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    [{"orgOrder":0,"company":"PANTHERx Rare Pharmacy","sponsor":"Blueprint Medicines","pharmaFlowCategory":"D","therapeuticArea":"Oncology","country":"U.S.A","productType":"Small molecule","year":"2020","type":"Partnership","leadProduct":"Avapritinib","moa":"PDGFRA","graph1":"Oncology","graph2":"Approved","graph3":"PANTHERx Rare Pharmacy","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Oncology","amount2New":0,"dosageForm":"Tablet","sponsorNew":"PANTHERx Rare Pharmacy \/ Blueprint Medicines","highestDevelopmentStatusID":"12","companyTruncated":"PANTHERx Rare Pharmacy \/ Blueprint Medicines"},{"orgOrder":0,"company":"PANTHERx Rare Pharmacy","sponsor":"Blueprint Medicines","pharmaFlowCategory":"D","therapeuticArea":"Oncology","country":"","productType":"Small molecule","year":"2020","type":"Agreement","leadProduct":"Pralsetinib","moa":"","graph1":"Oncology","graph2":"Approved","graph3":"PANTHERx Rare 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Pharmaceuticals"},{"orgOrder":0,"company":"PANTHERx Rare Pharmacy","sponsor":"Biocodex","pharmaFlowCategory":"D","therapeuticArea":"Neurology","country":"","productType":"Small molecule","year":"2022","type":"Partnership","leadProduct":"Stiripentol","moa":"GABA A receptor","graph1":"Neurology","graph2":"Approved","graph3":"PANTHERx Rare Pharmacy","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Neurology","amount2New":0,"dosageForm":"Capsule","sponsorNew":"PANTHERx Rare Pharmacy \/ PANTHERx Rare","highestDevelopmentStatusID":"12","companyTruncated":"PANTHERx Rare Pharmacy \/ PANTHERx Rare"},{"orgOrder":0,"company":"PANTHERx Rare Pharmacy","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Leniolisib","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"PANTHERx Rare Pharmacy","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"PANTHERx Rare Pharmacy \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"PANTHERx Rare Pharmacy \/ Not Applicable"},{"orgOrder":0,"company":"PANTHERx Rare Pharmacy","sponsor":"X4 Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2024","type":"Partnership","leadProduct":"Mavorixafor","moa":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"PANTHERx Rare Pharmacy","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"PANTHERx Rare Pharmacy \/ X4 Pharmaceuticals","highestDevelopmentStatusID":"12","companyTruncated":"PANTHERx Rare Pharmacy \/ X4 Pharmaceuticals"},{"orgOrder":0,"company":"PANTHERx Rare Pharmacy","sponsor":"ITF Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2024","type":"Partnership","leadProduct":"Givinostat","moa":"","graph1":"Technology","graph2":"Phase I\/II","graph3":"PANTHERx Rare Pharmacy","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Technology","amount2New":0.94999999999999996,"dosageForm":"","sponsorNew":"PANTHERx Rare Pharmacy \/ ITF Therapeutics","highestDevelopmentStatusID":"14","companyTruncated":"PANTHERx Rare Pharmacy \/ ITF Therapeutics"},{"orgOrder":0,"company":"PANTHERx Rare Pharmacy","sponsor":"Gilead Sciences","pharmaFlowCategory":"D","therapeuticArea":"Hepatology (Liver, Pancreatic, Gall Bladder)","country":"","productType":"Small molecule","year":"2024","type":"Partnership","leadProduct":"Seladelpar Lysine","moa":"","graph1":"Technology","graph2":"Phase I\/II","graph3":"PANTHERx Rare Pharmacy","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Technology","amount2New":0.94999999999999996,"dosageForm":"","sponsorNew":"PANTHERx Rare Pharmacy \/ Gilead Sciences","highestDevelopmentStatusID":"14","companyTruncated":"PANTHERx Rare Pharmacy \/ Gilead Sciences"}]

    Find Clinical Drug Pipeline Developments & Deals by PANTHERx Rare Pharmacy

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                            01

                            PANTHERx Rare Pharmacy

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                            Medlab Asia & Asia Health
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                            PANTHERx Rare Pharmacy

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                            Lead Product(s) : Seladelpar Lysine

                            Therapeutic Area : Hepatology (Liver, Pancreatic, Gall Bladder)

                            Study Phase : Approved

                            Sponsor : Gilead Sciences

                            Deal Size : Undisclosed

                            Deal Type : Partnership

                            DEALS_DEV arrow-down

                            PANTHERx® Rare Partnering with Gilead Sciences, Inc. for the Distribution of LIVDELZI®

                            Details : The partnership aims for the distribution of a novel selective, first-in-class, PPAR delta agonist, Livdelzi (seladelpar), to treat adults with primary biliary cholangitis (PBC).

