[{"orgOrder":0,"company":"Cycle Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Nitisinone","moa":"HPDD","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Cycle Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"Cycle Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Cycle Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Cycle Pharmaceuticals","sponsor":"Torrent Pharmaceuticals Limited","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"Dichlorphenamide","moa":"CA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Cycle Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"Cycle Pharmaceuticals \/ Torrent Pharmaceuticals Limited","highestDevelopmentStatusID":"15","companyTruncated":"Cycle Pharmaceuticals \/ Torrent Pharmaceuticals Limited"},{"orgOrder":0,"company":"Cycle Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Other Small Molecule","year":"2021","type":"Inapplicable","leadProduct":"Icatibant Acetate","moa":"Bradykinin B2 receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Cycle Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Cycle Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Cycle Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Cycle Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"UNITED KINGDOM","productType":"Other Small Molecule","year":"2023","type":"Inapplicable","leadProduct":"Fingolimod Hydrochloride","moa":"S1PR","graph1":"Neurology","graph2":"Approved FDF","graph3":"Cycle Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Neurology","amount2New":0,"dosageForm":"Oral Tablet, Orally Disintegrating","sponsorNew":"Cycle Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Cycle Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Cycle Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"UNITED KINGDOM","productType":"Other Small Molecule","year":"2023","type":"Inapplicable","leadProduct":"Fingolimod Hydrochloride","moa":"S1PR","graph1":"Neurology","graph2":"Approved FDF","graph3":"Cycle Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Neurology","amount2New":0,"dosageForm":"Oral Tablet, Orally Disintegrating","sponsorNew":"Cycle Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Cycle Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Cycle Pharmaceuticals","sponsor":"Torrent Pharmaceuticals Limited","pharmaFlowCategory":"DU","therapeuticArea":"Nephrology","country":"UNITED KINGDOM","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"Tiopronin","moa":"Urinary cystine concentration","graph1":"Nephrology","graph2":"Approved FDF","graph3":"Cycle Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Nephrology","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"Cycle Pharmaceuticals \/ Torrent Pharmaceuticals Limited","highestDevelopmentStatusID":"15","companyTruncated":"Cycle Pharmaceuticals \/ Torrent Pharmaceuticals Limited"},{"orgOrder":0,"company":"Cycle Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Nephrology","country":"UNITED KINGDOM","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"Tiopronin","moa":"Urinary cystine concentration","graph1":"Nephrology","graph2":"Approved FDF","graph3":"Cycle Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Nephrology","amount2New":0,"dosageForm":"Oral Tablet, Delayed Release","sponsorNew":"Cycle Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Cycle Pharmaceuticals \/ Inapplicable"}]

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                          01

                          AAN
                          Not Confirmed
                          AAN
                          Not Confirmed

                          Details : Venxxiva is indicated, in combination with high fluid intake, alkali, and diet modification, for the prevention of cystine stone formation with severe homozygous cystinuria.

                          Product Name : Venxxiva

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          March 18, 2025

                          Lead Product(s) : Tiopronin

                          Therapeutic Area : Nephrology

                          Highest Development Status : Approved FDF

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          02

                          AAN
                          Not Confirmed
                          AAN
                          Not Confirmed

                          Details : Ormalvi (dichlorphenamide) is a carbonic anhydrase inhibitor. The generic version has been approved and is indicated for the treatment of primary hyperkalemic periodic paralysis and related variants.

                          Product Name : Ormalvi

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          August 05, 2024

                          Lead Product(s) : Dichlorphenamide

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Torrent Pharmaceuticals Limited

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          03

                          AAN
                          Not Confirmed
                          AAN
                          Not Confirmed

                          Details : Tiopronin is an active reducing agent which undergoes thiol-disulfide exchange with cystine to form a mixed disulfide of tiopronin-cysteine. It is indicated for cystinuria.

                          Product Name : Tiopronin-Generic

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          May 13, 2024

                          Lead Product(s) : Tiopronin

                          Therapeutic Area : Nephrology

                          Highest Development Status : Approved FDF

                          Sponsor : Torrent Pharmaceuticals Limited

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          04

                          AAN
                          Not Confirmed
                          AAN
                          Not Confirmed

                          Details : Tascenso (fingolimod) is a sphingosine 1-phosphate receptor modulator indicated for the treatment of relapsing forms of MS, to include clinically isolated syndrome, relapsing-remitting disease, and active secondary progressive disease, in patients 10 yea...

                          Product Name : Tascenso

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          February 13, 2023

                          Lead Product(s) : Fingolimod Hydrochloride

                          Therapeutic Area : Neurology

                          Highest Development Status : Approved FDF

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          05

                          AAN
                          Not Confirmed
                          AAN
                          Not Confirmed

                          Details : Tascenso (fingolimod) is a sphingosine 1-phosphate receptor modulator indicated for the treatment of relapsing forms of MS, to include clinically isolated syndrome, relapsing-remitting disease, and active secondary progressive disease, in patients 10 yea...

                          Product Name : Tascenso

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          January 17, 2023

                          Lead Product(s) : Fingolimod Hydrochloride

                          Therapeutic Area : Neurology

                          Highest Development Status : Approved FDF

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          06

                          AAN
                          Not Confirmed
                          AAN
                          Not Confirmed

                          Details : SAJAZIR™ is a bradykinin B2 receptor antagonist indicated for treatment of acute attacks of hereditary angioedema (HAE) in adults 18 years of age and older.

                          Product Name : Sajazir

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          September 28, 2021

                          Lead Product(s) : Icatibant Acetate

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          07

                          AAN
                          Not Confirmed
                          AAN
                          Not Confirmed

                          Details : NITYR® is indicated for the treatment of patients with hereditary tyrosinemia type 1 (HT-1) in combination with dietary restriction of tyrosine and phenylalanine.

                          Product Name : Nityr

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          September 14, 2020

                          Lead Product(s) : Nitisinone

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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