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.
Drug: olipudase alfa (GZ402665)
Patients will receive intravenous (IV) infusion of olipudase alfa
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
Clinical Sciences & Operations
Study Director
Sanofi