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    [{"orgOrder":0,"company":"PANTHERx Rare Pharmacy","sponsor":"Alnylam Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2020","type":"Partnership","leadProduct":"Lumasiran","moa":"Glycolate oxidase","graph1":"Rare Diseases and Disorders","graph2":"Approved FDF","graph3":"PANTHERx Rare Pharmacy","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"PANTHERx Rare Pharmacy \/ Alnylam Pharmaceuticals","highestDevelopmentStatusID":"15","companyTruncated":"PANTHERx Rare Pharmacy \/ Alnylam Pharmaceuticals"},{"orgOrder":0,"company":"PANTHERx Rare Pharmacy","sponsor":"Blueprint Medicines","pharmaFlowCategory":"D","therapeuticArea":"Oncology","country":"U.S.A","productType":"Other Small Molecule","year":"2020","type":"Agreement","leadProduct":"Pralsetinib","moa":"RET receptor","graph1":"Oncology","graph2":"Approved FDF","graph3":"PANTHERx Rare Pharmacy","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Oncology","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"PANTHERx Rare Pharmacy \/ Blueprint Medicines","highestDevelopmentStatusID":"15","companyTruncated":"PANTHERx Rare Pharmacy \/ Blueprint Medicines"},{"orgOrder":0,"company":"PANTHERx Rare Pharmacy","sponsor":"Gilead Sciences","pharmaFlowCategory":"D","therapeuticArea":"Hepatology (Liver, Pancreatic, Gall Bladder)","country":"U.S.A","productType":"Other Small Molecule","year":"2024","type":"Partnership","leadProduct":"Seladelpar Lysine","moa":"PPA-delta receptor","graph1":"Hepatology (Liver, Pancreatic, Gall Bladder)","graph2":"Approved FDF","graph3":"PANTHERx Rare Pharmacy","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Hepatology","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"PANTHERx Rare Pharmacy \/ Gilead Sciences","highestDevelopmentStatusID":"15","companyTruncated":"PANTHERx Rare Pharmacy \/ Gilead Sciences"},{"orgOrder":0,"company":"PANTHERx Rare Pharmacy","sponsor":"ITF Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2024","type":"Partnership","leadProduct":"Givinostat HCl","moa":"HDAC","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PANTHERx Rare Pharmacy","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Suspension","sponsorNew":"PANTHERx Rare Pharmacy \/ ITF Therapeutics","highestDevelopmentStatusID":"15","companyTruncated":"PANTHERx Rare Pharmacy \/ ITF Therapeutics"},{"orgOrder":0,"company":"PANTHERx Rare Pharmacy","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Small Molecule","year":"2023","type":"Inapplicable","leadProduct":"Leniolisib Phosphate","moa":"PI3K delta","graph1":"Rare Diseases and Disorders","graph2":"Approved FDF","graph3":"PANTHERx Rare Pharmacy","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral Tablet, Film Coated","sponsorNew":"PANTHERx Rare Pharmacy \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"PANTHERx Rare Pharmacy \/ Inapplicable"},{"orgOrder":0,"company":"PANTHERx Rare Pharmacy","sponsor":"Blueprint Medicines","pharmaFlowCategory":"D","therapeuticArea":"Gastroenterology","country":"U.S.A","productType":"Other Small Molecule","year":"2020","type":"Partnership","leadProduct":"Avapritinib","moa":"PDGFA receptor","graph1":"Gastroenterology","graph2":"Approved FDF","graph3":"PANTHERx Rare Pharmacy","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Gastroenterology","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"PANTHERx Rare Pharmacy \/ Blueprint Medicines","highestDevelopmentStatusID":"15","companyTruncated":"PANTHERx Rare Pharmacy \/ Blueprint Medicines"},{"orgOrder":0,"company":"PANTHERx Rare Pharmacy","sponsor":"X4 Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Small Molecule","year":"2024","type":"Partnership","leadProduct":"Mavorixafor","moa":"CXCR-4 receptor","graph1":"Rare Diseases and Disorders","graph2":"Approved FDF","graph3":"PANTHERx Rare Pharmacy","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"PANTHERx Rare Pharmacy \/ X4 Pharmaceuticals","highestDevelopmentStatusID":"15","companyTruncated":"PANTHERx Rare Pharmacy \/ X4 Pharmaceuticals"}]

    Find Clinical Drug Pipeline Developments & Deals by PANTHERx Rare Pharmacy

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                            01

                            PANTHERx Rare Pharmacy

                            U.S.A arrow
                            STLE Annual Meeting
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                            PANTHERx Rare Pharmacy

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                            STLE Annual Meeting
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                            Lead Product(s) : Seladelpar Lysine

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

                            Study Phase : Approved FDF

                            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).

