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            Lead Product(s): Ivacaftor,Tezacaftor,Elexacaftor

            Therapeutic Area: Genetic Disease Product Name: Trikafta

            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 September 10, 2020

            Details:

            The primary endpoint of the study was safety and tolerability, and the results showed that TRIKAFTA was generally well tolerated and the safety data were consistent with those observed in previous Phase 3 studies.

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            Lead Product(s): Viltolarsen

            Therapeutic Area: Genetic Disease Product Name: Viltepso

            Highest Development Status: Approved Product Type: Large molecule

            Partner/Sponsor/Collaborator: NS PHARMA INC

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Agreement September 02, 2020

            Details:

            Option Care Health has been selected to participate in the limited distribution network of VILTEPSO™ (viltolarsen) for patients with Duchenne Muscular Dystrophy (“DMD”) who are amenable to exon 53 skipping therapy.

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            Lead Product(s): Ivacaftor,Tezacaftor,Elexacaftor

            Therapeutic Area: Genetic Disease Product Name: Trikafta

            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 September 01, 2020

            Details:

            U.S. Food and Drug Administration (FDA) accepted three supplemental New Drug Applications (sNDAs) for TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor), SYMDEKO® (tezacaftor/ivacaftor and ivacaftor) and KALYDECO® (ivacaftor).

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            Lead Product(s): Cysteamine Hydrochloride

            Therapeutic Area: Genetic Disease Product Name: Cystadrops

            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 August 25, 2020

            Details:

            CYSTADROPS demonstrated a significant reduction in cystine crystal deposits in the cornea of the eye and is the first and only FDA-approved cysteamine drop formulation with four times a day dosing.

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            Lead Product(s): Elexacaftor,Tezacaftor,Ivacaftor

            Therapeutic Area: Genetic Disease Product Name: Kaftrio

            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 August 21, 2020

            Details:

            E.C has granted marketing authorization for KAFTRIO® in a combination regimen with ivacaftor to treat people with cystic fibrosis ages 12 years and older with one F508del mutation and one minimal function mutation (F/MF), or two F508del mutations (F/F) in the CFTR gene.

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            Lead Product(s): L-Glutamine

            Therapeutic Area: Genetic Disease Product Name: Endari

            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 August 18, 2020

            Details:

            The application follows Emmaus’ announcement that the SFDA had granted Endari® a priority review designation as part of its program to expedite the review of drugs that are expected to have a significant impact on the treatment of a disease with unmet medical need.

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            Lead Product(s): Viltolarsen

            Therapeutic Area: Genetic Disease Product Name: Viltepso

            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 August 12, 2020

            Details:

            U.S. Food & Drug Administration (FDA) has approved VILTEPSO™ (viltolarsen) injection for patients with Duchenne muscular dystrophy (DMD) who are amenable to exon 53 skipping therapy.

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            Lead Product(s): L-Glutamine

            Therapeutic Area: Genetic Disease Product Name: Xyndari

            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 29, 2020

            Details:

            European Medicines Agency (EMA) issued scientific advice to Emmaus regarding the clinical development pathway for Xyndari™ for the treatment of sickle cell disease.

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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 July 12, 2020

            Details:

            In the STASEY study, Hemlibra was effective with no new safety signals identified in adults and adolescents with hemophilia A with factor VIII inhibitors, which was consistent with previous safety observations.

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            Lead Product(s): Triheptanoin

            Therapeutic Area: Genetic Disease Product Name: Dojolvi

            Highest Development Status: Approved Product Type: Small molecule

            Partner/Sponsor/Collaborator: Ultragenyx Pharmaceutical

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Partnership July 10, 2020

            Details:

            Orsini Pharmaceutical Services has been selected by Ultragenyx Pharmaceutical as a limited distribution specialty pharmacy partner for Dojolvi (triheptanoin). Dojolvi™ is indicated as a source of calories and fatty acids for the treatment of molecularly confirmed LC-FAOD.

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