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    [{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"Delandistrogene Moxeparvovec","moa":"","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Sarepta Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"15","companyTruncated":"Sarepta Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"Delandistrogene Moxeparvovec","moa":"Micro-dystrophin gene","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"15","companyTruncated":"Sarepta Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"F. Hoffmann-La Roche","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"Delandistrogene Moxeparvovec","moa":"Micro-dystrophin gene","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Roche","highestDevelopmentStatusID":"15","companyTruncated":"Sarepta Therapeutics \/ Roche"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"F. Hoffmann-La Roche","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"Delandistrogene Moxeparvovec","moa":"Micro-dystrophin gene","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Roche","highestDevelopmentStatusID":"15","companyTruncated":"Sarepta Therapeutics \/ Roche"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"Delandistrogene Moxeparvovec","moa":"","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"15","companyTruncated":"Sarepta Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"Delandistrogene Moxeparvovec","moa":"","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"15","companyTruncated":"Sarepta Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"Delandistrogene Moxeparvovec","moa":"","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"15","companyTruncated":"Sarepta Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"Delandistrogene Moxeparvovec","moa":"","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"15","companyTruncated":"Sarepta Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"Delandistrogene Moxeparvovec","moa":"","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"15","companyTruncated":"Sarepta Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"Delandistrogene Moxeparvovec","moa":"","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"15","companyTruncated":"Sarepta Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Teva Pharmaceutical Industries","sponsor":"Eton Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Other Small Molecule","year":"2025","type":"Acquisition","leadProduct":"Zinc Acetate Dihydrate","moa":"","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Teva Pharmaceutical Industries","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Teva Pharmaceutical Industries \/ Eton Pharmaceuticals","highestDevelopmentStatusID":"15","companyTruncated":"Teva Pharmaceutical Industries \/ Eton Pharmaceuticals"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"Clinigen Group","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Steroid","year":"2025","type":"Agreement","leadProduct":"Vamorolone","moa":"Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Santhera Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Santhera Pharmaceuticals \/ Clinigen Group","highestDevelopmentStatusID":"15","companyTruncated":"Santhera Pharmaceuticals \/ Clinigen Group"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody","year":"2025","type":"Inapplicable","leadProduct":"Evinacumab","moa":"ANGPTL3","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Ultragenyx Pharmaceutical \/ Inapplicable"},{"orgOrder":0,"company":"Acadia Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide","year":"2025","type":"Inapplicable","leadProduct":"Trofinetide","moa":"OATP1B1\/OATP1B3\/CYP3A4","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Acadia Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Acadia Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Acadia Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"X4 Pharmaceuticals","sponsor":"Norgine","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2025","type":"Licensing Agreement","leadProduct":"Mavorixafor","moa":"CXCR4","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"X4 Pharmaceuticals","amount2":0.26000000000000001,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0.26000000000000001,"dosageForm":"Capsule","sponsorNew":"X4 Pharmaceuticals \/ Norgine","highestDevelopmentStatusID":"15","companyTruncated":"X4 Pharmaceuticals \/ Norgine"},{"orgOrder":0,"company":"Idorsia Pharmaceuticals","sponsor":"R-Bridge Healthcare Fund","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Steroid","year":"2025","type":"Agreement","leadProduct":"Vamorolone","moa":"Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Idorsia Pharmaceuticals","amount2":0.029999999999999999,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0.029999999999999999,"dosageForm":"Suspension","sponsorNew":"Idorsia Pharmaceuticals \/ R-Bridge Healthcare Fund","highestDevelopmentStatusID":"15","companyTruncated":"Idorsia Pharmaceuticals \/ R-Bridge Healthcare Fund"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Steroid","year":"2025","type":"Inapplicable","leadProduct":"Vamorolone","moa":"Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Santhera Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Santhera Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Santhera Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Lupin Ltd","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"INDIA","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"Ivacaftor","moa":"CFTR","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Lupin Ltd","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Granule","sponsorNew":"Lupin Ltd \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Lupin Ltd \/ Inapplicable"},{"orgOrder":0,"company":"X4 Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"Mavorixafor","moa":"CXCR4","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"X4 Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"X4 Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"X4 Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2025","type":"Inapplicable","leadProduct":"Delandistrogene Moxeparvovec","moa":"Micro-dystrophin","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Sarepta Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Sarepta Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Lupin Ltd","sponsor":"Avas Pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"INDIA","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"Mexiletine","moa":"Sodium channel","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Lupin Ltd","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Lupin Ltd \/ Avas Pharmaceuticals","highestDevelopmentStatusID":"15","companyTruncated":"Lupin Ltd \/ Avas Pharmaceuticals"},{"orgOrder":0,"company":"CSL Behring","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2025","type":"Inapplicable","leadProduct":"Etranacogene Dezaparvovec","moa":"hFIX Padua gene","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"CSL Behring","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Suspension for Infusion","sponsorNew":"CSL Behring \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"CSL Behring \/ Inapplicable"}]