                            Brand Name : Livdelzi

                            Molecule Type : Small molecule

                            Upfront Cash : Undisclosed

                            August 16, 2024

                            Lead Product(s) : Seladelpar Lysine

                            Therapeutic Area : Hepatology (Liver, Pancreatic, Gall Bladder)

                            Highest Development Status : Approved

                            Sponsor : Gilead Sciences

                            Deal Size : Undisclosed

                            Deal Type : Partnership

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                            02

                            PANTHERx Rare Pharmacy

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                            Medlab Asia & Asia Health
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                            PANTHERx Rare Pharmacy

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                            Medlab Asia & Asia Health
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                            Lead Product(s) : Givinostat

                            Therapeutic Area : Genetic Disease

                            Study Phase : Approved

                            Sponsor : ITF Therapeutics

                            Deal Size : Undisclosed

                            Deal Type : Partnership

                            DEALS_DEV arrow-down

                            PANTHERx®Rare Partnering with ITF Therapeutics LLC for U.S. Distribution of DUVYZAT™

                            Details : PANTHERx partners with ITF for the U.S. distribution of Duvyzat (givinostat), the approved product to treat patients with all genetic variants of Duchenne muscular dystrophy aged six years and older.

                            Brand Name : Duvyzat

                            Molecule Type : Small molecule

                            Upfront Cash : Undisclosed

                            July 25, 2024

                            Lead Product(s) : Givinostat

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Approved

                            Sponsor : ITF Therapeutics

                            Deal Size : Undisclosed

                            Deal Type : Partnership

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                            03

                            PANTHERx Rare Pharmacy

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                            Medlab Asia & Asia Health
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                            Medlab Asia & Asia Health
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                            Lead Product(s) : Mavorixafor

                            Therapeutic Area : Genetic Disease

                            Study Phase : Approved

                            Sponsor : X4 Pharmaceuticals

                            Deal Size : Undisclosed

                            Deal Type : Partnership

                            DEALS_DEV arrow-down

                            PANTHERx Partners with X4 for XOLREMDI™ (mavorixafor) in WHIM Syndrome

                            Details : X4 Pharmaceuticals will distribute Xolremdi (mavorixafor), FDA-approved for patients 12 years and older with warts, hypogammaglobulinemia, infections, and myelokathexis syndrome.

                            Brand Name : Xolremdi

                            Molecule Type : Small molecule

                            Upfront Cash : Undisclosed

                            April 29, 2024

                            Lead Product(s) : Mavorixafor

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Approved

                            Sponsor : X4 Pharmaceuticals

                            Deal Size : Undisclosed

                            Deal Type : Partnership

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                            04

                            PANTHERx Rare Pharmacy

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                            Medlab Asia & Asia Health
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                            PANTHERx Rare Pharmacy

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                            Medlab Asia & Asia Health
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                            Lead Product(s) : Leniolisib

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Approved

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            PANTHERx® Rare Launches Drug for APDS

                            Details : Joenja (leniolisib) is an oral, selective PI3K-delta inhibitor which inhibits the signalling pathways that lead to increased production of PIP3, hyperactivity of the downstream mTOR/AKT pathway, and to the dysregulation of B and T cells in patients with ...

                            Brand Name : Joenja

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            March 28, 2023

                            Lead Product(s) : Leniolisib

                            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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                            05

                            PANTHERx Rare Pharmacy

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                            Medlab Asia & Asia Health
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                            PANTHERx Rare Pharmacy

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                            Medlab Asia & Asia Health
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                            Lead Product(s) : Stiripentol

                            Therapeutic Area : Neurology

                            Study Phase : Approved

                            Recipient : Biocodex

                            Deal Size : Undisclosed

                            Deal Type : Partnership

                            DEALS_DEV arrow-down

                            PANTHERx Rare Announces Partnership with Biocodex for the Distribution of DIACOMIT® (stiripentol) for the Tre...

