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2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Chugai Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Chugai\u2019s Enspryng Launched in Taiwan as First Indication for Neuromyelitis Optica Spectrum Disorder (NMOSD)","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"JAPAN","productType":"Large molecule","productStatus":"Approved","date":"October 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Approved"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Rocket Pharmaceuticals Announces FDA Acceptance of Biologics License Application with Priority Review for RP-L201 (marnetegragene autotemcel) for the Treatment of Severe Leukocyte Adhesion Deficiency-I (LAD-I)","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"October 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"St. Jude Children\u2019s Research Hospital","sponsor":"n-Lorem Foundation","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"n-Lorem Foundation Collaborates to Support Children With Extremely Rare Diseases","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery","country":"","productType":"Large molecule","productStatus":"New Molecular Entity","date":"October 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Discovery"},{"orgOrder":0,"company":"GC Biopharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FDA Grants Orphan Drug Designation to GC Biopharma's Drug Candidate for Thrombotic Thrombocytopenic Purpura","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Preclinical","country":"SOUTH KOREA","productType":"Large molecule","productStatus":"New Molecular Entity","date":"October 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Preclinical"},{"orgOrder":0,"company":"Inozyme Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Inozyme Pharma Highlights Inclusion of Generalized Arterial Calcification of Infancy (GACI) in Genomics England's Generation Study of Rare Conditions","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"October 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Annexon Biosciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Annexon Announces Clinical and Regulatory Progress for ANX005 Pivotal Program in Guillain-Barr\u00e9 Syndrome (GBS)","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"October 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase III"},{"orgOrder":0,"company":"American Regent","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"American Regent Introduces Levocarnitine Injection, USP; FDA-Approved, \"AP\" Rated, and Therapeutically Equivalent to Carnitor\u00ae\u00b9","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"October 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Approved"},{"orgOrder":0,"company":"Inovio Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"INOVIO Receives FDA Feedback that Data from Completed Phase 1\/2 Trial of INO-3107 Can Be Used to Submit a BLA Under Accelerated Approval Program","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Vaccine","productStatus":"New Molecular Entity","date":"October 2023","url1":"","url2":"","graph1":"Vaccine","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Pint Pharma","sponsor":"OrphanDC","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Empaveli is the First Treatment for PNH that Binds to the Complement Protein C3","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"AUSTRIA","productType":"Peptide","productStatus":"Approved","date":"October 2023","url1":"","url2":"","graph1":"Peptide","graph2":"Approved"},{"orgOrder":0,"company":"Novo Nordisk","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FDA Approves Rivfloza\u2122 for Children \u22659 Years Old and Adults Living with Primary Hyperoxaluria Type 1 (PH1), a Rare Genetic Condition","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"DENMARK","productType":"Large molecule","productStatus":"Approved","date":"October 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Approved"},{"orgOrder":0,"company":"AB Institut de Neuroci\u00e8ncies","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"EMA Gives Cannabidiol Orphan Drug Designation for the Treatment of Leigh syndrome","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Preclinical","country":"","productType":"Small molecule","productStatus":"New Molecular Entity","date":"September 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Preclinical"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"REGENXBIO Announces NAV\u00ae Technology Platform Will Support Bespoke Gene Therapy Consortium's First Rare Disease Clinical Portfolio","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Preclinical"},{"orgOrder":0,"company":"UT Southwestern Medical Center","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Public-private Consortium will Fund three Gene Therapy Clinical Trials at UT Southwestern and Children\u2019s Health","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"June 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Preclinical"},{"orgOrder":0,"company":"Myto Therapeutics","sponsor":"University of California","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"MYT-109, a Promising Treatment for Leigh Syndrome","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"November 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Preclinical"},{"orgOrder":0,"company":"EA Pharma","sponsor":"Eisai","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"All-case Surveillance Condition for Approval of \u201cActonel\u00ae 17.5 mg tablets\u201d for Treatment of Paget\u2019s Disease of Bone Cleared in Japan","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"FRANCE","productType":"Small