Year
DEALS // DEV.
Country
Therapeutic Area
Study Phase
Deal Type
Product Type
Dosage Form
Lead Product
Target
Lead Product(s) : Miglustat
Therapeutic Area : Genetic Disease
Study Phase : Approved FDF
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Breckenridge Announces Approval of its ANDA for Miglustat Capsules (generic for Zavesca®)
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.
Product Name : Miglustat-Generic
Product Type : Other Small Molecule
Upfront Cash : Inapplicable
April 21, 2022
Lead Product(s) : Miglustat
Therapeutic Area : Genetic Disease
Highest Development Status : Approved FDF
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Arimoclomol,Miglustat
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Approved FDF
Sponsor : Undisclosed
Deal Size : $150.0 million
Deal Type : Agreement
Zevra to Sell Rare Pediatric Disease Voucher for $150M
Details : Zevra sold it's Rare Pediatric Disease PRV for FDA approved of Miplyffa (arimoclomol). It is indicated for use in combination with miglustat for the treatment of neurological manifestations of NPC. C
Product Name : Miplyffa
Product Type : Other Small Molecule
Upfront Cash : Undisclosed
February 27, 2025
Lead Product(s) : Arimoclomol,Miglustat
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Approved FDF
Sponsor : Undisclosed
Deal Size : $150.0 million
Deal Type : Agreement
Lead Product(s) : Arimoclomol,Miglustat
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Phase II/ Phase III
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Zevra Launches MIPLYFFA™ in US for Niemann-Pick Disease Type C Treatment
Details : Miplyffa (arimoclomol) is a first in class oral HSF1 activator approved for Niemann-Pick disease type C in combination with miglustat.
Product Name : Miplyffa
Product Type : Other Small Molecule
Upfront Cash : Inapplicable
November 21, 2024
Lead Product(s) : Arimoclomol,Miglustat
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Phase II/ Phase III
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Arimoclomol,Miglustat
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Approved FDF
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
FDA Approves First Treatment for Niemann-Pick Disease, Type C
Details : Miplyffa (Arimoclomol) is indicated for use in combination with miglustat for the treatment of Niemann-Pick disease type C in adult and pediatric patients 2 years of age and older.
Product Name : Miplyffa
Product Type : Other Small Molecule
Upfront Cash : Inapplicable
September 20, 2024
Lead Product(s) : Arimoclomol,Miglustat
Therapeutic Area : Rare Diseases and Disorders
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 : Amicus Therapeutics
Deal Size : Undisclosed
Deal Type : Agreement
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.
Product Name : Pombiliti
Product Type : Enzyme
Upfront Cash : Undisclosed
September 28, 2023
Lead Product(s) : Cipaglucosidase Alpha,Miglustat
Therapeutic Area : Genetic Disease
Highest Development Status : Approved FDF
Sponsor : Amicus Therapeutics
Deal Size : Undisclosed
Deal Type : Agreement
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) : Miglustat
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Phase III
Sponsor : Beyond Batten Disease Foundation
Deal Size : Inapplicable
Deal Type : Inapplicable
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...
Product Name : Batten-1
Product Type : Other Small Molecule
Upfront Cash : Inapplicable
September 05, 2023
Lead Product(s) : Miglustat
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Phase III
Sponsor : Beyond Batten Disease Foundation
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