                            Details : DIACOMIT (stiripentol), is a molecular entity approved by the FDA for the adjunctive treatment of seizures associated with Dravet syndrome in 2 years of age and older taking clobazam. An orally administered, DIACOMIT is given as either a capsule or powde...

                            Brand Name : Diacomit

                            Molecule Type : Small molecule

                            Upfront Cash : Undisclosed

                            June 27, 2022

                            Lead Product(s) : Stiripentol

                            Therapeutic Area : Neurology

                            Highest Development Status : Approved

                            Recipient : Biocodex

                            Deal Size : Undisclosed

                            Deal Type : Partnership

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                            06

                            PANTHERx Rare Pharmacy

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                            Medlab Asia & Asia Health
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                            PANTHERx Rare Pharmacy

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                            Medlab Asia & Asia Health
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                            Lead Product(s) : Lumasiran

                            Therapeutic Area : Genetic Disease

                            Study Phase : Approved

                            Sponsor : Alnylam Pharmaceuticals

                            Deal Size : Undisclosed

                            Deal Type : Partnership

                            DEALS_DEV arrow-down

                            PANTHERx Rare® Pharmacy Chosen as Partner by Alnylam for OXLUMO to Treat Primary Hyperoxaluria Type 1 to Lowe...

                            Details : PANTHERx® Rare Pharmacy has been selected by Alnylam Pharmaceuticals as a very limited distribution pharmacy partner for new drug OXLUMO™ (lumasiran). OXLUMO is a subcutaneously administered RNAi therapeutic that targets the messenger RNA of the hydro...

                            Brand Name : Oxlumo

                            Molecule Type : Large molecule

                            Upfront Cash : Undisclosed

                            December 03, 2020

                            Lead Product(s) : Lumasiran

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Approved

                            Sponsor : Alnylam Pharmaceuticals

                            Deal Size : Undisclosed

                            Deal Type : Partnership

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                            07

                            PANTHERx Rare Pharmacy

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                            Medlab Asia & Asia Health
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                            PANTHERx Rare Pharmacy

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                            Medlab Asia & Asia Health
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                            Lead Product(s) : Pralsetinib

                            Therapeutic Area : Oncology

                            Study Phase : Approved

                            Sponsor : Blueprint Medicines

                            Deal Size : Undisclosed

                            Deal Type : Agreement

                            DEALS_DEV arrow-down

                            PANTHERx® Rare Pharmacy Selected by Blueprint Medicines to Distribute GAVRETO™ for Metastatic RET Fusion-Po...

                            Details : PANTHERx® Rare Pharmacy has been selected by Blueprint Medicines as a limited distribution partner for new drug GAVRETO™ (pralsetinib), which was granted ­accelerated approval for the treatment of adult patients with metastatic RET fusion-positive no...

                            Brand Name : Gavreto

                            Molecule Type : Small molecule

                            Upfront Cash : Undisclosed

                            September 14, 2020

                            Lead Product(s) : Pralsetinib

                            Therapeutic Area : Oncology

                            Highest Development Status : Approved

                            Sponsor : Blueprint Medicines

                            Deal Size : Undisclosed

                            Deal Type : Agreement

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                            08

                            PANTHERx Rare Pharmacy

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                            Medlab Asia & Asia Health
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                            PANTHERx Rare Pharmacy

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                            Lead Product(s) : Avapritinib

                            Therapeutic Area : Oncology

                            Study Phase : Approved

                            Sponsor : Blueprint Medicines

                            Deal Size : Undisclosed

                            Deal Type : Partnership

                            DEALS_DEV arrow-down

                            PANTHERx® Rare Pharmacy Selected by Blueprint Medicines Corporation to Distribute AYVAKIT™ (avapritinib) fo...

                            Details : With nationwide distribution of AYVAKIT, PANTHERx establishes a formidable presence in the precision oncology space.

                            Brand Name : Undisclosed

                            Molecule Type : Small molecule

                            Upfront Cash : Undisclosed

                            January 17, 2020

                            Lead Product(s) : Avapritinib

                            Therapeutic Area : Oncology

                            Highest Development Status : Approved

                            Sponsor : Blueprint Medicines

                            Deal Size : Undisclosed

                            Deal Type : Partnership

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