Velmanase alfa-tycv (Lamzede) - alpha-mannosidosis Skip to main contentSkip to FDA SearchSkip to footer links An official website of the United States government Here's how you know U.S. Food and Drug Administration Search MenuSearch FDASubmit search Home Drugs Drug Approvals and Databases Drugs@FDADrug Approval Package: LAMZEDEShareTweetLinkedinEmailPrintCompany: Chiesi Farmaceutici
Velmanase alfa approved for treatment of non-central nervous system manifestations of alpha-mannosidosis: A therapeutics bulletin of the American College of Medical Genetics and Genomics (ACMG) ACMG THERAPEUTICS BULLETINVelmanase alfa approved for treatment of non-centralnervous system manifestations of alpha-mannosidosis: A therapeutics bulletin of theAmerican College of Medical Genetics deficiency. Central nervoussystem traits include intellectual disability and cerebellarataxia. Alpha-mannosidosis may be categorized into 3subtypes—mild, moderate, and severe forms; however, theclinical manifestations fall along a continuum of diseaseseverity. Except for the severe early lethal form, progres-sion can be insidious with some individuals living into thesixth decade of life.2Management
Velmanase alfa (Lamzede) - alpha-mannosidosis 1 Published 12 September 2022 1 www.scottishmedicines.org.uk SMC2466 velmanase alfa 10mg powder for solution for infusion (Lamzede®) Chiesi Limited 05 August 2022 The Scottish Medicines Consortium (SMC) has completed its initial assessment of the evidence for the above product using the ultra-orphan framework: Indication under review: enzyme replacement therapy for the treatment of non-neurological manifestations in patients with mild to moderate alpha-mannosidosis. Key points: Alpha-mannosidosis is a very rare progressive lysosomal storage disorder which results in the accumulation of mannose-rich oligosaccharides in all
Velmanase alfa (Lamzede) - alpha-Mannosidosis 25 January 2018 EMA/205473/2018 Committee for Medicinal Products for Human Use (CHMP) Assessment report Lamzede International non-proprietary name: velmanase alfa Procedure No. EMEA/H/C/003922/0000 Note Assessment report as adopted by the CHMP with all information of a commercially confidential nature deleted. 30 Churchill Place ● Canary Wharf ) No 726/2004. The eligibility to the centralised procedure was agreed upon by the EMA/CHMP on 23 January 2014. Lamzede, was designated as an orphan medicinal product EU/3/04/260 on 26 January 2005 in the following condition: Treatment of alpha-Mannosidosis Assessment report EMA/205473/2018 Page 6/109 The applicant applied for the following indication: Lamzede is indicated for long-term enzyme
Audiological and radiological study of eight polish patients with alpha-mannosidosis. Alpha-mannosidase catalyze lysosomal cleaving of mannose residues from glycoproteins. The enzyme is encoded by the MAN2B1 gene. Biallelic pathogenic variants cause enzymatic deficiency, which clinically results in alpha-mannosidosis (AM), an autosomal recessively inherited condition. Typical features observed
Can velmanase alfa be the next widespread potential therapy for alphamannosidosis? Alpha-mannosidosis (AM) is an autosomal recessive lysosomal storage disorder caused by reduced activity of the enzyme alpha-mannosidase. The disease is characterized by immunodeficiency, facial and skeletal abnormalities, impaired hearing, and intellectual disability. The clinical subtype of AM shows considerable
Global CNS correction in a large brain model of human alpha-mannosidosis by intravascular gene therapy. Intravascular injection of certain adeno-associated virus vector serotypes can cross the blood-brain barrier to deliver a gene into the CNS. However, gene distribution has been much more limited within the brains of large animals compared to rodents, rendering this approach suboptimal , including in the cerebral cortex, an important target since mental retardation is an important component of many of the human neurogenetic diseases. The therapeutic potential of a hu.32 serotype vector was evaluated in the cat homologue of the human lysosomal storage disease alpha-mannosidosis, which has globally distributed lysosomal storage lesions in the brain. Treated alpha-mannosidosis cats had
Alpha-Mannosidosis: Therapeutic Strategies Alpha-mannosidosis (α-mannosidosis) is a rare lysosomal storage disorder with an autosomal recessive inheritance caused by mutations in the gene encoding for the lysosomal α-d-mannosidase. So far, 155 variants from 191 patients have been identified and in part characterized at the biochemical level. Similarly to other lysosomal storage diseases , there is no relationship between genotype and phenotype in alpha-mannosidosis. Enzyme replacement therapy is at the moment the most effective therapy for lysosomal storage disease, including alpha-mannosidosis. In this review, the genetic of alpha-mannosidosis has been described together with the results so far obtained by two different therapeutic strategies: bone marrow transplantation and enzyme replacement therapy
