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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
Partner/Sponsor/Collaborator: Amicus Therapeutics
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:
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.
Lead Product(s): Miglustat
Therapeutic Area: Genetic Disease Product Name: Batten-1
Highest Development Status: Phase IIIProduct Type: Small molecule
Partner/Sponsor/Collaborator: Beyond Batten Disease Foundation
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable May 09, 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
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 cipaglucosidase alfa.
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 May 10, 2022
Details:
Miglustat Capsules (generic for Zavesca), 100mg strength, is an inhibitor of the enzyme glucosylceramide synthase, which is a glucosyl transferase enzyme responsible for first step in synthesis of most glycosphingolipids.
Lead Product(s): Miglustat
Therapeutic Area: Genetic Disease Product Name: Miglustat-Generic
Highest Development Status: ApprovedProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable April 21, 2022
Details:
Latest long-term data from Phase 1/2 study of AT-GAA, represent very meaningful and durable improvements in functional outcomes, as well as persistent reductions in key biomarkers of muscle damage and disease substrate out to three years.
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 March 16, 2022
Details:
AT-GAA is investigational two-component therapy that consists of cipaglucosidase alfa, a unique recombinant human acid alpha-glucosidase enzyme with optimized carbohydrate structures, particularly bis-phosphorylated mannose-6 phosphate glycans, to enhance uptake into cells.
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 18, 2021
Details:
Janssen will provide drug supply for the clinical trials and Extended Access Program, and right of reference to data within the Zavesca New Drug Application (NDA) to support the development of BBDF-101.
Lead Product(s): Miglustat,Trehalose API
Therapeutic Area: Genetic Disease Product Name: BBDF 101
Highest Development Status: PreclinicalProduct Type: Small molecule
Partner/Sponsor/Collaborator: Beyond Batten Disease Foundation
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Collaboration March 02, 2021
Details:
AT-GAA showed a nominally statistically significant and clinically meaningful difference for superiority on the first key secondary endpoint of FVC compared to patients treated with alglucosidase alfa (cipaglucosidase alfa and 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 February 11, 2021
Details:
Orphazyme plans to present a series of data on its investigational drug arimoclomol during the 17th Annual WORLDSymposium Scientific Meeting, to be held virtually on February 8-12, 2021.
Lead Product(s): Arimoclomol,Miglustat
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Phase II/ Phase IIIProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable February 04, 2021
Details:
The long-term data demonstrate a continued positive impact on disease progression over two years. Furthermore, separate data from a post-hoc genetic sub-group analysis of patients provides more evidence of the efficacy of arimoclomol.
Lead Product(s): Arimoclomol,Miglustat
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Phase II/ Phase IIIProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable January 22, 2021
Details:
AT-GAA is an investigational therapy that consists of cipaglucosidase alfa (ATB200), a unique enzyme replacement therapy with optimized carbohydrate structures, administered in conjunction with miglustat (AT2221), an orally administered stabilizer of cipaglucosidase alfa.
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 December 01, 2020
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