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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): 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:

            Orsini Specialty Pharmacy been selected by Alnylam® Pharmaceuticals as a limited distribution partner for OXLUMO™. OXLUMO is the first and only treatment approved for primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients.

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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: Cell and Gene therapy

            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.

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            Lead Product(s): Voxelotor

            Therapeutic Area: Genetic Disease Product Name: Oxbryta

            Highest Development Status: Approved Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable December 02, 2020

            Details:

            Through the voxelotor early access program, physicians in countries with an early access regulatory and legal pathway may be able to request voxelotor for eligible SCD patients who do not have access to the medicine as part of a clinical trial.

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            Lead Product(s): Lonafarnib

            Therapeutic Area: Genetic Disease Product Name: Zokinvy

            Highest Development Status: Approved Product Type: Small molecule

            Partner/Sponsor/Collaborator: The Progeria Research Foundation

            Deal Size: $95.0 million Upfront Cash: Undisclosed

            Deal Type: Divestment November 23, 2020

            Details:

            The PRV was granted in conjunction with the recent approval by the U.S. Food and Drug Administration of ZokinvyTM (lonafarnib) for treatment of Progeria and processing-deficient Progeroid Laminopathies.

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            Lead Product(s): Lonafarnib

            Therapeutic Area: Genetic Disease Product Name: Zokinvy

            Highest Development Status: Approved Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable November 20, 2020

            Details:

            Zokinvy is a disease-modifying agent that has demonstrated a statistically significant survival benefit in children and young adults with Progeria. In patients with Progeria, Zokinvy reduced the incidence of mortality by 60% and increased average survival time by 2.5 years.

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            Lead Product(s): Lumasiran

            Therapeutic Area: Genetic Disease Product Name: Oxlumo

            Highest Development Status: Approved Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable November 19, 2020

            Details:

            OXLUMO is the first therapeutic approved for the treatment of PH1, and the only therapy proven to lower harmful oxalate levels that drive the progression of PH1 disease.

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            Lead Product(s): Ivacaftor

            Therapeutic Area: Genetic Disease Product Name: Kalydeco

            Highest Development Status: Approved Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable November 05, 2020

            Details:

            KALYDECO® (ivacaftor) will be now available to additional eligible patients in Germany and will be available shortly in countries that have entered into innovative long-term reimbursement agreements with Vertex, including the UK, Denmark and the Republic of Ireland.

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            Lead Product(s): Ivacaftor

            Therapeutic Area: Genetic Disease Product Name: Kalydeco

            Highest Development Status: Approved Product Type: Small molecule

            Partner/Sponsor/Collaborator: Royalty Pharma

            Deal Size: $650.0 million Upfront Cash: $575.0 million

            Deal Type: Acquisition November 02, 2020

            Details:

            Funding will support the CF Foundation’s work to fund research and drug development and advance high-quality, specialized CF care. KALYDECO® (ivacaftor) as First and Only CFTR Modulator, indicate to treat Eligible Infants With CF as Early as Four Months of Age.