molecule","productStatus":"Approved","date":"March 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Approved"},{"orgOrder":0,"company":"OMERS Life Sciences","sponsor":"Xencor","pharmaFlowCategory":"D","amount":"$215.0 million","upfrontCash":"Undisclosed","newsHeadline":"Xencor Sells Portion of Royalties and Milestones from Ultomiris\u00ae and Monjuvi\u00ae to OMERS Life Sciences for $215 Million","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"","productType":"Large molecule","productStatus":"Approved","date":"November 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Approved"},{"orgOrder":0,"company":"PaxMedica","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"PaxMedica Holds Important FDA Type-B Meeting to Review PAX-101 Pivotal Trial Data","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"October 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"IND Enabling"},{"orgOrder":0,"company":"Atsena Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Atsena Therapeutics Receives FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for ATSN-101 Gene Therapy for GUCY2D-associated Leber Congenital Amaurosis (LCA1)","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"November 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Pharming","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Pharming Group Provides Updates on EMA Regulatory Review of Leniolisib MAA and Plans to File For UK Regulatory Approval","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"NETHERLANDS","productType":"Small molecule","productStatus":"Approved","date":"November 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Palvella Therapeutics","sponsor":"Ligand Pharmaceuticals","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"$5.0 million","newsHeadline":"Palvella Therapeutics and Ligand Pharmaceuticals Expand Strategic Partnership to Accelerate Phase 3 Development of QTORIN\u2122 Rapamycin for Microcystic Lymphatic Malformations and Additional High Unmet Need Clinical Indications","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"December 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II"},{"orgOrder":0,"company":"ProQR Therapeutics","sponsor":"Laboratoires Thea","pharmaFlowCategory":"D","amount":"$186.3 million","upfrontCash":"$8.6 million","newsHeadline":"ProQR Therapeutics Announces Transaction Completed for Th\u00e9a to Acquire Sepofarsen and Ultevursen Ophthalmic Assets","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II\/ Phase III","country":"NETHERLANDS","productType":"Large molecule","productStatus":"New Molecular Entity","date":"December 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase II\/ Phase III"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Takeda\u2019s ADZYNMA (ADAMTS13, recombinant-krhn) Approved by U.S. FDA as the First and Only Recombinant ADAMTS13 Enzyme Replacement Therapy for the Treatment of Congenital Thrombotic Thrombocytopenic Purpura (cTTP)","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"JAPAN","productType":"Large molecule","productStatus":"Approved","date":"November 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Approved"},{"orgOrder":0,"company":"Caliway Biopharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Caliway Closes Oversubscribed Capital Raise and Secures Over $100M to Advance CBL-514 Pivotal Phase 3 Study in Subcutaneous Fat Reduction","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"TAIWAN","productType":"Small molecule","productStatus":"New Molecular Entity","date":"December 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II"},{"orgOrder":0,"company":"Longboard Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Longboard Pharmaceuticals Announces Positive Topline Data from the PACIFIC Study, a Phase 1b\/2a Clinical Trial, for Bexicaserin (LP352) in Participants with Developmental and Epileptic Encephalopathies (DEEs)","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2024","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Agios Announces Phase 3 ENERGIZE Study of Mitapivat Met Primary Endpoint and Both Key Secondary Endpoints in Adults with Non-Transfusion-Dependent Alpha- or Beta-Thalassemia","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"January 2024","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase III"},{"orgOrder":0,"company":"AskBio","sponsor":"Bayer AG","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"AskBio Phase Ib trial of AB-1005 Gene Therapy in Patients with Parkinson\u2019s Disease Meets Primary Endpoint","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2024","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase I"},{"orgOrder":0,"company":"NovelMed Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"USAN Approves Generic Name \u201cRuxoprubart\u201d for NM8074, an Antibody Therapy focused on Complement-Mediated Diseases","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"January 2024","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase II"},{"orgOrder":0,"company":"Zevra Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Zevra Therapeutics Receives FDA Acceptance of Resubmission of NDA for Arimoclomol as a Treatment for Niemann-Pick Disease Type C","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II\/ Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2024","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II\/ Phase III"},{"orgOrder":0,"company":"Caliway Biopharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Caliway Announces FDA Acceptance of CBL-514 IND Application for Placebo-Controlled Phase 2 Study to Treat Dercum's