Anemia Drug From Acceleron Pharma and Celgene Wins FDA Approval

Xconomy New York — 

[Updated 6:41 p.m. See below.] A drug that Celgene and Acceleron Pharma developed to treat low red blood cell levels in patients who have a rare blood disorder now has regulatory clearance to enter the market.

The FDA on Friday approved luspatercept (Rebloyzl) for patients who have beta thalassemia, an inherited disorder that leads to anemia, or low levels of oxygen-carrying red blood cells. Beta thalassemia patients require regular blood transfusions to keep their red blood cell levels up. Those transfusions can lead to a buildup of iron, which can damage organs. The excess iron must be removed by additional therapy.

Summit, NJ-based Celgene (NASDAQ: CELG) and Acceleron (NASDAQ: XLRN) of Cambridge, MA, developed luspatercept to regulate the late-stage maturation of red blood cells, boosting the production of these cells in patients who have rare types of anemia. The drug, which is given as a subcutaneous injection, is intended to reduce a patient’s need for blood transfusions.

Luspatercept’s approval is based on data from a Phase 3 test that showed the patient group treated with the drug had a greater than 33 percent reduction in red blood cell transfusions compared to a 4.5 percent reduction in the group given a placebo. The most common side effects included headache, bone pain, and joint pain. More serious side effects, such as deep vein thromboses and ischemic stroke, were reported in 3.6 percent of patients treated with luspatercept. Abnormally high blood pressure was reported in 10.7 percent of treated patients.

Luspatercept was discovered in the labs of Acceleron. The company began a drug development partnership with Celgene in 2008. Their collaboration on luspatercept dates to 2011. The FDA’s approval of the drug comes about three weeks ahead of the Dec. 4 target date for a decision. The two companies will co-promote the drug in the US, and they expect it will become available within a week.

Celgene and Acceleron aim to broaden use of luspatercept to other types of anemia. They have filed for FDA review of the drug as a treatment for anemia in patients who have myelodysplastic syndrome (MDS), a disorder that leads to red blood cells that are abnormally formed or poorly functioning. An FDA decision for that use of the drug is expected by April 4. The drug is also in mid-stage testing as a treatment for myelofibrosis, a rare type of bone marrow cancer that disrupts red blood cell production.

[Paragraph added with pricing information, analyst comment] Luspatercept will be priced at $3,441 for a 25 mg vial, according to Reuters. The amount of drug required per dose depends on a patient’s weight. SVB Leerink analyst Geoffrey Porges wrote in a Friday evening research note that the cost works out to roughly $10,000 per dose, with an annual cost in the neighborhood of $170,000. That’s “significantly higher than we expected,” Porges wrote. He projects the drug could reach $2.7 billion in sales by 2029—$2 billion for MDS and $700 million for beta thalassemia.

Photo by Flickr user Joel Kramer via a Creative Commons license