Study on JZP458 for Treating ALL/LBL Patients Allergic to E. Coli-derived Asparaginase

Adult and pediatric patients with Acute Lymphoblastic Leukemia (ALL) or Lymphoblastic Lymphoma (LBL) who are hypersensitive to E.coli-derived asparaginases (allergic reaction or silent inactivation) and meet the criteria below:

Inclusion Criteria (Initial Requests):

• Patient (or parent/legally authorized representative thereof) is able to understand and voluntarily give informed consent/assent

• Patient has a diagnosis of ALL or LBL

• Patient received appropriate standard treatment without success (or no standard treatment exists for the patient's condition), and no satisfactory alternative drug is available

• Patient had a ≥ Grade 2 allergic reaction (Common Terminology Criteria for Adverse Events [CTCAE] v5.0) to a long-acting E. coli-derived asparaginase OR have silent inactivation

• Patient, in Investigators' opinion, has fully recovered from their prior allergic reaction to E. coli-derived asparaginase Note: Patients must have completed antihistamine, epinephrine, and/or corticosteroid treatment for the allergic reaction ≥ 24 hours prior to JZP458 administration.

• Patient has adequate liver function, defined as: direct (conjugated) bilirubin ≤ 3X upper limit of normal (ULN); serum glutamic pyruvic transaminase (SGPT)(alanine aminotransferase [ALT]), and serum glutamic-oxaloacetic transaminase (SGOT)(aspartate transaminase [AST]) ≤ 5X ULN

• For female patients of childbearing potential (i.e., fertile, following menarche) and male patients who have female partners of childbearing potential:

  • Patient has agreed to use medically acceptable methods of contraception with their partners for the duration of treatment with JZP458 and for 30 days after the last dose of JZP458.
  • Medically acceptable methods of contraception that the patient may use include abstinence, progestogen-only oral hormonal contraception, where inhibition of ovulation is not the primary mode of action; a combination of male condoms with either cap, diaphragm, or sponge with spermicide (double barrier methods).

Exclusion Criteria (Initial Requests):

• Patient is currently taking part in or is eligible for a clinical trial for JZP458
• Patient previously received asparaginase Erwinia Chrysanthemi AND has access to a short-acting non-E. Coliasparaginase
• Patient experienced concern of a possible adverse event (AE) related to asparaginase Erwinia Chrysanthemi
• Patient to receive asparaginase Erwinia Chrysanthemi within 1 week of plans to start JZP458
• Patient is concurrently receiving another investigational agent and/or treated with an investigational device at the same time as JZP458(within 48 hours)
• Patient has a history of ≥ Grade 3 pancreatitis (per CTCAE v5.0)
• Patient has a history of ≥ Grade 3 hypersensitivity to asparaginase Erwinia Chrysanthemi
• Patient has a prior history of asparaginase-associated ≥ Grade 3 (per CTCAE v5.0) haemorrhagic event or asparaginase-associated thrombus requiring anticoagulation therapy, excluding catheter-related thrombotic events
• Female patients who are lactating and do not agree to stop breast-feeding

Eligibility Criteria (Re-Supply Requests)

• Patient remains eligible for inclusion in the Expanded Access Protocol (EAP)
• Patient continues to benefit from treatment with JZP458
• Patient has not encountered unacceptable toxicity
• Patient has not shown signs/symptoms of relapsed or refractory disease
• Patient has not encountered ≥ Grade 3hypersensitivity symptoms
• Patient is not pregnant or breastfeeding