menu
    • menu
    [{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Arimoclomol","moa":"","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Orphazyme","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Orphazyme \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Orphazyme \/ Not Applicable"},{"orgOrder":0,"company":"Orphazyme","sponsor":"CytRx Corporation","pharmaFlowCategory":"D","therapeuticArea":"Neurology","country":"U.S.A","productType":"Small molecule","year":"2020","type":"Licensing Agreement","leadProduct":"Arimoclomol","moa":"HSF-1","graph1":"Neurology","graph2":"Phase III","graph3":"Orphazyme","amount2":0.12,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Neurology","amount2New":0.12,"dosageForm":"Capsule","sponsorNew":"Orphazyme \/ Orphazyme A\/S","highestDevelopmentStatusID":"10","companyTruncated":"Orphazyme \/ Orphazyme A\/S"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Arimoclomol","moa":"HSF-1","graph1":"Neurology","graph2":"Phase III","graph3":"Orphazyme","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Neurology","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Orphazyme \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Orphazyme \/ Not Applicable"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Arimoclomol","moa":"Molecular chaperones","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Orphazyme","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Orphazyme \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Orphazyme \/ Not Applicable"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Arimoclomol","moa":"Molecular chaperones","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Orphazyme","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Orphazyme \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Orphazyme \/ Not Applicable"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Arimoclomol","moa":"Molecular chaperones","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Orphazyme","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Orphazyme \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Orphazyme \/ Not Applicable"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Michael J. Fox Foundation","pharmaFlowCategory":"D","therapeuticArea":"Neurology","country":"","productType":"Small molecule","year":"2020","type":"Collaboration","leadProduct":"Arimoclomol","moa":"HSF-1","graph1":"Neurology","graph2":"Preclinical","graph3":"Orphazyme","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Neurology","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Orphazyme \/ Michael J. Fox Foundation","highestDevelopmentStatusID":"4","companyTruncated":"Orphazyme \/ Michael J. Fox Foundation"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Arimoclomol","moa":"Molecular chaperones","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Orphazyme","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Orphazyme \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Orphazyme \/ Not Applicable"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Arimoclomol","moa":"Molecular chaperones","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Orphazyme","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Orphazyme \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Orphazyme \/ Not Applicable"},{"orgOrder":0,"company":"Orphazyme","sponsor":"CytRx Corporation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2020","type":"Public Offering","leadProduct":"Arimoclomol","moa":"Molecular chaperones","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Orphazyme","amount2":0.080000000000000002,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.080000000000000002,"dosageForm":"Capsule","sponsorNew":"Orphazyme \/ CytRx","highestDevelopmentStatusID":"10","companyTruncated":"Orphazyme \/ CytRx"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Arimoclomol","moa":"Molecular chaperones","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Orphazyme","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Orphazyme \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Orphazyme \/ Not Applicable"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Arimoclomol","moa":"Molecular chaperones","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Orphazyme","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"capsule","sponsorNew":"Orphazyme \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Orphazyme \/ Not Applicable"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"Arimoclomol","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Orphazyme","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Orphazyme \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Orphazyme \/ Not Applicable"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"Arimoclomol","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Orphazyme","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Orphazyme \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Orphazyme \/ Not Applicable"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Immunology","country":"","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"Arimoclomol","moa":"Molecular chaperones","graph1":"Immunology","graph2":"Phase II","graph3":"Orphazyme","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Immunology","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Orphazyme \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Orphazyme \/ Not Applicable"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"Arimoclomol","moa":"HSF-1","graph1":"Neurology","graph2":"Phase III","graph3":"Orphazyme","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Neurology","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Orphazyme \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Orphazyme \/ Not Applicable"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"Arimoclomol","moa":"Molecular chaperones","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Orphazyme","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Orphazyme \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Orphazyme \/ Not Applicable"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"Arimoclomol","moa":"Molecular chaperones","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Orphazyme","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Orphazyme \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Orphazyme \/ Not Applicable"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Arimoclomol","moa":"","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Orphazyme","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Orphazyme \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Orphazyme \/ Not Applicable"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Arimoclomol","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Orphazyme","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Orphazyme \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Orphazyme \/ Not Applicable"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Arimoclomol","moa":"HSP","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Orphazyme","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Orphazyme \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Orphazyme \/ Not Applicable"},{"orgOrder":0,"company":"Orphazyme","sponsor":"KemPharm","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Acquisition","leadProduct":"Arimoclomol","moa":"HSP","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Orphazyme","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Orphazyme \/ KemPharm","highestDevelopmentStatusID":"9","companyTruncated":"Orphazyme \/ KemPharm"}]

