The FDA has approved a new therapy for chronic immune thrombocytopenia (ITP). This treatment is an oral therapy to be taken with food, created by Dova Pharmaceuticals. It is called avatrombopag (Doptelet).
Avatrombopag is a thrombopoietin receptor agonist which works to increase the platelets in the body. It has been approved for adult, insufficiently treated, ITP patients. This is the treatment’s second approved indication. Previously (May of 2018), it had been approved for thrombocytopenia patients who had chronic liver disease and were scheduled for a dental procedure or other medical procedure. This expanded approval will give ITP patients more options.
The expanded indication came primarily as result of two Phase 2 investigations, and a few Phase 3 studies. In total, over 1,000 patients were observed across 24 different studies. These investigations supported the treatments tolerability and safety for ITP patients.
The most influential of these studies was a Phase 3 investigation. This study lasted six months, however after just 8 days a dramatic improvement was documented. The majority of patients who had been treated with Avatrombopag had at least a 50,000 per µL improvement in platelet numbers. These results were sustained over the six month period, showing significant benefit compared to placebo.
This therapy should be launched on the market for ITP patients by the middle of this month (July 2019).
A New Choice
The Platelet Disorder Support Association is an organization dedicated to patient advocacy for individuals living with ITP. Upon hearing about this newly approved treatment, the association put out a message encouraging patients to discuss their new treatment options with their physician.
For different individuals, different treatments may better complement their goals and their lifestyle. By having a conversation with their provider prior to the new therapy hitting the market, ITP patients will be more prepared to make a decision when the treatment becomes available to them.
For many patients with rare diseases such as ITP, there is no approved therapy. For those who do have an approved treatment option, there is likely only one. It is uncommon to have a choice of treatments for rare conditions. For a long time, this has been the case for thrombocytopenia patients.
Choice is described as a luxury in the medical field, and thankfully, adult ITP patients now have options.
“Every new treatment provides more choices and new hope to our community.”
You can read more about this newly approved therapy here.