Hematology and Immunology Strategic Portfolio Supports Sobi's Narrow Moat
Given Sobi's cash flow generation from its hemophilia business and mature product Synagis, the firm ended 2024 with a net debt/EBITDA ratio of 1.6 times. We expect the firm can support this level of ...
Hosted on MSN4h
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 (RPDD) to Arbor Biotechnologies' gene editing therapeutic, ABO-101, to treat ...
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 Oncology13h
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 ...
TMCnet18h
U.S. FDA Grants Orphan Drug Designation to Ariceum Therapeutics' Proprietary Radiopharmaceutical Cancer Therapy
Ariceum Therapeutics is a private, clinical stage radiopharmaceutical company focused on the diagnosis and precision treatment of certain neuroendocrine and other aggressive, hard-to-treat cancers.
TMCnet2d
Quince Therapeutics Receives Notice of Allowance Covering Innovative Method of Use for Lead Indication Ataxia-Telangiectasia
Quince Therapeutics, Inc. (Nasdaq: QNCX) is a late-stage biotechnology company dedicated to unlocking the power of a patient's own biology for the treatment of rare diseases. For more information on ...
Cumberland Pharmaceuticals' Duchenne Candidate Shows Improved Heart Function In Patients With Associated Heart Disease
Cumberland Pharmaceuticals' Phase 2 trial shows ifetroban improves heart function in Duchenne muscular dystrophy patients.
Nature14d
Drug discovery articles from across Nature Portfolio
Drug discovery is the process through which potential new medicines are identified. It involves a wide range of scientific disciplines, including biology, chemistry and pharmacology. Large-scale ...
Business Wire15d
Zai Lab Receives Orphan Drug Designation from the U.S. FDA for ZL-1310 (DLL3 ADC) for the Treatment of Small Cell Lung Cancer (SCLC)
SHANGHAI & CAMBRIDGE, Mass.--(BUSINESS WIRE)--Zai Lab Limited (NASDAQ: ZLAB; HKEX: 9688) today announced the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD ...
PharmaTimes15d
Tris Pharma announces positive results from ALLEVIATE-1 trial of cebranopadol
Phase 3 trial shows promising pain relief for acute pain patients Tris Pharma, Inc. has announced positive results from its ALLEVIATE-1 phase 3 clinical trial evaluating cebranopadol for treating ...