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      <title>By the numbers: US leads charge of cell and gene therapies</title>
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        <![CDATA[U.S. biotechs and regulators ushered in the era of gene therapy in 2023, experts at Bio Japan said, but medical reform is needed to pave the way for the “year of cell therapy” in 2024 and implement wider access to ultra-expensive cell and gene therapies.]]>
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      <guid>http://www.bioworld.com/articles/713702</guid>
      <pubDate>Tue, 22 Oct 2024 09:00:00 -0400</pubDate>
      <link>https://www.bioworld.com/articles/713702-by-the-numbers-us-leads-charge-of-cell-and-gene-therapies</link>
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        <media:title type="plain">3D rendering of CAR T therapy in cell</media:title>
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      <title>By the numbers: US leads charge of cell and gene therapies</title>
      <description>
        <![CDATA[U.S. biotechs and regulators ushered in the era of gene therapy in 2023, experts at Bio Japan said, but medical reform is needed to pave the way for the “year of cell therapy” in 2024 and implement wider access to ultra-expensive cell and gene therapies.]]>
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      <guid>http://www.bioworld.com/articles/713634</guid>
      <pubDate>Fri, 18 Oct 2024 09:00:00 -0400</pubDate>
      <link>https://www.bioworld.com/articles/713634-by-the-numbers-us-leads-charge-of-cell-and-gene-therapies</link>
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        <media:title type="plain">3D rendering of CAR T therapy in cell</media:title>
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      <title>Intercept faces a skeptical FDA in rare liver disease adcom</title>
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        <![CDATA[Intercept Pharmaceuticals Inc. lost its uphill battle to convince the U.S. FDA’s Gastrointestinal Drugs Advisory Committee that the risks of its second-line primary biliary cholangitis drug outweigh the benefits. The committee overwhelmingly said the data in the follow-up studies of treating the rare disease with Ocaliva (obeticholic acid), which has accelerated approval from the FDA, was insufficient.]]>
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      <guid>http://www.bioworld.com/articles/712531</guid>
      <pubDate>Fri, 13 Sep 2024 12:00:00 -0400</pubDate>
      <link>https://www.bioworld.com/articles/712531-intercept-faces-a-skeptical-fda-in-rare-liver-disease-adcom</link>
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      <title>FDA approves Argenx's Vyvgart for rare autoimmune disease CIDP</title>
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        <![CDATA[Argenx SE gained U.S. FDA approval of subcutaneously given Vyvgart Hytrulo (efgartigimod alfa and hyaluronidase-qvfc) for adult patients with chronic inflammatory demyelinating polyneuropathy (CIDP). The company’s stock (NASDAQ:ARGX) was up 11.7% to $440.59 at the close of trading June 24. About 24,000 people in the U.S. are being treated for CIDP, and patients are generally diagnosed between 40 and 60 years of age.]]>
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      <guid>http://www.bioworld.com/articles/709890</guid>
      <pubDate>Mon, 24 Jun 2024 12:00:00 -0400</pubDate>
      <link>https://www.bioworld.com/articles/709890-fda-approves-argenxs-vyvgart-for-rare-autoimmune-disease-cidp</link>
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        <media:title type="plain">Vyvgart Hytrulo</media:title>
        <media:description type="plain">Credit: Argenx </media:description>
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      <title>Day One’s Ojemda approved in US for rare pediatric cancer </title>
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        <![CDATA[Becoming the first type II RAF inhibitor for relapsed or refractory BRAF-altered pediatric low-grade glioma, Day One Biopharmaceuticals Inc.’s Ojemda (tovorafenib, DAY-101) gained U.S. FDA accelerated approval on April 23, a week earlier than its expected PDUFA date, bringing the Brisbane, Calif.-based company a rare pediatric disease priority review voucher.]]>
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      <guid>http://www.bioworld.com/articles/707897</guid>
      <pubDate>Tue, 23 Apr 2024 12:00:00 -0400</pubDate>
      <link>https://www.bioworld.com/articles/707897-day-ones-ojemda-approved-in-us-for-rare-pediatric-cancer</link>
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      <title>A first: Biologics dominate 2023 class of accelerated approvals</title>
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        <![CDATA[At first glance, the number of drugs that received accelerated approval from the U.S. FDA’s Center for Drug Evaluation and Research (CDER) in 2023 was nothing to write home about. Yes, CDER granted nine accelerated approvals last year, up from six in 2022. But the proportion of novel drugs with accelerated approval was 16% both years. And when compared with the 12 drugs in 2020 and the 14 that received accelerated approval in 2021, last year’s crop was a little lackluster. However, a deeper look at the 2023 class of accelerated approvals shows a historic milestone. For the first time since the path was created in 1992, the number of novel biologics getting accelerated approval at CDER outpaced the number of small-molecule drugs.]]>
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      <guid>http://www.bioworld.com/articles/706690</guid>
      <pubDate>Mon, 18 Mar 2024 12:00:00 -0400</pubDate>
      <link>https://www.bioworld.com/articles/706690-a-first-biologics-dominate-2023-class-of-accelerated-approvals</link>
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        <media:title type="plain">Vial and syringe with molecule structure</media:title>
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