Official Title
Compassionate Use Program for Olipudase Alfa Enzyme Replacement Therapy for Patients With Chronic Acid Sphingomyelinase Deficiency (ASMD)
Brief Summary

The objective of this program is to provide access to enzyme replacement therapy (ERT) with olipudase alfa for certain patients with ASMD, a severe, life threatening disease, that could not participate in the olipudase clinical trials. The program will provide access to olipudase alfa prior to registration and the availability of commercial product (including reimbursement where applicable) in the country of the patient.

Approved for marketing
Intermediate-size Population
Sphingomyelin Lipidosis

Drug: olipudase alfa (GZ402665)
Patients will receive intravenous (IV) infusion of olipudase alfa

Eligibility Criteria

Inclusion Criteria:

All patients (adult and pediatric) - Unsolicited request for the patient from a site with at least 3 years of experience in administration and safety management of ERT. - Written informed consent signed by the patient or the patient's parent(s)/guardian(s), where applicable. - Documented deficiency of acid sphingomyelinase in peripheral leukocytes, lymphocytes, or cultured fibroblasts.

Adult patients - Age ≥ 18 years. - Clinically documented advanced disease evidenced by defined thresholds for lung, spleen, liver, and hematologic parameters.

Pediatric patients

- Age >3 years and

Eligibility Gender
Eligibility Age
Minimum: 3 Years

Clinical Sciences & Operations
Study Director

NCT Number
MeSH Terms
Niemann-Pick Disease, Type A
Niemann-Pick Diseases
Niemann-Pick Disease, Type C