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                          01

                          Lead Product(s) : Avalglucosidase Alfa

                          Therapeutic Area : Rare Diseases and Disorders

                          Study Phase : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          Details : Nexviazyme (avalglucosidase alfa) is a USFDA approved GAA stimulator enzyme replacement therapy. It is being evaluated for the treatment of treatment-naïve Infantile-onset Pompe disease.

                          Brand Name : Nexviazyme

                          Molecule Type : Large molecule

                          Upfront Cash : Not Applicable

                          February 05, 2024

                          Lead Product(s) : Avalglucosidase Alfa

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          Sanofi Company Banner

                          02

                          Lead Product(s) : Olipudase Alfa-rpcp

                          Therapeutic Area : Rare Diseases and Disorders

                          Study Phase : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          Details : Xenpozyme, (olipudase Alfa-rpcp) hydrolytic lysosomal sphingomyelin-specific enzyme replacement therapy, is designed to replace deficient or defective acid sphingomyelinase, an enzyme that allows for the breakdown of the lipid sphingomyelin.

                          Brand Name : Xenpozyme

                          Molecule Type : Large molecule

                          Upfront Cash : Not Applicable

                          August 31, 2022

                          Lead Product(s) : Olipudase Alfa-rpcp

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          Sanofi Company Banner

                          03

                          Lead Product(s) : Olipudase Alfa

                          Therapeutic Area : Rare Diseases and Disorders

                          Study Phase : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          Details : Xenpozyme (Olipudase alfa), is an enzyme replacement therapy designed to replace deficient or defective acid sphingomyelinase (ASM), an enzyme that allows for the breakdown of the lipid sphingomyelin.

                          Brand Name : Xenpozyme

                          Molecule Type : Large molecule

                          Upfront Cash : Not Applicable

                          June 28, 2022

                          Lead Product(s) : Olipudase Alfa

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          Sanofi Company Banner

                          04

                          Lead Product(s) : Olipudase Alfa

                          Therapeutic Area : Rare Diseases and Disorders

                          Study Phase : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          Details : Recommendation based on positive results from two clinical trials in which Xenpozyme (olipudase alfa) provided improvement across multiple non-CNS clinical manifestations of ASMD in pediatric and adult patients.

                          Brand Name : Xenpozyme

                          Molecule Type : Large molecule

                          Upfront Cash : Not Applicable

                          May 19, 2022

                          Lead Product(s) : Olipudase Alfa

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          Sanofi Company Banner

                          05

                          Lead Product(s) : Olipudase Alfa

                          Therapeutic Area : Rare Diseases and Disorders

                          Study Phase : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          Details : Approval based on positive results of Xenpozyme® (olipudase alfa) from two separate clinical trials in children and adults demonstrating improvement in lung function (as measured by DLco) and reduction of spleen and liver volumes.

                          Brand Name : Xenpozyme

                          Molecule Type : Large molecule

                          Upfront Cash : Not Applicable

                          March 28, 2022

                          Lead Product(s) : Olipudase Alfa

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          Sanofi Company Banner

                          06

                          Aquaexpo 2024
                          Not Confirmed
                          Aquaexpo 2024
                          Not Confirmed

                          Details : Miplyffa (Arimoclomol) is indicated for use in combination with miglustat for the treatment of Niemann-Pick disease type C in adult and pediatric patients 2 years of age and older.

                          Brand Name : Miplyffa

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          September 20, 2024

                          Lead Product(s) : Arimoclomol,Miglustat

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          07

                          Enable Injections

                          Country arrow
                          Aquaexpo 2024
                          Not Confirmed

                          Enable Injections

                          Country arrow
                          Aquaexpo 2024
                          Not Confirmed

                          Details : The agreement aims for the international development and distribution for the enFuse Injector, for the subcutaneous delivery of Aspaveli (pegcetacoplan), a targeted C3 therapy, for PNH.

                          Brand Name : Aspaveli

                          Molecule Type : Peptide

                          Upfront Cash : Undisclosed

                          September 12, 2024

                          Lead Product(s) : Pegcetacoplan

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Approved

                          Sponsor : Swedish Orphan Biovitrum AB

                          Deal Size : Undisclosed

                          Deal Type : Agreement

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                          08

                          Aquaexpo 2024
                          Not Confirmed
                          Aquaexpo 2024
                          Not Confirmed

                          Details : Piasky (crovalimab) is a humanized C5 inhibitor monoclonal antibody, being developed for the treatment of adults and adolescents (12 years of age and above) with Paroxysmal Nocturnal Hemoglobinuria.

                          Brand Name : Piasky

                          Molecule Type : Large molecule

                          Upfront Cash : Not Applicable

                          August 27, 2024

                          Lead Product(s) : Crovalimab

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          09

                          Aquaexpo 2024
                          Not Confirmed
                          Aquaexpo 2024
                          Not Confirmed

                          Details : Epidiolex (cannabidiol) is an oral CB1 receptor NAM. It is now approved for the treatment of seizures associated with Lennox-Gastaut syndrome & Dravet syndrome in patients 2 years of age and older.

                          Brand Name : Epidiolex

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          August 22, 2024

                          Lead Product(s) : Cannabidiol

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          10

                          Aquaexpo 2024
                          Not Confirmed
                          Aquaexpo 2024
                          Not Confirmed

                          Details : Adzynma (apadamtase alfa /cinaxadamtase alfa) is the first and only ADAMTS13 enzyme replacement therapy, which is approved for the treatment of congenital thrombotic thrombocytopenic purpura.

                          Brand Name : Adzynma

                          Molecule Type : Large molecule

                          Upfront Cash : Not Applicable

                          August 08, 2024

                          Lead Product(s) : Apadamtase Alfa,Cinaxadamtase Alfa

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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