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Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Tablet","sponsorNew":"KYE Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"KYE Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"MEDUNIK CANADA","sponsor":"Jacobus Pharmaceutical Company, Inc","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"CANADA","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Amifampridine Phosphate","moa":"Voltage-gated potassium channel","graph1":"Rare Diseases and Disorders","graph2":"Approved FDF","graph3":"MEDUNIK CANADA","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Tablet","sponsorNew":"MEDUNIK CANADA \/ Jacobus Pharmaceutical Company, Inc","highestDevelopmentStatusID":"15","companyTruncated":"MEDUNIK CANADA \/ Jacobus Pharmaceutical Company, Inc"},{"orgOrder":0,"company":"Catalyst Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Amifampridine Phosphate","moa":"Voltage-gated potassium channel","graph1":"Rare Diseases and Disorders","graph2":"Approved FDF","graph3":"Catalyst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Catalyst Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Catalyst Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Stealth Biotherapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2025","type":"Inapplicable","leadProduct":"Elamipretide","moa":"Cardiolipin","graph1":"Rare Diseases and Disorders","graph2":"Approved FDF","graph3":"Stealth Biotherapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Stealth Biotherapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Stealth Biotherapeutics \/ Undisclosed"}]

    Find Clinical Drug Development Pipelines & Deals | PipelineProspector

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                            01

                            Breckenridge Pharmaceutical

                            U.S.A
                            OTS 2025
                            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) : Everolimus

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Approved FDF

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            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 : Miscellaneous

                            Upfront Cash : Inapplicable

                            January 29, 2025

                            Lead Product(s) : Everolimus

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Approved FDF

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Breckenridge Pharmaceutical

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                            02

                            Sanofi

                            France
                            CPhI WW Frankfurt
                            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) : Avalglucosidase Alpha

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Approved FDF

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            New Data Show Safety, Benefit, and Durability of Nexviazyme in Pompe Disease Patients

                            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.

                            Product Name : Nexviazyme

                            Product Type : Enzyme

                            Upfront Cash : Inapplicable

                            February 05, 2024

                            Lead Product(s) : Avalglucosidase Alpha

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Approved FDF

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Sanofi Company Banner

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                            03

                            Orion Corporation

                            Finland
                            OTS 2025
                            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) : Ganaxolone

                            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 : Miscellaneous

                            Upfront Cash : Inapplicable

                            July 31, 2023

                            Lead Product(s) : Ganaxolone

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Approved FDF

                            Sponsor : Marinus Pharmaceuticals

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            04

                            Zenara Pharma

                            India
                            OTS 2025
                            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) : Cannabidiol

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Approved FDF

                            Sponsor : Undisclosed

                            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

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Approved FDF

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Biophore CB

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                            05

                            Sanofi

                            France
                            CPhI WW Frankfurt
                            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) : Avalglucosidase Alpha

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Approved FDF

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            New Phase 3 Data Presented At WORLD Symposium™ Reinforce Nexviazyme® (avalglucosidase alfa) As Potential Ne...

                            Details : Nexviazyme (avalglucosidase alfa) is an enzyme replacement therapy (ERT) designed to target the mannose-6-phosphate (M6P) receptor, the key pathway for uptake and transport of ERT. It is being developed for treatment of people living with pompe disease.

                            Product Name : Nexviazyme

                            Product Type : Enzyme

                            Upfront Cash : Inapplicable

                            February 24, 2023

                            Lead Product(s) : Avalglucosidase Alpha

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Approved FDF

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Sanofi Company Banner

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                            06

                            Sanofi

                            France
                            CPhI WW Frankfurt
                            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

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Approved FDF

                            Sponsor : Undisclosed

                            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

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Approved FDF

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Sanofi Company Banner

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                            07

                            Sanofi

                            France
                            CPhI WW Frankfurt
                            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) : Avalglucosidase Alpha

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Approved FDF

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Nexviadyme® (avalglucosidase alfa) Approved by European Commission as a Potential New Standard of Care for th...

                            Details : Nexviadyme (avalglucosidase alfa), is the first and only newly approved medicine for Pompe disease in Europe since 2006, when the European Commission authorized the marketing of alglucosidase alfa, branded Myozyme®.

                            Product Name : Nexviadyme

                            Product Type : Enzyme

                            Upfront Cash : Inapplicable

                            June 28, 2022

                            Lead Product(s) : Avalglucosidase Alpha

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Approved FDF

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Sanofi Company Banner

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                            08

                            Sanofi

                            France
                            CPhI WW Frankfurt
                            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

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Approved FDF

                            Sponsor : Undisclosed

                            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

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Approved FDF

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Sanofi Company Banner

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                            09

                            Sanofi

                            France
                            CPhI WW Frankfurt
                            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

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Approved FDF

                            Sponsor : Undisclosed

                            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

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Approved FDF

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Sanofi Company Banner

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                            10

                            Sanofi

                            France
                            CPhI WW Frankfurt
                            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

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Approved FDF

                            Sponsor : Undisclosed

                            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

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Approved FDF

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Sanofi Company Banner

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