Sanofi’ Enjaymo Wins FDA Approval for Rare Autoimmune Anemia

Sanofi’s Enjaymo (sutimlimab-jome) has become the first FDA-approved treatment for cold agglutinin disease (CAD), a rare and chronic autoimmune blood disorder. The disease affects around 5,000 people in the US, and is characterized by the constant destruction of red blood cells (hemolysis).

An Autoimmune Anemia

Normally, red blood cells have a lifespan of around 120 days before they are destroyed in the spleen. However, antibodies called cold agglutinins bind to the surface of red blood cells, triggering a process where the body’s immune system mistakenly attacks these cells, causing their premature destruction. 

Patients with CAD typically experience symptoms of anemia, such as fatigue, weakness, shortness of breath, lightheadedness, and irregular heartbeat among other complications. 

CAD is termed “cold” because the antibodies are active at low temperatures.

Measures against the disease range from avoiding cold temperatures, treating hemolysis and anemia through blood transfusions, or modulating the body’s immune system.

Rituximab, a monoclonal antibody, is a first-line therapy that suppresses the white blood cells that produce the antibodies responsible for causing CAD. However, CAD often recurs after rituximab treatment. 

Related Article: Sanofi Bets $1 Billion on Preclinical Next-gen Immunotherapies

Enjaymo Mechanism of Action and Trial Results

Enjaymo is a monoclonal antibody that inhibits C1s in the classical complement pathway. By blocking C1s, Enjaymo prevents the activation of the complement cascade in the immune system which causes hemolysis. 

Enjaymo’s US approval is based on positive results from the Cardinal study, a 26-week Phase 3 trial in CAD patients who have a recent history of blood transfusions.

54% of patients met the primary endpoint of improved hemoglobin level and no blood transfusions after week 5 or other medications prohibited by the protocol.  

63% of patients experienced normalization of hemoglobin levels or an increase in baseline hemoglobin, while 71% remained transfusion-free after week 5. 92% of patients did not use other treatments for CAD.

The study also met secondary endpoints of mean increases in hemoglobin level and mean reductions in bilirubin levels.

“For people living with cold agglutinin disease, it is as if their body’s immune system is waging a war on itself,” said Catherine Broome, an associate professor of medicine at Georgetown University Lombardi Comprehensive Cancer Center and a principal investigator in the Cardinal study.

“In the pivotal study, patients treated with sutimlimab had an improvement in anemia as measured by hemoglobin and bilirubin levels during the 26-week study.”  

Enjaymo is administered intravenously weekly for the first two weeks, and then administered biweekly thereafter. The list price of Enjaymo in the U.S. is $1,800 per vial, but actual costs to patients is expected to be lower, said Sanofi.

Related Article: WHO Recommends the Use of Sanofi’s and Roche’s Rheumatoid Arthritis Drugs to Cut COVID-19 Deaths

Sanofi Rebrands

Last Thursday, Sanofi unveiled a new corporate brand and logo, saying that the rebrand unites the company under one purpose and a single identity. 

Sanofi’s business units Sanofi Pasteur and Sanofi Genzyme, which specialize in vaccines and specialty care respectively, and other acquired brands will be united under the Sanofi name and brand.

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Joy Lin

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