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MEDIA ADVISORY: Ultragenyx Announces Mepsevii® (vestronidase alfa) Receives Reimbursement Approval for Treatment of Mucopolysaccharidosis VII in Italy
NOVATO, Calif., Sept. 09, 2021 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc., a biopharmaceutical company focused on the development and
About this update from Ultragenyx Pharmaceutical Inc.
[{"type":"text","content":"NOVATO, Calif., Sept. 09, 2021 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc., a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultra-rare genetic diseases, today 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. INDICATION (IN THE E.U.) Mepsevii is indicated for the treatment of non-neurological manifestations of Mucopolysaccharidosis VII (MPS VII; Sly syndrome). For more detail, including complete Summary of Product Characteristics (SmPC), please visit https://www.ultragenyx.com/wp-content/uploads/2021/09/SmPC_Mepsevii_INN-vestronidase_alfa_IT.pdf. IMPORTANT SAFETY INFORMATION (IN THE E.U.)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 report...