Blueprint Medicines Corporation has just announced that they have submitted a supplemental New Drug Application (NDA) to the FDA. This application is for a therapy called AYVAKIT, which was developed for systemic mastocytosis (SM).
They have requested priority review, which would provide them a response in just six months. Within the next six days, the FDA will determine if the application can move forward in the review process.
SM
SM is caused by a mutation in the KIT D816V gene. It leads to uncontrolled activation of the mast cells in the body. This can cause a wide range of chronic and severe symptoms that are rather unpredictable.
Symptoms may include-
- Anaphylaxis
- Pruritus
- Maculopapular rash
- Brain fog
- Diarrhea
- Bone pain
- Fatigue
Most patients have a less advanced form of the disease which has severe symptoms and can drastically effect quality of life. Those with advanced form are at risk for hematological neoplasm, as well as mast cell leukemia. Individuals with this type of the condition can face organ damage due to the mast cells.
Unfortunately, current treatments often don’t eliminate symptoms for patients. It is clear that new options are needed to eliminate unpredictable onsets of pain and distress which impact day to day life.
AYVAKIT
This therapy is an inhibitor of D816V, which is the main driver of the disease. Researchers are working to develop this therapy as an option for those with both advanced and not advanced presentations of the condition. They hope that by eliminating the main driver of the condition, they could eliminate all symptoms.
This therapy has already been approved for metastatic or unresectable gastrointestinal stromal tumor patients who have certain mutations.
Looking Forward
Researchers are hopeful that this new application will move forward. It is based off data which shows positive response and remission rates for patients. Individuals had increased survival rates and minimal adverse events.
If eventually approved, this would be the very first precision treatment which targets the underlying driver of SM.
You can read more about this investigative therapy here.
