https://www.bioworld.com/rss en-us Niagen Bioscience Inc.’s proprietary lead small-molecule drug candidate, NB-4168, has been awarded European orphan drug designation and U.S. rare pediatric disease designation for the treatment of ataxia telangiectasia. http://www.bioworld.com/articles/732626 Tue, 14 Jul 2026 09:00:00 -0400 https://www.bioworld.com/articles/732626-niagens-nb-4168-designated-orphan-drug-in-eu DNA illustration SL Science Holding Ltd. has submitted an orphan drug designation request to the FDA for its γδ T-cell therapy product, Vδ2+ γδ T cells, for the treatment of glioblastoma multiforme. http://www.bioworld.com/articles/732565 Mon, 13 Jul 2026 09:00:00 -0400 https://www.bioworld.com/articles/732565-sl-science-seeks-us-orphan-drug-designation-for-t-cell-therapy Illustration of T cells attacking tumor At the BIO International Convention, nonprofit and for-profit companies discussed ways to fund orphan drug development, especially for ultra-rare diseases where development costs can be challenging. One of the simplest ways to keep costs down is to skip the discovery phase and use repurposed drugs or rescue shelved therapeutic assets as the Orphan Therapeutics Accelerator has done. The Cambridge, Mass.-based accelerator was founded in 2024 to rescue shelled therapeutic assets for ultrarare diseases. http://www.bioworld.com/articles/732173 Fri, 26 Jun 2026 12:00:00 -0400 https://www.bioworld.com/articles/732173-bio-2026-alternatives-to-funding-orphan-drugs Dollar sign inside vial, syringe Nippon Shinyaku Co. Ltd.’s NS-035 has been awarded Japanese orphan drug designation for the treatment of Fukuyama congenital muscular dystrophy. http://www.bioworld.com/articles/732135 Thu, 25 Jun 2026 09:00:00 -0400 https://www.bioworld.com/articles/732135-nippon-shinyakus-ns-035-designated-orphan-drug-in-japan 3d illustration of human body muscle tissue anatomy Elixiron Immunotherapeutics Inc. announced positive interim open-label phase II Alzheimer’s disease (AD) study findings of enrupatinib, an oral brain-penetrant colony-stimulating factor 1 receptor inhibitor, with plans to move the asset into a biomarker-based and placebo-controlled study. http://www.bioworld.com/articles/731984 Tue, 16 Jun 2026 12:00:00 -0400 https://www.bioworld.com/articles/731984-elixirons-enrupatinib-shows-upbeat-interim-phase-ii-ad-results Illustration of amyloid plaques in Alzheimer's disease Elixiron Immunotherapeutics Inc. announced positive interim open-label phase II Alzheimer’s disease (AD) study findings of enrupatinib, an oral brain-penetrant colony-stimulating factor 1 receptor inhibitor, with plans to move the asset into a biomarker-based and placebo-controlled study. http://www.bioworld.com/articles/731877 Mon, 15 Jun 2026 12:00:00 -0400 https://www.bioworld.com/articles/731877-elixirons-enrupatinib-shows-upbeat-interim-phase-ii-ad-results Illustration of amyloid plaques in Alzheimer's disease Agios Pharmaceuticals Inc.’s potential $665 million deal for Oscotec Inc.’s cevidoplenib, a phase III-ready oral SYK inhibitor to treat immune thrombocytopenia, marks a new chapter of development focused on cancer therapy resistance for Oscotec, CEO Yoon Tae-young said. http://www.bioworld.com/articles/731828 Tue, 09 Jun 2026 12:00:00 -0400 https://www.bioworld.com/articles/731828-disciplined-665m-deal-moves-oscotecs-cevidoplenib-to-agios Deal handshake with coin, chart background Gen1e Lifesciences Inc. has obtained both orphan drug and rare pediatric disease designations from the U.S. FDA for GEn-1123 for the treatment of Duchenne muscular dystrophy (DMD). http://www.bioworld.com/articles/731763 Tue, 09 Jun 2026 09:00:00 -0400 https://www.bioworld.com/articles/731763-gen1e-lifesciences-receives-us-fda-designations-for-gen-1123 Art concept for inflamed human tissue Agios Pharmaceuticals Inc.’s potential $665 million deal for Oscotec Inc.’s cevidoplenib, a phase III-ready oral SYK inhibitor to treat immune thrombocytopenia, marks a new chapter of development focused on cancer therapy resistance for Oscotec, CEO Yoon Tae-young said. http://www.bioworld.com/articles/731632 Fri, 05 Jun 2026 12:00:00 -0400 https://www.bioworld.com/articles/731632-disciplined-665m-deal-moves-oscotecs-cevidoplenib-to-agios Deal handshake with coin, chart background Sotio Biotech AS’s SOT-106 has been granted orphan drug designation by the FDA for the treatment of osteosarcoma. SOT-106 is a next-generation antibody-drug conjugate targeting leucine-rich repeat-containing 15 (LRRC15), a clinically validated target broadly expressed across sarcoma subtypes and in tumor-associated stroma. http://www.bioworld.com/articles/731653 Thu, 04 Jun 2026 09:00:00 -0400 https://www.bioworld.com/articles/731653-sotio-biotechs-sot-106-designated-orphan-drug-for-osteosarcoma Illustration of bone cancer in the knee Hanmi Pharmaceutical Co. Ltd. secured a $1.26 billion deal with Eli Lilly and Co. to out-license ex-Korea rights to sonefpeglutide (HM-15912), a Lapscovery-based glucagon-like peptide-2 analog in development for multiple indications, including an ongoing phase II study of short bowel syndrome. It was one of two billion-dollar Asian company deals signed by Lilly on June 1, with the second transaction involving Haisco Pharmaceutical Group Co. Ltd., of Beijing. http://www.bioworld.com/articles/731645 Tue, 02 Jun 2026 12:00:00 -0400 https://www.bioworld.com/articles/731645-hanmi-haisco-win-billion-dollar-partnerships-with-eli-lilly Hanmi signage Credit: Hanmi Pharmaceutical Co. Ltd. Hanmi Pharmaceutical Co. Ltd. secured a $1.26 billion deal with Eli Lilly and Co. to out-license ex-Korea rights to sonefpeglutide (HM-15912), a Lapscovery-based glucagon-like peptide-2 analog in development for multiple indications, including an ongoing phase II study of short bowel syndrome. It was one of two billion-dollar Asian company deals signed by Lilly on June 1, with the second transaction involving Haisco Pharmaceutical Group Co. Ltd., of Beijing. http://www.bioworld.com/articles/731459 Mon, 01 Jun 2026 12:00:00 -0400 https://www.bioworld.com/articles/731459-hanmi-haisco-win-billion-dollar-partnerships-with-eli-lilly Hanmi signage Credit: Hanmi Pharmaceutical Co. Ltd. Pilatus Bio Inc. is working to address a major under-addressed target of current checkpoint inhibitors: metabolic stress within solid tumors. “Traditional immunotherapies release immune ‘brakes,’ but they do not address the underlying metabolic stress in tumors,” Pilatus CEO and cofounder Raven Lin said. “That’s why more than 60% of solid tumor patients do not respond to treatment.” http://www.bioworld.com/articles/730671 Tue, 28 Apr 2026 12:00:00 -0400 https://www.bioworld.com/articles/730671-pilatus-plt-012-takes-immunometabolic-approach-to-reprogram-tme Illustration of monoclonal antibody treatment for cancer With three rare pediatric disease priority review vouchers (RPD PRVs) awarded just since the end of March, the nearly year-and-a-half lapse in the program’s reauthorization seems to have had little short-term impact. The three new vouchers bring the total RPD PRVs granted so far this year to seven – one more than the agency issued all last year and down two from the nine given in 