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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":"UNITED KINGDOM","productType":"Small molecule","productStatus":"Approved","date":"June 2020","year":"2020","type":"Agreement","leadProduct":"Ivacaftor","moa":"CFTR","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"NHS England","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase IV","highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","productSubType":"Small molecule","amount2New":0,"dosageForm":"","sponsorNew":"NHS England \/ Vertex Pharmaceuticals","highestDevelopmentStatusID":"11","companyTruncated":"NHS Englan.."},{"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":"","productType":"Large molecule","productStatus":"Approved","date":"November 2020","year":"2020","type":"Not Applicable","leadProduct":"Recombinant coagulation factor IX","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Medexus Pharmaceuticals","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase IV","highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Medexus Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"11","companyTruncated":"Medexus Ph.."},{"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":"","productType":"Small molecule","productStatus":"Approved","date":"July 2023","year":"2023","type":"Not Applicable","leadProduct":"Risdiplam","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"F. Hoffmann-La Roche","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase IV","highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","productSubType":"Small molecule","amount2New":0,"dosageForm":"","sponsorNew":"F. Hoffmann-La Roche \/ Not Applicable","highestDevelopmentStatusID":"11","companyTruncated":"F. Hoffman.."},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Santhera's Raxone\u00ae Meets Phase 4 Endpoint in Optic Neuropathy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase IV","country":"","productType":"Small molecule","productStatus":"Approved","date":"June 2021","year":"2021","type":"Not Applicable","leadProduct":"Idebenone","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Santhera Pharmaceuticals","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase IV","highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","productSubType":"Small molecule","amount2New":0,"dosageForm":"","sponsorNew":"Santhera Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"11","companyTruncated":"Santhera P.."}]

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                            01

                            Biogen

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                            Biogen

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                            Virtual Booth Boost your online visibility by uploading your products, APIs, FDFs, intermediates, excipients, and services for free on PharmaCompass. Rank higher among suppliers and expand your reach across the internet efficiently and cost-effectively.

                            Lead Product(s) : Nusinersen Sodium

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase IV

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            New Biomarker Data Add Further Evidence Supporting the Potential Benefit of SPINRAZA® (nusinersen...

                            Details : Spinraza (nusinersen) is a survival motor neuron-2 (SMN2)-directed antisense oligonucleotide indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients.

                            Brand Name : Spinraza

                            Molecule Type : Large molecule

                            Upfront Cash : Not Applicable

                            March 06, 2024

                            Lead Product(s) : Nusinersen Sodium

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase IV

                            Sponsor : Not Applicable

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                            02

                            F. Hoffmann-La Roche

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                            Virtual Booth Boost your online visibility by uploading your products, APIs, FDFs, intermediates, excipients, and services for free on PharmaCompass. Rank higher among suppliers and expand your reach across the internet efficiently and cost-effectively.

                            Lead Product(s) : Risdiplam

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase IV

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            CHMP Recommends Roche’s Evrysdi for Babies Under Two Months Old with Spinal Muscular Atrophy (SM...

                            Details : 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.

                            Brand Name : Evrysdi

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            July 21, 2023

                            Lead Product(s) : Risdiplam

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase IV

                            Sponsor : Not Applicable

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                            F. Hoffmann-La Roche

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                            Lead Product(s) : Risdiplam

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase IV

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            Four-Year Follow up Data for Roche’s Evrysdi Show Continued Increase in Number of Children with ...

                            Details : 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.

                            Brand Name : Evrysdi

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            June 30, 2023

                            Lead Product(s) : Risdiplam

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase IV

                            Sponsor : Not Applicable

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                            Takeda Pharmaceutical

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                            Virtual Booth Boost your online visibility by uploading your products, APIs, FDFs, intermediates, excipients, and services for free on PharmaCompass. Rank higher among suppliers and expand your reach across the internet efficiently and cost-effectively.

                            Lead Product(s) : Lanadelumab

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase IV

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            Interim Phase 4 Data Support TAKHZYRO® (lanadelumab) as an Effective Treatment to Reduce Attacks ...

                            Details : 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 ...

                            Brand Name : Takhzyro

                            Molecule Type : Large molecule

                            Upfront Cash : Not Applicable

                            February 28, 2022

                            Lead Product(s) : Lanadelumab

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase IV

                            Sponsor : Not Applicable

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                            05

                            Santhera Pharmaceuticals

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                            Lead Product(s) : Idebenone

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase IV

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            Santhera's Raxone® Meets Phase 4 Endpoint in Optic Neuropathy

                            Details : Raxone (idebenone) is an antioxidant benzoquinone, small molecule drug candidate which is indicated for the treatment of Leber’s Hereditary Optic Neuropathy.

                            Brand Name : Raxone

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            June 23, 2021

                            Lead Product(s) : Idebenone

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase IV

                            Sponsor : Not Applicable

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                            06

                            Biogen

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                            Lead Product(s) : Nusinersen Sodium

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase IV

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            Biogen Announces First Patient Treated in Respond Study Evaluating Benefit of Spinraza® in Patien...

                            Details : 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 gl...

                            Brand Name : Spinraza

                            Molecule Type : Large molecule

                            Upfront Cash : Not Applicable

                            January 08, 2021

                            Lead Product(s) : Nusinersen Sodium

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase IV

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            07

                            Medexus Pharmaceuticals

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                            Medexus Pharmaceuticals

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                            Virtual Booth Boost your online visibility by uploading your products, APIs, FDFs, intermediates, excipients, and services for free on PharmaCompass. Rank higher among suppliers and expand your reach across the internet efficiently and cost-effectively.

                            Lead Product(s) : Recombinant coagulation factor IX

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase IV

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            Medexus Reaches 50% Enrollment in IXINITY® Phase 4 Clinical Trial Targeting Label Expansion for P...

                            Details : 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.

                            Brand Name : Ixinity

                            Molecule Type : Large molecule

                            Upfront Cash : Not Applicable

                            November 16, 2020

                            Lead Product(s) : Recombinant coagulation factor IX

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase IV

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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