    Find Clinical Drug Development Pipelines & Deals | PipelineProspector

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                            01

                            Lupin Ltd

                            India arrow
                            BIO CEO & Investor
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                            Lupin Ltd

                            India arrow
                            BIO CEO & Investor
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                            Lead Product(s) : Mexiletine

                            Therapeutic Area : Genetic Disease

                            Study Phase : Approved FDF

                            Sponsor : Avas Pharmaceuticals

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Lupin and Avas Launch NaMuscla in Italy

                            Details : NaMuscla (mexiletine hydrochloride) is a Sodium channel alpha subunit blocker, being evaluated for the symptomatic treatment of myotonia in adults with non-dystrophic myotonic (NDM) disorders.

                            Product Name : Namuscla

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            January 28, 2025

                            Lead Product(s) : Mexiletine

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Approved FDF

                            Sponsor : Avas Pharmaceuticals

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            02

                            Sarepta Therapeutics

                            U.S.A arrow
                            RNA Therapeutics
                            Not Confirmed

                            Sarepta Therapeutics

                            U.S.A arrow
                            RNA Therapeutics
                            Not Confirmed
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                            Lead Product(s) : Delandistrogene Moxeparvovec

                            Therapeutic Area : Genetic Disease

                            Study Phase : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Sarepta’s Embark Part 2 Shows ELEVIDYS Benefits for DMD

                            Details : Elevidys (delandistrogene moxeparvovec) is a single-dose gene transfer therapy, designed to to treat Duchenne muscular dystrophy by producing micro-dystrophin in skeletal muscle.

                            Product Name : Elevidys

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            January 27, 2025

                            Lead Product(s) : Delandistrogene Moxeparvovec

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            03

                            X4 Pharmaceuticals

                            U.S.A arrow
                            BIO CEO & Investor
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                            X4 Pharmaceuticals

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                            Lead Product(s) : Mavorixafor

                            Therapeutic Area : Genetic Disease

                            Study Phase : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            X4 Pharmaceuticals Announces EMA Validation of Mavorixafor for WHIM Syndrome

                            Details : Xolremdi (mavorixafor) is a selective CXC chemokine receptor 4 (CXCR4) antagonist, which is FDA approved for the treatment of WHIM syndrome in patients 12 years of age and older.

                            Product Name : Xolremdi

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            January 24, 2025

                            Lead Product(s) : Mavorixafor

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            04

                            CSL Behring

                            U.S.A arrow
                            RNA Therapeutics
                            Not Confirmed

                            CSL Behring

                            U.S.A arrow
                            RNA Therapeutics
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : Etranacogene Dezaparvovec

                            Therapeutic Area : Genetic Disease

                            Study Phase : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            First Patients Receive Hemgenix Gene Therapy for Hemophilia B in Denmark

                            Details : Hemgenix (etranacogene dezaparvovec-drlb) is an AAV5 based gene therapy designed to deliver a copy of a gene encoding hFIX-Padua in patients with Hemophilia B.

                            Product Name : Hemgenix

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            January 21, 2025

                            Lead Product(s) : Etranacogene Dezaparvovec

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            05

                            Idorsia Pharmaceuticals

                            Switzerland arrow
                            RNA Therapeutics
                            Not Confirmed

                            Idorsia Pharmaceuticals

                            Switzerland arrow
                            RNA Therapeutics
                            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) : Vamorolone

                            Therapeutic Area : Genetic Disease

                            Study Phase : Approved FDF

                            Sponsor : R-Bridge Healthcare Fund

                            Deal Size : $30.0 million

                            Deal Type : Agreement

                            DEALS_DEV arrow-down

                            Santhera Pharmaceuticals Updates Royalty Agreement for AGAMREE® (Vamorolone)

                            Details : Idorsia transfers rights to royalties and milestones for Agamree (vamorolone), a dissociative steroid with novel mode of action in patients with Duchenne muscular dystrophy, to R-Bridge Healthcare.

