Elosulfase alfa

How it works

It supplies the deficient lysosomal enzyme N-acetylgalactosamine-6-sulfatase, enabling catabolism of the glycosaminoglycan keratan sulfate that otherwise accumulates in tissues.

Prescribing in practice

• Acute and potentially life-threatening hypersensitivity and anaphylactic reactions can occur during or after infusion, so it must be given under supervision with resuscitation facilities available and pre-treatment with antihistamine considered.
• Given by intravenous infusion under the care of a specialist metabolic centre experienced in managing lysosomal storage disorders.
• The infusion rate may need to be slowed, interrupted or the dose split if infusion-related reactions develop.

Monitoring

Monitor closely for hypersensitivity and infusion-related reactions throughout administration and for an appropriate period afterwards, with periodic clinical assessment of mobility and respiratory function.

Counselling the patient

• Report any rash, wheeze, swelling, fever or difficulty breathing during or after the infusion immediately.
• Treatment is long-term and requires regular hospital infusion appointments.

Evidence & guidelines

Approval was supported by a randomised placebo-controlled trial demonstrating improvement in endurance (six-minute walk test) in Morquio A patients, and use is guided by specialist metabolic protocols and the SPC.