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Applicable","newsHeadline":"The Lancet Neurology Publishes Pivotal Phase 3 PROPEL Study Results of AT-GAA in Late-Onset Pompe Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"November 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"},{"orgOrder":0,"company":"Amicus Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Amicus Therapeutics Announces Positive Long-Term Data from Phase 1\/2 Study of AT-GAA in Pompe Disease at the 2022 MDA Clinical & Scientific Conference","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"March 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"},{"orgOrder":0,"company":"Orsini 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III","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"April 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"},{"orgOrder":0,"company":"Theranexus","sponsor":"Beyond Batten Disease Foundation","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Theranexus and BBDF Win FDA Approval on Efficacy Endpoints for the Phase III Trial to Evaluate Batten-1 in CLN3 Batten Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"FRANCE","productType":"Small molecule","productStatus":"Approved","date":"May 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"},{"orgOrder":0,"company":"Amicus Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Amicus Therapeutics Announces Approval and Launch of New Pompe Disease Therapy in the European 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for Pompe Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"September 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"}]
Find Clinical Drug Pipeline Developments & Deals for Miglustat
Under the agreement, Orsini will dispense Pombiliti™ (cipaglucosidase alfa-atga), a hydrolytic lysosomal glycogen-specific enzyme, and Opfolda™ (miglustat), a two-component treatment approved for certain patients with late-onset Pompe disease.
The U.S. FDA has approved Pombiliti™ (cipaglucosidase alfa-atga) + Opfolda™ (miglustat) 65mg capsules. This two-component therapy is indicated for adults living with late-onset Pompe disease (LOPD) weighing ≥40 kg.
MHRA of the United Kingdom has granted marketing authorizations for Pombiliti® (cipaglucosidase alfa) + Opfolda® (miglustat) for adults living with late-onset Pompe disease (acid α-glucosidase [GAA] deficiency).
Upon approval of enzyme replacement therapy (cipaglucosidase alfa) + opfolda (miglustat), the enzyme stabilizer component of AT-GAA, will be the first two-component therapy available in the EU for the treatment of adults living with late-onset pompe disease.
Batten-1 is a novel and exclusive proprietary drug containing the active ingredient miglustat. The mechanism of action of this substance blocks the accumulation of glycosphingolipids and neuroinflammation. It is being investigated for CLN3 batten disease.
Upon approval of enzyme replacement therapy (cipaglucosidase alfa) + opfolda (miglustat), the enzyme stabilizer component of AT-GAA, will be the first two-component therapy available in the EU for the treatment of adults living with late-onset pompe disease.
Pombiliti (cipaglucosidase alfa) is administered with miglustat for the treatment of pompe disease. AT-GAA is a recombinant human acid alpha-glucosidase enzyme with optimized carbohydrate structures to enhance uptake into cells. Miglustat is a stabilizes AT-GAA.
AT-GAA (cipaglucosidase alfa) is administered with miglustat for the treatment of pompe disease. AT-GAA is a recombinant human acid alpha-glucosidase enzyme with optimized carbohydrate structures to enhance uptake into cells. Miglustat is a stabilizes AT-GAA.
AT-GAA is an investigational two-component therapy that consists of cipaglucosidase alfa (ATB200), a unique recombinant human acid alpha-glucosidase (rhGAA) enzyme with optimized carbohydrate structures.
AT-GAA is an investigational two-component therapy that consists of cipaglucosidase alfa, a unique rhGAA enzyme with optimized carbohydrate structures, particularly bis-M6P glycans, to enhance uptake into cells, administered in conjunction with miglustat.
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