                            Product Name : Agamree

                            Product Type : Steroid

                            Upfront Cash : Undisclosed

                            January 16, 2025

                            Lead Product(s) : Vamorolone

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Approved FDF

                            Sponsor : R-Bridge Healthcare Fund

                            Deal Size : $30.0 million

                            Deal Type : Agreement

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                            06

                            Acadia Pharmaceuticals

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                            BIO CEO & Investor
                            Not Confirmed

                            Acadia Pharmaceuticals

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                            BIO CEO & Investor
                            Not Confirmed
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                            Lead Product(s) : Trofinetide

                            Therapeutic Area : Genetic Disease

                            Study Phase : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Acadia Pharmaceuticals Submits EMA Application for Trofinetide in Rett Syndrome

                            Details : Daybue (trofinetide) is a inhibitor of CYP3A4, indicated for Rett syndrome, a rare neurodevelopmental disorder in 2 year and older. It is already approved by FDA for the same indication.

                            Product Name : Daybue

                            Product Type : Peptide

                            Upfront Cash : Inapplicable

                            January 14, 2025

                            Lead Product(s) : Trofinetide

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            07

                            Santhera Pharmaceuticals

                            Switzerland arrow
                            BIO CEO & Investor
                            Not Confirmed

                            Santhera Pharmaceuticals

                            Switzerland arrow
                            BIO CEO & Investor
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : Vamorolone

                            Therapeutic Area : Genetic Disease

                            Study Phase : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Santhera Receives Positive Recommendation for AGAMREE® in DMD

                            Details : Agamree (vamorolone) is a novel drug candidate with a mode of action based on binding to the mineralcorticosteroid receptor, being developed for the treatment Duchenne muscular dystrophy.

                            Product Name : Agamree

                            Product Type : Steroid

                            Upfront Cash : Inapplicable

                            January 14, 2025

                            Lead Product(s) : Vamorolone

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            08

                            X4 Pharmaceuticals

                            U.S.A arrow
                            BIO CEO & Investor
                            Not Confirmed

                            X4 Pharmaceuticals

                            U.S.A arrow
                            BIO CEO & Investor
                            Not Confirmed
                            • fda
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                            • WHO-GMP
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                            Lead Product(s) : Mavorixafor

                            Therapeutic Area : Genetic Disease

                            Study Phase : Approved FDF

                            Sponsor : Norgine

                            Deal Size : $260.5 million

                            Deal Type : Licensing Agreement

                            DEALS_DEV arrow-down

                            X4 Pharmaceuticals, Norgine Sign License for Mavorixafor in Europe, Australia

                            Details : Under the licensing agreement, Norgine will hold exclusive rights to commercialize Xolremdi (mavorixafor) in Europe, Australia, etc. It is being indicated for the treatment of WHIM syndrome.

                            Product Name : Xolremdi

                            Product Type : Other Small Molecule

                            Upfront Cash : $29.1 million

                            January 13, 2025

                            Lead Product(s) : Mavorixafor

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Approved FDF

                            Sponsor : Norgine

                            Deal Size : $260.5 million

                            Deal Type : Licensing Agreement

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                            09

                            Lupin Ltd

                            India arrow
                            RNA Therapeutics
                            Not Confirmed

                            Lupin Ltd

                            India arrow
                            RNA Therapeutics
                            Not Confirmed
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                            Lead Product(s) : Ivacaftor

                            Therapeutic Area : Genetic Disease

                            Study Phase : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Lupin Secures Tentative U.S. FDA Approval for Ivacaftor Oral Granules

                            Details : Kalydeco-Generic (ivacaftor) is an oral, CFTR potentiator, small molecule drug candidate. It is now approved for the treatment of cystic fibrosis in patients aged 1 month and older.

                            Product Name : Kalydeco-Generic

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            January 10, 2025

                            Lead Product(s) : Ivacaftor

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            10

                            Santhera Pharmaceuticals

                            Switzerland arrow
                            BIO CEO & Investor
                            Not Confirmed

                            Santhera Pharmaceuticals

                            Switzerland arrow
                            BIO CEO & Investor
                            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) : Vamorolone

                            Therapeutic Area : Genetic Disease

                            Study Phase : Approved FDF

                            Sponsor : Clinigen Group

                            Deal Size : Undisclosed

                            Deal Type : Agreement

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                            Santhera, Clinigen Sign Supply, Distribution Deal for AGAMREE® (Vamorolone)

                            Details : Under the agreement, Clinigen Group will supply and distribute Agamree (vamorolone) in countries where the product is not otherwise commercially available.

                            Product Name : Agamree

                            Product Type : Steroid

                            Upfront Cash : Undisclosed

                            January 06, 2025

                            Lead Product(s) : Vamorolone

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Approved FDF

                            Sponsor : Clinigen Group

                            Deal Size : Undisclosed

                            Deal Type : Agreement

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