CSBio CSBio

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            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.

            Lead Product(s): Efanesoctocog Alfa

            Therapeutic Area: Genetic Disease Product Name: Altuviiio

            Highest Development Status: Approved Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable September 25, 2023

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            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.

            Lead Product(s): Ganaxolone

            Therapeutic Area: Genetic Disease Product Name: Ztalmy

            Highest Development Status: Approved Product Type: Small molecule

            Partner/Sponsor/Collaborator: Marinus Pharmaceuticals

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable July 31, 2023

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            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).

            Lead Product(s): Eliglustat

            Therapeutic Area: Genetic Disease Product Name: Cerdelga

            Highest Development Status: Approved Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable April 01, 2023

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            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.

            Lead Product(s): Avalglucosidase Alfa

            Therapeutic Area: Genetic Disease Product Name: Nexviazyme

            Highest Development Status: Approved Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 24, 2023

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            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 haemophilia A.

            Lead Product(s): Efanesoctocog Alfa

            Therapeutic Area: Genetic Disease Product Name: Altuviiio

            Highest Development Status: Approved Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 23, 2023

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            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®.

            Lead Product(s): Avalglucosidase Alfa

            Therapeutic Area: Genetic Disease Product Name: Nexviadyme

            Highest Development Status: Approved Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable June 28, 2022

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            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.

            Lead Product(s): Avalglucosidase Alfa-ngpt

            Therapeutic Area: Genetic Disease Product Name: Nexviazyme

            Highest Development Status: Approved Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 08, 2022

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            Nexviazyme® (avalglucosidase alfa-ngpt), is an enzyme replacement therapy designed to target the mannose6-phosphate (M6P) receptor, ) for treatment of late-onset Pompe disease in patients one year of age and older.

            Lead Product(s): Avalglucosidase Alfa

            Therapeutic Area: Genetic Disease Product Name: Nexviazyme

            Highest Development Status: Approved Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable November 12, 2021

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            Nexviazyme, (avalglucosidase alfa-ngpt) has demonstrated improvements for people living with late-onset Pompe disease. In pivotal Phase 3 trial, showed improvements in respiratory function and walking distance measures in people with LOPD and established its safety profile.

            Lead Product(s): Avalglucosidase Alfa

            Therapeutic Area: Genetic Disease Product Name: Nexviazyme

            Highest Development Status: Approved Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable August 06, 2021

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            The launch of Sapropterin Dihydrochloride Powder for Oral Solution, 100mg, a therapeutic equivalent generic version of Kuvan® (sapropterin dihydrochloride) Powder for Oral Solution, 100 mg, USP, is approved by the U.S. Food and Drug Administration (USFDA).

            Lead Product(s): Sapropterin Hydrochloride

            Therapeutic Area: Genetic Disease Product Name: Sapropterin Dihydrochloride-Generic

            Highest Development Status: Approved Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable April 07, 2021

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