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Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Lotion","sponsorNew":"Quoin Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Quoin Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Quoin Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Miscellaneous","year":"2022","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":"Lotion","sponsorNew":"Quoin Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Quoin Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Quoin 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Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Lotion","sponsorNew":"Quoin Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Quoin Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Quoin Pharmaceuticals","sponsor":"Maxim Group LLC","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Public Offering","leadProduct":"QRX003","moa":"Serine protease","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Quoin Pharmaceuticals","amount2":0.01,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0.01,"dosageForm":"Lotion","sponsorNew":"Quoin Pharmaceuticals \/ Maxim Group LLC","highestDevelopmentStatusID":"9","companyTruncated":"Quoin Pharmaceuticals \/ Maxim Group 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Undisclosed"},{"orgOrder":0,"company":"Quoin Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Miscellaneous","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":"Lotion","sponsorNew":"Quoin Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Quoin Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Quoin Pharmaceuticals","sponsor":"Endo Ventures Limited","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Agreement","leadProduct":"QRX003","moa":"Serine protease","graph1":"Rare Diseases and 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Phase III","graph3":"Quoin Pharmaceuticals","amount2":0.10000000000000001,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0.10000000000000001,"dosageForm":"Lotion","sponsorNew":"Quoin Pharmaceuticals \/ AIGH Capital Management | Soleus Capital | Nantahala Capital","highestDevelopmentStatusID":"9","companyTruncated":"Quoin Pharmaceuticals \/ AIGH Capital Management | Soleus Capital | Nantahala Capital"}]

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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 : Undisclosed

                          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

                          February 09, 2022

                          Lead Product(s) : Olipudase Alpha

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Undisclosed

                          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 : Undisclosed

                          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

                          December 06, 2021

                          Lead Product(s) : Olipudase Alpha

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Undisclosed

                          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 : Undisclosed

                          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 : Undisclosed

                          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 : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : Caplacizumab is a Antibody drug candidate, which is currently being evaluated in phase II/ phase III clinical studies for the treatment of Purpura, Thrombotic Thrombocytopenic.

                          Product Name : Undisclosed

                          Product Type : Antibody, Unconjugated

                          Upfront Cash : Inapplicable

                          August 29, 2019

                          Lead Product(s) : Caplacizumab

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Undisclosed

                          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 : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : Olipudase Alfa is a Enzyme drug candidate, which is currently being evaluated in phase II/ phase III clinical studies for the treatment of Niemann-Pick Disease, Type A.

                          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 : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          06

                          OTS 2025
                          Not Confirmed
                          OTS 2025
                          Not Confirmed

                          Details : In a private placement, QRX003, a serine protease for Netherton Syndrome, secures funding to advance research and development efforts.

                          Product Name : QRX003

                          Product Type : Miscellaneous

                          Upfront Cash : $16.5 million

                          October 10, 2025

                          Lead Product(s) : QRX003

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : AIGH Capital Management | Soleus Capital | Nantahala Capital

                          Deal Size : $104.5 million

                          Deal Type : Private Placement

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                          07

                          OTS 2025
                          Not Confirmed
                          OTS 2025
                          Not Confirmed

                          Details : Avacopan is a Small Molecule drug candidate drug candidate, which is currently being evaluated in Phase II/ Phase III clinical studies for the treatment of Granulomatosis with Polyangiitis.

                          Product Name : Undisclosed

                          Product Type : Miscellaneous

                          Upfront Cash : Inapplicable

                          September 16, 2025

                          Lead Product(s) : Avacopan

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          blank

                          08

                          OTS 2025
                          Not Confirmed
                          OTS 2025
                          Not Confirmed

                          Details : QRX003 is a Other Small Molecule drug candidate, which is currently being evaluated in phase II/ phase III clinical studies for the treatment of Netherton Syndrome.

                          Product Name : Undisclosed

                          Product Type : Miscellaneous

                          Upfront Cash : Inapplicable

                          May 01, 2025

                          Lead Product(s) : QRX003

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          09

                          OTS 2025
                          Not Confirmed
                          OTS 2025
                          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, Unconjugated

                          Upfront Cash : Inapplicable

                          April 29, 2025

                          Lead Product(s) : Elamipretide

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          10

                          OTS 2025
                          Not Confirmed
                          OTS 2025
                          Not Confirmed

                          Details : MY008211A is a Other Small Molecule drug candidate, which is currently being evaluated in phase II/ phase III clinical studies for the treatment of Hemoglobinuria, Paroxysmal.

                          Product Name : Undisclosed

                          Product Type : Miscellaneous

                          Upfront Cash : Inapplicable

                          April 18, 2025

                          Lead Product(s) : MY008211A

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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