FDA grants orphan drug status to Arbor Biotechnologies’ PH1
The US Food and Drug Administration (FDA) has granted orphan drug designation (ODD) and rare paediatric disease designation ...
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RARE Reports New Upbeat Data From Sanfilippo Syndrome Drug Study
Ultragenyx Pharmaceutical RARE reported new findings from an ongoing, pivotal study of its investigational candidate, UX111 (ABO-102) AAV gene therapy, for Sanfilippo syndrome type A (MPS IIIA). Per ...
Medexus targets $100M annual revenue for GRAFAPEX within 5 years post-launch
Q3 2025 Management View CEO Ken d'Entremont highlighted the successful FDA approval of GRAFAPEX, the branded name for treosulfan for injection in the U.S., which holds Orphan Drug Designation, ...
Targeted Oncology10h
FDA Grants Key Oncology Approvals and Designations in January 2025
On January 7, 2025, the FDA granted priority review to the new drug application for sunvozertinib (DZD9008), an oral EGFR ...
Atsena Therapeutics to Present ATSN-201 Safety and Efficacy Data at The Macula Society’s 48th Annual Meeting
Atsena Therapeutics, a clinical-stage gene therapy company focused on bringing the life-changing power of genetic medicine to reverse or prevent blindness, today announced that safety and efficacy ...
NurExone Biologic Inc: NurExone Forms Exo-Top Inc. in the U.S. for Exosome Manufacture and Commercialization
NurExone Biologic Inc. (TSXV: NRX) (OTCQB: NRXBF) (Germany: J90) ("NurExone" or the "Company") is pleased to announce the formation of a U.S.-based ...
PharmaTimes14h
FDA grants Orphan Drug Designation to Ariceum cancer therapy
Ariceum Therapeutics, a private biotech company based in Berlin, has announced that the US Food and Drug Administration (FDA) ...
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U.S. FDA Grants Orphan Drug Designation to Ariceum Therapeutics’ Proprietary Radiopharmaceutical Cancer Therapy
Ac-satoreotide is a first-in-class Actinium-labelled somatostatin receptor 2 antagonist targeting extensive-stage Small Cell Lung Cancer or Merkel Cell ...
PharmiWeb18h
Global Frontotemporal Disorders Treatment Market Poised for Growth, Reaching USD 451.4 Million by 2028 at a CAGR of 4.6%
The global market for frontotemporal disorders treatment, valued at USD 343.8 million in 2022, is projected to experience steady year-on-year growth, with an anticipated Compound Annual Growth Rate ...