EAP for Deucrictibant Immediate-release

• Provision of informed consent: Participants must provide written informed consent. Adolescents (≥12 to \<18 years old, or as per local law) require consent of parent or legally designated representative/guardian and must assent. If an adolescent reaches adulthood during the EAP, they must sign the adult informed consent form (ICF) to remain in the EAP.
• Male or female, aged ≥12 to ≤75 years at the time of providing written informed consent/assent.

• Confirmed diagnosis of HAE.

  1. for participants with hereditary angioedema with normal C1 inhibitor (HAE-nC1INH): documented genetic mutation associated with HAE-nC1INH

     OR

  2. if no documented mutation:

     • clinical diagnosis with family history of HAE-nC1INH,
     • attacks not responding to treatment with high-dose antihistamine (cetirizine 40 milligrams [mg]/day or equivalent high-dose second-generation antihistamine medication) and no clinical attack symptom relief if treated with corticosteroid, montelukast, and/or omalizumab.
     • documented effective attack symptom relief with on-demand icatibant treatment
• No satisfactory treatment options are available among currently approved HAE therapies for treatment of acute attacks ((i.e. Berinert (C1 esterase inhibitor [human]), Ekterly (sebetralstat), Firazyr (icatibant), Kalbitor (ecallantide), Ruconest (C1 esterase inhibitor [recombinant])) based on inadequate response, contraindication, safety/tolerability concerns, and/or other reasons, as documented by the treating physician in the participant's medical records and maintained as part of the EAP enrollment documentation.
• Not eligible for an ongoing clinical study or for whom participation in a clinical study is not possible or feasible for other reasons, including but not limited to clinical study eligibility criteria, geographic accessibility, study availability, timing considerations, participant decision to participate, or other participant-specific factors, as documented by the treating physician in the participant's medical records and maintained as part of the EAP enrollment documentation.
• Residence in the US or US territories.

Exclusion Criteria:

• Pregnancy or nursing: any female participant who is pregnant, planning to become pregnant during the EAP, or currently breastfeeding.
• Any diagnosis of angioedema other than HAE.
• Significant comorbidity: any clinically significant comorbidity or systemic dysfunction (e.g., cardiovascular, gastrointestinal, renal, neurologic, respiratory) that, in the opinion of the treating physician, would interfere with the participant's safety or ability to participate in this EAP.
• Severe hepatic impairment (Child-Pugh Class C).
• Substance abuse: history of alcohol or drug abuse within the past year, or current evidence of substance dependence or abuse.
• Prior treatment with deucrictibant IR resulting in discontinuation due to lack of efficacy, safety concerns, or tolerability issues.
• Participation in another investigational drug study, or treatment with any investigational drug within 30 days or 5 half-lives (whichever is longer) prior to enrolment.
• Prior gene therapy use for any indication at any time.
• Current use of medications with systemic absorption that are strong CYP3A4 inhibitors (e.g., clarithromycin, itraconazole, ketoconazole, ritonavir) or strong CYP3A4 inducers (e.g., carbamazepine, phenytoin) within 30 days (or 5 half-lives, whichever longer) prior to enrolment.