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Home » Topics » Regulatory » Breakthrough therapy

Breakthrough therapy
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Knee pain illustration

Allay’s $57.5M extends postsurgical pain relief with ATX-101

June 10, 2025
By Karen Carey
No Comments
As it advances its nonopioid analgesic ATX-101 breakthrough therapy through a phase IIb registration trial, Allay Therapeutics secured $57.5 million in a series D round, which included an investment from the company’s Japanese partner.
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Knee pain illustration

Allay’s $57.5M extends postsurgical pain relief with ATX-101

June 6, 2025
By Karen Carey
No Comments
As it advances its nonopioid analgesic ATX-101 breakthrough therapy through a phase IIb registration trial, Allay Therapeutics secured $57.5 million in a series D round, which included an investment from the company’s Japanese partner. ATX-101, a configuration of sodium ion channel blocker bupivacaine and a biopolymer, which is in a phase IIb registration study, is designed to offer pain relief following total knee arthroplasty.
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Vykat XR

First Prader-Willi treatment Vykat XR FDA-approved: Soleno soars

March 27, 2025
By Karen Carey
Becoming the first treatment for rare genetic disorder Prader-Willi syndrome, breakthrough therapy drug DCCR (diazoxide choline) gained U.S. FDA approval as Vykat XR the evening before its March 27 PDUFA date, sending shares of developer Soleno Therapeutics Inc. up 38%.
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3D illustration of mesenchymal stem cells

Nature Cell wins FDA breakthrough designation of Jointstem

March 25, 2025
By Marian (YoonJee) Chu
Nature Cell Co. Ltd. won U.S. FDA breakthrough therapy designation of its autologous adipose-derived mesenchymal stem cell therapy, Jointstem, March 20, becoming the first Korean company to earn the label in the field of cell therapy.
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Polycythemia vera illustration

Protagonist’s rusfertide hits phase III goals in rare leukemia

March 5, 2025
By Marian (YoonJee) Chu
Rusfertide could become a blockbuster therapy for polycythemia vera, H.C. Wainwright analyst Douglas Tsao wrote March 4 after the injectable hepcidin mimetic peptide hit its primary endpoint and all four key secondary endpoints in the ongoing phase III Verify study.
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Vaccine vial and syringe

Moderna’s Mresvia approved as first mRNA-based RSV vaccine

May 31, 2024
By Karen Carey
Moderna Inc. gained U.S. FDA approval on May 31, nearly three weeks past its original PDUFA date, for its respiratory syncytial virus (RSV) vaccine Mresvia (mRNA-1345), which had both breakthrough therapy and fast track designations in the U.S.
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Acute myeloid leukemia

Glycomimetics wilts on missed acute myeloid leukemia phase III

May 6, 2024
By Lee Landenberger
Top-line data from Glycomimetics Inc.’s pivotal phase III study of uproleselan in 388 patients with relapsed/refractory acute myeloid leukemia missed its primary endpoint. The data crippled the stock, which closed at the low point of its 10-year existence.
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Day One’s Ojemda approved in US for rare pediatric cancer

April 23, 2024
By Karen Carey
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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Doctor examining child's leg

China accepts Sperogenix NDA filed for rare muscle disorder drug

April 2, 2024
By Marian (YoonJee) Chu
Beijing- and Shanghai-based Sperogenix Therapeutics Ltd. said that China’s regulatory agency accepted the NDA filing and granted priority review of Agamree (vamorolone) for Duchenne muscular dystrophy on March 26.
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Doctor examining child's leg

China accepts Sperogenix NDA filed for rare muscle disorder drug

April 1, 2024
By Marian (YoonJee) Chu
Beijing- and Shanghai-based Sperogenix Therapeutics Ltd. said that China’s regulatory agency accepted the NDA filing and granted priority review of Agamree (vamorolone) for Duchenne muscular dystrophy on March 26.
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