[{"orgOrder":0,"company":"AstraZeneca","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"UNITED KINGDOM","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"Eculizumab","moa":"Completent C5","graph1":"Rare Diseases and Disorders","graph2":"Approved FDF","graph3":"AstraZeneca","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"AstraZeneca \/ Not Applicable","highestDevelopmentStatusID":"15","companyTruncated":"AstraZeneca \/ Not Applicable"},{"orgOrder":0,"company":"Samsung Bioepis","sponsor":"Teva Pharmaceutical Industries","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"SOUTH KOREA","productType":"Antibody","year":"2025","type":"Licensing Agreement","leadProduct":"Eculizumab","moa":"Complement C5a","graph1":"Rare Diseases and Disorders","graph2":"Approved FDF","graph3":"Samsung Bioepis","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Samsung Bioepis \/ Teva Pharmaceutical Industries","highestDevelopmentStatusID":"15","companyTruncated":"Samsung Bioepis \/ Teva Pharmaceutical Industries"},{"orgOrder":0,"company":"AstraZeneca","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"UNITED KINGDOM","productType":"Antibody","year":"2025","type":"Inapplicable","leadProduct":"Eculizumab","moa":"Complement C5a","graph1":"Rare Diseases and Disorders","graph2":"Approved FDF","graph3":"AstraZeneca","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"AstraZeneca \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"AstraZeneca \/ Inapplicable"},{"orgOrder":0,"company":"Breckenridge Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"Everolimus","moa":"mTOR","graph1":"Rare Diseases and Disorders","graph2":"Approved FDF","graph3":"Breckenridge Pharmaceutical","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral Tablet, Orally Disintegrating","sponsorNew":"Breckenridge Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Breckenridge Pharmaceutical \/ Inapplicable"},{"orgOrder":0,"company":"Merck & Co","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"Belzutifan","moa":"HIF-2 alpha","graph1":"Rare Diseases and Disorders","graph2":"Approved FDF","graph3":"Merck & Co","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral Tablet, Film Coated","sponsorNew":"Merck & Co \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Merck & Co \/ Inapplicable"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"Chenodeoxycholic Acid","moa":"Cholesterol synthesis","graph1":"Rare Diseases and Disorders","graph2":"Approved FDF","graph3":"Mirum Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral Tablet, Film Coated","sponsorNew":"Mirum Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Mirum Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Zevra Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Small Molecule","year":"2025","type":"Agreement","leadProduct":"Arimoclomol","moa":"||TFEB","graph1":"Rare Diseases and Disorders","graph2":"Approved FDF","graph3":"Zevra Therapeutics","amount2":0.14999999999999999,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Rare Diseases","amount2New":0.14999999999999999,"dosageForm":"Oral Capsule","sponsorNew":"Zevra Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Zevra Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Eton Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"Zinc Acetate Dihydrate","moa":"Intestinal cell metallothionein","graph1":"Rare Diseases and Disorders","graph2":"Approved FDF","graph3":"Eton Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Eton Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Eton Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Alembic Pharmaceuticals Limited","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"INDIA","productType":"HPAPI","year":"2020","type":"Inapplicable","leadProduct":"Bimatoprost","moa":"FP receptor","graph1":"Rare Diseases and Disorders","graph2":"Approved FDF","graph3":"Alembic Pharmaceuticals Limited","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Ophthalmic Solution","sponsorNew":"Alembic Pharmaceuticals Limited \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Alembic Pharmaceuticals Limited \/ Inapplicable"},{"orgOrder":0,"company":"Novo Nordisk","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"DENMARK","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"Nedosiran","moa":"LDH","graph1":"Rare Diseases and Disorders","graph2":"Approved FDF","graph3":"Novo Nordisk","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Novo Nordisk \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Novo Nordisk \/ Inapplicable"},{"orgOrder":0,"company":"Neurotech Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2025","type":"Inapplicable","leadProduct":"Revakinagene Taroretcel","moa":"CNTF","graph1":"Rare Diseases and Disorders","graph2":"Approved FDF","graph3":"Neurotech Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravitreal Implant","sponsorNew":"Neurotech Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Neurotech Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Lupin Ltd","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"INDIA","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"Amifampridine Phosphate","moa":"Potassium channel","graph1":"Rare Diseases and Disorders","graph2":"Approved FDF","graph3":"Lupin Ltd","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"Lupin Ltd \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Lupin Ltd \/ Inapplicable"}]

Find Clinical Drug Development Pipelines & Deals | PipelineProspector

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                          01

                          Details : Afinitor Disperz (everolimus) is an mTOR inhibitor small molecule drug candidate, which is currently being evaluated for the treatment of tuberous sclerosis complex.

