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receptor","graph1":"Rare Diseases and Disorders","graph2":"Approved FDF","graph3":"PANTHERx Rare Pharmacy","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"PANTHERx Rare Pharmacy \/ X4 Pharmaceuticals","highestDevelopmentStatusID":"15","companyTruncated":"PANTHERx Rare Pharmacy \/ X4 Pharmaceuticals"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"Chenodeoxycholic Acid","moa":"Cholesterol","graph1":"Rare Diseases and Disorders","graph2":"Approved FDF","graph3":"Mirum Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral Tablet, Film Coated","sponsorNew":"Mirum Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Mirum Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Horizon Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"IRELAND","productType":"Antibody","year":"2022","type":"Inapplicable","leadProduct":"Inebilizumab","moa":"CD19","graph1":"Rare Diseases and Disorders","graph2":"Approved FDF","graph3":"Horizon Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Horizon Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Horizon Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Horizon Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and 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Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Horizon Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Horizon Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Horizon Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"IRELAND","productType":"Antibody","year":"2022","type":"Inapplicable","leadProduct":"Inebilizumab","moa":"CD19","graph1":"Rare Diseases and Disorders","graph2":"Approved FDF","graph3":"Horizon Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Horizon Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Horizon Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Horizon Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and 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Disorders","country":"U.S.A","productType":"Antibody","year":"2021","type":"Acquisition","leadProduct":"Inebilizumab","moa":"CD19","graph1":"Rare Diseases and Disorders","graph2":"Approved FDF","graph3":"Viela Bio","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Viela Bio \/ Horizon Therapeutics","highestDevelopmentStatusID":"15","companyTruncated":"Viela Bio \/ Horizon Therapeutics"},{"orgOrder":0,"company":"Viela Bio","sponsor":"Horizon Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Antibody","year":"2021","type":"Acquisition","leadProduct":"Inebilizumab","moa":"CD19","graph1":"Rare Diseases and Disorders","graph2":"Approved FDF","graph3":"Viela Bio","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Viela Bio \/ Horizon Therapeutics","highestDevelopmentStatusID":"15","companyTruncated":"Viela Bio \/ Horizon Therapeutics"},{"orgOrder":0,"company":"Viela Bio","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Antibody","year":"2020","type":"Inapplicable","leadProduct":"Inebilizumab","moa":"CD19","graph1":"Rare Diseases and Disorders","graph2":"Approved FDF","graph3":"Viela Bio","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Viela Bio \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Viela Bio \/ Inapplicable"},{"orgOrder":0,"company":"Sentynl Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"Fosdenopterin","moa":"cPMP","graph1":"Rare Diseases and Disorders","graph2":"Approved FDF","graph3":"Sentynl Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Sentynl Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Sentynl Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Zevra Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Small Molecule","year":"2025","type":"Agreement","leadProduct":"Arimoclomol Citrate","moa":"||TFEB","graph1":"Rare Diseases and Disorders","graph2":"Approved FDF","graph3":"Zevra Therapeutics","amount2":0.14999999999999999,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Rare Diseases","amount2New":0.14999999999999999,"dosageForm":"Oral Capsule","sponsorNew":"Zevra Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Zevra Therapeutics \/ Undisclosed"}]

    Find Clinical Drug Development Pipelines & Deals | PipelineProspector

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                            01

                            Breckenridge Pharmaceutical

                            U.S.A
                            German Wound Congress
                            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 : Inapplicable

                            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 : Other Small Molecule

                            Upfront Cash : Inapplicable

                            January 29, 2025

                            Lead Product(s) : Everolimus

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Breckenridge Pharmaceutical

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                            02

                            Orion Corporation

                            Finland
                            German Wound Congress
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
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                            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 : Other Small Molecule

                            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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                            03

                            Zenara Pharma

                            India
                            German Wound Congress
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : Cannabidiol

                            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

                            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
                            BIO International Convention
                            Exhibiting
                            • 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 : 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

                            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
                            BIO International Convention
                            Exhibiting
                            • 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 : 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

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Sanofi Company Banner

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                            06

                            Sanofi

                            France
                            BIO International Convention
                            Exhibiting
                            • 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 : 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

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Sanofi Company Banner

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                            07

                            Sanofi

                            France
                            BIO International Convention
                            Exhibiting
                            • 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 : 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

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Sanofi Company Banner

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                            08

                            Zevra Therapeutics

                            U.S.A arrow
                            German Wound Congress
                            Not Confirmed

                            Zevra Therapeutics

                            U.S.A arrow
                            German Wound Congress
                            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) : Arimoclomol Citrate,Miglustat

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Approved FDF

                            Sponsor : Undisclosed

                            Deal Size : $150.0 million

                            Deal Type : Agreement

                            DEALS_DEV arrow-down

                            Zevra Sells Rare Disease Review Voucher For $150 Million

                            Details : Zevra sold it's Rare Pediatric Disease PRV for FDA approved of Miplyffa (arimoclomol). It is indicated for use in combination with miglustat for the treatment of neurological manifestations of NPC. C

                            Product Name : Miplyffa

                            Product Type : Other Small Molecule

                            Upfront Cash : Undisclosed

                            April 07, 2025

                            Lead Product(s) : Arimoclomol Citrate,Miglustat

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Approved FDF

                            Sponsor : Undisclosed

                            Deal Size : $150.0 million

                            Deal Type : Agreement

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                            09

                            Ionis Pharmaceuticals

                            U.S.A arrow
                            German Wound Congress
                            Not Confirmed

                            Ionis Pharmaceuticals

                            U.S.A arrow
                            German Wound Congress
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : Olezarsen Sodium

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Approved FDF

                            Sponsor : Swedish Orphan Biovitrum AB

                            Deal Size : Undisclosed

                            Deal Type : Licensing Agreement

                            DEALS_DEV arrow-down

                            Ionis, Sobi Expand Partnership for Olezarsen Commercialization Outside U.S.

                            Details : Under the licensing agreement, Sobi will hold the exclusive rights in countries outside the U.S., Canada to commercialize Tryngolza (olezarsen sodium) as a potential treatment for FCS.

                            Product Name : Tryngolza

                            Product Type : Oligonucleotide

                            Upfront Cash : Undisclosed

                            March 26, 2025

                            Lead Product(s) : Olezarsen Sodium

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Approved FDF

                            Sponsor : Swedish Orphan Biovitrum AB

                            Deal Size : Undisclosed

                            Deal Type : Licensing Agreement

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                            10

                            Lupin Ltd

                            India arrow
                            German Wound Congress
                            Not Confirmed

                            Lupin Ltd

                            India arrow
                            German Wound Congress
                            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) : Amifampridine Phosphate

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Lupin Gains Tentative USFDA Approval for Amifampridine Tablets

                            Details : Firdapse-Generic (amifampridine) is an oral, nonspecific, voltage-dependent, potassium channel blocker that is indicated to treat Lambert-Eaton myasthenic syndrome.

                            Product Name : Firdapse-Generic

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            March 17, 2025

                            Lead Product(s) : Amifampridine Phosphate

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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