This section highlights FDA-related milestones and regulatory updates for drugs developed by Blueprint Medicines (BPMC).
Over the past two years, Blueprint Medicines has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
Avapritinib and AYVAKIT. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
Avapritinib - FDA Regulatory Timeline and Events
Avapritinib is a drug developed by Blueprint Medicines for the following indication: For Indolent Systemic Mastocytosis.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- Avapritinib
- Announced Date:
- June 12, 2025
- Indication:
- For Indolent Systemic Mastocytosis
Announcement
Blueprint Medicines announced data presentations reflecting over a decade of collaboration with clinical experts and patient advocates to transform the treatment of systemic mastocytosis (SM).
AI Summary
Blueprint Medicines announced new data presentations showcasing more than a decade of teamwork with clinical experts and patient advocates to transform the treatment of systemic mastocytosis (SM). The presentations emphasize that AYVAKIT®/AYVAKYT® (avapritinib) remains a durable standard of care for both indolent and advanced forms of SM.
The collected evidence comes from large, long-term trials and real-world studies, highlighting significant improvements in symptom control and long-term survival. These studies demonstrate that by tackling the root cause of SM, treatment with AYVAKIT/AYVAKYT can reduce the real-world burden of the disease while enhancing patients’ overall quality of life. This decade-long collaboration underlines Blueprint Medicines’ commitment to innovation and improving outcomes for those living with SM.
Read Announcement- Drug:
- Avapritinib
- Announced Date:
- May 1, 2025
- Indication:
- For Indolent Systemic Mastocytosis
Announcement
Blueprint Medicines Corporation provided corporate updates.
AI Summary
Blueprint Medicines Corporation provided a corporate update highlighting their recent strong performance in the first quarter of 2025. The company reported 61% year-over-year growth, achieving $149.4 million in AYVAKIT net product revenues. Along with this solid performance, they raised their full-year AYVAKIT revenue guidance to between $700 million and $720 million, building on a commitment to drive further commercial growth.
In addition, the update detailed progress in their research and development efforts. The company has initiated proof-of-concept studies for BLU-808, an investigational drug targeting allergic rhinoconjunctivitis and chronic urticaria. Blueprint Medicines emphasized that their strong revenue growth, sound cash position, and disciplined capital allocation will support further innovation and help reach their goal of $2 billion in AYVAKIT revenue by 2030.
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AYVAKIT (avapritinib) PIONEER - FDA Regulatory Timeline and Events
AYVAKIT (avapritinib) PIONEER is a drug developed by Blueprint Medicines for the following indication: Non-advanced systemic mastocytosis.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- AYVAKIT (avapritinib) PIONEER
- Announced Date:
- May 30, 2024
- Indication:
- Non-advanced systemic mastocytosis
Announcement
Blueprint Medicines Corporation announced multiple upcoming datasets across two key conferences that reinforce the significant real-world burden of systemic mastocytosis (SM), and highlight the durable clinical outcomes of AYVAKIT®/AYVAKYT® (avapritinib) across the spectrum of the disease
AI Summary
Blueprint Medicines Corporation announced that it will present multiple datasets at two major conferences. The new data emphasize the heavy real-world burden of systemic mastocytosis (SM) and highlight how AYVAKIT®/AYVAKYT® (avapritinib) offers durable clinical benefits for patients across the disease spectrum. The upcoming presentations will share results from studies that report long-term benefits and a favorable safety profile of AYVAKIT in both indolent and advanced forms of SM. The data, gathered over follow-ups of two to three years, show that the treatment not only improves symptoms and quality of life but also offers sustained efficacy for patients. This reinforces Blueprint’s goal of establishing AYVAKIT as a global standard of care, underscoring the urgent need for early diagnosis and effective treatment of SM.
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