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Find Approved Drugs for Genetic Disease in Clinical Development

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            Lead Product(s): Antihemophilic Factor (Recombinant), PEGylated

            Therapeutic Area: Genetic Disease Product Name: Adynovate

            Highest Development Status: Approved Product Type: Large molecule

            Partner/Sponsor/Collaborator: Healthcare Royalty

            Deal Size: $150.0 million Upfront Cash: $150.0 million

            Deal Type: Agreement December 22, 2020

            Details:

            Net proceed will be used towards the funding of clinical trials for its early and late stage immune-oncology programs. For the nine-month period ended September 30, 2020, Nektar recognized $30.5 million in aggregate royalties from net sales of ADYNOVATE and MOVANTIK.

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            Lead Product(s): Antihemophilic factor recombinant

            Therapeutic Area: Genetic Disease Product Name: ADVATE

            Highest Development Status: Approved Product Type: Large molecule

            Partner/Sponsor/Collaborator: Takeda Pharmaceutical

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Agreement December 16, 2020

            Details:

            ADVATE is a recombinant antihemophilic factor indicated for use in children and adults with hemophilia A. The arrangement between Prime and Takeda provides an opportunity to assess clinical outcomes and cost implications with an existing treatment for hemophilia A.

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            Lead Product(s): Berotralstat

            Therapeutic Area: Genetic Disease Product Name: Orladeyo

            Highest Development Status: Approved Product Type: Small molecule

            Partner/Sponsor/Collaborator: Royalty Pharma

            Deal Size: $325.0 million Upfront Cash: $125.0 million

            Deal Type: Funding December 07, 2020

            Details:

            BioCryst plans to invest the combined proceeds to support the launch of ORLADEYO in the U.S. and Europe and to advance the development of BCX9930 into clinical trials in multiple complement-mediated diseases.

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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 December 07, 2020

            Details:

            With nearly three years of follow-up, Hemlibra maintained low treated bleed rates and was well tolerated in people with haemophilia A of all ages, with and without factor VIII inhibitors.

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            Lead Product(s): Lumasiran

            Therapeutic Area: Genetic Disease Product Name: Oxlumo

            Highest Development Status: Approved Product Type: Large molecule

            Partner/Sponsor/Collaborator: Alnylam Pharmaceuticals

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Partnership December 03, 2020

            Details:

            PANTHERx® Rare Pharmacy has been selected by Alnylam Pharmaceuticals as a very limited distribution pharmacy partner for new drug OXLUMO™ (lumasiran). OXLUMO is a subcutaneously administered RNAi therapeutic that targets the messenger RNA of the hydroxyacid oxidase 1 gene.

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            Lead Product(s): Lumasiran

            Therapeutic Area: Genetic Disease Product Name: Oxlumo

            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 December 03, 2020

            Details:

            Alnylam has achieved full patient enrollment in its ILLUMINATE-C Phase 3 study of lumasiran, an RNAi therapeutic targeting hydroxyacid oxidase 1– the gene encoding glycolate oxidase (GO) – for the treatment of adults and children with advanced primary hyperoxaluria type 1.