Recently, CSL Behring announced that the company has chosen to end their distribution agreement with Ferring Pharmaceuticals for STIMATE庐 nasal spray, effectively thereby removing this medication as a valuable treatment option for patients with von Willebrand Disease (VWD) and mild hemophilia A. Background For over […]

Cardinal Health Issues Nationwide Recall of Select Monoject™ Flush Prefilled Saline Syringes

August 21, 2021

Read the U.S. Food & Drug Administration’s announcement here. On August 4, 2021, Cardinal Health initiated a nationwide recall of approximately 267 million MonojectTM Flush Prefilled Saline Syringes (0.9% Sodium Chloride). The products have been found to reintroduce air into the syringe after the air has been expelled. This could result in injection of air […]

Gilead Warns of Counterfeit HIV Medication Being Distributed in the United States

August 6, 2021

The following is sourced from Gilead. Read the update in its entirety “Gilead Warns of Counterfeit HIV Medication Being Distributed in the United States”. Click to view the original press release. Foster City, Calif., August 5, 2021聽鈥 Gilead Sciences has become aware of tampered and counterfeit versions of its once-daily single tablet HIV treatment regimen […]

Biomarin: Clinical Development Program Update

July 20, 2021

The following is sourced from BioMarin. Read the update in its entirety “BioMarin Hemophilia A Clinical聽Development Program: An Update for the Hemophilia Community” View PDF | Download PDF BioMarin Hemophilia A Clinical Development Program: An Update for the Hemophilia Community BioMarin is pleased to update the community regarding our ongoing gene therapy clinical trial program […]

Sigilon Therapeutics Announces Clinical Hold on SIG-001 Phase 1/2 Study in Hemophilia A

July 12, 2021

The following is sourced from a press release from Sigilon Therapeutics. Read the update in its entirety聽here. The U.S. Food and Drug Administration (FDA) has placed a clinical hold on Sigilon Therapeutics鈥 phase 1/2 study of SIG-001 in patients with severe or moderately severe hemophilia A. SIG-001 is a cell-based therapy, designed to carry significant […]

BioMarin: Clinical Development Program Update

July 2, 2021

BioMarin is pleased to update the community regarding our ongoing gene therapy clinical trial program in hemophilia A. BioMarin’s investigational gene therapy for hemophilia A has not been approved for use; it is in ongoing clinical trials evaluating its safety and efficacy. BioMarin Hemophilia A Clinical聽Development Program: An Update for the Hemophilia Community View PDF […]

BioMarin: Clinical Development Program Update

May 21, 2021

Update for Hemophilia Associations BioMarin is pleased to update the community regarding our gene therapy clinical trial program in hemophilia A. BioMarin鈥檚 investigational gene therapy for hemophilia A has not been approved for use; it is in ongoing clinical trials evaluating its safety and efficacy. BioMarin Hemophilia Update For Patient Associations View PDF | Download […]

CSL Behring Announces Closing of Global Commercialization and License Agreement with uniQure for etranacogene dezaparvovec

May 7, 2021

Read the full press release from CSL Behring here. Global biotherapeutics leader CSL Behring 聽announced the closing of its global Commercialization and License agreement with uniQure for etranacogene dezaparvovec, a novel gene therapy for the treatment of hemophilia B. Etranacogene dezaparvovec is currently in Phase 3 clinical trials and has been shown聽 to result in […]

uniQure Announces Closing of Commercialization and License Agreement with CSL Behring

May 6, 2021

Read the original press release here uniQure, a leading gene therapy company advancing transformative therapies for patients with severe medical needs, announced that the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976 for the global licensing agreement with CSL Behring for etranacogene dezaparvovec, an investigational gene therapy for patients with hemophilia B, expired […]

uniQure Announces FDA Removes Clinical Hold on Hemophilia B Gene Therapy Program

April 26, 2021

Read the full release here. uniQure, a leading gene therapy company advancing transformative therapies for patients with severe medical needs, announced that the U.S. Food and Drug Administration (FDA) has removed the clinical hold on the Company鈥檚 hemophilia B gene therapy program after determining the Company satisfactorily addressed all issues identied by the FDA related […]


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