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Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"KORU Medical Systems \/ Undisclosed"},{"orgOrder":0,"company":"KBio","sponsor":"National Institute of Allergy and Infectious Diseases","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"EV68-228-N","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"KBio","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"KBio \/ National Institute of Allergy and Infectious Diseases","highestDevelopmentStatusID":"6","companyTruncated":"KBio \/ National Institute of Allergy and Infectious Diseases"},{"orgOrder":0,"company":"Savara","sponsor":"Jefferies","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Large molecule","year":"2024","type":"Public Offering","leadProduct":"Molgramostim","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Savara","amount2":0.10000000000000001,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0.10000000000000001,"dosageForm":"","sponsorNew":"Savara \/ Jefferies","highestDevelopmentStatusID":"10","companyTruncated":"Savara \/ Jefferies"},{"orgOrder":0,"company":"Savara","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"Molgramostim","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Savara","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Savara \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Savara \/ Not Applicable"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"RCT1100","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"ReCode Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"ReCode Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"ReCode Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Chenodeoxycholic Acid","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Mirum Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Mirum Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Mirum Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Cyclo Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Hydroxypropyl-Beta Cyclodextrin","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Cyclo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Cyclo Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Cyclo Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"ChromaDex, Inc","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Nicotinamide Riboside Chloride","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"ChromaDex, Inc","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"ChromaDex, Inc \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"ChromaDex, Inc \/ Not Applicable"},{"orgOrder":0,"company":"Quoin Pharmaceuticals","sponsor":"University College Cork","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Small molecule","year":"2024","type":"Agreement","leadProduct":"Sirolimus","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Quoin Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Quoin Pharmaceuticals \/ University College Cork","highestDevelopmentStatusID":"4","companyTruncated":"Quoin Pharmaceuticals \/ University College Cork"}]

    Find Clinical Drug Development Pipelines & Deals | PipelineProspector

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                            01

                            Sanofi

                            France
                            Pharma, Lab & Chemical Expo
                            Not Confirmed
                            • 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 Alfa

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Approved

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            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.

                            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

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                            02

                            Sanofi

                            France
                            Pharma, Lab & Chemical Expo
                            Not Confirmed
                            • 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 Alfa-rpcp

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Approved

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            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.

                            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

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                            03

                            Sanofi

                            France
                            Pharma, Lab & Chemical Expo
                            Not Confirmed
                            • 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 Alfa

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Approved

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            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.

                            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

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                            04

                            Sanofi

                            France
                            Pharma, Lab & Chemical Expo
                            Not Confirmed
                            • 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 Alfa

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Approved

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            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.

                            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

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                            05

                            Sanofi

                            France
                            Pharma, Lab & Chemical Expo
                            Not Confirmed
                            • 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 Alfa

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Approved

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            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.

                            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

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                            06

                            Sanofi

                            France
                            Pharma, Lab & Chemical Expo
                            Not Confirmed
                            • 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 Alfa

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase II/ Phase III

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            EMA Accepts Regulatory Submission for Olipudase Alfa, the First Potential Therapy for ASMD

                            Details : Olipudase alfa is an investigational enzyme replacement therapy designed to replace deficient or defective ASM, allowing for the breakdown of sphingomyelin.

                            Brand Name : GZ402665

                            Molecule Type : Large molecule

                            Upfront Cash : Not Applicable

                            December 06, 2021

                            Lead Product(s) : Olipudase Alfa

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase II/ Phase III

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            07

                            Soligenix

                            U.S.A arrow
                            Pharma, Lab & Chemical Expo
                            Not Confirmed

                            Soligenix

                            U.S.A arrow
                            Pharma, Lab & Chemical Expo
                            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) : Dusquetide

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase II

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            Soligenix Begins Phase 2 Trial of SGX945 for Behçet’s Disease Treatment

                            Details : SGX945 (dusquetide) is a peptide, P62 inhibitor administered intravenously via infusion, which is being investigated for treating Aphthous Ulcers in Behçet's Disease.

                            Brand Name : SGX945

                            Molecule Type : Peptide

                            Upfront Cash : Not Applicable

                            November 14, 2024

                            Lead Product(s) : Dusquetide

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase II

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            08

                            BPGbio

                            U.S.A arrow
                            Pharma, Lab & Chemical Expo
                            Not Confirmed

                            BPGbio

                            U.S.A arrow
                            Pharma, Lab & Chemical Expo
                            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) : Ubidecarenone

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase II

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            BPGbio Receives FDA Pediatric Disease Designation for Epidermolysis Bullosa Treatment

                            Details : BPM31510T (benzoquinone ubidecarenone) is an investigational treatment for epidermolysis bullosa (EB), received Rare Pediatric Disease Designation for respective indication.

                            Brand Name : BPM31510T

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            October 31, 2024

                            Lead Product(s) : Ubidecarenone

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase II

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            09

                            Aro Biotherapeutics

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                            Pharma, Lab & Chemical Expo
                            Not Confirmed

                            Aro Biotherapeutics

                            U.S.A arrow
                            Pharma, Lab & Chemical Expo
                            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) : ABX1100

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase I

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            Aro Biotherapeutics Opens Phase 1b Trial for ABX1100 in Late-Onset Pompe Disease

                            Details : ABX1100, an investigational treatment for Pompe disease, is comprised of a CD71 receptor-binding Centyrin conjugated to a siRNA that specifically interferes with expression of GYS1 mRNA.

                            Brand Name : ABX1100

                            Molecule Type : Large molecule

                            Upfront Cash : Not Applicable

                            October 29, 2024

                            Lead Product(s) : ABX1100

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase I

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            10

                            AstraZeneca

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                            Pharma, Lab & Chemical Expo
                            Not Confirmed

                            AstraZeneca

                            United Kingdom arrow
                            Pharma, Lab & Chemical Expo
                            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) : Benralizumab

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Approved

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            Fasenra Approved in the EU for Eosinophilic Granulomatosis with Polyangiitis

                            Details : Fasenra (benralizumab) is IL-5Rα inhibitor antibody candidate, which is indicated for the treatment of eosinophilic granulomatosis with polyangiitis.

                            Brand Name : Fasenra

                            Molecule Type : Large molecule

                            Upfront Cash : Not Applicable

                            October 29, 2024

                            Lead Product(s) : Benralizumab

                            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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