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            Epidiolex (cannabidiol) is an oral CB receptor CB1 receptor negative allosteric modulator. It is now approved for the treatment of seizures associated with Lennox-Gastaut syndrome & Dravet syndrome in patients 2 years of age and older.

            Lead Product(s): Cannabidiol

            Therapeutic Area: Rare Diseases and Disorders Product Name: Epidiolex

            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 23, 2023

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            The acquisition expands chiesi’s rare disease medicine portfolio by adding Amryt's pipeline including, Filsuvez (oleogel-S10), a topical therapeutic gel, is a potential treatment for the cutaneous manifestations of Epidermolysis Bullosa.

            Lead Product(s): Oleogel-S10

            Therapeutic Area: Rare Diseases and Disorders Product Name: Filsuvez

            Highest Development Status: Approved Product Type: Small molecule

            Partner/Sponsor/Collaborator: Chiesi Group

            Deal Size: $1,480.0 million Upfront Cash: $1,250.0 million

            Deal Type: Acquisition April 12, 2023

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            Epidyolex (cannabidiol), plant-derived cannabis-based medicine which is act CB receptor agonist, administered as an oral solution, is indicated for the treatment of seizures associated with Lennox-Gastaut syndrome, Dravet syndrome, or tuberous sclerosis complex in patients 1 year of age and older.

            Lead Product(s): Cannabidiol

            Therapeutic Area: Rare Diseases and Disorders Product Name: Epidyolex

            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 January 31, 2023

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            Filsuvez (oleogel-S10), a topical therapeutic gel, is a potential treatment for the cutaneous manifestations of Epidermolysis Bullosa, a rare and distressing genetic skin disorder affecting young children and adults for which there is currently no approved treatment.

            Lead Product(s): Oleogel-S10

            Therapeutic Area: Rare Diseases and Disorders Product Name: Filsuvez

            Highest Development Status: Approved Product Type: Small molecule

            Partner/Sponsor/Collaborator: Chiesi Farmaceutici

            Deal Size: $1,480.0 million Upfront Cash: $1,250.0 million

            Deal Type: Acquisition January 08, 2023

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            UPLIZNA (inebilizumab-cdon), is indicated for the treatment of neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody positive.

            Lead Product(s): Inebilizumab

            Therapeutic Area: Rare Diseases and Disorders Product Name: Uplizna

            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 20, 2022

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            The acquisition will enable Amgen to strengthens the potential of new medicines in Horizon’s pipeline for rare, autoimmune and severe inflammatory diseases and Horizon’s first-in-class medicines like TEPEZZA, KRYSTEXXA and UPLIZNA.

            Lead Product(s): Teprotumumab

            Therapeutic Area: Rare Diseases and Disorders Product Name: Tepezza

            Highest Development Status: Approved Product Type: Large molecule

            Partner/Sponsor/Collaborator: Amgen Inc

            Deal Size: $27,800.0 million Upfront Cash: $27,800.0 million

            Deal Type: Acquisition December 12, 2022

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            UPLIZNA (inebilizumab-cdon) is indicated for the treatment of neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody positive. By targeting CD19, UPLIZNA reduces levels of plasmablasts in addition to memory B cells.

            Lead Product(s): Inebilizumab

            Therapeutic Area: Rare Diseases and Disorders Product Name: Uplizna

            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 26, 2022

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            Filsuvez (Oleogel-S10) is a herbal medicinal product which contains birch triterpenes from birch bark. This sterile gel is being developed to speed up healing of skin wounds in adults and children with severe subtypes of epidermolysis bullosa.

            Lead Product(s): Oleogel-S10

            Therapeutic Area: Rare Diseases and Disorders Product Name: Filsuvez

            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 October 25, 2022

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            UPLIZNA (Inebilizumab-cdon) is the first and only anti-CD19 B-cell-depleting humanized monoclonal antibody approved by the U.S. Food and Drug Administration (FDA) and European Commission (EC) for the treatment of adult patients with anti-aquaporin-4 antibody positive NMOSD.

            Lead Product(s): Inebilizumab

            Therapeutic Area: Rare Diseases and Disorders Product Name: Uplizna

            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 12, 2022

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            Key highlights include efficacy of Oleogel-S10 (filsuvez) on accelerated wound healing, data on procedural pain and dressing change frequency, and a 12-month interim analysis of the open-label phase, in patients with Dystrophic Epidermolysis Bullosa.

            Lead Product(s): Birch Bark Extract

            Therapeutic Area: Rare Diseases and Disorders Product Name: Filsuvez

            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 July 07, 2022

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