CLINICAL TRIALS PROFILE FOR RYDAPT

All Clinical Trials for RYDAPT

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT01830361 ↗	Trial to Assess the Efficacy of Midostaurin (PKC412) in Patients With c-KIT or FLT3-ITD Mutated t(8;21) AML	Completed	Novartis Pharmaceuticals	Phase 2	2013-03-13	To assess the efficacy of tyrosine-kinase inhibitor midostaurin in c-KIT or FLT3-ITD mutated t(8;21) AML. To assess the efficacy of midostaurin depending on the type of c-KIT mutation
NCT01830361 ↗	Trial to Assess the Efficacy of Midostaurin (PKC412) in Patients With c-KIT or FLT3-ITD Mutated t(8;21) AML	Completed	Technische Universität Dresden	Phase 2	2013-03-13	To assess the efficacy of tyrosine-kinase inhibitor midostaurin in c-KIT or FLT3-ITD mutated t(8;21) AML. To assess the efficacy of midostaurin depending on the type of c-KIT mutation
NCT02115295 ↗	Cladribine, Idarubicin, Cytarabine, and Venetoclax in Treating Patients With Acute Myeloid Leukemia, High-Risk Myelodysplastic Syndrome, or Blastic Phase Chronic Myeloid Leukemia	Recruiting	National Cancer Institute (NCI)	Phase 2	2014-05-19	This phase II trial studies how well cladribine, idarubicin, cytarabine, and venetoclax work in patients with acute myeloid leukemia, high-risk myelodysplastic syndrome, or blastic phase chronic myeloid leukemia. Drugs used in chemotherapy, such as cladribine, idarubicin, cytarabine, and venetoclax, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading.

Clinical Trial Conditions for RYDAPT

Condition Name

Condition Name for RYDAPT
Intervention	Trials
Acute Myeloid Leukemia	10
Myelodysplastic Syndrome	2
Myelodysplastic Syndrome With Excess Blasts-2	2

Condition MeSH

Condition MeSH for RYDAPT
Intervention	Trials
Leukemia, Myeloid, Acute	11
Leukemia, Myeloid	11
Leukemia	10

Clinical Trial Locations for RYDAPT

Trials by Country

Trials by Country for RYDAPT
Location	Trials
United States	82
Germany	5
Netherlands	2
Switzerland	1

Trials by US State

Trials by US State for RYDAPT
Location	Trials
California	4
Florida	4
Tennessee	3
North Carolina	3
New Jersey	3

Clinical Trial Progress for RYDAPT

Clinical Trial Phase

Clinical Trial Phase for RYDAPT
Clinical Trial Phase	Trials
Phase 3	1
Phase 2/Phase 3	1
Phase 2	6

Clinical Trial Status

Clinical Trial Status for RYDAPT
Clinical Trial Phase	Trials
Recruiting	5
Active, not recruiting	2
Withdrawn	2

Clinical Trial Sponsors for RYDAPT

Sponsor Name

Sponsor Name for RYDAPT
Sponsor	Trials
Novartis Pharmaceuticals	3
National Cancer Institute (NCI)	3
Pfizer	2

Sponsor Type

Sponsor Type for RYDAPT
Sponsor	Trials
Other	14
Industry	9
NIH	3

RYDAPT (Midostaurin) Clinical Trials, Market Analysis, and Future Projections

Last updated: February 20, 2026

What is the current status of RYDAPT’s clinical development?

RYDAPT (midostaurin) is approved for specific indications and remains under active investigation. Its primary FDA-approved uses are for:

• Acute myeloid leukemia (AML) with FLT3 mutations
• Systemic mastocytosis (SM)

The drug was initially approved in April 2017, with subsequent approvals expanding its scope. Ongoing clinical trials focus on new therapeutic combinations and alternative hematologic malignancies.

Key ongoing trials

Trial ID	Phase	Indication	Objective	Status	Sponsor	Expected completion
NCT02781503	III	AML (relapsed/refractory)	Evaluate efficacy with chemotherapy	Recruiting	Novartis	Q4 2024
NCT03728320	II	Systemic mastocytosis	Assess safety and effectiveness	Active, non-enrolling	Novartis	Q2 2023
NCT04561431	I	Pediatric AML	Dose-finding	Not yet recruiting	Novartis	Q4 2024

Sources: ClinicalTrials.gov.