                          Product Name : Afinitor Disperz-Generic

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          January 29, 2025

                          Lead Product(s) : Everolimus

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Approved FDF

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Breckenridge Pharmaceutical

                          02

                          ASPEN
                          Not Confirmed
                          ASPEN
                          Not Confirmed

                          Details : Firdapse-Generic (amifampridine) is an oral, nonspecific, voltage-dependent, potassium channel blocker that is indicated to treat Lambert-Eaton myasthenic syndrome.

                          Product Name : Firdapse-Generic

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          March 17, 2025

                          Lead Product(s) : Amifampridine Phosphate

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Approved FDF

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          03

                          ASPEN
                          Not Confirmed
                          ASPEN
                          Not Confirmed

                          Details : Encelto (revakinagene taroretcel) leverages is a gene therapy which delivers CNTF through an ocular implant, it is approved for the treatment of chronic retinal diseases, such as MacTel.

                          Product Name : Encelto

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          March 06, 2025

                          Lead Product(s) : Revakinagene Taroretcel

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Approved FDF

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          04

                          ASPEN
                          Not Confirmed
                          ASPEN
                          Not Confirmed

                          Details : Galzin (zinc acetate) is FDA-approved for the maintenance treatment of patients with Wilson disease who have been initially treated with a chelating agent.

                          Product Name : Galzin

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          March 03, 2025

                          Lead Product(s) : Zinc Acetate Dihydrate

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Approved FDF

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          05

                          ASPEN
                          Not Confirmed
                          ASPEN
                          Not Confirmed

                          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 : Other Small Molecule

                          Upfront Cash : Undisclosed

                          February 27, 2025

                          Lead Product(s) : Arimoclomol,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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                          06

                          ASPEN
                          Not Confirmed
                          ASPEN
                          Not Confirmed

                          Details : Ctexli (chenodiol) is an oral cholesterol synthesis inhibitor which is being evaluated for the treatment of cerebrotendinous xanthomatosis.

                          Product Name : Ctexli

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          February 21, 2025

                          Lead Product(s) : Chenodeoxycholic Acid

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Approved FDF

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          07

                          ASPEN
                          Not Confirmed
                          ASPEN
                          Not Confirmed

                          Details : Welireg (belzutifan), an oral HIF-2α inhibitor, being evaluated for the treatment of adult and pediatric patients with advanced, unresectable, or metastatic pheochromocytoma and paraganglioma.

                          Product Name : Welireg

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          February 18, 2025

                          Lead Product(s) : Belzutifan

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Approved FDF

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          08

                          ASPEN
                          Not Confirmed
                          ASPEN
                          Not Confirmed

                          Details : Soliris (eculizumab) is a complement C5 inhibitor antibody which is indicated to treat paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome.

                          Product Name : Soliris

                          Product Type : Antibody

                          Upfront Cash : Inapplicable

                          January 17, 2025

                          Lead Product(s) : Eculizumab

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Approved FDF

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          09

                          ASPEN
                          Not Confirmed
                          ASPEN
                          Not Confirmed

                          Details : Under the licensing agreement, Teva will be responsible for the commercialization of the product in the U.S. It is being indicated for the treatment of paroxysmal nocturnal hemoglobinuria.

                          Product Name : Epysqli

                          Product Type : Antibody

                          Upfront Cash : Undisclosed

                          January 10, 2025

                          Lead Product(s) : Eculizumab

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Approved FDF

                          Sponsor : Teva Pharmaceutical Industries

                          Deal Size : Undisclosed

                          Deal Type : Licensing Agreement

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                          10

                          ASPEN
                          Not Confirmed
                          ASPEN
                          Not Confirmed

                          Details : Rivfloza (nedosiran) is a once-monthly subcutaneous ribonucleic acid interference (RNAi) therapy indicated to lower urinary oxalate levels in children 9 years of age and older and adults with primary hyperoxaluria type 1 .

                          Product Name : Rivfloza

                          Product Type : Oligonucleotide

                          Upfront Cash : Inapplicable

                          October 02, 2023

                          Lead Product(s) : Nedosiran

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Approved FDF

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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