Expanded Access to Bezuclastinib for Patients With NonAdvanced Systemic Mastocytosis or Advanced Systemic Mastocytosis

Intermediate-Size Patient Population Expanded Access to Bezuclastinib for Patients With NonAdvanced Systemic Mastocytosis or Advanced Systemic Mastocytosis

The purpose of this expanded access program (EAP) protocol is to provide investigational bezuclastinib to patients with a diagnosis of nonadvanced systemic mastocytosis (NonAdvSM) or advanced systemic mastocytosis (AdvSM) who have received and failed or been intolerant to at least one standard approved therapy and/or have no comparable or satisfactory alternative therapy options.

Key Inclusion Criteria:

• Able to provide written informed consent and commit to EAP assessments.
• ≥18 years of age.
• Able to swallow tablets.
• Diagnosed with ASM, SM-AHN, MCL, BMM, ISM, or SSM according to the 2022 WHO Classification for SM
• Not receiving adequate disease control on current therapy(ies).
• Have clinically acceptable laboratory screening results.

Exclusion Criteria:

• Patients who are eligible for and/or enrolled in an on-going bezuclastinib clinical trial.
• Patients who discontinued investigational use of bezuclastinib in previous clinical trials due to toxicity or withdrawal of consent.
• Pregnant or currently breastfeeding.
• Prior or ongoing clinically significant illness or medical or physical condition

Other protocol-defined criteria apply.