Orphan Drug Designations - Pharma

Relevant Publications

Feasibility of disease terminology systems for mapping orphan conditions and therapeutic indications of designated orphan medicines in the European Union.

Issue Release: 2024

Analysis of US Food and Drug Administration new drug and biologic approvals, regulatory pathways, and review times, 1980-2022.

Issue Release: 2024

Pozelimab, a human monoclonal immunoglobulin for the treatment of CHAPLE disease.

Issue Release: 2024

Comparative Analysis of Post-Authorization Measures for Advanced Medicinal Products Authorized in the European Union and in the United States of America Between 2009 and 2023.

Issue Release: 2024

Expediting treatments in the 21st century: orphan drugs and accelerated approvals.

Issue Release: 2024

Randomized controlled trial data for successful new drug application for rare diseases in the United States.

Issue Release: 2023

Pozelimab: First Approval.

Issue Release: 2023

A comprehensive study of the rare diseases and conditions targeted by orphan drug designations and approvals over the forty years of the Orphan Drug Act.

Issue Release: 2023

Traits, trends and hits of orphan drug designations in cystic fibrosis.

Issue Release: 2023

Special FDA designations for drug development: orphan, fast track, accelerated approval, priority review, and breakthrough therapy.

Issue Release: 2023

Successfully Navigating FDA Orphan Drug and Rare Pediatric Disease Designations for AAV9-hPCCA Gene Therapy: The NIH Pave-GT Experience.

Issue Release: 2023

Long-term evaluation of clinical success and safety of omadacycline in nontuberculous mycobacteria infections: a retrospective, multicenter cohort of real-world health outcomes.

Issue Release: 2023

Cross-national comparative study of orphan drug policies in Saudi Arabia, the United States, and the European Union.

Issue Release: 2023

Clinical benefit, development, innovativeness, trials, epidemiology, and price for cancer drugs and indications with multiple special FDA designations.

Issue Release: 2023

Discovery, synthesis, activities, structure-activity relationships, and clinical development of combretastatins and analogs as anticancer drugs. A comprehensive review.

Issue Release: 2023

Decisions on Non-oncology Breakthrough Therapy Designation Requests in 2017-2019.

Issue Release: 2023

Drugs and biologics receiving FDA orphan drug designation: an analysis of the most frequently designated products and their repositioning strategies.

Issue Release: 2022

Toripalimab: the First Domestic Anti-Tumor PD-1 Antibody in China.

Issue Release: 2022

Initial and supplementary indication approval of new targeted cancer drugs by the FDA, EMA, Health Canada, and TGA.

Issue Release: 2022

Orphan drug development in alpha-1 antitypsin deficiency.

Issue Release: 2022

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