Disease","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"TAIWAN","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2024","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II"},{"orgOrder":0,"company":"Soligenix","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FDA Grants Soligenix \"Fast Track\" Designation for Dusquetide in the Treatment of Oral Lesions of Behcet's Disease","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Peptide","productStatus":"New Molecular Entity","date":"January 2024","url1":"","url2":"","graph1":"Peptide","graph2":"IND Enabling"},{"orgOrder":0,"company":"Drug Farm","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Drug Farm Receives Rare Pediatric Disease Designation from the U.S. FDA for DF-003 to Treat ROSAH Syndrome","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"CHINA","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2024","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase I"},{"orgOrder":0,"company":"LadRx","sponsor":"XOMA","pharmaFlowCategory":"D","amount":"$58.6 million","upfrontCash":"$5.0 million","newsHeadline":"FDA Acceptance of Zevra's Arimoclomol NDA Filing for Niemann-Pick Disease Type C (NPC) Results in XOMA Making a $1 Million Milestone Payment to LadRx","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II\/ Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2024","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II\/ Phase III"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"ReCode Therapeutics Doses First Patient in Phase 1 Clinical Study of Novel mRNA-based Therapy for the Treatment of Primary Ciliary Dyskinesia","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"January 2024","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase I"},{"orgOrder":0,"company":"Atsena Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Atsena Therapeutics Receives Rare Pediatric Disease Designation from FDA for ATSN-101 Gene Therapy for GUCY2D-associated Leber Congenital Amaurosis (LCA1)","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2024","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Blue Owl Capital","pharmaFlowCategory":"D","amount":"$500.0 million","upfrontCash":"$500.0 million","newsHeadline":"BridgeBio Pharma Secures up to $1.25 Billion of Capital from Blue Owl and CPP Investments to Accelerate the Development and Launch of Genetic Medicines","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2024","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Kyowa Kirin","pharmaFlowCategory":"D","amount":"$477.6 million","upfrontCash":"$387.4 million","newsHeadline":"Kyowa Kirin Successfully Completes Acquisition of Orchard Therapeutics, a Global Gene Therapy Leader for Rare Diseases","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"Approved","date":"January 2024","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Approved"},{"orgOrder":0,"company":"NS Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"The European Commission Grants Orphan Drug Designation to NS-229 for the Treatment of Eosinophilic Granulomatosis with Polyangiitis","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2024","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II"},{"orgOrder":0,"company":"Jazz Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Jazz Pharmaceuticals Receives Health Canada Approval for Epidiolex\u00ae (Cannabidiol Oral Solution) for the Treatment of Seizures Associated with Three Rare Forms of Epilepsy","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"IRELAND","productType":"Small molecule","productStatus":"Approved","date":"November 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Approved"},{"orgOrder":0,"company":"Avicanna","sponsor":"Undisclosed","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Avicanna Announces Supply and Licensing Agreement with Multinational Pharmaceutical Company","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II\/ Phase III","country":"CANADA","productType":"Small molecule","productStatus":"Approved","date":"February 2024","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II\/ Phase III"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"New Data Demonstrate Clinical Safety, Benefit and Durability of Nexviazyme\u00ae Across a Wide-Range of Pompe Disease Patient Groups","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"FRANCE","productType":"Large molecule","productStatus":"Approved","date":"February 2024","url1":"","url2":"","graph1":"Large molecule","graph2":"Approved"},{"orgOrder":0,"company":"Amylyx Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Amylyx Pharmaceuticals Announces Completion of Enrollment in HELIOS, a Phase 2 Study of AMX0035 for the Treatment of Wolfram Syndrome","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"February 2024","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II"},{"orgOrder":0,"company":"NovelMed Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Ruxoprubart (NM8074) Scores FDA Orphan Drug Designation for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2024","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase II"},{"orgOrder":0,"company":"Azitra","sponsor":"ThinkEquity","pharmaFlowCategory":"D","amount":"$5.0 million","upfrontCash":"Undisclosed","newsHeadline":"Azitra, Inc. Announces Pricing of Public Offering","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2024","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase I"},{"orgOrder":0,"company":"AstraZeneca","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Voydeya Recommended for Approval in the EU by CHMP as Add-On Treatment to Ravulizumab or Eculizumab for Adults with Pnh Who Have Residual Haemolytic Anaemia","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"Approved","date":"February 2024","url1":"","url2":"","graph1":"Small molecule","graph2":"Approved"}]

