BioMarin's ROCTAVIAN Wins Food and Drug Administration Approval As First Gene Therapy for Severe Hemophilia A

Author:

Philippidis Alex

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

Reference11 articles.

1. BioMarin Pharmaceutical. U.S. Food and Drug Administration Approves BioMarin's ROCTAVIANÔ (valoctocogene roxaparvovec-rvox), the First and Only Gene Therapy for Adults with Severe Hemophilia A. June 29, 2023. Available from: https://investors.biomarin.com/2023-06-29-U-SFood-and-Drug-Administration-Approves-BioMar ins-ROCTAVIAN-TM-valoctocogene-roxaparvovecrvox-the-First-and-Only-Gene-Therapy-for-Adultswith-Severe-Hemophilia-A [Last accessed: July 14, 2023].

2. Myshko D. FDA Approves $2.9 Million Gene Therapy for Hemophilia A Formulary Watch. June 29, 2023. Available from: https://www .formularywatch.com/view/fda-approves-2-9- million-gene-therapy-for-hemophilia-a [Last accessed: June 14, 2023].

3. Scribe Therapeutics. Scribe Therapeutics Expands Collaboration with Sanofito Advance In Vivo Genetic Medicines for Sickle Cell and Other Genomic Diseases. July 17, 2023. Available from: https://www.scribetx.com/press-release-scribetherapeutics-expands-collaboration-with-sanofi-toadvance-in-vivo-genetic-medicines-for-sickle-cell-a nd-other-genomic-diseases/ [Last accessed: July 17, 2023].

4. Astellas Pharma and 4D Molecular Therapeutics. Astellas and 4D Molecular Therapeutics (4DMT) Enter into License Agreement to Use 4DMT's Proprietary Intravitreal R100 Vector for Rare Ophthalmic Targets. July 11, 2023. Available from: https://www.astellas.com/en/news/28136 [Last accessed: July 11, 2023].

5. Sarepta Therapeutics. Sarepta Therapeutics Announces FDA Approval of ELEVIDYS, the First Gene Therapy to Treat Duchenne Muscular Dystrophy. June 22, 2023. Available from: https: //investorrelations.sarepta.com/news-releases/ news-release-details/sarepta-therapeuticsannounces-fda-approval-elevidys-first-gene [Last accessed: June 14, 2023].

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