Qfitlia approved as the first therapy in the US to treat hemophilia A or B with or without inhibitors

• Qfitlia (fitusiran), the first antithrombin-lowering therapy in hemophilia, offers consistent protection with as few as six injections a year via a prefilled pen or vial and syringe
• Unique mechanism helps reduce the frequency of bleeding episodes for people with hemophilia   

Paris, March 28, 2025. The US Food and Drug Administration (FDA) has approved Qfitlia (fitusiran), the first antithrombin-lowering (AT) therapy for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients (aged 12 or older) with hemophilia A or B with or without factor VIII or IX inhibitors. The approval is based on data from the ATLAS phase 3 studies that demonstrated clinically meaningful bleed protection as measured by annualized bleeding rates (ABR) across hemophilia patients with or without inhibitors.

Phil Gattone
President and CEO, National Bleeding Disorders Foundation
“Current treatment options can make people with hemophilia feel they need to choose between effective bleed control and convenient dosing schedules, leading to trade-offs when it comes to disease management. Qfitlia takes a novel approach to providing protection for people living with hemophilia while reducing the frequency of dosing for patients and their families.”

By lowering AT, a protein that inhibits blood clotting, Qfitlia helps increase thrombin generation to restore hemostasis in people with hemophilia. Qfitlia uses small-interfering RNA technology, which enables low treatment frequency, subcutaneous dosing, and low volume injections.

Brian Foard
Executive Vice President, Head of Specialty Care, Sanofi
“This approval highlights our commitment to advancing innovation and improving care for the rare blood disorders community. Qfitlia has the potential to meaningfully change the hemophilia landscape through effective bleed protection, infrequent dosing, and simplified administration. Our robust portfolio of hemophilia treatment options continues to grow as we focus on offering protection with reduced treatment burden that best fits an individual’s needs.”

Guy Young, MD
Director, Hemostasis and Thrombosis Center at Children's Hospital, Los Angeles
“Qfitlia delivers the fewest doses of any prophylactic therapy in hemophilia, and its unique mechanism allows it to be used to treat all types of hemophilia, including with inhibitors and hemophilia B, where unmet medical needs remain. By targeting antithrombin, which can be reliably measured with an FDA-cleared blood assay, Qfitlia is proven to help rebalance hemostasis and improve bleed rates and protection.”
In the ATLAS clinical development program, Qfitlia demonstrated low bleed rates across subgroups with as few as six injections a year. Key results include:

• Significant bleed reduction by 71% in ABR for patients without inhibitors treated with Qfitlia prophylaxis compared to clotting factor concentrate on-demand (estimated mean: ABR 9.0 vs. 31.4, respectively; p<0.0001) and by 73% in ABR compared to bypassing agent on-demand for patients with inhibitors (estimated mean: ABR 5.1 vs. 19.1, respectively; p=0.0006)
• Median observed ABR during the open-label extension study was 3.8 (IQR: 0.0–11.2) in patients without inhibitors and 1.9 (IQR: 0.0–5.6) in patients with inhibitors
• Median observed annualized spontaneous bleeding rate during the open-label extension study was 1.9 (interquartile range (IQR): 0.0-7.5) in patients without inhibitors and 1.9 (IQR: 0.0-3.7) in patients with inhibitors
• Nearly half of patients in the open-label extension study experienced one or fewer bleeds (31% 0 bleeds and 47% 0-1 bleeds)

There is also the potential for significant adverse reactions, including thrombotic events, acute and recurrent gallbladder disease, and hepatotoxicity. The most common adverse reactions (incidence >10%) are viral infection, nasopharyngitis, and bacterial infection.
In conjunction with the Qfitlia approval, the FDA also cleared the Siemens Healthineers’ INNOVANCE® Antithrombin assay as a companion diagnostic for Qfitlia to measure AT levels. Through the Qfitlia Testing Program with Labcorp, the FDA-cleared companion diagnostic will be available to patients prescribed Qfitlia to measure AT levels at no cost.

