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Find Clinical Drug Development Pipelines & Deals | PipelineProspector

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            Lead Product(s): Burosumab

            Therapeutic Area: Genetic Disease Product Name: Crysvita

            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 October 05, 2020

            Details:

            CRYSVITA, created by Kyowa Kirin, is a recombinant fully human monoclonal IgG1 antibody against the FGF23. More people in Europe are now eligible for treatment with CRYSVITA, the only therapy that targets the underlying pathophysiology of XLH.

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            Lead Product(s): Porcine recombinant Factor VIII

            Therapeutic Area: Genetic Disease Product Name: Obizur

            Highest Development Status: Approved Product Type: Large molecule

            Partner/Sponsor/Collaborator: HLS Therapeutics

            Deal Size: $49.3 million Upfront Cash: $30.8 million

            Deal Type: Acquisition September 30, 2020

            Details:

            HLS has acquired certain entities that hold the rights to a diversified portfolio of royalty interests on global sales of four different products, this includes Takeda Pharmaceutical's Obizur, a porcine recombinant Factor VIII for acquired hemophilia A.

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            Lead Product(s): Burosumab

            Therapeutic Area: Genetic Disease Product Name: Crysvita

            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 July 24, 2020

            Details:

            The European Commission has already granted a conditional marketing authorisation for CRYSVITA for the treatment of XLH with radiographic evidence of bone disease in children one year of age and older and adolescents with growing skeletons.

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            Lead Product(s): Emicizumab

            Therapeutic Area: Genetic Disease Product Name: Hemlibra

            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 July 13, 2020

            Details:

            In the STASEY study, Hemlibra was effective with no new safety signals identified in adults and adolescents with hemophilia A with factor VIII inhibitors, which was consistent with the previously reported safety profile

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            Lead Product(s): Lanadelumab

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            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 06, 2020

            Details:

            Data from the Phase 3 HELP Study™ Open-label Extension suggest that TAKHZYRO® (lanadelumab) is well-tolerated and can prevent hereditary angioedema (HAE) attacks over an extended treatment period, with sustained and consistent reduction in monthly attack rates.

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            Lead Product(s): Lanadelumab

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            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 May 29, 2020

            Details:

            The pre-filled syringe presentation is designed to enhance the treatment administration experience for HAE patients receiving TAKHZYRO.