Year
DEALS // DEV.
Country
Therapeutic Area
Study Phase
Deal Type
Product Type
Dosage Form
Lead Product
Target
Lead Product(s) : Cipaglucosidase Alpha,Miglustat
Therapeutic Area : Genetic Disease
Study Phase : Approved FDF
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Amicus Therapeutics Announces FDA Approval and Launch of New Treatment for Pompe Disease
Details : 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.
Product Name : AT-GAA
Product Type : Enzyme
Upfront Cash : Inapplicable
September 28, 2023
Lead Product(s) : Cipaglucosidase Alpha,Miglustat
Therapeutic Area : Genetic Disease
Highest Development Status : Approved FDF
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Cipaglucosidase Alpha,Miglustat
Therapeutic Area : Genetic Disease
Study Phase : Approved FDF
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Amicus Therapeutics Announces Approval and Launch of New Pompe Disease Therapy in the United Kingdom
Details : 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).
Product Name : AT-GAA
Product Type : Enzyme
Upfront Cash : Inapplicable
August 15, 2023
Lead Product(s) : Cipaglucosidase Alpha,Miglustat
Therapeutic Area : Genetic Disease
Highest Development Status : Approved FDF
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Cipaglucosidase Alpha,Miglustat
Therapeutic Area : Genetic Disease
Study Phase : Approved FDF
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Amicus Therapeutics Announces Approval and Launch of New Pompe Disease Therapy in the European Union
Details : 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 dise...
Product Name : AT-GAA
Product Type : Enzyme
Upfront Cash : Inapplicable
June 27, 2023
Lead Product(s) : Cipaglucosidase Alpha,Miglustat
Therapeutic Area : Genetic Disease
Highest Development Status : Approved FDF
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Cipaglucosidase Alpha,Miglustat
Therapeutic Area : Genetic Disease
Study Phase : Phase III
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : 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 dise...
Product Name : AT-GAA
Product Type : Enzyme
Upfront Cash : Inapplicable
April 26, 2023
Lead Product(s) : Cipaglucosidase Alpha,Miglustat
Therapeutic Area : Genetic Disease
Highest Development Status : Phase III
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Cipaglucosidase Alpha,Miglustat
Therapeutic Area : Genetic Disease
Study Phase : Approved FDF
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : 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...
Product Name : AT-GAA
Product Type : Enzyme
Upfront Cash : Inapplicable
March 27, 2023
Lead Product(s) : Cipaglucosidase Alpha,Miglustat
Therapeutic Area : Genetic Disease
Highest Development Status : Approved FDF
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Cipaglucosidase Alpha,Miglustat
Therapeutic Area : Genetic Disease
Study Phase : Phase III
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Amicus Therapeutics Announces Presentations and Posters at the 19th Annual WORLDSymposium™ 2023
Details : 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...
Product Name : AT-GAA
Product Type : Enzyme
Upfront Cash : Inapplicable
February 13, 2023
Lead Product(s) : Cipaglucosidase Alpha,Miglustat
Therapeutic Area : Genetic Disease
Highest Development Status : Phase III
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Migalastat
Therapeutic Area : Genetic Disease
Study Phase : Approved FDF
Sponsor : Blackstone Life Sciences
Deal Size : $30.0 million
Deal Type : Financing
Amicus Therapeutics and Blackstone Enter into $430 Million Strategic Financing Collaboration
Details : The proceeds will be used to fund the ongoing operations of the company, including Galafold (migalastat), a small molecule and the first and only oral therapy for adults with Fabry disease and an amenable GLA variant.
Product Name : Galafold
Product Type : Other Small Molecule
Upfront Cash : Undisclosed
February 10, 2023
Lead Product(s) : Migalastat
Therapeutic Area : Genetic Disease
Highest Development Status : Approved FDF
Sponsor : Blackstone Life Sciences
Deal Size : $30.0 million
Deal Type : Financing
Lead Product(s) : Cipaglucosidase Alpha,Miglustat
Therapeutic Area : Genetic Disease
Study Phase : Phase III
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
U.S. FDA Defers Action on Filing for AT-GAA in Late-onset Pompe Disease
Details : 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...
Product Name : AT-GAA
Product Type : Enzyme
Upfront Cash : Inapplicable
October 28, 2022
Lead Product(s) : Cipaglucosidase Alpha,Miglustat
Therapeutic Area : Genetic Disease
Highest Development Status : Phase III
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Cipaglucosidase Alpha,Miglustat
Therapeutic Area : Genetic Disease
Study Phase : Phase III
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Amicus Therapeutics Receives Notification of PDUFA Date Extensions for AT-GAA
Details : AT-GAA, consists of cipaglucosidase alfa, a unique rhGAA enzyme with optimized carbohydrate structures, particularly bis-phosphorylated mannose-6 phosphate glycans, to enhance uptake into cells, administered in conjunction with miglustat, a stabilizer of...
Product Name : AT-GAA
Product Type : Enzyme
Upfront Cash : Inapplicable
October 05, 2022
Lead Product(s) : Cipaglucosidase Alpha,Miglustat
Therapeutic Area : Genetic Disease
Highest Development Status : Phase III
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Cipaglucosidase Alpha,Miglustat
Therapeutic Area : Genetic Disease
Study Phase : Phase III
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Amicus Therapeutics Provides EU Regulatory Update for AT-GAA
Details : 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.
Product Name : AT-GAA
Product Type : Enzyme
Upfront Cash : Inapplicable
July 11, 2022
Lead Product(s) : Cipaglucosidase Alpha,Miglustat
Therapeutic Area : Genetic Disease
Highest Development Status : Phase III
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable