Veranova A CDMO that manages complexity with confidence

Veranova A CDMO that manages complexity with confidence

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III","country":"GERMANY","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Genentech","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Genentech Announces Positive Data From Global Phase III Program for Crovalimab in PNH, a Rare, Life-Threatening Blood Condition","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Atsena Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Atsena Therapeutics to Present Positive Interim Encore Data from the Phase I\/II Clinical Trial of ATSN-101 for the Treatment of GUCY2D-associated Leber Congenital Amaurosis (LCA1) at the 46th Annual Macula Society Meeting","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Chugai Pharmaceutical","sponsor":"F. Hoffmann-La Roche","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Chugai Announces Positive Data from Global Phase III Program for Crovalimab in PNH, a Rare Life-Threatening Blood Condition","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"JAPAN","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Evozyne","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"D","amount":"$400.0 million","upfrontCash":"Undisclosed","newsHeadline":"Evozyne Announces Multi-Target Research Collaboration and License Agreement with Takeda to Develop Next-Generation Gene Therapies","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"April 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Discovery"},{"orgOrder":0,"company":"Relief Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Relief Therapeutics Announces Enrollment of First Three Patients in Proof-of-Concept Clinical Trial of RLF-TD011 for the Treatment of Epidermolysis Bullosa","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"SWITZERLAND","productType":"Small molecule","productStatus":"Approved","date":"February 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase I"},{"orgOrder":0,"company":"Pharming","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Pharming Provides Update on EMA Regulatory Review of Leniolisib For APDS in Europe","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"NETHERLANDS","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Zevra Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Zevra Therapeutics Announces Two Abstracts Accepted for Presentation at the 19th Annual WORLDSymposium 2023","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Apellis Pharmaceuticals","sponsor":"J.P. Morgan Securities LLC","pharmaFlowCategory":"D","amount":"$350.0 million","upfrontCash":"Undisclosed","newsHeadline":"Apellis Pharmaceuticals Announces Pricing of Public Offering of Common Stock and Pre-Funded Warrants","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Peptide","productStatus":"Approved","date":"February 2023","url1":"","url2":"","graph1":"Peptide","graph2":"Approved"},{"orgOrder":0,"company":"Apellis Pharmaceuticals","sponsor":"J.P. Morgan Securities","pharmaFlowCategory":"D","amount":"$402.5 million","upfrontCash":"Undisclosed","newsHeadline":"Apellis Pharmaceuticals Announces Closing of Public Offering of Common Stock and Pre-Funded Warrants and Full Exercise of Option to Purchase Additional Shares","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Peptide","productStatus":"Approved","date":"February 2023","url1":"","url2":"","graph1":"Peptide","graph2":"Approved"},{"orgOrder":0,"company":"Palvella Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Palvella Therapeutics Announces Positive Topline Results from Phase 2 Study of QTORIN 3.9% Rapamycin Anhydrous Gel (QTORIN rapamycin) for the Treatment of Microcystic Lymphatic Malformations, a Serious, Rare Genetic Skin Disease with No FDA-approved Thera","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"March 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II"},{"orgOrder":0,"company":"Endo International","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Endo Provides Top-Line Results from Phase 2 Study of Collagenase Clostridium Histolyticum (CCH) in Participants With Plantar Fibromatosis","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"IRELAND","productType":"Large molecule","productStatus":"New Molecular Entity","date":"March 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Nobelpharma America","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Rare Disease Treatment, HYFTOR\u00ae (sirolimus topical gel) 0.2% Coverage Expanded by Medicaid, Including in Key U.S. States","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"March 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Approved"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"ReCode Therapeutics Announces First Participants Dosed in a Phase 1 Healthy Volunteer Clinical Trial of Novel Disease-Modifying Genetic Medicine, RCT1100 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":"March 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase I"},{"orgOrder":0,"company":"Disc medicine","sponsor":"National Institutes of Health","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Disc Medicine Announces Collaboration with National Institutes of Health for Phase 2 Clinical Study of Bitopertin in Patients with Diamond-Blackfan Anemia (DBA)","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"March 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"IND Enabling"},{"orgOrder":0,"company":"Pharming","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Pharming Announces US FDA Approval of Joenja\u00ae (leniolisib) as The First and Only Treatment Indicated for APDS","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"NETHERLANDS","productType":"Small molecule","productStatus":"Approved","date":"March 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Approved"}]

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Sanofi

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Pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme.

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Xenpozyme TM (Olipudase Alfa-rpcp) Approved by FDA as First Disease-specific Treatment for ASMD (Non-CNS Manifestations)

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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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