Sanofi's Enjaymo, once a jewel in its Bioverativ buyout, finally wins its FDA nod for a rare blood disorder

Patients with the rare blood disorder cold agglutinin disease (CAD), now have a drug that will help limit the blood transfusions they need.

On Friday, the FDA approved Sanofi’s Enjaymo (sutimlimab) for hemolysis—the destruction of red blood cells—which accompanies the disease. Enjaymo becomes the first approved treatment for CAD.

Winning approval for sutimlimab will give Sanofi a chance to recoup some more of the $11.6 billion (€10.5 billion) it spent to acquire Bioverativ in 2018. Bioverativ itself acquired sutimlimab in 2017 via a $400 million-plus deal for True North Therapeutics.

At the time of Sanofi’s takeover of Bioverativ, Citi analyst Peter Verdult saw sutimlimab as “the most promising pipeline asset” at the acquired company. 

It's been a long time coming, though. Sutimlimab, a monoclonal antibody, was up for approval in 2020, but the FDA hit Sanofi with a complete response letter in November of that year, citing deficiencies in an inspection of a contract manufacturer's plant. There were no clinical or safety deficiencies in the rejection.

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Enjaymo is given intravenously, with weekly doses the first two weeks followed by doses every two weeks. It's priced at $1,800 per vial before payer discounts, and dosing depends on body weight.

For a patient who weighs between 39 kg (85 pounds) and 75 kg (165 pounds) the cost—once established on the treatment—would be $280,000 annually, according to a company spokesman.

Enjaymo succeeded in a second phase 3 study presented last June. Of 22 CAD patients given the drug, 16 had a 1.5-g/dL or more improvement in hemoglobin levels and were free from transfusions and the need for other non-permitted therapies after five weeks. Hemoglobin improvements were statistically significant over placebo.

Cold agglutinin disease is an autoimmune disorder that affects roughly 5,000 people in the United States. It is caused by antibodies—cold agglutinins—binding to the surface of red blood cells, which in turn triggers a process that causes the immune system to attack and rupture healthy red blood cells.

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Considering red blood cells carry oxygen throughout the body, the disease can cause a host of symptoms including fatigue, weakness, shortness of breath, light-headedness, chest pain, irregular heartbeat and other complications.

“Until now, people living with cold agglutinin disease haven’t had an approved treatment option to manage the constant destruction of red blood cells,” Bill Sibold, the head of Specialty Care, said in a release. “(Enjaymo) helps stop the chain reaction from the start.”

On the way to approval, Enjaymo won FDA breakthrough therapy and orphan drug designations, as well as priority review. It's also under review for approval in Europe and Japan.