This week the FDA approved a novel treatment for sickle cell disease, a serious and sometimes deadly disease that affects around 100,000 Americans. The treatment was found to exceptionally improve the health and quality of life in the “end of life” patients involved in the trials, and experts believe that upon release, it may do much to improve the lives of sickle cell patients throughout the world.
Oxbryta is an inhibitor of deoxygenated sickle hemoglobin polymerization and was granted Accelerated Approval. This classification allows the FDA to quickly approve drugs for serious conditions. The drug’s trial consisted of 274 patients with sickle cell disease who received different doses of the medication or a placebo, a press release explained.
Results showed that the medication was able to increase the hemoglobin response rate in patients far more than those in the placebo group. This is important as sickle cell affects the hemoglobin, a type of red blood cell, and leads to some of the disease’s devastating effects.
Sickle cell anemia is an inherited blood disease where the normally round and flexible blood cells become rigid and take on a sickle cell shape. As a result, they cannot flow through the blood vessels as well and are unable to effectively carry oxygen throughout the body. The disease can cause pain, delayed growth, infections, swelling and even some vision problems.
9/09/05-SICKLE-Isaiah Adeshigbin is a four-year-old boy has sickle cell disease, an inherited blood ... [+]
In an interview, Dr. Lanetta Bronte-Hall, the president for The Foundation for Sickle Cell Disease Research explained just how fundamentally important this recent approval will be for the Sickle Cell community. The foundation had 5 patients enrolled in the trial as part of a process called compassionate use. These patients were essentially at the end of their life and had severe forms of the disease. Because of this, the FDA allowed the medication to be used in these patients before proper efficacy and safety issues had been addressed. A patient affectionately referred to as ‘Mr.C’ has been on the drug for 5 years, and was the first patient on the drug in the US.
According to Bronte-Hall, the drug allows for greater oxygen delivery and indirectly increases the hemoglobin. As a result, patients not only reported having more energy but also signs of increased blood flow such as increased color in the face and physical warmth upon touch.
“Their experiences were just truly amazing,” explained Bronte-Hall, referring to the patients who were a part of the drug trial. “The quality of life has truly been amazing. They have had a 40 percent reduction in ER visits and hospitalization. They gave reports about going to the beach, one patient washed 5 loads of clothes. It really is going to be a medication that will greatly enhance the lives of patients. “
As reported by the FDA press release, common side effects reported in patients included headaches, diarrhea, abdominal pain, nausea, fatigue, and fever.
