CLINICAL TRIALS PROFILE FOR CRESEMBA

All Clinical Trials for CRESEMBA

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT03816176 ↗	A Study to Evaluate Isavuconazonium Sulfate for the Treatment of Invasive Aspergillosis (IA) or Invasive Mucormycosis (IM) in Pediatric Participants	Recruiting	Astellas Pharma Global Development, Inc.	Phase 2	2019-08-22	The purpose of this study is to evaluate the safety, tolerability, and efficacy of isavuconazonium sulfate in pediatric participants.
NCT04096157 ↗	A Study to Assess Isavuconazole Following a Single Dose of Isavuconazonium Sulfate Intravenous Solution Via Nasogastric (NG) Tube Compared to a Single Dose of Oral Capsules Under Fasting Conditions in Healthy Participants	Completed	Astellas Pharma Global Development, Inc.	Phase 1	2019-09-24	The purpose of this study is to evaluate the bioequivalence of isavuconazole following a single dose of isavuconazonium sulfate intravenous (IV) solution via nasogastric (NG) tube (test formulation) compared to a single dose of isavuconazonium sulfate capsules for oral administration (i.e., oral capsules administered to nonintubated participants)(reference formulation). In addition, this study will evaluate the safety and tolerability of isavuconazole and the general pharmacokinetic (PK) parameters of isavuconazole when administered as a single dose of isavuconazonium sulfate IV solution via NG tube (test formulation) and a single dose of isavuconazonium sulfate capsules for oral administration (i.e., oral capsules administered to nonintubated participants) (reference formulation) under fasting conditions in healthy male and female participants.
NCT04548648 ↗	LCCC1841: A Phase 2 Trial of Acalabrutinib in Relapsed/Refractory Primary and Secondary CNS Lymphomas	Recruiting	AstraZeneca	Phase 2	2020-10-15	The purpose of this study is to see if acalabrutinib is effective in treating a type of cancer call central nervous system (CNS) lymphoma. Acalabrutinib has not been approved by the Food and Drug Administration (FDA) for the treatment of CNS lymphoma. However, FDA has approved its use for treatment of another type of lymphoma called mantle cell lymphoma. Currently, there are no standard FDA approved treatments for treatment of CNS lymphoma. Acalabrutinib acts similar to another cancer drug called ibrutinib. Ibrutinib was tested in several research trials for management of CNS lymphomas, and the results were promising. Acalabrutinib and ibrutinib attack a similar target found in CNC lymphoma. Research studies show that acalabrutinib does a better job in attacking this target than ibrutinib, and this might be beneficial for using this drug in treating CNS lymphoma. The purpose of this study is test whether giving acalabrutinib is safe and could help controlling with CNS lymphoma. The study doctors will be looking to see if acalabrutinib can shrink the cancer. In this research study, participants will be given acalabrutinib and isavuconazol, because it helps in preventing fungal infections. Fungal infection is a common side effect of acalabrutinib. Treatment with acalabrutinib and isavuconazole will continue unless the cancer progresses or participants experience bad side effects.
NCT04548648 ↗	LCCC1841: A Phase 2 Trial of Acalabrutinib in Relapsed/Refractory Primary and Secondary CNS Lymphomas	Recruiting	UNC Lineberger Comprehensive Cancer Center	Phase 2	2020-10-15	The purpose of this study is to see if acalabrutinib is effective in treating a type of cancer call central nervous system (CNS) lymphoma. Acalabrutinib has not been approved by the Food and Drug Administration (FDA) for the treatment of CNS lymphoma. However, FDA has approved its use for treatment of another type of lymphoma called mantle cell lymphoma. Currently, there are no standard FDA approved treatments for treatment of CNS lymphoma. Acalabrutinib acts similar to another cancer drug called ibrutinib. Ibrutinib was tested in several research trials for management of CNS lymphomas, and the results were promising. Acalabrutinib and ibrutinib attack a similar target found in CNC lymphoma. Research studies show that acalabrutinib does a better job in attacking this target than ibrutinib, and this might be beneficial for using this drug in treating CNS lymphoma. The purpose of this study is test whether giving acalabrutinib is safe and could help controlling with CNS lymphoma. The study doctors will be looking to see if acalabrutinib can shrink the cancer. In this research study, participants will be given acalabrutinib and isavuconazol, because it helps in preventing fungal infections. Fungal infection is a common side effect of acalabrutinib. Treatment with acalabrutinib and isavuconazole will continue unless the cancer progresses or participants experience bad side effects.