                            Product Name : Livdelzi

                            Product Type : Other Small Molecule

                            Upfront Cash : Undisclosed

                            August 16, 2024

                            Lead Product(s) : Seladelpar Lysine

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

                            Highest Development Status : Approved FDF

                            Sponsor : Gilead Sciences

                            Deal Size : Undisclosed

                            Deal Type : Partnership

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                            02

                            PANTHERx Rare Pharmacy

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                            STLE Annual Meeting
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                            PANTHERx Rare Pharmacy

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                            STLE Annual Meeting
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                            Lead Product(s) : Givinostat HCl

                            Therapeutic Area : Genetic Disease

                            Study Phase : Approved FDF

                            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.

                            Product Name : Duvyzat

                            Product Type : Other Small Molecule

                            Upfront Cash : Undisclosed

                            July 25, 2024

                            Lead Product(s) : Givinostat HCl

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Approved FDF

                            Sponsor : ITF Therapeutics

                            Deal Size : Undisclosed

                            Deal Type : Partnership

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                            03

                            PANTHERx Rare Pharmacy

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                            STLE Annual Meeting
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                            STLE Annual Meeting
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                            Lead Product(s) : Mavorixafor

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Approved FDF

                            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.

                            Product Name : Xolremdi

                            Product Type : Other Small Molecule

                            Upfront Cash : Undisclosed

                            April 29, 2024

                            Lead Product(s) : Mavorixafor

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Approved FDF

                            Sponsor : X4 Pharmaceuticals

                            Deal Size : Undisclosed

                            Deal Type : Partnership

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                            04

                            PANTHERx Rare Pharmacy

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                            STLE Annual Meeting
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                            Lead Product(s) : Leniolisib Phosphate

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            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 ...

                            Product Name : Joenja

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            March 28, 2023

                            Lead Product(s) : Leniolisib Phosphate

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            05

                            PANTHERx Rare Pharmacy

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                            STLE Annual Meeting
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                            PANTHERx Rare Pharmacy

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                            STLE Annual Meeting
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                            Lead Product(s) : Pralsetinib

                            Therapeutic Area : Oncology

                            Study Phase : Approved FDF

                            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...

                            Product Name : Gavreto

                            Product Type : Other Small Molecule

                            Upfront Cash : Undisclosed

                            September 14, 2020

                            Lead Product(s) : Pralsetinib

                            Therapeutic Area : Oncology

                            Highest Development Status : Approved FDF

                            Sponsor : Blueprint Medicines

                            Deal Size : Undisclosed

                            Deal Type : Agreement

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                            06

                            PANTHERx Rare Pharmacy

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                            STLE Annual Meeting
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                            Lead Product(s) : Lumasiran

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Approved FDF

                            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...

                            Product Name : Oxlumo

                            Product Type : Oligonucleotide

                            Upfront Cash : Undisclosed

                            March 12, 2020

                            Lead Product(s) : Lumasiran

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Approved FDF

                            Sponsor : Alnylam Pharmaceuticals

                            Deal Size : Undisclosed

                            Deal Type : Partnership

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                            07

                            PANTHERx Rare Pharmacy

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                            STLE Annual Meeting
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                            Lead Product(s) : Avapritinib

                            Therapeutic Area : Gastroenterology

                            Study Phase : Approved FDF

                            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.

                            Product Name : Undisclosed

                            Product Type : Other Small Molecule

                            Upfront Cash : Undisclosed

                            January 17, 2020

                            Lead Product(s) : Avapritinib

                            Therapeutic Area : Gastroenterology

                            Highest Development Status : Approved FDF

                            Sponsor : Blueprint Medicines

                            Deal Size : Undisclosed

                            Deal Type : Partnership

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