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Find Approved Drugs for Genetic Disease in Clinical Development

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                          Top Deals by Deal Size (USD bn)

                          01

                          Lead Product(s) : Penicillamine

                          Therapeutic Area : Genetic Disease

                          Study Phase : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          Details : Generic version of penicillamine is a copper chelating agent, small molecule drug candidate, which approved by USFDA for the treatment of Wilson's disease.

                          Brand Name : Cuprimine-Generic

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          December 03, 2020

                          Lead Product(s) : Penicillamine

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          Granules India

                          02

                          Lead Product(s) : Efanesoctocog Alfa

                          Therapeutic Area : Genetic Disease

                          Study Phase : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          Details : Altuviiio (efanesoctocog alfa) is a novel and investigational recombinant factor VIII therapy with the potential to deliver near-normal factor activity levels, extending bleed protection in a once-weekly dose for people with haemophilia A.

                          Brand Name : Altuviiio

                          Molecule Type : Large molecule

                          Upfront Cash : Not Applicable

                          September 25, 2023

                          Lead Product(s) : Efanesoctocog Alfa

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          Sanofi Company Banner

                          03

                          Details : Ztalmy (ganaxolone) is a positive allosteric modulation of the gamma-aminobutyric acid type A (GABAA) receptor in the CNS. It is used for the treatment of epileptic seizures associated with CDKL5 deficiency disorder.

                          Brand Name : Ztalmy

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          July 31, 2023

                          Lead Product(s) : Ganaxolone

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved

                          Sponsor : Marinus Pharmaceuticals

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          Fermion Orion Company Banner

                          04

                          Lead Product(s) : Eliglustat

                          Therapeutic Area : Genetic Disease

                          Study Phase : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          Details : Cerdelga (eliglustat tartrate) is a prescription medicine used for the long-term treatment of Gaucher disease type 1 (GD1) in adults who are CYP2D6 extensive metabolizers (EMs), intermediate metabolizers (IMs), or poor metabolizers (PMs).

                          Brand Name : Cerdelga

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          April 01, 2023

                          Lead Product(s) : Eliglustat

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          Sanofi Company Banner

                          05

                          Lead Product(s) : Avalglucosidase Alfa

                          Therapeutic Area : Genetic Disease

                          Study Phase : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          Details : Nexviazyme (avalglucosidase alfa) is an enzyme replacement therapy (ERT) designed to target the mannose-6-phosphate (M6P) receptor, the key pathway for uptake and transport of ERT. It is being developed for treatment of people living with pompe disease.

                          Brand Name : Nexviazyme

                          Molecule Type : Large molecule

                          Upfront Cash : Not Applicable

                          February 24, 2023

                          Lead Product(s) : Avalglucosidase Alfa

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          Sanofi Company Banner

                          06

                          Lead Product(s) : Efanesoctocog Alfa

                          Therapeutic Area : Genetic Disease

                          Study Phase : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          Details : Altuviiio (efanesoctocog alfa) is a novel and investigational recombinant factor VIII therapy with the potential to deliver near-normal factor activity levels for most of the week, extending bleed protection in a once-weekly dose for people with haemophi...

                          Brand Name : Altuviiio

                          Molecule Type : Large molecule

                          Upfront Cash : Not Applicable

                          February 23, 2023

                          Lead Product(s) : Efanesoctocog Alfa

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          Sanofi Company Banner

                          07

                          Lead Product(s) : Avalglucosidase Alfa

                          Therapeutic Area : Genetic Disease

                          Study Phase : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          Details : Nexviadyme (avalglucosidase alfa), is the first and only newly approved medicine for Pompe disease in Europe since 2006, when the European Commission authorized the marketing of alglucosidase alfa, branded Myozyme®.

                          Brand Name : Nexviadyme

                          Molecule Type : Large molecule

                          Upfront Cash : Not Applicable

                          June 28, 2022

                          Lead Product(s) : Avalglucosidase Alfa

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          Sanofi Company Banner

                          08

                          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.

                          Brand Name : Miglustat-Generic

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          April 21, 2022

                          Lead Product(s) : Miglustat

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          Breckenridge Pharmaceutical

                          09

                          Details : Cuprimine-Generic (penicillamine) is a copper chelating agent, small molecule drug candidate, which is being evauated for the treatment of Wilson's disease.

                          Brand Name : Cuprimine-Generic

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          April 20, 2022

                          Lead Product(s) : Penicillamine

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          Breckenridge Pharmaceutical

                          10

                          Lead Product(s) : Avalglucosidase Alfa-ngpt

                          Therapeutic Area : Genetic Disease

                          Study Phase : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          Details : During the extension period of COMET, patients who had initially received Nexviazyme continued their treatment, while patients who were previously treated with alglucosidase alfa switched to Nexviazyme 20 mg/kg.

                          Brand Name : Nexviazyme

                          Molecule Type : Large molecule

                          Upfront Cash : Not Applicable

                          February 08, 2022

                          Lead Product(s) : Avalglucosidase Alfa-ngpt

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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