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                            Virtual BoothSanofi is a pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme.

                            Lead Product(s) : Caplacizumab

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase III

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                            Caplacizumab and Immunosuppressive Therapy Without Firstline Therapeutic Plasma Exchange in Adults With Immune...

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                            July 21, 2022

                            Lead Product(s) : Caplacizumab

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                            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) : Adalimumab

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase III

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                            A Study Assessing the Efficacy and Safety of Adalimumab in Active Ulcer(s) of Pyoderma Gangrenosum in Particip...

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                            October 17, 2017

                            Lead Product(s) : Adalimumab

                            Therapeutic Area : Rare Diseases and Disorders

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                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : Adalimumab

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase III

                            Sponsor : Eisai

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            A Study of Adalimumab in Japanese Subjects With Intestinal Behçet's Disease

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Antibody

                            Upfront Cash : Inapplicable

                            November 18, 2010

                            Lead Product(s) : Adalimumab

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase III

                            Sponsor : Eisai

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            04

                            Azafaros

                            Netherlands arrow
                            ESCRS
                            Not Confirmed

                            Azafaros

                            Netherlands arrow
                            ESCRS
                            Not Confirmed
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                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : AZ-3102

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Nizubaglustat Safety and Efficacy In GM1/GM2 Gangliosidosis Pediatric Patients

                            Details : AZ-3102 is a Other Small Molecule drug candidate, which is currently being evaluated in Phase III clinical studies for the treatment of Gangliosidoses, GM2.

                            Product Name : Undisclosed

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            July 24, 2025

                            Lead Product(s) : AZ-3102

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            05

                            Createrna Science and Technology

                            China arrow
                            ESCRS
                            Not Confirmed

                            Createrna Science and Technology

                            China arrow
                            ESCRS
                            Not Confirmed
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                            Lead Product(s) : MY008211A

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Study of Safety and Efficacy of MY008211A in Patients With Residual Anemia Despite Anti-C5 Antibody Treatment

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            April 17, 2025

                            Lead Product(s) : MY008211A

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            06

                            Createrna Science and Technology

                            China arrow
                            ESCRS
                            Not Confirmed

                            Createrna Science and Technology

                            China arrow
                            ESCRS
                            Not Confirmed
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                            Lead Product(s) : MY008211A

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Study of Safety and Efficacy of MY008211A in Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients Who Are Naive ...

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            April 17, 2025

                            Lead Product(s) : MY008211A

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            07

                            Palvella Therapeutics

                            U.S.A arrow
                            ESCRS
                            Not Confirmed

                            Palvella Therapeutics

                            U.S.A arrow
                            ESCRS
                            Not Confirmed
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                            • EDQM
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                            Lead Product(s) : Sirolimus

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Palvella Expands Phase 3 SELVA Trial of Qtorin Rapamycin Gel to Younger Kids

                            Details : Qtorin rapamycin has the potential to be the first approved therapy and standard of care in the U.S. for the treatment of microcystic lymphatic malformations.

                            Product Name : Qtorin Rapamycin

                            Product Type : Antibiotic

                            Upfront Cash : Inapplicable

                            February 10, 2025

                            Lead Product(s) : Sirolimus

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            08

                            AB2 Bio

                            Switzerland arrow
                            ESCRS
                            Not Confirmed

                            AB2 Bio

                            Switzerland arrow
                            ESCRS
                            Not Confirmed
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                            Lead Product(s) : Tadekinig Alpha

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase III

                            Sponsor : Nippon Shinyaku

                            Deal Size : $686.0 million

                            Deal Type : Licensing Agreement

                            DEALS_DEV arrow-down

                            AB2 Bio Signs U.S. Option and Licensing Agreement with Nippon Shinyaku for Tadekinig Alfa

                            Details : Under the licensing agreement, Nippon received an option to acquire exclusive U.S. rights to commercialize r-hIL-18BP (tadekinig alfa) to treat NLRC4-MAS.

                            Product Name : Undisclosed

                            Product Type : Protein

                            Upfront Cash : $6.0 million

                            January 27, 2025

                            Lead Product(s) : Tadekinig Alpha

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase III

                            Sponsor : Nippon Shinyaku

                            Deal Size : $686.0 million

                            Deal Type : Licensing Agreement

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                            09

                            Chigenovo

                            Country arrow
                            ESCRS
                            Not Confirmed

                            Chigenovo

                            Country arrow
                            ESCRS
                            Not Confirmed
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                            Lead Product(s) : ZVS101e

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Efficacy and Safety of ZVS101e in Patients With Bietti 's Crystalline Dystrophy

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            December 20, 2024

                            Lead Product(s) : ZVS101e

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            10

                            Ultragenyx Pharmaceutical

                            U.S.A arrow
                            ESCRS
                            Not Confirmed

                            Ultragenyx Pharmaceutical

                            U.S.A arrow
                            ESCRS
                            Not Confirmed
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                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : Betamethasone

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Ultragenyx Announces First Patient Dosed in Ph 3 Study for GTX-102 in Angelman Syndrome

                            Details : GTX-102 is an investigational antisense oligonucleotide therapy designed to inhibit the expression of UBE3A, currently being evaluated for the treatment of angelman syndrome.

                            Product Name : Undisclosed

                            Product Type : Steroid

                            Upfront Cash : Inapplicable

                            December 19, 2024

                            Lead Product(s) : Betamethasone

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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