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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.
Lead Product(s): Migalastat
Therapeutic Area: Genetic Disease Product Name: Galafold
Highest Development Status: ApprovedProduct Type: Small molecule
Partner/Sponsor/Collaborator: Blackstone Life Sciences
Deal Size: $30.0 million Upfront Cash: Undisclosed
Deal Type: Financing October 02, 2023
Details:
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.
Lead Product(s): Cipaglucosidase Alfa,Miglustat
Therapeutic Area: Genetic Disease Product Name: Pombiliti
Highest Development Status: ApprovedProduct Type: Large molecule
Recipient: Orsini Specialty Pharmacy
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Agreement September 28, 2023
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.
Lead Product(s): Cipaglucosidase Alfa,Miglustat
Therapeutic Area: Genetic Disease Product Name: Pombiliti
Highest Development Status: ApprovedProduct Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable September 28, 2023
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).
Lead Product(s): Cipaglucosidase Alfa,Miglustat
Therapeutic Area: Genetic Disease Product Name: Pombiliti
Highest Development Status: ApprovedProduct Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable August 15, 2023
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 disease.
Lead Product(s): Cipaglucosidase Alfa,Miglustat
Therapeutic Area: Genetic Disease Product Name: Pombiliti
Highest Development Status: ApprovedProduct Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable June 27, 2023
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 disease.
Lead Product(s): Cipaglucosidase Alfa,Miglustat
Therapeutic Area: Genetic Disease Product Name: Pombiliti
Highest Development Status: Phase IIIProduct Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable April 26, 2023
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 AT-GAA.
Lead Product(s): Cipaglucosidase Alfa,Miglustat
Therapeutic Area: Genetic Disease Product Name: Pombiliti
Highest Development Status: ApprovedProduct Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable March 27, 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-GAA.
Lead Product(s): Cipaglucosidase Alfa,Miglustat
Therapeutic Area: Genetic Disease Product Name: AT-GAA
Highest Development Status: Phase IIIProduct Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable February 13, 2023
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.
Lead Product(s): Cipaglucosidase Alfa,Miglustat
Therapeutic Area: Genetic Disease Product Name: AT-GAA
Highest Development Status: Phase IIIProduct Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable November 07, 2022
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.
Lead Product(s): Cipaglucosidase Alfa,Miglustat
Therapeutic Area: Genetic Disease Product Name: AT-GAA
Highest Development Status: Phase IIIProduct Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable October 28, 2022
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