A drug created by Agios Pharmaceuticals, TIBSOVO, was recently granted the Breakthrough Therapy designation by the FDA for the treatment of relapsed or refractory myelodysplastic syndrome (MDS) with an IDH1 mutation in adults. This designation means that the development and review of this drug will be expedited, as it presents the possibility to bring substantial improvements to treatment. Agios Pharmaceuticals recently re-opened the part of their study that focuses on MDS in order to give people with this syndrome the best possible treatment.
About Myelodysplastic Syndrome (MDS)
Myelodysplastic syndrome is a group of conditions that are characterized by the prevention of bone marrow cells from creating the amount of red blood cells needed by the body. In those with MDS, these cells never fully grow into healthy and mature cells. If they even leave the bone marrow, they have very short lives. Severity of this disease varies from person to person, but about half of those with MDS see it progress into acute myeloid leukemia. People of any age are susceptible to this syndrome, but it tends to affect those over 60.
There are five types of MDS, which are based on an analysis of the bone marrow. These types are refractory anemia, refractory anemia with sideroblasts, refractory anemia with excess blasts, refractory anemia with excess blasts in transformation, and chronic myelomonocytic leukemia.
There is no known cause of this disease. Some believe that there is a genetic predisposition that can be triggered later in life by external factors like radiation, chemotherapy, or certain chemicals like benzene.
This is a progressive disease, so people may not experience any symptoms in the beginning. As the level of blood cells decrease, people will begin to experience the symptoms of anemia, such as fatigue, heart palpitations, shortness of breath, chest pain, and pale skin. A lack of white blood cells is called neutropenia, and the main symptom of this is an increased risk of an infection. People typically experience these infections in the lungs, sinuses, skin, and urinary tract. A low platelet count may make people bruise and bleed more easily.
About TIBSOVO
TIBSOVO is an isocitrate dehydrogenase-1 (IDH1) inhibitor. It has shown some side effects, with the most severe being symptoms of differentiation syndrome. Other adverse effects include diarrhea, fatigue, rash, leukocytosis, arthralgia, dyspnea, edema, nausea, mucositis, pyrexia, cough, constipation, and electrocardiogram QT prolongation.
About the Study
The re-opening of the arm of the study on MDS is based on the Phase 1 results of 12 patients. They re-opened this part of the study in order to pursue FDA filings and approval. The results of this first phase found that TIBSOVO was well tolerated and led to durable remissions. The median treatment duration was slightly less than one year, at 11.4 months. 75% of patients responded to the treatment, with 42% responding completely. While there were some adverse effects, they did not result in the discontinuation of treatment.
The next phase of this trial is meant to evaluate the safety, activity, tolerability, pharmacokinetics, and pharmacodynamics of TIBSOVO. It will enroll up to 25 patients across 22 sites in the United States and France.
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