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            Lead Product(s): Copper histidinate

            Therapeutic Area: Genetic Disease

            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 January 16, 2020

            Details:

            If Cyprium’s new drug application is approved, the company may be eligible to receive a priority review voucher.

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            Lead Product(s): Berotralstat

            Therapeutic Area: Genetic Disease

            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 January 12, 2020

            Details:

            NDA submitted to U.S Food and Drug Administration in December 2019. APeX-J trial in Japan met its primary endpoint for prevention of HAE attacks, and berotralstat was well-tolerated.

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            Lead Product(s): Lumasiran

            Therapeutic Area: Genetic Disease

            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 January 10, 2020

            Details:

            The rolling submission allows completed sections of an NDA to be reviewed by the FDA on an ongoing basis. Alnylam has submitted the non-clinical components to the FDA.