CONTENTS:
- A New Drug Application Has Been Submitted for FDA Review for a Pediatric ITP Therapy
- Video: Can I have surgery or dental work?
A New Drug Application Has Been Submitted for FDA Review for a Pediatric ITP Therapy
Avatrombopag, more commonly referred to as Doptelet, is an ITP treatment that belongs to a class of drugs known as thrombopoetin-receptor agonists (TPO-RAs), that stimulate the bone marrow to produce more platelets in the bloodstream. Other available TPO-RAs used to treat ITP include Eltrombopag (Promacta/Revolade) and Romiplostim (Nplate). To date, Doptelet is only available for adults with ITP who have failed to respond to other standard therapies. Clinical trials have been on-going to assess the effect of the drug in children, and results have shown favorable results.
On December 12, 2024, it was announced that the Food and Drug Administration (FDA) accepted two supplemental new drug applications for a pediatric ITP treatment:
- Doptelet (oral pill)
- Doptelet (oral suspension). This refers to a capsule where its granules can be sprinkled onto a soft food or liquid prior to ingestion.
This means the FDA has agreed to perform an extensive review of Doptelet (in either form) to determine if it is indeed safe and effective in individuals living in the US between the ages of 1-18 years of age who have had ITP for at least three months and have had an unsuccessful response to prior standard therapies. The FDA has suggested a Prescription Drug User Fee Act (PDUFA) target action date of July 24, 2025, which refers to the date they anticipate their review will be completed and shared with the public. This would be a priority review. If approved, this will mean that children and adolescents with ITP will have one more treatment available to them to manage their condition and improve bleeding risks and their overall quality of life.
Video: Can I have surgery or dental work?
