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Inc","highestDevelopmentStatusID":"8","companyTruncated":"Zevra Therapeutics \/ Rho Inc"}]

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                            01

                            Zevra Therapeutics

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                            Lead Product(s) : Sodium Phenylbutyrate

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase I

                            Recipient : Jerry Vockley

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Open-Label Study of ACER-001 in Treatment of Combined D,L-2 Hydroxyglutaric Aciduria

                            Details : Sodium Phenylbutyrate is a Other Small Molecule drug candidate, which is currently being evaluated in Phase I clinical studies for the treatment of 2-Hydroxyglutaricaciduria.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            August 15, 2025

                            Lead Product(s) : Sodium Phenylbutyrate

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase I

                            Recipient : Jerry Vockley

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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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 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 : Miscellaneous

                            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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                            03

                            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 : Miscellaneous

                            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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                            04

                            Zevra Therapeutics

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                            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 : Sodium Phenylbutyrate is a Other Small Molecule drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of Medium chain acyl CoA dehydrogenase Deficiency.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            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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                            05

                            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 : Undisclosed

                            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 : Miscellaneous

                            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 : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            06

                            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 : 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 : Miscellaneous

                            Upfront Cash : Inapplicable

                            September 20, 2024

                            Lead Product(s) : Arimoclomol Citrate,Miglustat

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Approved FDF

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            07

                            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 : Miscellaneous

                            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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                            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 : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            FDA Advisory Committee Supports Arimoclomol as Effective for Niemann-Pick Disease Type C

                            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 : Miscellaneous

                            Upfront Cash : Inapplicable

                            August 02, 2024

                            Lead Product(s) : Arimoclomol Citrate

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase II/ Phase III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            09

                            Zevra Therapeutics

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                            Zevra Therapeutics

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                            Lead Product(s) : Arimoclomol Citrate

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase II/ Phase III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            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 : Miscellaneous

                            Upfront Cash : Inapplicable

                            July 09, 2024

                            Lead Product(s) : Arimoclomol Citrate

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase II/ Phase III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            10

                            Zevra Therapeutics

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                            Zevra Therapeutics

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                            Lead Product(s) : Dexmethylphenidate

                            Therapeutic Area : Sleep

                            Study Phase : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            Zevra Announces Positive Phase 2 Results of KP1077 for Hypersomnia at SLEEP 2024

                            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.

                            Product Name : Undisclosed

                            Product Type : Controlled Substance

                            Upfront Cash : Inapplicable

                            June 03, 2024

                            Lead Product(s) : Dexmethylphenidate

                            Therapeutic Area : Sleep

                            Highest Development Status : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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