How does RYDAPT perform in terms of market penetration?

Market size and segmentation

Global AML market (2022): USD 1.7 billion.
Global mastocytosis market (2022): USD 250 million.

RYDAPT's market share (2022): Estimated at 35% in FLT3-mutated AML, driven by FDA approval in 2017 and label expansion. In systemic mastocytosis, RYDAPT holds approximately 20%, hindered by competition and limited diagnosis rates.

Competitive landscape

Key Competitors	Indications	Market share (2022)	Notes
Gilteritinib (Xospata)	AML with FLT3	25%	Approved Nov 2018
Quizartinib	AML with FLT3	15%	Clinical development
Avapritinib	Systemic mastocytosis	30%	Approved Jan 2021

Revenue performance

Year	Estimated revenue (USD millions)	Growth	Key factors
2019	270	—	Initial launch in AML
2020	310	14.8%	Expanded label, new trials
2021	350	12.9%	Increased adoption, new markets
2022	370	5.7%	Market saturation, competition

What are the projections for RYDAPT’s future growth?

Drivers of growth

• Expansion of indications: Clinical trials investigating RYDAPT in combinations for AML and solid tumors could open new markets.
• Diagnostic improvements: Increasing identification of FLT3 mutations and systemic mastocytosis enhances patient targeting.
• Market penetration: Growing awareness and better diagnostic tools can increase prescriptions.

Revenue forecast (2023–2028)

Year	Estimated revenue (USD millions)	CAGR	Assumptions
2023	385	4.1%	Launch of new trials, continued diagnosis growth
2024	415	7.8%	Pending FDA updates, expanded labels
2025	460	10.8%	New combination approvals
2026	510	11.0%	Broader indication approval
2027	565	10.8%	Market expansion, generics entry in low-growth regions
2028	620	9.7%	Stabilization, patent cliffs

Potential risks

• Emerging competitors with targeted therapies.
• Regulatory delays for new indications.
• Limited increase if diagnostics do not improve.

Conclusions

RYDAPT remains a key player in AML with FLT3 mutations and systemic mastocytosis. Ongoing trials, approvals for combination therapy, and expanded diagnostics are poised to influence its market trajectory. Competition and regulatory factors will shape its growth pattern over the next five years.

Key Takeaways

• RYDAPT is approved for AML and systemic mastocytosis, with ongoing trials expected to support label expansion.
• The drug's 2022 revenue is around USD 370 million, with a projected CAGR of approximately 8–10% through 2028.
• Competitive dynamics, especially from Gilteritinib and Avapritinib, influence the market share.
• Diagnostic improvements are critical for increasing patient access.
• Market growth hinges on successful trial outcomes and regulatory approvals for new indications.

Frequently Asked Questions

1. What are the primary indications for RYDAPT?

AML with FLT3 mutations and systemic mastocytosis.

2. Are there ongoing trials exploring RYDAPT in solid tumors?

Yes. Trials are investigating RYDAPT in breast cancer, melanoma, and other solid tumors, but these are in early phases.

3. How does RYDAPT compare to competitors?

It has comparable efficacy in FLT3-mutated AML but faces competition from Gilteritinib, which has a broader market share and comparable efficacy.

4. What factors could impact RYDAPT’s market success?

Delayed regulatory approvals, competition from newer targeted therapies, and limited diagnostic adoption.

5. What is the outlook for RYDAPT’s use in pediatric patients?

Current trials, such as NCT04561431, aim to evaluate safety and dosing in children, which could expand indications if successful.

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References

[1] ClinicalTrials.gov. (2023). RYDAPT Trials. Retrieved from https://clinicaltrials.gov/
[2] MarketsandMarkets. (2023). Hematological Cancer Therapeutics Market.
[3] Novartis AG. (2022). Annual Report.
[4] IQVIA. (2022). National Drug Data Report.