menu
    • menu
    [{"orgOrder":0,"company":"NHS England","sponsor":"Vertex Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Small molecule","year":"2020","type":"Agreement","leadProduct":"Ivacaftor","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"NHS England","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"NHS England \/ Vertex Pharmaceuticals","highestDevelopmentStatusID":"11","companyTruncated":"NHS England \/ Vertex Pharmaceuticals"},{"orgOrder":0,"company":"Medexus Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2020","type":"Not Applicable","leadProduct":"Recombinant coagulation factor IX","moa":"","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Medexus Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Medexus Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"11","companyTruncated":"Medexus Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Risdiplam","moa":"","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"F. Hoffmann-La Roche \/ Not Applicable","highestDevelopmentStatusID":"11","companyTruncated":"F. Hoffmann-La Roche \/ Not Applicable"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"Idebenone","moa":"","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Santhera Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Santhera Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"11","companyTruncated":"Santhera Pharmaceuticals \/ Not Applicable"}]

    Find Clinical Drug Development Pipelines & Deals | PipelineProspector

    Xls
    Filters Filter
    ×
    FILTER:
    filter
    Company Name
      filter

      Year

        filter

        DEALS // DEV.

          filter

          Country

            filter
            Sponsor
              filter

              Therapeutic Area

                filter

                Study Phase

                  filter

                  Deal Type

                    filter

                    Product Type

                      filter

                      Dosage Form

                        filter

                        Lead Product

                          filter

                          Target

                            Loading...
                            refresh Filters
                            refresh Reset Filter
                            refresh Reset Filter
                            price-trend All Developments

                            Therapeutic Area by Lead Product

                            Study Phase by Lead Product

                            Company by Lead Product

                            Top Deals by Deal Size (USD bn)

                            01

                            Biogen

                            U.S.A arrow
                            PackExpo
                            Not Confirmed

                            Biogen

                            U.S.A arrow
                            PackExpo
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
                            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

                            DEALS_DEV arrow-down

                            New Biomarker Data Support Potential Benefit of SPINRAZA in Infants

                            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

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            02

                            F. Hoffmann-La Roche

                            Switzerland arrow
                            PackExpo
                            Not Confirmed

                            F. Hoffmann-La Roche

                            Switzerland arrow
                            PackExpo
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
                            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

                            DEALS_DEV arrow-down

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

                            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

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            03

                            F. Hoffmann-La Roche

                            Switzerland arrow
                            PackExpo
                            Not Confirmed

                            F. Hoffmann-La Roche

                            Switzerland arrow
                            PackExpo
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
                            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

                            DEALS_DEV arrow-down

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

                            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

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            04

                            Takeda Pharmaceutical

                            Japan arrow
                            PackExpo
                            Not Confirmed

                            Takeda Pharmaceutical

                            Japan arrow
                            PackExpo
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
                            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

                            DEALS_DEV arrow-down

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

                            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 are expected to be published in 2024.

                            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

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            05

                            Santhera Pharmaceuticals

                            Switzerland arrow
                            PackExpo
                            Not Confirmed

                            Santhera Pharmaceuticals

                            Switzerland arrow
                            PackExpo
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
                            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) : Idebenone

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase IV

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            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

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            06

                            Biogen

                            U.S.A arrow
                            PackExpo
                            Not Confirmed

                            Biogen

                            U.S.A arrow
                            PackExpo
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
                            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

                            DEALS_DEV arrow-down

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

                            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 globally, with researchers aiming to enrol up to 60 ...

                            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

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            07

                            Medexus Pharmaceuticals

                            Canada arrow
                            PackExpo
                            Not Confirmed

                            Medexus Pharmaceuticals

                            Canada arrow
                            PackExpo
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
                            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 Pediatric Hem...

                            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

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            08

                            Biogen

                            U.S.A arrow
                            PackExpo
                            Not Confirmed

                            Biogen

                            U.S.A arrow
                            PackExpo
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
                            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

                            DEALS_DEV arrow-down

                            Biogen to Initiate Phase 4 Respond Study Evaluating Benefit of Spinraza in Patients Treated with Zolgensma

                            Details : 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.

                            Brand Name : Spinraza

                            Molecule Type : Large molecule

                            Upfront Cash : Not Applicable

                            July 21, 2020

                            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

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            09

                            Vertex Pharmaceuticals

                            U.S.A arrow
                            PackExpo
                            Not Confirmed

                            Vertex Pharmaceuticals

                            U.S.A arrow
                            PackExpo
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
                            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) : Ivacaftor,Tezacaftor,Elexacaftor

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase IV

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            Vertex Pharmaceuticals Reports Positive Phase 3 Study Results for TRIKAFTA® in People Ages 12 and Older With ...

                            Details : 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 F508de...

                            Brand Name : Trikafta

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            July 20, 2020

                            Lead Product(s) : Ivacaftor,Tezacaftor,Elexacaftor

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase IV

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            10

                            NHS England

                            Country arrow
                            PackExpo
                            Not Confirmed

                            NHS England

                            Country arrow
                            PackExpo
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
                            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) : Ivacaftor,Tezacaftor,Elexacaftor

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase IV

                            Sponsor : Vertex Pharmaceuticals

                            Deal Size : Undisclosed

                            Deal Type : Agreement

                            DEALS_DEV arrow-down

                            Vertex Announces Expansion of Reimbursement Agreement With NHS England

                            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 collect...

                            Brand Name : Kaftrio

                            Molecule Type : Small molecule

                            Upfront Cash : Undisclosed

                            June 30, 2020

                            Lead Product(s) : Ivacaftor,Tezacaftor,Elexacaftor

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase IV

                            Sponsor : Vertex Pharmaceuticals

                            Deal Size : Undisclosed

                            Deal Type : Agreement

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth