Search results for "Marketing authorization"
showing 7 items of 7 documents
Myotonic dystrophy type 1 drug development: A pipeline toward the market
2021
Highlights • Myotonic dystrophy, a neuromuscular disease, affects at least around half a million people worldwide. • Close to two dozen preclinical and clinical drug development programs active. • Drugs encompass new chemical entities, repurposing, oligonucleotide, and gene therapy. • Tideglusib, mexiletine, and metformin are close to reaching marketing authorization.
Trends in individual reimbursement of orphan drugs in Latvia in 2008–2011
2014
Orphan drugs (ODs) are medicinal products intended for diagnosis, prevention or treatment of life-threatening or very serious diseases affecting less than 5 in 10 000 people in the European Union (EU). These drugs are called “orphans” because the pharmaceutical industry has little interest, under normal market conditions, in developing and marketing products intended for only a small number of patients suffering from very rare conditions. Because of the small market, ODs are often very expensive. Whereas decisions surrounding orphan designation and marketing authorization of ODs are taken at the EU level, decisions governing pricing and reimbursement of ODs are a member state responsibility…
Manufacturing, characterization and control of cell-based medicinal products: challenging paradigms toward commercial use
2015
During the past decade, a large number of cell-based medicinal products have been tested in clinical trials for the treatment of various diseases and tissue defects. However, licensed products and those approaching marketing authorization are still few. One major area of challenge is the manufacturing and quality development of these complex products, for which significant manipulation of cells might be required. While the paradigms of quality, safety and efficacy must apply also to these innovative products, their demonstration may be demanding. Demonstration of comparability between production processes and batches may be difficult for cell-based medicinal products. Thus, the development …
Biowaiver monograph for immediate-release solid oral dosage forms: acetylsalicylic acid.
2012
A biowaiver monograph for acetylsalicylic acid (ASA) is presented. Literature and experimental data indicate that ASA is a highly soluble and highly permeable drug, leading to assignment of this active pharmaceutical ingredient (API) to Class I of the Biopharmaceutics Classification System (BCS). Limited bioequivalence (BE) studies reported in the literature indicate that products that have been tested are bioequivalent. Most of the excipients used in products with a marketing authorization in Europe are not considered to have an impact on gastrointestinal motility or permeability. Furthermore, ASA has a wide therapeutic index. Thus, the risks to the patient that might occur if a nonbioequi…
Pharmacy and pharmacology of biosimilars
2008
Biosimilar medicines are biological medicinal products that can obtain a marketing authorization in the EU after the original product (biological reference medicine) has run out of patent. As a prerequisite, studies including clinical trials are to be conducted to compare the quality, safety, and efficacy of the biosimilar and reference medicine. Due to the specific characteristics of biopharmaceuticals like complex 3-dimensional (glyco) protein structure, immunogenicity, production in living organisms, which causes heterogeneity, complex manufacturing process and analysis, interchangeability of the biosimilar with its reference drug product is not guaranteed. In addition, INN (internationa…
Regulatory evaluation of Glybera in Europe — two committees, one mission
2013
Representing the first gene therapy to be approved in the Western world, alipogene tiparvovec (Glybera; Uniqure) has recently been said to have had a “substantial impact from a regulatory perspective” (Nature Rev. Drug Discov. 11, 664; 2012) 1 . The therapy was granted marketing authorization in the European Union for the treatment of lipoprotein lipase deficiency, which results in a clinically heterogeneous condition with a risk of potentially life-threatening pancreatitis 2 , at the end of 2012. The decision followed a positive opinion by the European Medicines Agency (EMA)’s Committee for Medicinal Products for Human Use (CHMP) 3
Development of extracellular vesicle-based medicinal products: A position paper of the group “Extracellular Vesicle translatiOn to clinicaL perspecti…
2021
International audience; Extracellular vesicles (EV) are emergent therapeutic effectors that have reached clinical trial investigation. To translate EV-based therapeutic to clinic, the challenge is to demonstrate quality, safety, and efficacy, as required for any medicinal product. EV research translation into medicinal products is an exciting and challenging perspective. Recent papers, provide important guidance on regulatory aspects of pharmaceutical development, defining EVs for therapeutic applications and critical considerations for the development of potency tests. In addition, the ISEV Task Force on Regulatory Affairs and Clinical Use of EV-based Therapeutics as well as the Exosomes C…