Cipaglucosidase alfa; Recombinant Human acid alpha-glucosidase

Specification

• Cat#: THP-1102
• Product Name: Cipaglucosidase alfa; Recombinant Human acid alpha-glucosidase
• Cas No: 2359727-71-0
• Description: Cipaglucosidase alfa is a recombinant human acid alpha-glucosidase used to treat late-onset Pompe disease in adults.
• Sequences: QQGASRPGPRDAQAHPGRPRAVPTQCDVPPNSRFDCAPDKAITQEQCEARGCCYIPAKQGLQGAQMGQPWCFFPPSYPSYKLENLSSSEMGYTATLTRTTPTFFPKDILTLRLDVMMETENRLHFTIKDPANRRYEVPLETPHVHSRAPSPLYSVEFSEEPFGVIVRRQLDGRVLLNTTVAPLFFADQFLQLSTSLPSQYITGLAEHLSPLMLSTSWTRITLWNRDLAPTPGANLYGSHPFYLALEDGGSAHGVFLLNSNAMDVVLQPSPALSWRSTGGILDVYIFLGPEPKSVVQQYLDVVGYPFMPPYWGLGFHLCRWGYSSTAITRQVVENMTRAHFPLDVQWNDLDYMDSRRDFTFNKDGFRDFPAMVQELHQGGRRYMMIVDPAISSSGPAGSYRPYDEGLRRGVFITNETGQPLIGKVWPGSTAFPDFTNPTALAWWEDMVAEFHDQVPFDGMWIDMNEPSNFIRGSEDGCPNNELENPPYVPGVVGGTLQAATICASSHQFLSTHYNLHNLYGLTEAIASHRALVKARGTRPFVISRSTFAGHGRYAGHWTGDVWSSWEQLASSVPEILQFNLLGVPLVGADVCGFLGNTSEELCVRWTQLGAFYPFMRNHNSLLSLPQEPYSFSEPAQQAMRKALTLRYALLPHLYTLFHQAHVAGETVARPLFLEFPKDSSTWTVDHQLLWGEALLITPVLQAGKAEVTGYFPLGTWYDLQTVPVEALGSLPPPPAAPREPAIHSEGQWVTLPAPLDTINVHLRAGYIIPLQGPGLTTTESRQQPMALAVALTKGGEARGELFWDDGESLEVLERGAYTQVIFLARNNTIVNELVRVTSEGAGLQLQKVTVLGVATAPQQVLSNGVPVSNFTYSPDTKVLDICVSLLMGEQFLVSWC
• Species: Human
• Molecular Weight: 99400.0 Da (approximate)
• Introduction: Cipaglucosidase alfa is a novel recombinant human acid alpha-glucosidase (GAA) investigated for the treatment of patients with Pompe disease, a rare inherited metabolic disorder characterized by a deficiency in GAA. Other types of enzyme replacement therapy for the treatment of Pompe disease include alglucosidase alfa and avalglucosidase alfa. Cipaglucosidase alfa is conjugated with mannose-6-phosphate (M6P) N-glycans that bind to the cation-independent mannose-6-phosphate receptor (CI-MPR) in skeletal muscle, one of the main affected tissues in Pompe disease. Compared to alglucosidase alfa, cipaglucosidase alfa has a higher M6P content. In December 2022, the EMA's Committee for Medicinal Products for Human Use (CHMP) recommended cipaglucosidase alfa be granted marketing authorization for the treatment of Pompe disease, and the EMA fully approved the drug on March 27, 2023. Cipaglucosidase alfa is coadministered with miglustat, a small-molecule pharmacological chaperone that stabilizes the conformation of the enzyme. In September 2023, the FDA also approved cipaglucosidase alfa for similar indications.
• Formula: C4489H6796N1197O1298S32
• Biologic Classification: Protein Based Therapies Recombinant Enzymes
• Physical Appearance: Liquid

Pharmaceutical Information

• Targets: 1. Cation-independent mannose-6-phosphate receptor; 2. Alpha glucosidase (GAA)