Clinical Trial Conditions for CRESEMBA

Condition Name

Condition Name for CRESEMBA
Intervention	Trials
Aspergillosis Invasive	1
Healthy Subjects	1
Invasive Aspergillosis	1
Invasive Fungal Disease	1

Condition MeSH

Condition MeSH for CRESEMBA
Intervention	Trials
Aspergillosis	2
COVID-19	1
Coronavirus Infections	1
Lymphoma	1

Clinical Trial Locations for CRESEMBA

Trials by Country

Trials by Country for CRESEMBA
Location	Trials
United States	15
United Kingdom	2
Germany	2
Belgium	1
Spain	1

Trials by US State

Trials by US State for CRESEMBA
Location	Trials
California	2
North Carolina	2
Missouri	1
Michigan	1
Louisiana	1

Clinical Trial Progress for CRESEMBA

Clinical Trial Phase

Clinical Trial Phase for CRESEMBA
Clinical Trial Phase	Trials
Phase 4	1
Phase 3	1
Phase 2	2

Clinical Trial Status

Clinical Trial Status for CRESEMBA
Clinical Trial Phase	Trials
Recruiting	3
Completed	1
Not yet recruiting	1

Clinical Trial Sponsors for CRESEMBA

Sponsor Name

Sponsor Name for CRESEMBA
Sponsor	Trials
Astellas Pharma Global Development, Inc.	3
UNC Lineberger Comprehensive Cancer Center	1
Jeffrey Jenks, MD, MPH	1

Sponsor Type

Sponsor Type for CRESEMBA
Sponsor	Trials
Industry	5
Other	2

Cresemba: A Comprehensive Update on Clinical Trials, Market Analysis, and Projections

Introduction to Cresemba

Cresemba, also known as isavuconazonium sulfate, is a potent azole antifungal medication developed by Basilea Pharmaceutica and marketed by Astellas Pharma. It is used to treat invasive aspergillosis (IA) and invasive mucormycosis (IM), two severe and potentially life-threatening fungal infections.

Clinical Trials and Efficacy

Trial 2 (VITAL Study)

The VITAL study, a Phase 3 open-label, noncomparative trial, evaluated the safety and efficacy of Cresemba in patients with invasive fungal diseases, including those with renal impairment and those affected by rare molds, yeasts, or dimorphic fungi. Specifically, 37 patients with proven or probable invasive mucormycosis were assessed, showing promising results in treating this rare and dangerous infection[1].

Pediatric Clinical Studies

Two pediatric clinical studies, including a Phase 2 open-label, non-comparative, multicenter study (NCT03816176), were conducted to evaluate the safety, efficacy, and pharmacokinetics of Cresemba in children aged 1 to 17 years with IA or IM. The study involved 31 patients and demonstrated a successful response rate of 54.8% at the end of treatment. The all-cause case fatality rates were 6.5% through Day 42 and 9.7% through Day 84, with none considered treatment-related[3][4].

FDA Approval and Market Exclusivity

Recent FDA Approval

On December 8, 2023, the FDA approved Cresemba for the treatment of IA and IM in patients aged one or older. This approval includes both an injectable formulation for patients aged one or older and a capsule formulation for patients aged six or older, weighing at least 16 kg[2][3][4].

Orphan Drug Status and Market Exclusivity

Following the FDA approval, Cresemba was granted orphan drug exclusivity, providing seven years of market exclusivity in the United States for the treatment of IA and IM. Additionally, pediatric market exclusivity extends this protection by an additional six months[2].