Efficacy and safety of Velmanase alfa in the treatment of patients with alpha-mannosidosis: results from the core and extension phase analysis of a phase III multicentre, double-blind, randomised, placebo-controlled trial. This phase III, double-blind, randomised, placebo-controlled trial (and extension phase) was designed to assess the efficacy and safety of velmanase alfa (VA) in alpha -mannosidosis (AM) patients. Twenty-five patients were randomised to weekly 1 mg/kg VA or placebo for 52 weeks. At study conclusion, placebo patients switched to VA; 23 patients continued receiving VA in compassionate-use/follow-on studies and were evaluated in the extension phase [last observation (LO)]. Co-primary endpoints were changes in serum oligosaccharide (S-oligo) and in the 3-min stair-climb test
Comprehensive long-term efficacy and safety of recombinant human alpha-mannosidase (velmanase alfa) treatment in patients with alpha-mannosidosis. Long-term outcome data provide important insights into the clinical utility of enzyme replacement therapies. Such data are presented for velmanase alfa in the treatment of alpha-mannosidosis (AM). Patient data (n = 33; 14 adults, 19 paediatric) from
Analysis of Velmanase Alfa (Lamzede®)'s Effects in the Body of Children With Alpha-Mannosidosis Under the Age 3 The goal of this observational study is to learn the effects of the drug velmanase alfa (Lamzede®) in the bodies of children under the age of 3 with Alpha-Mannosidosis.The main questions it aims to answer are:study the effect of velmanase alfa on a marker of the disease called GlcNAc
Clinical Improvement of Alpha-mannosidosis Cat Following a Single Cisterna Magna Infusion of AAV1. Lysosomal storage diseases (LSDs) are debilitating neurometabolic disorders for most of which long-term effective therapies have not been developed. Gene therapy is a potential treatment but a critical barrier to treating the brain is the need for global correction. We tested the efficacy of cisterna magna infusion of adeno-associated virus type 1 (AAV1) expressing feline alpha-mannosidase gene in the postsymptomatic alpha-mannosidosis (AMD) cat, a homologue of the human disease. Lysosomal alpha-mannosidase (MANB) activity in the cerebrospinal fluid (CSF) and serum were increased above the control values in untreated AMD cats. Clinical neurological signs were delayed in onset and reduced
Alpha-mannosidosis: correlation between phenotype, genotype and mutant MAN2B1 subcellular localisation Alpha-mannosidosis is caused by mutations in MAN2B1, leading to loss of lysosomal alpha-mannosidase activity. Symptoms include intellectual disabilities, hearing impairment, motor function disturbances, facial coarsening and musculoskeletal abnormalities. To study the genotype-phenotype relationship for alpha-mannosidosis 66 patients were included. Based on the predicted effect of the mutations and the subcellular localisation of mutant MAN2B1 in cultured cells, the patients were divided into three subgroups. Clinical and biochemical data were collected. Correlation analyses between each of the three subgroups of genotype/subcellular localisation and the clinical and biochemical data were
Alpha-mannosidosis: Characterisation of CNS pathology and correlation between CNS pathology and cognitive function. Alpha-mannosidosis (AM) (OMIM 248500) is a rare lysosomal storage disease. The understanding of the central nervous system (CNS) pathology is limited. This study is the first describing the CNS pathology and the correlation between the CNS pathology and intellectual disabilities
What are the ENT and upper respiratory manifestations of AlphaMannosidosis? What are the ENT and upper respiratory manifestations of AlphaMannosidosis? Print | PDF PROSPERO This information has been provided by the named contact for this review. CRD has accepted this information in good faith and registered the review in PROSPERO. The registrant confirms that the information supplied
Enzyme replacement therapy for alpha-mannosidosis: 12Â months follow-up of a single centre, randomised, multiple dose study. Alpha-mannosidosis (OMIM 248500) is a rare lysosomal storage disease (LSD) caused by alpha-mannosidase deficiency. Manifestations include intellectual disabilities, facial characteristics and hearing impairment. A recombinant human alpha-mannosidase (rhLAMAN) has been
Interventional Study to Assess Efficacy and Safety of Velmanase Alfa in Patients With AlphaMannosidosis Randomized, double-blind, placebo-controlled, parallel group study where subjects will receive velmanase alfa or placebo for 24 weeks.Each subject undergoes to 8 complete visits at the clinic for clinical, laboratory and functional assessments. Study treatment is administered weekly through