Find Drugs for Genetic Disease in Phase III Clinical Development in UNITED STATES

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            Lead Product(s): Giroctocogene fitelparvovec

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Phase III Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Sangamo Therapeutics

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable June 18, 2020

            Details:

            All five patients with severe hemophilia A who received the 3e13 vg/kg dose showed sustained factor VIII (FVIII) activity levels, with a median of 64.2% via chromogenic assay (patient-level geometric means after week 9 post-infusion).

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            Lead Product(s): Eflornithine HCl,Sulindac

            Therapeutic Area: Genetic Disease Product Name: CPP-1X/sul

            Highest Development Status: Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable September 11, 2020

            Details:

            Although the trial did not demonstrate that overall disease progression was significantly lower with CPP-1X/sul compared to eflornithine or sulindac alone, in a subgroup of patients with intact colons there was a 70% decreased risk of disease progression with CPP-1X/sul.

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            Lead Product(s): Daratumumab,Hyaluronidase,Cyclophosphamide

            Therapeutic Area: Genetic Disease Product Name: Darzalex Faspro

            Highest Development Status: Phase III Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable September 10, 2020

            Details:

            The sBLA is supported by positive results from the Phase 3 ANDROMEDA study. ANDROMEDA evaluated subcutaneous daratumumab in combination with bortezomib, cyclophosphamide and dexamethasone compared to VCd alone and met its primary endpoint.

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            Lead Product(s): Episalvan

            Therapeutic Area: Genetic Disease Product Name: Filsuvez

            Highest Development Status: Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable September 09, 2020

            Details:

            The primary endpoint of the trial was to compare the efficacy of FILSUVEZ® versus control gel according to the proportion of patients with complete closure of the target wound within 45 days of treatment. The primary endpoint was achieved with statistical significance.

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            Lead Product(s): Vosoritide

            Therapeutic Area: Genetic Disease Product Name: BMN 111

            Highest Development Status: Phase III Product Type: Peptide

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable September 08, 2020

            Details:

            The findings demonstrated that the adjusted mean difference in AGV between children in the vosoritide group and placebo group was 1.57cm per year in favor of vosoritide , a substantial proportion of the approximately 2 cm/yr AGV deficit relative to average-stature children.

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            Lead Product(s): Odevixibat

            Therapeutic Area: Genetic Disease Product Name: A4250

            Highest Development Status: Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable September 08, 2020

            Details:

            Odevixibat was well tolerated, with an overall adverse event incidence similar to placebo. There were no drug-related serious adverse events (SAEs) reported during the study.

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            Lead Product(s): Odevixibat

            Therapeutic Area: Genetic Disease Product Name: A4250

            Highest Development Status: Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable September 07, 2020

            Details:

            Albireo’s lead candidate, odevixibat, is being developed to treat rare pediatric cholestatic liver diseases and is in Phase 3 development in progressive familial intrahepatic cholestasis and biliary atresia, with a third Phase 3 trial being planned in Alagille syndrome.

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            Lead Product(s): Vutrisiran

            Therapeutic Area: Genetic Disease Product Name: ALN-TTRSC02

            Highest Development Status: Phase III Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable September 03, 2020

            Details:

            The Company will discuss the potential for a biannual subcutaneous dosing regimen for vutrisiran, as well as discuss ongoing Phase 3 development for patisiran and vutrisiran across the APOLLO-B, HELIOS-A, and HELIOS-B studies.

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            Lead Product(s): Maralixibat

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable August 29, 2020

            Details:

            Maralixibat demonstrates significant transplant-free survival for patients with progressive familial intrahepatic cholestasis (PFIC2) who achieved serum bile acid (sBA) control in five-year analysis.

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            Lead Product(s): Casimersen

            Therapeutic Area: Genetic Disease Product Name: SRP-4045

            Highest Development Status: Phase III Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable August 25, 2020

            Details:

            U.S. Food and Drug Administration (FDA) has accepted the Company’s New Drug Application (NDA) seeking accelerated approval for casimersen (SRP-4045) and provided a regulatory action date of February 25, 2021.

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