Market Analysis

Global Sales Performance

Cresemba has shown strong sales performance globally, with total in-market sales amounting to USD 505 million in the 12 months between July 2023 and June 2024. This represents a 20% year-on-year growth, highlighting the significant medical need and market demand for this antifungal medication[5].

Regional Performance

In the Asia Pacific region and China, Cresemba's sales have been particularly robust, triggering multiple milestone payments to Basilea Pharmaceutica. This region, along with Europe and other countries, contributes to Cresemba being approved and marketed in over 70 countries worldwide[5].

Market Projections

Forecasted Revenue

GlobalData forecasts predict that Cresemba will generate substantial revenue, with an estimated $450 million in 2023 alone. Given the drug's strong sales performance and expanding market reach, these projections are likely to be met or exceeded[2].

Growth Momentum

The continued strong growth momentum of Cresemba is driven by its efficacy in treating life-threatening invasive mold infections. As more patients and healthcare providers become aware of its benefits, the market is expected to continue growing, especially in regions with high medical needs[5].

Safety and Adverse Events

Clinical Study Findings

In the pediatric clinical studies, treatment-emergent adverse events (TEAEs) occurred in 93.5% of patients, with 29.0% experiencing drug-related TEAEs. Serious TEAEs were reported in 58.1% of patients, but only one was assessed as drug-related by the investigator. These findings indicate that while Cresemba is generally well-tolerated, careful monitoring is necessary[4].

Conclusion

Cresemba has emerged as a critical treatment option for invasive aspergillosis and invasive mucormycosis, backed by robust clinical trial data and recent FDA approvals. Its strong market performance and projected growth underscore its importance in addressing severe fungal infections globally.

Key Takeaways

• Clinical Efficacy: Cresemba has shown promising results in treating IA and IM in both adult and pediatric populations.
• FDA Approval: Recent approval includes both injectable and capsule formulations for patients aged one or older.
• Market Exclusivity: Seven years of orphan drug exclusivity plus an additional six months of pediatric exclusivity.
• Global Sales: Total in-market sales of USD 505 million between July 2023 and June 2024, with a 20% year-on-year growth.
• Market Projections: Forecasted revenue of $450 million in 2023, with continued growth momentum expected.
• Safety Profile: Generally well-tolerated, but with a notable incidence of adverse events requiring careful monitoring.

FAQs

What is Cresemba used for?

Cresemba is used to treat invasive aspergillosis (IA) and invasive mucormycosis (IM), two severe and potentially life-threatening fungal infections.

What is the recent FDA approval for Cresemba?

The FDA approved Cresemba on December 8, 2023, for the treatment of IA and IM in patients aged one or older, including both injectable and capsule formulations.

What market exclusivity does Cresemba have?

Cresemba has seven years of orphan drug exclusivity and an additional six months of pediatric market exclusivity in the United States.

How has Cresemba performed in the market?

Cresemba has shown strong sales performance globally, with total in-market sales of USD 505 million between July 2023 and June 2024, representing a 20% year-on-year growth.

What are the safety concerns associated with Cresemba?

While generally well-tolerated, Cresemba has a notable incidence of treatment-emergent adverse events, with 93.5% of patients experiencing TEAEs and 58.1% experiencing serious TEAEs.

What are the projections for Cresemba's future market performance?

GlobalData forecasts predict that Cresemba will continue to grow, driven by its efficacy and expanding market reach, with an estimated $450 million in revenue for 2023.

Sources

1. CRESEMBA® (isavuconazonium sulfate) - Invasive Mucormycosis Efficacy. CRESEMBA.
2. Astellas wins FDA orphan drug status for approved antifungal Cresemba. Pharmaceutical Technology.
3. FDA Approves Expanded Use of CRESEMBA® (isavuconazonium sulfate) in Children with Invasive Aspergillosis and Invasive Mucormycosis. PR Newswire.
4. FDA Approves Expanded Use of CRESEMBA® (isavuconazonium sulfate) in Children with Invasive Aspergillosis and Invasive Mucormycosis. Astellas Pharma.
5. Strong Cresemba® (isavuconazole) sales performance in Asia Pacific and China triggers fourth milestone payment for this region to Basilea this year. BioSpace.