Finally Severe Aplastic Anemia Patients will Have Access to Eltrombopag

Eltrombopag (Promacta) has now been approved for all individuals in the US with severe aplastic anemia. The drug will be accessible to both pediatric and adult patients. This is huge news.

Previously, the drug was only approved for certain populations of SAA patients. Primarily, it was used for those who had an insufficient response to immunosuppressive therapy (IST), the typical treatment for this disease. However, a recent study showed that the vast majority of patients experience the best outcomes when they utilize both IST and eltrombopag.

The Study

The study was completed by Novartis. They examined the effect of eltrombopag on both patients who had been treated with IST and those who had not received the immunosuppressive therapy. For 44% of these patients, complete response was achieved within 6 months. Historically, that rate is 27% higher than those who were solely prescribed IST.

This drug has previously been approved for other patient populations such as those with thrombocytopenia and hepatitis C. But it was only accessible for a very small group of those living with severe aplastic anemia. This new approval could significantly improve patient outcomes.

Additionally, Eltrombopag was granted Breakthrough Therapy designation by the FDA. This designation further indicates the drug’s potential.

Looking Forward

This approval means greater access to better treatment for all SAA patients. Hopefully, prescribing both of these treatments will reduce the amount of patients who face initial unresponsiveness. Ultimately, the goal has always been to get more patients more effective treatments faster.

This is great progress toward that goal for SAA patients.

You can read more about this new approval and the impact it could have on those living with SAA here.


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