Ultragenyx Pharmaceutical Inc. announced that Mepsevii® (vestronidase alfa) has been approved for reimbursement by the Agenzia Italiana del Farmaco (Italian Medicines Agency – AIFA) for the treatment of pediatric and adult patients with Mucopolysaccharidosis VII (MPS VII; Sly syndrome). MPS VII is a progressive, rare, genetic disorder that effects an estimated 200 people in the developed world. People with MPS VII do not produce enough beta-glucuronidase, an enzyme that plays a key part in the breakdown of specific sugars in the body. The lack of this enzyme leads to storage of sugars within the cells, which can cause progressive damage in many organs. Mepsevii is indicated for the treatment of non-neurological manifestations of Mucopolysaccharidosis VII (MPS VII; Sly syndrome). Contraindications: Life-threatening hypersensitivity (anaphylactic reaction) to the active substance or to any of the excipients. Special warnings and precautions for use: The effects of treatment with vestronidase alfa should be periodically evaluated and discontinuation of treatment should be considered in cases where clear benefits (including stabilisation of disease manifestations) are not observed. Discontinuation of treatment may cause significant worsening of the patient’s clinical status. As end organ damage progresses over time, it is more difficult for the treatment to reverse the damage or to show improvements. It should be considered by the treating physician that the administration of vestronidase alfa does not affect the irreversible complications (e.g. skeletal deformities). Vestronidase alfa, at the exposure observed in humans, is not expected to cross the blood-brain-barrier and therefore it is not likely to impact the neurological manifestations of the disease. Hypersensitivity reactions including anaphylaxis: Serious hypersensitivity reactions, including anaphylaxis, have been reported with vestronidase alfa; appropriate medical support should be readily available when vestronidase alfa is administered. Infusion should be avoided if the patient has an acute febrile or respiratory illness at the time. Spinal/Cervical cord compression: Spinal or cervical cord compression is a known and serious complication of MPS VII. Patients with MPS VII receiving vestronidase alfa should be monitored for signs and symptoms of spinal cord compression or neck instability including neck or back pain, weakness of limbs, changes in reflexes or urinary and faecal incontinence. Appropriate clinical treatment should be immediately sought. Sodium restricted diet: Mepsevii is considered high in sodium. This should be taken into consideration during dilution of the medicinal product for patients on a controlled sodium diet or for those patients with congestive heart failure needing to restrict sodium and total water intake. Traceability: the name and the batch number of the administered product should be clearly recorded. Undesirable effects: The most common adverse reactions from 4 clinical trials in 23 patients treated with vestronidase alfa were rash (17.4%), urticaria (17.4%), infusion site extravasation (17.4%), anaphylactoid reaction (13%), infusion site swelling (8.7%), pruritus (8.7%) and diarrhoea (8.7%). Most adverse reactions were mild to moderate in severity. There was a single adverse reaction of febrile convulsion observed in one patient (4.3%); the patient recovered without sequelae. See the SmPC for further information.