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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 May 10, 2021

            Details:

            The Committee indicated that, if a patient is receiving a stable dose of Crysvita (burosumab), their treating physician can then recommend that administration be performed by the patient or their carer following training.

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            Lead Product(s): Lanadelumab

            Therapeutic Area: Genetic Disease Product Name: Takhzyro

            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 13, 2020

            Details:

            Results were consistent with the safety and efficacy of TAKHZYRO in the pivotal trial. The HELP Study OLE was designed to evaluate the long-term safety (primary endpoint) and efficacy of TAKHZYRO for up to 2.5 years.

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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.