2024. http://www.bioworld.com/articles/730209 Thu, 09 Apr 2026 12:00:00 -0400 https://www.bioworld.com/articles/730209-pediatric-prvs-top-the-voucher-popularity-contest Pediatric exam With the U.S. FDA’s approval of GSK plc’s ileal bile acid transporter (IBAT) inhibitor, Lynavoy (linerixibat), patients with primary biliary cholangitis no longer need off-label treatments for a debilitating internal itch symptom called cholestatic pruritus. http://www.bioworld.com/articles/729745 Thu, 19 Mar 2026 12:00:00 -0400 https://www.bioworld.com/articles/729745-gsks-ibat-inhibitor-wins-us-fda-nod-for-cholestatic-pruritus Red wooden approved stamp Otsuka Pharmaceutical Co. Ltd. reaped positive results from an open-label phase II study of repinatrabit (JNT-517) in adolescents with phenylketonuria (PKU), a rare disease marked by the inability to break down the amino acid phenylalanine. http://www.bioworld.com/articles/729717 Tue, 17 Mar 2026 11:00:00 -0400 https://www.bioworld.com/articles/729717-otsukas-repinatrabit-shows-phase-ii-efficacy-in-pku-adolescents Molecule illustration Otsuka Pharmaceutical Co. Ltd. reaped positive results from an open-label phase II study of repinatrabit (JNT-517) in adolescents with phenylketonuria (PKU), a rare disease marked by the inability to break down the amino acid phenylalanine. http://www.bioworld.com/articles/729496 Fri, 13 Mar 2026 12:00:00 -0400 https://www.bioworld.com/articles/729496-otsukas-repinatrabit-shows-phase-ii-efficacy-in-pku-adolescents Molecule illustration At the current pace of innovation in the U.S. rare disease space, developing and approving therapies for just half of the 10,000-plus known rare diseases would take more than 160 years, Bradley Campbell, president and CEO of Amicus Therapeutics Inc., recently told the Senate Committee on Aging. http://www.bioworld.com/articles/729300 Wed, 04 Mar 2026 12:00:00 -0500 https://www.bioworld.com/articles/729300-fdas-rare-disease-toolbox-not-fully-used Magnifying glass, clock and capsules A lot of distance lies between talking regulatory flexibility and actually being flexible. That message was driven home again after Uniqure NV disclosed in its latest earnings report March 2 that the U.S. FDA wants a sham-controlled study before it will consider approval of the company’s gene therapy AMT-130 in Huntington’s, a rare disease currently affecting about 41,000 people in the U.S. http://www.bioworld.com/articles/729285 Tue, 03 Mar 2026 12:00:00 -0500 https://www.bioworld.com/articles/729285-experts-regulatory-roadblocks-stalling-rare-disease-therapies Red and white roadblock Tessera Therapeutics Inc.’s lead in vivo gene editing program, TSRA-196, has been awarded orphan drug and fast track designations by the FDA for adults with α-1 antitrypsin deficiency (AATD). http://www.bioworld.com/articles/729019 Tue, 24 Feb 2026 08:00:00 -0500 https://www.bioworld.com/articles/729019-tesseras-tsra-196-designated-orphan-drug-for-aatd DNA double helix under a magnifying glass Affinia Therapeutics Inc.’s AFTX-201 has been awarded orphan drug designation by the EMA for BAG3-associated dilated cardiomyopathy. http://www.bioworld.com/articles/728941 Thu, 19 Feb 2026 08:00:00 -0500 https://www.bioworld.com/articles/728941-affinias-aftx-201-designated-eu-orphan-drug 3D illustration of heart cross section The pressure on U.S. drug prices continues, with the CMS lining up the drugs for round 3 of negotiations, which will set maximum fair prices to go into effect in 2028. The slate includes 15 drugs and, for the first time, opens the negotiations to Part B drugs, as well as Part D. Consequently, seven