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FDA Approves Upstaza for AADC Deficiency | ASGCT - American Society of Gene & Cell Therapy

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Cannon, PhD - November 14, 2024</div> <div class="callout"> <p><p>FDA approved Kebilidi (eladocagene exuparvovec), the first&nbsp;approved gene therapy in the U.S.&nbsp;to be directly administered into the brain.</p> </p> </div> <p> <img src="/ASGCT/media/about/vector.png" alt="" </p> <p> <p paraeid="{2c09ad07-d67f-4b6c-a2bb-12a2145db64f}{177}" paraid="726394259">The <a href="https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-treatment-aromatic-l-amino-acid-decarboxylase-deficiency" target="_blank">FDA approved&nbsp;Kebilidi</a> (eladocagene exuparvovec), a gene therapy for the treatment of aromatic l-amino acid decarboxylase (AADC) deficiency.&nbsp;This is the first approved gene therapy in the U.S.&nbsp;to be directly administered into the brain.</p> <p paraeid="{2c09ad07-d67f-4b6c-a2bb-12a2145db64f}{205}" paraid="415328792"><a href="https://medlineplus.gov/genetics/condition/aromatic-l-amino-acid-decarboxylase-deficiency/#resources" rel="noreferrer noopener" target="_blank">AADC deficiency</a> is a fatal, rare inherited disorder caused by mutations in the DDC gene that reduce activity of the AADC enzyme. This causes neurons to produce less dopamine, contributing to developmental delays, intellectual disabilities, abnormal movements, and autonomic nervous system dysfunction. &nbsp;</p> <p paraeid="{698bb6a5-0291-4f7f-bb7f-52b886eeca16}{27}" paraid="926466496">Signs of AADC deficiency typically appear in the first six months of life, and include involuntary movements, weak muscle tone, and sleep disturbances. People with AADC deficiency also have an increased risk of infection, which can lead to life-threatening complications.&nbsp;</p> <p paraeid="{698bb6a5-0291-4f7f-bb7f-52b886eeca16}{51}" paraid="1521540859">Kebilidi, developed by PTC Therapeutics, is a one-time therapy indicated for patients 18 months and older with a clinical, molecular, and genetically confirmed diagnosis of AADC deficiency with a severe phenotype. It&#39;s administered via four infusions in one surgical session into a large structure in the brain involved in motor control. The treatment is a&nbsp;recombinant adeno-associated virus serotype 2 (AAV2)-based gene therapy that delivers a functioning DDC gene directly into the brain to increase levels of the AADC enzyme and restore dopamine production.&nbsp;</p> <p paraeid="{698bb6a5-0291-4f7f-bb7f-52b886eeca16}{119}" paraid="584491243">The safety and effectiveness of Kebilidi were demonstrated in an open-label, single-arm clinical study in 13 pediatric patients with confirmed diagnosis of AADC deficiency. At the beginning of the study, all patients had no gross motor function&nbsp;and decreased AADC activity in the plasma. Twelve of the 13 patients completed motor milestone assessments at week 48 after receiving the treatment and the efficacy of Kebilidi was demonstrated based on the gross motor function improvement in eight of the 12 patients.</p> <p paraeid="{698bb6a5-0291-4f7f-bb7f-52b886eeca16}{160}" paraid="1874742410">This approval represents an exciting development for children affected by this devastating disease. Congratulations to everyone who worked to bring this gene therapy to those children and their families.&nbsp;</p> <p paraeid="{698bb6a5-0291-4f7f-bb7f-52b886eeca16}{160}" paraid="1874742410"><em>Paula Cannon, PhD, is the president of ASGCT (2024-25) and a Distinguished Professor of Molecular Microbiology &amp; Immunology at USC&#39;s Keck School of Medicine.</em></p> </p> <h2 class="blog-landing--title">Related Articles</h2> <div class="article-grid--wrapper"> <a class="article-grid-item" href="/Publications/News/November-2024/FDA-approves-obe-cel-for-B-cell-acute-lymphoblasti"> <div class="article-grid-item--thumb" style="background-image: url('/ASGCT/media/about/CAR-T-cells-(3)_1.png')"><span class="sr-only"></span></div> <div class="article-grid-item--content"> <div class="article-grid-item--tags">Cell & Gene Therapy Approval</div> <h2>FDA Approves Obe-cel for B-Cell Acute Lymphoblastic Leukemia (ALL)</h2> <div class="article-grid--author">Paula Cannon, PhD - November 11, 2024</div> </div> </a> <a class="article-grid-item" href="/Publications/News/August-2024/Tecelra-Approval"> <div class="article-grid-item--thumb" style="background-image: url('')"><span class="sr-only"></span></div> <div class="article-grid-item--content"> <div class="article-grid-item--tags">Cell & Gene Therapy Approval</div> <h2>FDA Approves Tecelra for Metastatic Synovial Sarcoma</h2> <div class="article-grid--author">Paula Cannon, PhD - August 06, 2024</div> </div> </a> <a class="article-grid-item" href="/Publications/News/March-2024/Libmeldy-metachromatic-leukodystrophy-stem-cell-ge"> <div class="article-grid-item--thumb" style="background-image: url('/ASGCT/media/about/brain-for-MLD-approval-blog.png')"><span class="sr-only"></span></div> <div class="article-grid-item--content"> <div class="article-grid-item--tags">Cell & Gene Therapy Approval</div> <h2>FDA Approves Lenmeldy for Metachromatic Leukodystrophy </h2> <div class="article-grid--author">Jeffrey S. Chamberlain, PhD - March 18, 2024</div> </div> </a> <a class="article-grid-item" href="/Publications/News/June-2023/FDA-Approves-First-Gene-Therapy-Severe-HemophiliaA"> <div class="article-grid-item--thumb" style="background-image: url('/ASGCT/media/about/Hemo_Large_4.jpeg')"><span class="sr-only"></span></div> <div class="article-grid-item--content"> <div class="article-grid-item--tags">Cell & Gene Therapy Approval</div> <h2>FDA Approves First Gene Therapy to Treat Severe Hemophilia A</h2> <div class="article-grid--author">Jeffrey Chamberlain, PhD - June 29, 2023</div> </div> </a> </div> </main> <aside> <div class="asideBox"> <h4>Related Content</h4> <ul> <li><a href='/Publications/News/November-2024/FDA-approves-obe-cel-for-B-cell-acute-lymphoblasti'>FDA Approves Obe-cel for B-cell Acute Lymphoblastic Leukemia (ALL)</a></li> <li><a href='/Publications/News/August-2024/Tecelra-Approval'>Tecelra Approval</a></li> <li><a href='/Publications/News/March-2024/Libmeldy-metachromatic-leukodystrophy-stem-cell-ge'>FDA Approves Lenmeldy for Metachromatic Leukodystrophy</a></li> <li><a href='/Publications/News/June-2023/FDA-Approves-First-Gene-Therapy-Severe-HemophiliaA'>FDA Approves First Gene Therapy to Treat Severe Hemophilia A</a></li> <li><a href='/Publications/News/June-2023/First-Gene-Therapy-for-Duchenne-Muscular-Dystrophy'>First Gene Therapy for Duchenne Muscular Dystrophy</a></li> </ul> </div> </aside> </div> </section> <div class="event-row"> <div class="year">2025</div> <div class="content"> <h2>Class Considerations on Immunogenicity for AAV GT Products</h2> <p>January 22-23, 2025 | Virtual</p> </div> <div class="action"><a class="btn btn-white" href="https://www.asgct.org/asgct-events/january-2025/class-considerations-on-immunogenicity-for-aav-gen">Learn More</a></div> </div> <footer> <div class="container"> <div class="footerColContainer"> <div class="footerCol"> <p><img alt="logo-footer.png" class="footerLogo" src="/getmedia/c77b7df1-12a2-4998-9809-0a880c107341/ASGCT_Web_Horz_White.aspx" title="logo-footer.png" /></p> <p>20800 Swenson Dr.<br /> Suite 300<br /> Waukesha, WI 53186</p> </div> <div class="footerCol"> <ul> <li><a href="/about">About</a></li> <li><a href="/membership">Membership</a></li> <li><a href="https://annualmeeting.asgct.org" target="_blank">Annual Meeting</a></li> <li><a href="http://www.cell.com/molecular-therapy-family/home" target="_blank">Journal</a></li> </ul> </div> <div class="footerCol"> <ul> <li><a href="/education">Education</a></li> <li><a href="/advocacy">Advocacy</a></li> <li><a href="https://asgct.org/awards" target="_blank">Awards</a></li> <li><a href="https://asgct.org/about/privacy-policy" target="_blank">Privacy Policy</a></li> </ul> </div> <div class="footerCol"> <p class="altStyle">Connect with ASGCT</p> <p><strong>Phone:</strong> 414.278.1341<br /> <a href="mailto:info@asgct.org">info@asgct.org</a><div class="footerSocial"><div id="ctl00_bkSiteFooter_rptFooterBlocks_ctl04_widget1_ctl00_divSocialLinks" class="socialLinks footerSocial"> <ul> <li><a href="https://twitter.com/ASGCTherapy" target="_blank"><i class="bk-icon-twitter"></i></a></li> <li><a href="https://www.facebook.com/asgctherapy" target="_blank"><i class="bk-icon-facebook"></i></a></li> <li><a href="https://www.instagram.com/asgctherapy" target="_blank"><i class="bk-icon-instagram"></i></a></li> <li><a href="https://www.youtube.com/user/ASGCT" target="_blank"><i class="bk-icon-youtube"></i></a></li> <li><a href="https://www.linkedin.com/company/asgct/" target="_blank"><i class="bk-icon-linkedin"></i></a></li> </ul> </div></div></p> </div> </div> </div> </footer> <div class="postFooter"> <div class="container"> <p>&copy; 2000-2024 All rights reserved. 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