A Long-Term Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency

The study duration is planned to be 9 years. This study is an extension study for patients
who have completed a previous study with olipudase alfa (DFI13803 for pediatric patients and
DFI13412 for adult patients).

Inclusion criteria:

- The patient completed the treatment period of a previous study of olipudase alfa with
an acceptable safety profile in the opinion of the investigator and sponsor.

- The patient and/or the patient's parent(s)/legal guardian(s) is willing and able to
provide signed written informed consent.

- The patient who is female and of childbearing potential must have a negative urine
pregnancy test for beta human chorionic gonadotropin (β HCG).

- Female patients of childbearing potential and sexually mature male patients must be
willing to practice true abstinence in line with their preferred and usual lifestyle
or use 2 acceptable effective methods of contraception up to 15 days following their
last dose of study drug.

Exclusion criteria:

- The patient has any new condition or worsening of an existing condition which in the
opinion of the investigator would make the patient unsuitable for enrollment, or could
interfere with the patient participating in or completing the study.

- The patient, in the opinion of the investigator, is unable to adhere to the
requirements of the study.

- The patient is unwilling or unable to abstain from the use of alcohol for 1 day prior
to and 3 days after each olipudase alfa infusion for the duration of the treatment
period.

- The patient is unwilling or unable to avoid, for 10 days before and 3 days after liver
biopsies, medications or herbal supplements that are potentially hepatotoxic (eg, 3
hydroxy 3 methylglutaryl coenzyme A reductase inhibitors, erythromycin, valproic acid,
antidepressants, kava, echinacea) or may cause or prolong bleeding (eg,
anticoagulants, ibuprofen, aspirin, garlic supplements, ginkgo, ginseng).

- The patient requires medication(s) that may decrease olipudase alfa activity (eg,
fluoxetine, chlorpromazine; tricyclic antidepressants [eg, imipramine, desipramine]).

The above information is not intended to contain all considerations relevant to a patient's
potential participation in a clinical trial.