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                            Breckenridge Pharmaceutical

                            U.S.A
                            EPSC
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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) : Everolimus,Inapplicable

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            Breckenridge Gains FDA Nod for Everolimus, Afinitor Disperz Generic

                            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,Inapplicable

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

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                            Breckenridge Pharmaceutical

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                            02

                            Orion Corporation

                            Finland
                            EPSC
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : Ganaxolone,Inapplicable

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Approved FDF

                            Sponsor : Marinus Pharmaceuticals

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            European Commission Approval of Ztalmy (ganaxolone) for the Adjunctive Treatment of Epileptic Seizures Associa...

                            Details : Ztalmy (ganaxolone) is a positive allosteric modulation of the gamma-aminobutyric acid type A (GABAA) receptor in the CNS. It is used for the treatment of epileptic seizures associated with CDKL5 deficiency disorder.

                            Product Name : Ztalmy

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            July 31, 2023

                            Lead Product(s) : Ganaxolone,Inapplicable

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Approved FDF

                            Sponsor : Marinus Pharmaceuticals

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            03

                            Zenara Pharma

                            India
                            EPSC
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : Cannabidiol,Inapplicable

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Biophore, Its Subsidiary Zenara Pharma, Receive First Approval for Cannabidiol in India

                            Details : Cannabinoid oral solution has received approval for the treatment of several neurological disorders. It functions by stimulating two receptors, cannabinoid receptor type 1 and type 2, within the endocannabinoid system, maintaining body's homeostatis.

                            Product Name : Undisclosed

                            Product Type : Controlled Substance

                            Upfront Cash : Inapplicable

                            May 15, 2023

                            Lead Product(s) : Cannabidiol,Inapplicable

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Biophore CB

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                            04

                            Sanofi

                            France
                            ChemOutsourcing
                            Attending
                            • fda
                            • EDQM
                            • WHO-GMP
                            Virtual BoothSanofi is a pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme.

                            Lead Product(s) : Olipudase Alpha,Inapplicable

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Xenpozyme TM (Olipudase Alfa-rpcp) Approved by FDA as First Disease-specific Treatment for ASMD (Non-CNS Manif...

                            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.

                            Product Name : Xenpozyme

                            Product Type : Enzyme

                            Upfront Cash : Inapplicable

                            August 31, 2022

                            Lead Product(s) : Olipudase Alpha,Inapplicable

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            05

                            Sanofi

                            France
                            ChemOutsourcing
                            Attending
                            • fda
                            • EDQM
                            • WHO-GMP
                            Virtual BoothSanofi is a pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme.

                            Lead Product(s) : Olipudase Alpha,Inapplicable

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Xenpozyme® (olipudase alfa) Approved by European Commission as First and Only treatment for ASMD

                            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.

                            Product Name : Xenpozyme

                            Product Type : Enzyme

                            Upfront Cash : Inapplicable

                            June 28, 2022

                            Lead Product(s) : Olipudase Alpha,Inapplicable

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            Virtual Booth

                            06

                            Sanofi

                            France
                            ChemOutsourcing
                            Attending
                            • fda
                            • EDQM
                            • WHO-GMP
                            Virtual BoothSanofi is a pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme.

                            Lead Product(s) : Olipudase Alpha,Inapplicable

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            CHMP Recommends Approval of Xenpozyme® (Olipudase Alfa), the First and Only Treatment for ASMD

                            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.

                            Product Name : Xenpozyme

                            Product Type : Enzyme

                            Upfront Cash : Inapplicable

                            May 19, 2022

                            Lead Product(s) : Olipudase Alpha,Inapplicable

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            07

                            Sanofi

                            France
                            ChemOutsourcing
                            Attending
                            • fda
                            • EDQM
                            • WHO-GMP
                            Virtual BoothSanofi is a pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme.

                            Lead Product(s) : Olipudase Alpha,Inapplicable

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Xenpozyme® (olipudase alfa), Approved in Japan, First and Only Approved Therapy Indicated to Treat Acid Sphin...

                            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.

                            Product Name : Xenpozyme

                            Product Type : Enzyme

                            Upfront Cash : Inapplicable

                            March 28, 2022

                            Lead Product(s) : Olipudase Alpha,Inapplicable

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            Virtual Booth

                            08

                            Abeona Therapeutics

                            U.S.A arrow
                            EPSC
                            Not Confirmed

                            Abeona Therapeutics

                            U.S.A arrow
                            EPSC
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
                            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) : Prademagene Zamikeracel,Inapplicable

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Approved FDF

                            Sponsor : Undisclosed

                            Deal Size : $155.0 million

                            Deal Type : Divestment

                            DEALS_DEV arrow-down

                            Abeona Closes Sale of Rare Pediatric Disease Priority Review Voucher for $155 Million

                            Details : Zevaskyn (prademagene zamikeracel), the first and only U.S. approved autologous cell-based gene therapy for the treatment of wounds in adult and pediatric patients with recessive dystrophic epidermolysis bullosa.

                            Product Name : Zevaskyn

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Undisclosed

                            July 02, 2025

                            Lead Product(s) : Prademagene Zamikeracel,Inapplicable

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Approved FDF

                            Sponsor : Undisclosed

                            Deal Size : $155.0 million

                            Deal Type : Divestment

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                            09

                            Abeona Therapeutics

                            U.S.A arrow
                            EPSC
                            Not Confirmed

                            Abeona Therapeutics

                            U.S.A arrow
                            EPSC
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
                            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) : Prademagene Zamikeracel,Inapplicable

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Approved FDF

                            Sponsor : Undisclosed

                            Deal Size : $155.0 million

                            Deal Type : Agreement

                            DEALS_DEV arrow-down

                            Abeona To Sell Priority Review Voucher for $155M in New Agreement

                            Details : Abeona sold it's Rare Pediatric Disease PRV for FDA approved of Zevaskyn (prademagene zamikeracel). It is being indicated for the wounds in adult and pediatric patients with recessive DEB.

                            Product Name : Zevaskyn

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Undisclosed

                            May 12, 2025

                            Lead Product(s) : Prademagene Zamikeracel,Inapplicable

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Approved FDF

                            Sponsor : Undisclosed

                            Deal Size : $155.0 million

                            Deal Type : Agreement

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                            10

                            Abeona Therapeutics

                            U.S.A arrow
                            EPSC
                            Not Confirmed

                            Abeona Therapeutics

                            U.S.A arrow
                            EPSC
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
                            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) : Prademagene Zamikeracel,Inapplicable

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            FDA Approves ZEVASKYN, First Cell-Based Gene Therapy for RDEB Patients

                            Details : Zevaskyn consists of a patient’s own skin cells (keratinocytes) that have been genetically modified, to produce functional Type VII collagen.

                            Product Name : Zevaskyn

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            April 29, 2025

                            Lead Product(s) : Prademagene Zamikeracel,Inapplicable

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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