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Rylaze

Phase 2FDA approved 2021

Acute Lymphoblastic Leukemia | Small molecule | Oncology |Jazz Pharmaceuticals plc|Last Updated: Nov 18, 2023

Development status

FDA approval Jun 30, 2021
Approval holderJazz Pharms
ApplicationBLA761179
Highest phase Phase 2

Label indication RYLAZE is indicated as a component of a multi-agent chemotherapeutic regimen for the treatment of acute lymphoblastic leukemia (ALL) and lymphoblastic lymphoma (LBL) in adult and pediatric patients 1 month or older who have developed hypersensitivity to E. coli -derived asparaginase. FDA label

Target and mechanism

ModalitySmall molecule

Also known as IM JZP-458, JZP-458

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLED
Total Trials1
Total Enrollment229

FDA Designations

No designations recorded

Clinical trial landscape

Rylaze · 1 trial · 2 indications

Phase 2 1
NCT04145531An Open-Label Study of JZP-458 (RC-P) in Patients With Acute Lymphoblastic Leukemia (ALL)/Lymphoblastic Lymphoma (LBL)Acute Lymphoblastic Leukemia
COMPLETED229 Analytics
PHASE2COMPLETED
An Open-Label Study of JZP-458 (RC-P) in Patients With Acute Lymphoblastic Leukemia (ALL)/Lymphoblastic Lymphoma (LBL)
Acute Lymphoblastic LeukemiaUnlock trial analytics

Study Endpoints

Primary Endpoints

Response Rate During the First Course of JZP-458 Administration
Baseline up to 2 weeks

The response rate was defined as the number (proportion) of patients with the last 72-hour nadir serum asparaginase activity (NSAA) level ≥ 0.1 IU/mL during the first course of IM JZP-458. Blood samples were collected for serum asparaginase activity level determination.

Number of Participants With Any Treatment-emergent Adverse Events (TEAEs)
Date of written informed consent up to 30 days after last dose of last course, up to approximately 2 years 7 months

An adverse event (AE) is any untoward medical occurrence associated with the use of a drug in humans, whether or not considered related to study drug. AEs were classified by the Investigator using the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE), version 5.0.

Secondary Endpoints

Number of Participants With Last 48-hour NSAA Level ≥ 0.1 IU/mL During The First Course (6 Doses) of JZP-458 Administration
Baseline up to 2 weeks
Number of Participants With Last NSAA Levels ≥ 0.4 IU/mL During The First Course (6 Doses) of JZP-458 Administration
Baseline up to 2 weeks
Mean Serum Asparaginase Activity Levels in First Course of JZP-458 Administration
Up to 2 weeks (6 doses)
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSEQUENTIAL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
JZP-458EXPERIMENTALPart A (IM JZP-458) of the study will have 2 IM cohorts: * Cohort 1: a JZP-458 repeat dose/confirmatory cohort; a final IM JZP-458 dose level will be selected, and * Cohort 2: an expansion cohort to confirm the efficacy and safety of the final IM JZP-458 dose level and schedule Part B (IV JZP-458 Dose Confirmation) will be conducted to define the optimal dose of the IV administration of JZP-458 for further study in ALL/LBL patients as a repeated dose. Additional courses of JZP-458 (IM or IV depending on patient's allocation at study enrollment) will be administered based on each patient's original treatment plan for as long as the patient derives clinical benefit.

Interventions

NameTypeDescription
IM JZP-458DRUGIM JZP-458 will be administered in Part A, Cohorts 1 \& 2
IV JZP-458DRUGIV JZP-458 will be administered in Part B
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Eligibility Criteria

SexALL
Healthy VolunteersNo
Study Sites74

Inclusion Criteria: 1. Pediatric and adult patients with a diagnosis of ALL or LBL. 2. Have had an allergic reaction to a long-acting E. coli-derived asparaginase OR have silent inactivation. 3. Have 1 or more courses of E. coli-derived asparaginase remaining in his/her treatment plan. 4. Patients ...

Countries:United StatesCanada
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Frequently asked questions about Rylaze

What is Rylaze used for?

Rylaze is used for acute lymphoblastic leukemia, a cancer of the blood and bone marrow. It is also studied in lymphoblastic lymphoma. Jazz Pharmaceuticals plc develops Rylaze, which has been evaluated in a Phase 2 clinical trial in patients with these conditions.

Who makes Rylaze?

Rylaze is made by Jazz Pharmaceuticals plc, which trades under the ticker JAZZ. Jazz Pharmaceuticals is responsible for the development and manufacturing of Rylaze for acute lymphoblastic leukemia and related conditions.

What phase is Rylaze in?

Rylaze is in Phase 2 clinical development. A completed Phase 2 trial, NCT04145531, evaluated Rylaze in patients with acute lymphoblastic leukemia and lymphoblastic lymphoma. The trial enrolled 229 patients across the United States and Canada.

What clinical trials is Rylaze in?

Rylaze has been studied in one clinical trial, NCT04145531, titled an open-label study of JZP-458 (RC-P) in patients with acute lymphoblastic leukemia and lymphoblastic lymphoma. This Phase 2 trial is completed and enrolled 229 patients in the United States and Canada.

Is Rylaze the same as JZP-458?

Yes, Rylaze is also known as JZP-458 and IM JZP-458. These are alternative names for the same investigational drug developed by Jazz Pharmaceuticals plc for acute lymphoblastic leukemia and lymphoblastic lymphoma.