EAP of Apitegromab for Patients With Spinal Muscular Atrophy

Expanded Access Protocol of Apitegromab for Patients With Spinal Muscular Atrophy

The purpose of this expanded access program (EAP) is to provide access to apitegromab for eligible patients with spinal muscular atrophy (SMA) prior to approval by the local regulatory agency . A medical doctor must decide whether the potential benefit outweighs the risk of receiving an investigational therapy based on the individual patient's medical history and program eligibility criteria.

Inclusion Criteria:

1. ≥2 years of age.
2. Documented diagnosis of 5q SMA.

Exclusion Criteria:

1. Previous history of a hypersensitivity reaction to a monoclonal antibody or recombinant protein bearing an Fc domain (eg, a soluble receptor-Fc fusion protein), apitegromab, or excipients of apitegromab.
2. Enrolled in a clinical study for any investigational drug.
3. Pregnant or breastfeeding.