[{"orgOrder":0,"company":"Zevra Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Arimoclomol","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Zevra Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Zevra Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Zevra Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Zevra Therapeutics","sponsor":"RARE-X","pharmaFlowCategory":"D","therapeuticArea":"Sleep","country":"","productType":"Small molecule","year":"2023","type":"Collaboration","leadProduct":"Serdexmethylphenidate","moa":"","graph1":"Sleep","graph2":"Phase II","graph3":"Zevra Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph 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Therapeutics \/ Zevra Therapeutics"},{"orgOrder":0,"company":"Zevra Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Sleep","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Serdexmethylphenidate","moa":"","graph1":"Sleep","graph2":"Phase II","graph3":"Zevra Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Sleep","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Zevra Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Zevra Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Zevra Therapeutics","sponsor":"Acer Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2023","type":"Acquisition","leadProduct":"Sodium Phenylbutyrate","moa":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"Zevra Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Zevra Therapeutics \/ Zevra Therapeutics","highestDevelopmentStatusID":"12","companyTruncated":"Zevra Therapeutics \/ Zevra Therapeutics"},{"orgOrder":0,"company":"Zevra Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Arimoclomol","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Zevra Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Zevra Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Zevra Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Zevra Therapeutics","sponsor":"Not 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molecule","year":"2024","type":"Financing","leadProduct":"Sodium Phenylbutyrate","moa":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"Zevra Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Zevra Therapeutics \/ Perceptive Advisors","highestDevelopmentStatusID":"12","companyTruncated":"Zevra Therapeutics \/ Perceptive Advisors"},{"orgOrder":0,"company":"Zevra Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Sleep","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Serdexmethylphenidate","moa":"","graph1":"Technology","graph2":"Phase I\/II","graph3":"Zevra Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Technology","amount2New":0.94999999999999996,"dosageForm":"","sponsorNew":"Zevra Therapeutics \/ Not 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Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Zevra Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Arimoclomol","moa":"","graph1":"Technology","graph2":"Phase I\/II","graph3":"Zevra Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Technology","amount2New":0.94999999999999996,"dosageForm":"","sponsorNew":"Zevra Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"14","companyTruncated":"Zevra Therapeutics \/ Not Applicable"}]

Find Clinical Drug Pipeline Developments & Deals by Zevra Therapeutics

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                          Therapeutic Area by Lead Product

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                          Top Deals by Deal Size (USD bn)

                          01

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          Not Confirmed

                          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.

                          Brand Name : Miplyffa

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          September 20, 2024

                          Lead Product(s) : Arimoclomol,Miglustat

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          02

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          Not Confirmed

                          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.

                          Brand Name : BRX-345

                          Molecule Type : Small molecule

                          Upfront Cash : Undisclosed

                          August 08, 2024

                          Lead Product(s) : Arimoclomol

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Cantor

                          Deal Size : Undisclosed

                          Deal Type : Public Offering

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                          03

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          Not Confirmed

                          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.

                          Brand Name : BRX-345

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          August 02, 2024

                          Lead Product(s) : Arimoclomol

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          04

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          Not Confirmed

                          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.

                          Brand Name : BRX-345

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          July 09, 2024

                          Lead Product(s) : Arimoclomol

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          05

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          Not Confirmed

                          Details : KP1077 (serdexmethylphenidate) is a prodrug of d-methylphenidate, which acts as dopamine & norepinephrine transporter antagonists. It is being evaluated for the treatment of idiopathic hypersomnia.

                          Brand Name : KP1077

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          June 03, 2024

                          Lead Product(s) : Serdexmethylphenidate

                          Therapeutic Area : Sleep

                          Highest Development Status : Phase II

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          06

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          Not Confirmed

                          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.

                          Brand Name : Olpruva

                          Molecule Type : Small molecule

                          Upfront Cash : Undisclosed

                          April 10, 2024

                          Lead Product(s) : Sodium Phenylbutyrate

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved

                          Sponsor : Perceptive Advisors

                          Deal Size : Undisclosed

                          Deal Type : Financing

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                          07

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          Not Confirmed

                          Details : KP1077 (serdexmethylphenidate) is a prodrug of d-methylphenidate, which acts as dopamine & norepinephrine transporter antagonists. It is being evaluated for the treatment of idiopathic hypersomnia.

                          Brand Name : KP1077

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          March 26, 2024

                          Lead Product(s) : Serdexmethylphenidate

                          Therapeutic Area : Sleep

                          Highest Development Status : Phase II

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          08

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          Not Confirmed

                          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.

                          Brand Name : BRX-345

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          March 04, 2024

                          Lead Product(s) : Arimoclomol

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          09

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          Not Confirmed

                          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.

                          Brand Name : Undisclosed

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          January 08, 2024

                          Lead Product(s) : Arimoclomol

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          10

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          Not Confirmed

                          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.

                          Brand Name : Undisclosed

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          December 27, 2023

                          Lead Product(s) : Arimoclomol

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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