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    [{"orgOrder":0,"company":"Zevra Therapeutics","sponsor":"Perceptive Advisors","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2024","type":"Financing","leadProduct":"Sodium Phenylbutyrate","moa":"Glutamine","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Zevra Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Suspension","sponsorNew":"Zevra Therapeutics \/ Perceptive Advisors","highestDevelopmentStatusID":"15","companyTruncated":"Zevra Therapeutics \/ Perceptive Advisors"},{"orgOrder":0,"company":"Zevra Therapeutics","sponsor":"Acer Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2023","type":"Acquisition","leadProduct":"Sodium Phenylbutyrate","moa":"Glutamine","graph1":"Genetic Disease","graph2":"Approved 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Inapplicable","highestDevelopmentStatusID":"5","companyTruncated":"Zevra Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Zevra Therapeutics","sponsor":"Zevra Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"Sodium Phenylbutyrate","moa":"Glutamine","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Zevra Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Zevra Therapeutics \/ Zevra Therapeutics","highestDevelopmentStatusID":"8","companyTruncated":"Zevra Therapeutics \/ Zevra Therapeutics"}]

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                            01

                            Zevra Therapeutics

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                            Lead Product(s) : Arimoclomol Citrate,Miglustat

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Approved FDF

                            Sponsor : Undisclosed

                            Deal Size : $150.0 million

                            Deal Type : Agreement

                            DEALS_DEV arrow-down

                            Zevra Sells Rare Disease Review Voucher For $150 Million

                            Details : Zevra sold it's Rare Pediatric Disease PRV for FDA approved of Miplyffa (arimoclomol). It is indicated for use in combination with miglustat for the treatment of neurological manifestations of NPC. C

                            Product Name : Miplyffa

                            Product Type : Other Small Molecule

                            Upfront Cash : Undisclosed

                            April 07, 2025

                            Lead Product(s) : Arimoclomol Citrate,Miglustat

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Approved FDF

                            Sponsor : Undisclosed

                            Deal Size : $150.0 million

                            Deal Type : Agreement

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                            02

                            Zevra Therapeutics

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                            Lead Product(s) : Arimoclomol Citrate,Miglustat

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Approved FDF

                            Sponsor : Undisclosed

                            Deal Size : $150.0 million

                            Deal Type : Agreement

                            DEALS_DEV arrow-down

                            Zevra to Sell Rare Pediatric Disease Voucher for $150M

                            Details : Zevra sold it's Rare Pediatric Disease PRV for FDA approved of Miplyffa (arimoclomol). It is indicated for use in combination with miglustat for the treatment of neurological manifestations of NPC. C

                            Product Name : Miplyffa

                            Product Type : Other Small Molecule

                            Upfront Cash : Undisclosed

                            February 27, 2025

                            Lead Product(s) : Arimoclomol Citrate,Miglustat

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Approved FDF

                            Sponsor : Undisclosed

                            Deal Size : $150.0 million

                            Deal Type : Agreement

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                            03

                            Zevra Therapeutics

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                            Lead Product(s) : Sodium Phenylbutyrate

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Recipient : Jerry Vockley

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Study of Sodium Phenylbutyrate (ACER-001) for the Treatment of Pediatric and Adults Patients With Medium Chain...

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            January 14, 2025

                            Lead Product(s) : Sodium Phenylbutyrate

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Recipient : Jerry Vockley

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            04

                            Zevra Therapeutics

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                            ChemOutsourcing
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                            Zevra Therapeutics

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                            Lead Product(s) : Arimoclomol Citrate,Miglustat

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase II/ Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Zevra Launches MIPLYFFA™ in US for Niemann-Pick Disease Type C Treatment

                            Details : Miplyffa (arimoclomol) is a first in class oral HSF1 activator approved for Niemann-Pick disease type C in combination with miglustat.

