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Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"QRX003","moa":"Serine protease","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Quoin Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Topical Lotion","sponsorNew":"Quoin Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Quoin Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Quoin Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"QRX003","moa":"Serine protease","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase 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Inapplicable"},{"orgOrder":0,"company":"Retinagenix Holdings","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Undisclosed","year":"2020","type":"Inapplicable","leadProduct":"ZA","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Retinagenix Holdings","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Retinagenix Holdings \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Retinagenix Holdings \/ Inapplicable"},{"orgOrder":0,"company":"Shanghai Jiaolian Drug Research and Development Co., Ltd","sponsor":"Shanghai Pharmaceuticals Holding Co.,Ltd","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and 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                          Top Deals by Deal Size (USD bn)

                          01

                          Lead Product(s) : Olipudase Alpha

                          Therapeutic Area : Rare Diseases and Disorders

                          Study Phase : Phase II/ Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : Investigational data from long-term, follow-up studies showed that olipudase alfa provided sustained improvement in lung function (as measured by DLco) and reduction of spleen and liver volumes over time in patients with ASMD.

                          Product Name : Xenpozyme

                          Product Type : Enzyme

                          Upfront Cash : Inapplicable

                          September 02, 2022

                          Lead Product(s) : Olipudase Alpha

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          02

                          Lead Product(s) : Olipudase Alpha

                          Therapeutic Area : Rare Diseases and Disorders

                          Study Phase : Phase II/ Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : Olipudase alfa is an investigational enzyme replacement therapy designed to replace deficient or defective ASM, allowing for the breakdown of sphingomyelin.

                          Product Name : Xenpozyme

                          Product Type : Enzyme

                          Upfront Cash : Inapplicable

                          June 12, 2021

                          Lead Product(s) : Olipudase Alpha

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          03

                          Lead Product(s) : Olipudase Alpha

                          Therapeutic Area : Rare Diseases and Disorders

                          Study Phase : Phase II/ Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : Olipudase alfa is the first and only investigational enzyme replacement therapy in late-stage development for the treatment of ASMD.

                          Product Name : Xenpozyme

                          Product Type : Enzyme

                          Upfront Cash : Inapplicable

                          January 30, 2020

                          Lead Product(s) : Olipudase Alpha

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          04

                          Lead Product(s) : Caplacizumab

                          Therapeutic Area : Rare Diseases and Disorders

                          Study Phase : Phase II/ Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Antibody

                          Upfront Cash : Inapplicable

                          August 29, 2019

                          Lead Product(s) : Caplacizumab

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          05

                          Lead Product(s) : Olipudase Alfa

                          Therapeutic Area : Rare Diseases and Disorders

                          Study Phase : Phase II/ Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Enzyme

                          Upfront Cash : Inapplicable

                          December 09, 2013

                          Lead Product(s) : Olipudase Alfa

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          06

                          BIOHK2025
                          Not Confirmed
                          BIOHK2025
                          Not Confirmed

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          May 01, 2025

                          Lead Product(s) : QRX003

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          07

                          BIOHK2025
                          Not Confirmed
                          BIOHK2025
                          Not Confirmed

                          Details : MTP-131 (elamipretide) is a novel mitochondria-targeted drug that is targeted to cardiolipin, being investigated for the treatment of barth syndrome.

                          Product Name : Undisclosed

                          Product Type : Peptide

                          Upfront Cash : Inapplicable

                          April 29, 2025

                          Lead Product(s) : Elamipretide

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          08

                          BIOHK2025
                          Not Confirmed
                          BIOHK2025
                          Not Confirmed

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          April 18, 2025

                          Lead Product(s) : MY008211A

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          09

                          BIOHK2025
                          Not Confirmed
                          BIOHK2025
                          Not Confirmed

                          Details : QRX003 is a topical lotion, that contains a broad-spectrum serine protease inhibitor, being investigated for the treatment of pediatric netherton syndrome.

                          Product Name : Undisclosed

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          April 02, 2025

                          Lead Product(s) : QRX003

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          10

                          Biomed Industries

                          Country arrow
                          BIOHK2025
                          Not Confirmed

                          Biomed Industries

                          Country arrow
                          BIOHK2025
                          Not Confirmed

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Peptide

                          Upfront Cash : Inapplicable

                          February 27, 2025

                          Lead Product(s) : Bionetide

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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