Transcript
Onscreen text:
PATIENT STORIES
ANDREW
For US Healthcare Professionals Only
Real patient and spouse used with consent.
[AYVAKIT 25 mg logo]
AYVAKIT® (avapritinib) is indicated for the treatment of adult patients with indolent systemic mastocytosis (ISM).
Limitations of Use: AYVAKIT is not recommended for the treatment of patients with ISM with platelet counts of <50 x 109/L.
Please see additional Important Safety Information near the end of this video. Visit AYVAKIT.com to see the full Prescribing Information for AYVAKIT.
Andrew:
My name is Andrew. I was born and raised in Salem, Oregon. I've lived here all my life.
Onscreen text:
Andrew was compensated by Blueprint Medicines.
Andrew:
I am married to Shannon. We’ve been married for almost 24 years now. We have 3 kids. Being outside, being in nature, for me, is rejuvenating. It’s quieting, even the arduous parts of it.
Andrew:
I was diagnosed with indolent systemic mastocytosis in 2017.
Onscreen text:
Andrew, Salem, OR
Diagnosed with indolent systemic mastocytosis (ISM) in 2017
Andrew does not provide medical advice.
Andrew was paid by Blueprint Medicines.
Andrew:
I think, like probably a lot of people with ISM, I didn’t quite realize that I had this disease. In fact, I had no idea. I just knew that my eyes were watery and itchy. I started to notice uncomfortable, sort of itchiness in my skin joint pain, spots on my body, GI issues, and fatigue setting in, uh, more than it had in the past. So, I was at a routine skin check with my dermatologist. He did a biopsy… on one of the marks on my skin which, uh, he was suspicious about. And he explained that, uh, the biopsy had come back positive for this thing called mastocytosis. I had never heard of it before. So we found a specialist in Boston who was an immunologist.
Onscreen text:
“I had never heard of it before.”
Andrew:
Before I flew out, the doctor asked that I have a test for a KIT mutation, which came back positive. I also had a bone marrow biopsy. And she was able to diagnose me with indolent systemic mastocytosis. She recommended that I connect with a local hematology oncologist, uh, here in Oregon.
Andrew:
It was a pretty frightening, uh, moment for me.
Onscreen text:
“It was a pretty frightening moment for me”
Andrew:
It was scary.
So that’s when we started exploring, you know, treating all these different, disparate symptoms. Treat this symptom and that symptom and this symptom. Um, was sort of the best we could do at that point. It certainly made, uh, spontaneity a problem in terms of family activities and being different places.
My family and I were on a five-day rafting trip. And on the second night there I had a very strong reaction. I remember getting up and taking a few steps and uh, then collapsing. It was hard to breathe. Uh, my breath was very shallow.
Fortunately, my friend who was there with me, um, had some epinephrine. That seemed to bring me back and I sort of recovered.
If anything, that really focused me on…if there was any sort of treatment for this disease. If there’s any way to get my hands around what’s going on with my body and this ISM diagnosis.
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“That really focused me on…if there was any sort of treatment for this disease."
Andrew:
And then when I sat in my hematologist’s exam room... We talked together about this drug called AYVAKIT.
Onscreen text:
“We talked together about this drug called AYVAKIT.”
Andrew:
Seems to be the only drug that treats, you know, the disease that I have, ISM.
Onscreen text:
AYVAKIT is the first and only FDA-approved drug for adults with ISM. AYVAKIT is not recommended in people with low platelet counts (less than 50 x 109/L).
Andrew:
And we had a long talk about it. He took some time to research it himself. We decided it would be worth it for me to give it a shot.
Andrew:
I started taking AYVAKIT in November of 2023. I wanted something that targeted the source of my ISM, in addition to supportive care.
Onscreen text:
“I wanted something that targeted the source of my ISM…”
AYVAKIT® (avapritinib) targets KIT D816V, the underlying driver of disease in ~95% of patients.
Andrew:
Since starting AYVAKIT, I've noticed changes in my ISM symptoms.
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In a clinical trial, patients receiving AYVAKIT had greater symptom reduction vs placebo at 24 weeks. Both groups continued best supportive care.
Symptoms evaluated included brain fog, headache, dizziness, spots, itching, flushing, abdominal pain, nausea, bone pain and fatigue.
Andrew:
The solution’s going to be different for everyone, but this has been a great option for me.
Onscreen text:
Treat the source of ISM
Images and voices of patient and spouse used with consent.
Indication and Important Safety Information (Voice-over):
INDICATION
AYVAKIT® (avapritinib) is indicated for the treatment of adult patients with indolent systemic mastocytosis (ISM).
Limitations of Use: AYVAKIT is not recommended for the treatment of patients with ISM with platelet counts of less than 50,000 per microliter.
IMPORTANT SAFETY INFORMATION
Cognitive adverse reactions can occur in patients receiving AYVAKIT and occurred in 7.8% of patients with ISM who received AYVAKIT plus best supportive care (BSC) versus 7% of patients who received placebo plus BSC; less than 1% were Grade 3. Depending on the severity, withhold AYVAKIT and then resume at the same dose, or permanently discontinue AYVAKIT.
AYVAKIT may cause photosensitivity reactions. In all patients treated with AYVAKIT in clinical trials (n=1049), photosensitivity reactions occurred in 2.5% of patients. Advise patients to limit direct ultraviolet exposure during treatment with AYVAKIT and for one week after discontinuation of treatment.
AYVAKIT can cause fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to a fetus. Advise females and males of reproductive potential to use an effective contraception during treatment with AYVAKIT and for 6 weeks after the final dose. Advise women not to breastfeed during treatment with AYVAKIT and for 2 weeks following the final dose.
The most common adverse reactions (greater than or equal to 10%) in patients with ISM were eye edema, dizziness, peripheral edema, and flushing.
Avoid coadministration of AYVAKIT with strong or moderate CYP3A inhibitors or inducers. If contraception requires estrogen, limit ethinyl estradiol to less than or equal to 20 micrograms unless a higher dose is necessary.
To report suspected adverse reactions, contact Blueprint Medicines Corporation at 1-888-258-7768 or the FDA at 1-800-FDA-1088 or visit www.fda.gov/medwatch.
Please click here to see the full Prescribing Information for AYVAKIT.
07/2025 USAVA25.0118.1