                            Product Name : Miplyffa

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            November 21, 2024

                            Lead Product(s) : Arimoclomol Citrate,Miglustat

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase II/ Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            05

                            Zevra Therapeutics

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                            ChemOutsourcing
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                            Zevra Therapeutics

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                            Lead Product(s) : Sodium Phenylbutyrate

                            Therapeutic Area : Genetic Disease

                            Study Phase : Approved FDF

                            Sponsor : Perceptive Advisors

                            Deal Size : Undisclosed

                            Deal Type : Financing

                            DEALS_DEV arrow-down

                            Zevra Refinances Debt with Up to $100M in Committed Capital Under New Credit Facility

                            Details : The net proceeds will be used for launching Olpruva, a new formulation of sodium phenylbutyrate approved as an oral suspension by the FDA, to treat patients with urea cycle disorders.

                            Product Name : Olpruva

                            Product Type : Other Small Molecule

                            Upfront Cash : Undisclosed

                            October 04, 2024

                            Lead Product(s) : Sodium Phenylbutyrate

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Approved FDF

                            Sponsor : Perceptive Advisors

                            Deal Size : Undisclosed

                            Deal Type : Financing

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                            06

                            Zevra Therapeutics

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                            ChemOutsourcing
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                            Zevra Therapeutics

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                            Lead Product(s) : Arimoclomol Citrate,Miglustat

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            FDA Approves First Treatment for Niemann-Pick Disease, Type C

                            Details : Miplyffa (Arimoclomol) is indicated for use in combination with miglustat for the treatment of Niemann-Pick disease type C in adult and pediatric patients 2 years of age and older.

                            Product Name : Miplyffa

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            September 20, 2024

                            Lead Product(s) : Arimoclomol Citrate,Miglustat

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            07

                            Zevra Therapeutics

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                            ChemOutsourcing
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                            Zevra Therapeutics

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                            ChemOutsourcing
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                            Lead Product(s) : Arimoclomol Citrate

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase II/ Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Zevra Announces FDA Advisory Committee Meeting for Arimoclomol in Niemann-Pick Disease

                            Details : BRX-345 (arimoclomol) is a first in class oral HSF1 activator being investigated for Niemann-Pick disease type C. The FDA has accepted the resubmitted NDA and set a PDUFA date of September 21, 2024.

                            Product Name : Miplyffa

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            September 07, 2024

                            Lead Product(s) : Arimoclomol Citrate

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase II/ Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            08

                            Zevra Therapeutics

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                            Lead Product(s) : Arimoclomol Citrate

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase II/ Phase III

                            Sponsor : Cantor Fitzgerald

                            Deal Size : Undisclosed

                            Deal Type : Public Offering

                            DEALS_DEV arrow-down

                            Zevra Therapeutics Announces Proposed Public Offering of Common Stock

                            Details : The net proceeds from this offering will used to support the pre-commercial launch activities for BRX-345 (arimoclomol) for the treatment of Niemann-Pick Disease Type C.

                            Product Name : Miplyffa

                            Product Type : Other Small Molecule

                            Upfront Cash : Undisclosed

                            August 08, 2024

                            Lead Product(s) : Arimoclomol Citrate

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase II/ Phase III

                            Sponsor : Cantor Fitzgerald

                            Deal Size : Undisclosed

                            Deal Type : Public Offering

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                            09

                            Zevra Therapeutics

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                            • 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 Citrate

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase II/ Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Zevra Therapeutics Receives FDA Acceptance of NDA Resubmission for Arimoclomol Treatment

                            Details : Arimoclomol is a first in class investigational HSF1 activator, which is under phase 2/3 clinical development for the treatment of Niemann-Pick disease type C.

                            Product Name : Miplyffa

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            August 01, 2024

                            Lead Product(s) : Arimoclomol Citrate

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase II/ Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            10

                            Zevra Therapeutics

                            U.S.A arrow
                            ChemOutsourcing
                            Not Confirmed

                            Zevra Therapeutics

                            U.S.A arrow
                            ChemOutsourcing
                            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 Citrate

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase II/ Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Zevra Therapeutics Provides FDA Update on PDUFA Action Date for Arimoclomol Treatment

                            Details : BRX-345 (arimoclomol) is a first in class investigational HSF1 activator, which is being evaluated in phase 2/3 clinical trials for the treatment of Niemann-Pick disease type C.

                            Product Name : Miplyffa

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            April 03, 2024

                            Lead Product(s) : Arimoclomol Citrate

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase II/ Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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