of the 15 selected drugs are biologics. http://www.bioworld.com/articles/728341 Wed, 28 Jan 2026 12:00:00 -0500 https://www.bioworld.com/articles/728341-us-cms-names-its-choices-for-third-round-of-price-negotiations Drug capsule and dollar sign Adolore Biotherapeutics Inc. has announced that the FDA has granted orphan drug designation to the company’s Kv7-activating rdHSV-CA8* gene therapy for treatment of primary and secondary erythromelalgia. http://www.bioworld.com/articles/728034 Fri, 16 Jan 2026 08:00:00 -0500 https://www.bioworld.com/articles/728034-adolores-ca8-gene-therapy-designated-orphan-drug DNA double helix illustration with section being removed in red Askbio Inc., a subsidiary of Bayer AG, has received IND clearance from the FDA for AB-1009, an AAV gene therapy being developed for the treatment of late-onset Pompe disease. http://www.bioworld.com/articles/727855 Fri, 09 Jan 2026 08:00:00 -0500 https://www.bioworld.com/articles/727855-fda-clears-askbios-ind-for-ab-1009-for-late-onset-pompe-disease Arbele Pte Ltd.’s ARB-1002 has been awarded orphan drug designation by the FDA for the treatment of pancreatic cancer. ARB-1002 is an antibody-drug conjugate comprising an anti-CDH17 monoclonal antibody chemically linked to a potent cytotoxic agent. http://www.bioworld.com/articles/727744 Thu, 08 Jan 2026 08:00:00 -0500 https://www.bioworld.com/articles/727744-arbeles-arb-1002-designated-orphan-drug-for-pancreatic-cancer Immusoft of CA Inc., a wholly owned subsidiary of Immusoft Corp., has announced that the FDA has granted orphan drug designation to ISP-002, the company’s investigational engineered B-cell therapy for the treatment of mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome. http://www.bioworld.com/articles/727154 Tue, 16 Dec 2025 08:00:00 -0500 https://www.bioworld.com/articles/727154-immusofts-isp-002-designated-us-orphan-drug-for-mps-ii Missing puzzle piece and broken DNA chain The EU finally reached agreement on an update of the 20-year-old pharmaceutical legislation, more than five years after the EU Commission first put forward the case for reform and following two and a half years of negotiations on the new rules. http://www.bioworld.com/articles/726988 Mon, 15 Dec 2025 12:00:00 -0500 https://www.bioworld.com/articles/726988-eu-agrees-to-pharma-legislation-but-the-industry-isnt-happy EU flag, pills, syringe Vigencell Inc. plans to seek conditional approval in South Korea for VT-EBV-N, an antigen-specific killer T-cell therapy for natural killer T-cell lymphoma, after gaining positive top-line data from a phase II study Nov. 25. http://www.bioworld.com/articles/726794 Tue, 02 Dec 2025 00:00:00 -0500 https://www.bioworld.com/articles/726794-vigencells-cell-therapy-for-lymphoma-meets-phase-ii-endpoint Kedrion SpA’s investigational plasma-derived treatment for congenital aceruloplasminemia has been awarded European orphan drug designation by the EMA. The company is working to advance this treatment toward clinical development in Europe. http://www.bioworld.com/articles/726650 Fri, 28 Nov 2025 08:00:00 -0500 https://www.bioworld.com/articles/726650-kedrions-aceruloplasminemia-treatment-designated-eu-orphan-drug Bags of plasma Vigencell Inc. plans to seek conditional approval in South Korea for VT-EBV-N, an antigen-specific killer T-cell therapy for natural killer T-cell lymphoma, after gaining positive top-line data from a phase II study Nov. 25. http://www.bioworld.com/articles/726510 Wed, 26 Nov 2025 12:00:00 -0500 https://www.bioworld.com/articles/726510-vigencells-cell-therapy-for-lymphoma-meets-phase-ii-endpoint