Find Approved Drugs for Genetic Disease in Clinical Development

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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): Ravulizumab

            Therapeutic Area: Genetic Disease Product Name: Ultomiris

            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 30, 2020

            Details:

            PNH with Extravascular Hemolysis (EVH): Alexion plans to initiate a Phase 3 study of ALXN2040 as an add-on therapy for PNH patients with EVH by the end of 2020.

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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): Burosumab

            Therapeutic Area: Genetic Disease Product Name: Crysvita

            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 24, 2020

            Details:

            The European Commission has already granted a conditional marketing authorisation for CRYSVITA for the treatment of XLH with radiographic evidence of bone disease in children one year of age and older and adolescents with growing skeletons.

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            Lead Product(s): Crizanlizumab

            Therapeutic Area: Genetic Disease Product Name: Adakveo

            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 24, 2020

            Details:

            CHMP opinion supported by data showing Adakveo significantly reduced the rate of vaso-occlusive crises, with patients on Adakveo spending fewer days in hospital.

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