Anemia caused by the autoimmune disease cold agglutinin disease now has its first approved treatment, a Sanofi drug designed to block a key pathway that causes the disease to progress.This FDA’s regulatory nod For the drug, sutimlimab puts the French pharma giant ahead of a rival company that aims to treat the rare disease in a similar way.
Sanofi’s new drug, which is injected every two weeks, will be marketed under the name “Enjaymo.”
exist cold agglutinin disease, the patient’s immune system relentlessly attacks red blood cells, causing them to burst. The “cold” part of the disease’s name refers to the destruction of these cells at low temperatures, the FDA said in its approval announcement late Friday. At these temperatures, antibodies that normally attack bacteria attach to red blood cells and cause them to bind together or agglutinate. These antibodies also mark red blood cells for destruction by the immune system.
Disease symptoms usually worsen in winter when the body is exposed to cold. The FDA estimates that cold agglutinin disease affects 1 in 1 million people each year, usually older adults between the ages of 40 and 80. A sharp drop in red blood cell counts can lead to anemia, which can be controlled with blood transfusions.
Cold agglutinin disease is a disorder of the complement system, part of the innate immune system. Sanofi’s drug is a monoclonal antibody designed to selectively target and block a protein called C1 in the complement system. This is done to stop the deposition of antibodies on red blood cells, thereby halting the cascade of events that lead to cold agglutinin disease. Sanofi’s drug is designed to block C1 without affecting other complement system pathways.
The FDA’s approval of Enjaymo is based on results from an open-label Phase 3 study that enrolled 24 patients with cold agglutinin disease who had received blood transfusions within the six months prior to study entry. The participants received Sanofi’s experimental therapy for up to six months. According to the results, 54 percent of patients showed a response, which was defined as an increase in hemoglobin, no red blood cell transfusions after the first five weeks of treatment, and no other therapy was needed to treat the disease. The most common side effects reported in the study included respiratory infections, viral infections, diarrhea, indigestion, and swelling of the lower legs and hands.Sanofi presented data Key research from the American Society of Hematology 2019 Annual Meeting.
After an initial dose of twice weekly, Enjaymo is administered every two weeks based on patient weight: 6,500 mg for patients between 39 kg and 75 kg (approximately 86 to 165 lb) and 75 kg for patients weighing more than 7,500 mg . Sanofi suit Wholesale price is $1,800 per bottle before any rebates or discounts. Patients weighing between 39kg and 75kg needed six vials for the infusion, meaning a single dose would cost $10,800. Given that cold agglutinin disease is a chronic condition, the annual cost of treatment is $280,800. This is especially true for people who weigh more than 75 kg, as these patients require an additional vial per dose.
Njemo is Originally developed by True North Therapeuticsa biotechnology Acquired by Bioverativ 2017. the following year, Sanofi to buy Bioverativ for $11.6 billion. The drug’s approval came later than Nofi had hoped. By the end of 2020, the FDA Refuse The pharma giant’s biologics license application cited field problems with third-party manufacturers of the drug. Sanofi said regulatory reviews of Enjaymo were still ongoing in Europe and Japan.
Apellis Pharmaceuticals is also addressing complement system diseases, but by addressing a different complement system protein called C3.The Waltham, Massachusetts-based company’s drug pegcetacoplan won FDA approval last year to treat paroxysmal nocturnal hemoglobinuria, another rare blood disorder in which the body attacks red blood cells. The company said that pegcetacoplan has the potential to treat other diseases. In 2020, the company was established Ally with Sobi, the company paid $250 million upfront to participate in drug development for other indications. The partners expect to begin a Phase 3 study in cold agglutinin disease in the first quarter of this year.
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