Qfitlia can offer the fewest doses of all prophylactic therapies, and it will have a comparable price to other prophylactic hemophilia treatments. HemAssist is launching alongside Qfitlia to provide comprehensive patient support services, including insurance and financial assistance as well as educational resources. This program is for patients prescribed Qfitlia or other hemophilia treatments from Sanofi’s portfolio.
The FDA granted Qfitlia Orphan Drug Designation for hemophilia A and B, Fast Track Designation for hemophilia A and B with and without factor VIII or IX inhibitors, and Breakthrough Therapy Designation for hemophilia B with factor IX inhibitors. A regulatory submission for Qfitlia for the treatment of hemophilia A or B in adults and adolescents with or without inhibitors is under review in Brazil. A regulatory decision is expected in China in the second half of 2025.

Read the full press release: https://www.news.sanofi.us/2025-03-28-Qfitlia-approved-as-the-first-therapy-in-the-US-to-treat-hemophilia-A-or-B-with-or-without-inhibitors

Kedrion Expands Ryplazim Distribution Network for PLGD-1 Patients 

FORT LEE, NJ., Mar. 20, 2025 /PRNewswire/ -- Kedrion Biopharma Inc. expands the distribution network for Ryplazim, a plasma-derived human plasminogen indicated for treating patients with plasminogen deficiency type 1 (PLGD-1). This expansion ensures timely access to this vital medication for patients across the US. PLGD-1 is a serious condition causing abnormal fibrin-rich lesions on mucosal surfaces, potentially leading to severe consequences like vision and hearing loss, airway obstruction, and infertility. Ryplazim, the only FDA approved therapy for PLGD-1 patients, offers an important treatment option. 

The recent FDA approval of the technology transfer and manufacturing capacity expansion of Ryplazim has enabled Kedrion to broaden its limited distribution network due to the increase of supply. This crucial step means more patients can have access to this treatment. The expanded network now includes a new distribution partner and two new specialty pharmacies.

CuraScript SD a provider of comprehensive distribution solutions, joins existing partners FFF Enterprises and The Alliance Pharmacy in distributing Ryplazim to patients with PLGD-1.

CVS Health and Soleo Health have been added to the list of specialty pharmacies carrying Ryplazim, increasing access points for patients. These pharmacies join Nufactor, enhancing the network to ensure wider availability.

This collaborative effort underscores Kedrion's commitment to improving patient care and broadening access to essential therapies. 

To learn more about accessing Ryplazim and its potential benefits, please visit www.Ryplazim.com.

Read the full press release: https://lnkd.in/ez242whw

This is a letter from Takeda Pharmaceuticals U.S.A., Inc on March 18 for the bleeding disorders community.

Global Discontinuation of HEMOFIL® M [Antihemophilic Factor (Human), Method M, Monoclonal Purified] and RECOMBINATE® [Antihemophilic Factor (Recombinant)]

Dear Valued Patient,
The purpose of this letter is to inform you that Takeda has decided to globally discontinue HEMOFIL® M
[Antihemophilic Factor (Human), Method M, Monoclonal Purified] and RECOMBINATE® [Antihemophilic Factor (Recombinant)].

This was not a decision we made lightly. As the treatment landscape evolves, we decided to discontinue these medicines as hemophilia patients continue to transition to alternate treatment options in the space, including those within our own hematology portfolio. It is important to note there is no quality issue with either HEMOFIL M or RECOMBINATE and that their safety and efficacy remains consistent with the product Prescribing Information.

We understand that this directly impacts you and are here to support the hemophilia community during this transition. We intend to supply HEMOFIL M and RECOMBINATE to patients already receiving these medicines until inventory is depleted or expired in mid-2026. Exact timing will vary based on potency and demand.

​Transitioning to Alternative Treatment
We recommend beginning to have discussions with your healthcare team to ensure ample time for creating a longer-term, alternative treatment plan.
​
For more than 70 years, we’ve pioneered innovations and worked tirelessly to improve the standard of care for hemophilia patients. We are proud to offer alternative treatment options within the Takeda factor VIII portfolio, namely ADVATE® [Antihemophilic Factor (Recombinant)] and ADYNOVATE® [Antihemophilic Factor (Recombinant), PEGylated], that may meet your individual needs and are similar to HEMOFIL M and RECOMBINATE. Please visit ADVATE.com and ADYNOVATE.com for more information.