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            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.

            Lead Product(s): Avalglucosidase Alfa

            Therapeutic Area: Rare Diseases and Disorders Product Name: Nexviazyme

            Highest Development Status: Approved Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 05, 2024

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            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.

            Lead Product(s): Olipudase Alfa-rpcp

            Therapeutic Area: Rare Diseases and Disorders Product Name: Xenpozyme

            Highest Development Status: Approved Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable August 31, 2022

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            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.

            Lead Product(s): Olipudase Alfa

            Therapeutic Area: Rare Diseases and Disorders Product Name: Xenpozyme

            Highest Development Status: Approved Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable June 28, 2022

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            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.

            Lead Product(s): Olipudase Alfa

            Therapeutic Area: Rare Diseases and Disorders Product Name: Xenpozyme

            Highest Development Status: Approved Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable May 19, 2022

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            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.

            Lead Product(s): Olipudase Alfa

            Therapeutic Area: Rare Diseases and Disorders Product Name: Xenpozyme

            Highest Development Status: Approved Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable March 28, 2022

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

            Lead Product(s): Olipudase Alfa

            Therapeutic Area: Rare Diseases and Disorders Product Name: GZ402665

            Highest Development Status: Phase II/ Phase III Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable December 06, 2021

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            Details:

            Voydeya (danicopan) is an investigational oral medicine in development as an add-on to C5 inhibitor therapy eculizumab or ravulizumab for patients with paroxysmal nocturnal hemoglobinuria (PNH) who have residual haemolytic anaemia.

            Lead Product(s): Danicopan,Ravulizumab

            Therapeutic Area: Rare Diseases and Disorders Product Name: Voydeya

            Highest Development Status: Approved Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 26, 2024

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            NM8074 (ruxoprubart) is an alternative pathway (AP) blocker anti-Bb antibody. It is being evaluated for the treatment of paroxysmal nocturnal hemoglobinuria.

            Lead Product(s): Ruxoprubart

            Therapeutic Area: Rare Diseases and Disorders Product Name: NM8074

            Highest Development Status: Phase II Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 12, 2024

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            Relyvrio (sodium phenylbutyrate and taurursodiol) is an oral, fixed-dose combination. It is a HDAC inhibitor which is being evaluated in phase 2 clinical trials for the treatment of Wolfram syndrome.

            Lead Product(s): Sodium Phenylbutyrate,Tauroursodeoxycholic Acid

            Therapeutic Area: Rare Diseases and Disorders Product Name: Relyvrio

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 08, 2024

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            Arimoclomol is a first in class investigational HSF1 activator, which is under phase 2/3 clinical development for the treatment of Niemann-Pick disease type C.

            Lead Product(s): Arimoclomol

            Therapeutic Area: Rare Diseases and Disorders Product Name: Undisclosed

            Highest Development Status: Phase II/ Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 08, 2024

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