Bristol Myers Squibb (BMS) has been developing luspatercept-aamt – marketed as Reblozyl – as a treatment for anemia in non-transfusion dependent (NTD) beta thalassemia. They submitted a supplemental biologics license application (sBLA) to the FDA and were hoping to hear back with a decision by the end of this month. However, the FDA requested additional data from the pharmaceutical company and subsequently added three months onto their decision deadline.

Next Steps for Luspatercept-aamt

BMS – working with Merck – has developed this erythroid maturation agent as a treatment for NTD beta thalassemia. Most recently, it was evaluated in combination with the best palliative care in the Phase II BEYOND trial, which produced the following results:

• Participants saw a mean increase in hemoglobin of 77.1%
  • This can be compared to 0% in the placebo group
  • These participants experienced this in the 12 weeks that they were instructed to take off from blood transfusions
• Luspatercept-aamt improved quality of life (QOL)
• The drug was well-tolerated

BMS and Merck took these results and used them for an sBLA. However, the FDA asked for more data after this initial submission. Because of this, they will no longer be making their decision on the sBLA in March of 2022. Instead, it will come in three months at the end of June. You can learn more about the FDA’s decision here.

About Beta Thalassemia

Beta thalassemia is a blood disease caused by an issue with hemoglobin, which is a deficiency in this case. This disease occurs as three different types, which are minor, intermedia, and major. These types relate to the severity of the symptoms, with people who have minor beta thalassemia often being asymptomatic and those who have major needing lifelong medical care. This disease is caused by a mutation on the hemoglobin beta gene.

Those who have a mutation on one copy of this gene often experience minor beta thalassemia while those who have mutations on both copies experience the intermedia or major types. The symptoms of this disease include fatigue, weakness, shortness of breath, dizziness, headaches, blood clots, pallor, and splenomegaly, which is an enlargement of the spleen. Treatments for beta thalassemia depend on the type one has. Those with major require blood transfusions, which can be harmful if they cause iron overloads. If the spleen is heavily affected, surgery can be done to correct it. There is also an FDA approved treatment called Thiotepa.