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.
NCT02115295 ↗	Cladribine, Idarubicin, Cytarabine, and Venetoclax in Treating Patients With Acute Myeloid Leukemia, High-Risk Myelodysplastic Syndrome, or Blastic Phase Chronic Myeloid Leukemia	Recruiting	M.D. Anderson Cancer Center	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.
NCT02723435 ↗	Midostaurin in Treating Older Patients With Mutated Acute Myeloid Leukemia Post-Transplant	Withdrawn	National Cancer Institute (NCI)	Phase 2	1969-12-31	This phase 2 trial studies the side effects and how well midostaurin works in treating older patients with acute myeloid leukemia with change in genetic material post-hematopoietic cell transplantation. Midostaruin may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Giving midostaruin post-transplant may improve patient outcomes.

Clinical Trial Conditions for Rydapt

Condition Name

Condition Name for Rydapt
Intervention	Trials
Acute Myeloid Leukemia	10
Untreated Adult Acute Myeloid Leukemia	2
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
Syndrome	3

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
Florida	4
California	4
Massachusetts	3
Georgia	3
Washington	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
Jazz Pharmaceuticals	2

Sponsor Type

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

RYDAPT: Clinical Trials, Market Analysis, and Projections

Introduction to RYDAPT

RYDAPT, also known as midostaurin, is a targeted therapy developed by Novartis for the treatment of acute myeloid leukemia (AML) with a specific genetic mutation in the FLT3 gene. Here, we will delve into the clinical trials, market analysis, and future projections for this drug.

Clinical Trials and Approval

RYDAPT was approved by the FDA based on evidence from a single randomized, double-blind, placebo-controlled trial involving 717 patients with newly diagnosed AML and a confirmed FLT3 mutation. This trial, known as the RATIFY trial, was conducted across 177 centers in 13 countries, including those in Europe, North America, and Australia[1].

Trial Design and Outcomes

In the trial, patients were randomized to receive either RYDAPT or a placebo, both in combination with standard chemotherapy. RYDAPT was administered at a dose of 50 mg twice daily on days 8 to 21 of each chemotherapy cycle. The primary endpoint was overall survival (OS), measured from the date of randomization until death by any cause. The results showed that patients who received RYDAPT in combination with chemotherapy had a significant survival benefit compared to those who received chemotherapy alone[1].

Patient Demographics and Safety Profile

The trial included a diverse population, with detailed demographics by sex and race. The safety profile of RYDAPT was evaluated, showing common adverse effects such as increased alanine aminotransferase (ALT), hypernatremia, and hypocalcemia[1].

Market Analysis

Current Market Size and Growth Projections

The global RYDAPT market is anticipated to experience significant growth. As of 2024, the market size is estimated to be around $457.3 million, with projections indicating it will reach $1463.8 million by 2030, at a Compound Annual Growth Rate (CAGR) of 21.4%[2].

Regional Market Share

North America is currently the largest market for RYDAPT, followed by Europe, which holds a substantial share of around 24% of the sales market[2].

Application and Type Segmentation

The RYDAPT market is segmented by application, primarily in hospitals and pharmacies, and by type, including AML, acute myeloid leukemia with myelodysplasia-related changes (AML-MRC), systemic mastocytosis (SM), and other related conditions[2].

Market Drivers and Challenges

First-to-Market Advantage

RYDAPT's first-to-market advantage has allowed it to be rapidly integrated into the treatment paradigm for FLT3+ AML. However, this advantage is expected to be challenged by the emergence of more specific and effective FLT3 tyrosine kinase inhibitors (TKIs)[3].

Label Expansion and Competitive Landscape

Novartis is exploring label expansion for RYDAPT to include wild-type (WT) AML patients, which could significantly boost its market share by expanding the target population. This strategy leverages RYDAPT's weak affinity for FLT3, allowing it to be used in a broader patient population compared to more specific FLT3 TKIs[3].

Future Revenue Growth

Potential in WT AML

The potential approval of RYDAPT for WT AML could free it from competition with other FLT3 TKIs, significantly increasing its revenue. This expansion would more than double the existing patient population, providing a substantial growth opportunity[3].

Ongoing and Future Clinical Trials

In addition to its current indications, RYDAPT is being evaluated in various ongoing trials, including a Phase I trial assessing an immunopeptidome-defined multi-peptide vaccine against AML stem and progenitor cells. This trial aims to assess immunogenicity, safety, and preliminary clinical efficacy, with the goal of preventing relapse through T cell-mediated elimination of minimal residual disease (MRD)[4].

Key Takeaways

• Clinical Trials: RYDAPT was approved based on a randomized, double-blind, placebo-controlled trial showing significant overall survival benefits in patients with FLT3-mutated AML.
• Market Growth: The global RYDAPT market is projected to grow from $457.3 million in 2024 to $1463.8 million by 2030 at a CAGR of 21.4%.
• Regional Share: North America and Europe are the leading regions, with Europe holding around 24% of the sales market share.
• Label Expansion: Potential label expansion to WT AML could significantly boost RYDAPT's market share and revenue.
• Ongoing Trials: RYDAPT is being evaluated in various ongoing trials, including those focused on immunotherapy and prevention of relapse.

FAQs

What is RYDAPT used for?

RYDAPT is used to treat adults with acute myeloid leukemia (AML) that has a mutation in the FLT3 gene, in combination with chemotherapy[1].

How is RYDAPT administered?

RYDAPT is administered as a capsule, with two capsules (total of 50 mg) taken twice a day with food[1].

What are the common side effects of RYDAPT?

Common side effects include increased ALT, hypernatremia, and hypocalcemia[1].

What is the projected market size of RYDAPT by 2030?

The global RYDAPT market is projected to reach $1463.8 million by 2030[2].

Is RYDAPT being explored for other indications?

Yes, RYDAPT is being explored for label expansion to include wild-type (WT) AML and is part of ongoing trials evaluating its use in combination with immunotherapy[3][4].

Sources

1. FDA: Drug Trials Snapshots: RYDAPT - FDA
2. Valuates Reports: Global Rydapt Market Insights, Forecast to 2030
3. Clinical Trials Arena: The Driving Force behind Future Revenue Growth of Novartis' Rydapt
4. LARVOL Sigma: Rydapt (midostaurin) News
5. Cognitive Market Research: Rydapt Market Report 2024 (Global Edition)

Last updated: 2024-12-31