    Find Clinical Drug Pipeline Developments & Deals by Orphazyme

    Menu

    Orphazyme

    EVENTS

    • Webinars & Exhibitions

    DIGITAL CONTENT

    DEVELOPMENTS

    PharmaCompass
    Xls
    Filters Filter
    Ă—
    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

                            Orphazyme

                            Denmark arrow
                            Pharmtech & Ingredients
                            Not Confirmed

                            Orphazyme

                            Denmark arrow
                            Pharmtech & Ingredients
                            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) : Arimoclomol

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase II/ Phase III

                            Sponsor : KemPharm

                            Deal Size : Undisclosed

                            Deal Type : Acquisition

                            DEALS_DEV arrow-down

                            KemPharm Announces Strategic Acquisition of Arimoclomol from Orphazyme, Expanding its Rare CNS Diseases Pipeli...

                            Details : The acquisition of arimoclomol aligns perfectly with the strategy to build KemPharm’s value via the advancement and commercialization of novel treatments that address rare CNS conditions, including the lead clinical candidate, KP1077 in idiopathic hype...

                            Brand Name : Undisclosed

                            Molecule Type : Small molecule

                            Upfront Cash : $12.8 million

                            May 15, 2022

                            Lead Product(s) : Arimoclomol

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase II/ Phase III

                            Sponsor : KemPharm

                            Deal Size : Undisclosed

                            Deal Type : Acquisition

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            02

                            Orphazyme

                            Denmark arrow
                            Pharmtech & Ingredients
                            Not Confirmed

                            Orphazyme

                            Denmark arrow
                            Pharmtech & Ingredients
                            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) : Arimoclomol

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II/ Phase III

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            Orphazyme Announces Withdrawal of European Marketing Authorisation Application for Arimoclomol for the Treatme...

                            Details : Decision to withdraw the European Marketing Authorisation Application for BRX-345 (arimoclomol) for the treatment of Niemann-Pick disease Type C (NPC) ahead of a final vote and opinion by the CHMP on the MAA scheduled for later this month.

                            Brand Name : BRX-345

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            March 22, 2022

                            Lead Product(s) : Arimoclomol

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II/ Phase III

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            03

                            Orphazyme

                            Denmark arrow
                            Pharmtech & Ingredients
                            Not Confirmed

                            Orphazyme

                            Denmark arrow
                            Pharmtech & Ingredients
                            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) : Arimoclomol

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II/ Phase III

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            Orphazyme Provides Update for Planned NDA Resubmission for Arimoclomol for the Treatment of Niemann-Pick disea...

                            Details : Orphazyme has made progress towards resubmission of the NDA for BRX-345 (arimoclomol citrate), amplifies the production of heat shock proteins for the treatment of NPC to the FDA and plans to request a Type C Meeting in Q2 2022.

                            Brand Name : BRX-345

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            February 11, 2022

                            Lead Product(s) : Arimoclomol

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II/ Phase III

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            04

                            Orphazyme

                            Denmark arrow
                            Pharmtech & Ingredients
                            Not Confirmed

                            Orphazyme

                            Denmark arrow
                            Pharmtech & Ingredients
                            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) : Arimoclomol

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II/ Phase III

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            Orphazyme to Present Additional Data on Arimoclomol’s Mechanism of Action During 2022 Annual WORLDSymposiumâ...

                            Details : Arimoclomol, is an investigational drug candidate that amplifies the production of heat shock proteins (HSPs). Arimoclomol is administered orally, and has now been studied in Phase 1, Phase 2, and pivotal Phase 2/3 trials.

                            Brand Name : Undisclosed

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            February 07, 2022

                            Lead Product(s) : Arimoclomol

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II/ Phase III

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            05

                            Orphazyme

                            Denmark arrow
                            Pharmtech & Ingredients
                            Not Confirmed

                            Orphazyme

                            Denmark arrow
                            Pharmtech & Ingredients
                            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) : Arimoclomol

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II/ Phase III

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            Orphazyme Provides Regulatory Update Following Type A Meeting with FDA on Arimoclomol in Niemann-Pick Disease ...

