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[{"orgOrder":0,"company":"NHS England","sponsor":"Vertex Pharmaceuticals","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Vertex Announces Expansion of Reimbursement Agreement With NHS England","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase IV","country":"","productType":"Small molecule","productStatus":"Approved","date":"June 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"NHS England"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Vertex Pharmaceuticals Reports Positive Phase 3 Study Results for TRIKAFTA\u00ae in People Ages 12 and Older With Cystic Fibrosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase IV","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"July 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Vertex Pharmaceuticals"},{"orgOrder":0,"company":"Biogen","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Biogen to Initiate Phase 4 Respond Study Evaluating Benefit of Spinraza in Patients Treated with Zolgensma","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase IV","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"July 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Biogen"},{"orgOrder":0,"company":"Medexus Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Medexus Reaches 50% Enrollment in IXINITY\u00ae Phase 4 Clinical Trial Targeting Label Expansion for Pediatric Hemophilia B Patients","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase IV","country":"CANADA","productType":"Large molecule","productStatus":"Approved","date":"November 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Medexus Pharmaceuticals"},{"orgOrder":0,"company":"Biogen","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Biogen Announces First Patient Treated in Respond Study Evaluating Benefit of Spinraza\u00ae in Patients Treated with Zolgensma\u00ae","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase IV","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"January 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"Biogen"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Interim Phase 4 Data Support TAKHZYRO\u00ae (lanadelumab) as an Effective Treatment to Reduce Attacks in Hereditary Angioedema Patients","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase IV","country":"JAPAN","productType":"Large molecule","productStatus":"Approved","date":"February 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Takeda Pharmaceutical"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Four-Year Follow up Data for Roche\u2019s Evrysdi Show Continued Increase in Number of Children with A Severe Form of Spinal Muscular Atrophy (SMA) Able to Sit, Stand and Walk","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase IV","country":"SWITZERLAND","productType":"Small molecule","productStatus":"Approved","date":"June 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"F. Hoffmann-La Roche"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"CHMP Recommends Roche\u2019s Evrysdi for Babies Under Two Months Old with Spinal Muscular Atrophy (SMA)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase IV","country":"SWITZERLAND","productType":"Small molecule","productStatus":"Approved","date":"July 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"F. Hoffmann-La Roche"}]

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            Evrysdi (risdiplam) is a survival motor neuron 2 (SMN2) splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to survival motor neuron (SMN) protein deficiency.

            Lead Product(s): Risdiplam

            Therapeutic Area: Genetic Disease Product Name: Evrysdi

            Highest Development Status: Phase IV Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable July 21, 2023

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            Evrysdi (risdiplam) is a survival motor neuron 2 (SMN2) splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to survival motor neuron (SMN) protein deficiency.

            Lead Product(s): Risdiplam

            Therapeutic Area: Genetic Disease Product Name: Evrysdi

            Highest Development Status: Phase IV Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable June 30, 2023

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            The observational Phase 4 EMPOWER study, evaluating real-world HAE attack rates before and after treatment with TAKHZYRO in patients with HAE types I and II, is ongoing. Full results of the EMPOWER study are expected to be published in 2024.

            Lead Product(s): Lanadelumab

            Therapeutic Area: Genetic Disease Product Name: Takhzyro

            Highest Development Status: Phase IV Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 28, 2022

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            The RESPOND study aims to evaluate if Spinraza can benefit SMA patients who have experienced a ‘suboptimal’ response to Zolgensma (onasemnogene abeparvovec). It will be conducted at around 20 sites globally, with researchers aiming to enrol up to 60 children with SMA.

            Lead Product(s): Nusinersen

            Therapeutic Area: Genetic Disease Product Name: Spinraza

            Highest Development Status: Phase IV Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 08, 2021

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            Medexus reached the 50% enrollment target in its Phase 4 clinical trial investigating IXINITY® as a prophylactic treatment for pediatric patients under 12 years of age with hemophilia B.

            Lead Product(s): Recombinant coagulation factor IX

            Therapeutic Area: Genetic Disease Product Name: Ixinity

            Highest Development Status: Phase IV Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable November 16, 2020

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            The RESPOND study will seek to understand if the proven efficacy of SPINRAZA and its continuous production of SMN protein may also benefit patients previously treated with gene therapy.

            Lead Product(s): Nusinersen

            Therapeutic Area: Genetic Disease Product Name: Spinraza

            Highest Development Status: Phase IV Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable July 21, 2020

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            The results of this study demonstrate that the triple combination provides significant additional benefit compared to existing CFTR modulator therapy for F/G and F/RF patients and supports the benefit of the medicine for patients with at least one F508del mutation.

            Lead Product(s): Ivacaftor,Tezacaftor,Elexacaftor

            Therapeutic Area: Genetic Disease Product Name: Trikafta

            Highest Development Status: Phase IV Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable July 20, 2020

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            NHS England

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            Details:

            As part of the agreement with NHS England, Vertex has committed to submit ORKAMBI®, SYMKEVI® and KAFTRIO® to the National Institute for Health and Care Excellence (NICE) within an agreed upon timetable, allowing for a period of real-world data collection on the medicines.

            Lead Product(s): Ivacaftor,Tezacaftor,Elexacaftor

            Therapeutic Area: Genetic Disease Product Name: Kaftrio

            Highest Development Status: Phase IV Product Type: Small molecule

            Partner/Sponsor/Collaborator: Vertex Pharmaceuticals

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Agreement June 30, 2020

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