FDA grants priority review of sutimlimab, potential first approved treatment of hemolysis in adult

*Sutimlimab targets C1-activated hemolysis in cold agglutinin disease (CAD)

PARIS – May 14, 2020 - The U.S. Food and Drug Administration (FDA) has granted priority review of Sanofi’s Biologics License Application (BLA) for sutimlimab for the treatment of hemolysis in adult patients with cold agglutinin disease (CAD). Sutimlimab, an investigational monoclonal antibody, targets the underlying cause of hemolysis in CAD by selectively inhibiting complement C1s.

If approved, sutimlimab would be the first and only approved treatment for these patients. The target action date for the FDA decision is November 13, 2020. 

CAD is a chronic autoimmune hemolytic anemia that causes the body’s immune system to mistakenly attack healthy red blood cells and cause their rupture (hemolysis). CAD patients may experience chronic anemia, profound fatigue, acute hemolytic crisis, and other potential complications, including an increased risk of thromboembolic events and early death.1,2,3 An estimated 5,000 people in the U.S. live with CAD.

“People living with cold agglutinin disease currently have no approved treatment option and experience chronic anemia and profound fatigue, which have a persistent and serious impact on their lives,” said John Reed, M.D., Ph.D., Global Head of Research and Development at Sanofi. “Results from our 26-week pivotal Phase 3 study clearly demonstrated that sutimlimab had a clinically meaningful effect on complement-mediated hemolysis, which is the cause of anemia and fatigue. If approved, sutimlimab will be the first and only FDA-approved treatment to uniquely address C1-activated hemolysis and help alleviate the chronic disease burden for people with CAD.”

The BLA submission is based on results from part A (n=24) of the open label, single arm pivotal Phase 3 CARDINAL study in patients with primary CAD. The data were presented in the Late-Breaking Abstracts Session at the 61st Annual Meeting of the American Society of Hematology and demonstrated sutimlimab met its primary composite efficacy endpoint defined as the proportion of patients who demonstrated an increase from baseline in Hgb level ≥2 g/dL or normalization of Hgb level ≥12 g/dL at the treatment assessment time point (mean value from weeks 23, 25, and 26) and no blood transfusion from week 5 through week 26. 

The trial showed sutimlimab also met its secondary endpoints by indicating improvements in disease process, including improvements in hemoglobin, normalization of bilirubin, and improvements in Functional Assessment of Chronic Illness Therapy-Fatigue Score.

Targeting C1s in the classical complement pathway

Sutimlimab is designed to selectively target and inhibit C1s in the classical complement pathway, which is part of the innate immune system. By blocking C1s, it is thought that sutimlimab halts C1-activated hemolysis in CAD. The inhibition of the classical pathway at C1s aims to retain immune surveillance functional activities of the alternative or lectin complement pathways.

Sanofi is evaluating sutimlimab in the on-going Phase 3 CADENZA trial for CAD patients who have not recently had a blood transfusion and separately, investigating sutimlimab for patients with immune thrombocytopenic purpura. Sutimlimab has been granted Breakthrough Therapy and Orphan Drug designation by the FDA. Sutimlimab is currently under clinical development and its safety and efficacy have not been evaluated by any regulatory authority.


In Support of Rare Disease Patients Impacted by COVID-19, NORD Launches Premium and Limited Medical

NORD’s COVID-19 Premium & Medical Relief Program assists eligible rare disease patients facing financial challenges with out of pocket costs of health insurance premiums and limited medical assistance. 

Washington, DC, May 12, 2020— United with more than 25 million Americans living with rare diseases, the National Organization for Rare Disorders (NORD®) today launched its COVID-19 Premium & Medical Relief Program to provide vital support to members of the rare disease community affected by the COVID-19 pandemic. The program will assist those with a confirmed rare disease diagnosis who have been directly impacted by the pandemic through a job loss, reduced work hours, furlough, quarantine, etc.  

“COVID-19 poses significant threats to the rare disease community, given the susceptibility of those with chronic conditions and the economic challenges that affect their ability to weather this storm. We are hearing from patients who have been furloughed or laid off and desperately need financial support for insurance, COBRA payments, co-pays and medical expenses,” said Pamela Gavin, Chief Strategy Officer for NORD. “Through this new program, we are providing premium and limited medical assistance to rare patients who need it most during this crisis.” 

NORD recognizes the needs of the community and has developed this program to provide critical support. The COVID-19 Premium & Medical Relief Program helps with certain out-of-pocket costs associated with health insurance premiums, and supplies eligible uninsured and under-insured patients with support for limited medical expenses. These expenses include medical visits and telehealth consults, laboratory and diagnostic testing, physical therapy and medical equipment. 

We are grateful to Sanofi-Genzyme for their generous donation to this patient assistance program, which is available to all eligible rare disease patients affected by COVID-19. NORD has been proud to serve the rare disease community with assistance programs providing support to patients since 1987. In order to continue to meet the community’s needs during this unprecedented time, NORD is seeking additional donations to its COVID-19 relief programs. Please help us to help our rare community. 

For more information on NORD’s COVID-19 patient assistance programs, including eligibility requirements, please visit our website, contact NORD by telephone at 203.242.0497, or via email at COVID19assistance@rarediseases.org

To stay informed, the NORD COVID-19 resource center presents regularly updated information and vetted links relevant to the rare community during this pandemic. 


we have exciting news about the cadf summit

Hi CADdys!

We are working on getting the funding for a CAD live and in-person, inaugural Summit! Though you are not making a commitment, we need you to give us your feedback to help us plan. Those offering their input will be first on the list for attendance, room choices, etc. when we are ready to go. We would like everyone (worldwide) to answer, who lives in the US, Canada and overseas. Based on the situation we find ourselves in with COVID-19, this will probably take place in 2021, but we have to start planning now to get the funding, so please answer as quickly as possible.

In addition, we have future plans for a newsletter, other communication, and CADF growth, so included with the survey for our meeting is a profile section we need you to fill out. We would like to accomplish both sections at once, so if you would take the time to do both, we would appreciate it.

If you are not interested in the 2021 CADF meeting, please go straight to Section II and answer those questions. If at any time, you wish to opt-out of communications, you can.    

Click the link open for submissions May 1 - May 31, 2020


corona virus and cad

 Read a message from Sigbjørn Berentsen, MD, PhD on the Coronavirus and Cold Agglutinin Disease. Click below to view or download PDF.

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