                            Details : arimoclomol, a heat shock protein co-inducer that promotes nascent protein folding, in patients with rapidly progressive SOD1 amyotrophic lateral sclerosis (ALS) and Niemann-Pick disease type C.

                            Brand Name : Undisclosed

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            October 31, 2021

                            Lead Product(s) : Arimoclomol

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II/ Phase III

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            06

                            Orphazyme

                            Denmark arrow
                            Pharmtech & Ingredients
                            Not Confirmed

                            Orphazyme

                            Denmark arrow
                            Pharmtech & Ingredients
                            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) : Arimoclomol

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II/ Phase III

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            Orphazyme Presents 36-Month Data Supporting Durable Response to Arimoclomol During Parseghian Scientific Confe...

                            Details : NPC patients receiving routine clinical care was estimated to be a mean increase of 5.2 points after three years, based on a statistical model combining placebo data from the NPC-002 double-blind study and prospective data from the observational NPC-001 ...

                            Brand Name : Undisclosed

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            June 28, 2021

                            Lead Product(s) : Arimoclomol

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II/ Phase III

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            07

                            Orphazyme

                            Denmark arrow
                            Pharmtech & Ingredients
                            Not Confirmed

                            Orphazyme

                            Denmark arrow
                            Pharmtech & Ingredients
                            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) : Arimoclomol

                            Therapeutic Area : Neurology

                            Study Phase : Phase III

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            Orphazyme Announces Topline Data from Pivotal Trial of Arimoclomol in Amyotrophic Lateral Sclerosis (ALS)

                            Details : Orphazyme announced that ORARIALS-01 pivotal trial of arimoclomol in amyotrophic lateral sclerosis (ALS) did not meet its primary and secondary endpoints to show benefit in people living with ALS. No important safety signals were reported in the trial.

                            Brand Name : Miplyffa

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            May 07, 2021

                            Lead Product(s) : Arimoclomol

                            Therapeutic Area : Neurology

                            Highest Development Status : Phase III

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            08

                            Orphazyme

                            Denmark arrow
                            Pharmtech & Ingredients
                            Not Confirmed

                            Orphazyme

                            Denmark arrow
                            Pharmtech & Ingredients
                            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) : Arimoclomol

                            Therapeutic Area : Immunology

                            Study Phase : Phase II

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            Orphazyme Announces Topline Results from Pivotal Trial of Arimoclomol for Inclusion Body Myositis (IBM)

                            Details : Orphazyme's phase 2/3 trial evaluating arimoclomol for the treatment of inclusion body myositis (IBM), a progressively debilitating muscle-wasting disease, did not meet its primary and secondary endpoints.

                            Brand Name : Undisclosed

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            March 29, 2021

                            Lead Product(s) : Arimoclomol

                            Therapeutic Area : Immunology

                            Highest Development Status : Phase II

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            09

                            Orphazyme

                            Denmark arrow
                            Pharmtech & Ingredients
                            Not Confirmed

                            Orphazyme

                            Denmark arrow
                            Pharmtech & Ingredients
                            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) : Arimoclomol,Miglustat

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II/ Phase III

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            Orphazyme to Showcase Data on Arimoclomol in Niemann-Pick Disease Type C During the 2021 Annual WORLDSymposium...

                            Details : Orphazyme plans to present a series of data on its investigational drug arimoclomol during the 17th Annual WORLDSymposium Scientific Meeting, to be held virtually on February 8-12, 2021.

                            Brand Name : Undisclosed

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            February 04, 2021

                            Lead Product(s) : Arimoclomol,Miglustat

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II/ Phase III

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth

                            10

                            Orphazyme

                            Denmark arrow
                            Pharmtech & Ingredients
                            Not Confirmed

                            Orphazyme

                            Denmark arrow
                            Pharmtech & Ingredients
                            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) : Arimoclomol,Miglustat

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II/ Phase III

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            Orphazyme’s Arimoclomol Expected to Be Used with Zavesca After Likely FDA Approval in Niemann-Pick Disease T...

                            Details : The long-term data demonstrate a continued positive impact on disease progression over two years. Furthermore, separate data from a post-hoc genetic sub-group analysis of patients provides more evidence of the efficacy of arimoclomol.

                            Brand Name : Undisclosed

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            January 22, 2021

                            Lead Product(s) : Arimoclomol,Miglustat

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II/ Phase III

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            blank

                            Stock Recap
                            #PipelineProspector

                            Data Compilation
                            #PharmaFlow

                            Virtual Booth