Orphan drugs are designated drug substances that are intended to treat rare or 'orphan' diseases. More than 7000 rare diseases are known that collectively affect some 6-7% of the developed world's population, but individually, any single rare disease may only affect a handful of people making them unattractive for the biopharmaceutical industry to target. Providing an up-to-date mongraph, this book covers the basic science, drug discovery and regulatory elements behind orphan drugs.
Orphan drugs are designated drug substances that are intended to treat rare or 'orphan' diseases. More than 7000 rare diseases are known that collectively affect some 6-7% of the developed world's population, but individually, any single rare disease may only affect a handful of people making them unattractive for the biopharmaceutical industry to target. Providing an up-to-date mongraph, this book covers the basic science, drug discovery and regulatory elements behind orphan drugs.
David C. Pryde is a medicinal chemist based at Pfizer Global Research and Development Neusentis research unit. He earned his B.Sc. in Pure and Applied Chemistry from the University of Strathclyde, Scotland (1991), PhD from the University of Nottingham, England, under the supervision of Professor Gerry Pattenden in the field of biomimetic radical cyclisations (1994) and carried out postdoctoral research with Professor Albert Meyers at Colorado State University on stereoselective metallation chemistry before joining Pfizer in 1997. He has worked in several therapeutic areas from cardiovascular and pain, to sexual health and anti-infectives and identified development candidates in each of these areas. His research interests include all aspects of medicinal chemistry, synthetic biologics and chemical biology methodology. He has coauthored more than 50 papers and patents. Michael J Palmer is a medicinal chemist currently specialising in Neglected Disease Research, and in particular malaria. He earned his B.Sc. in chemistry at the University of Nottingham in 1975 and his M.Sc. from the University of East Anglia in 1981 under the supervision of Pr. Alan Katritzky and Pr. Sandy McKillop. He worked for Pfizer Global R and D, Sandwich for 36 years, spanning many therapeutic areas and during which time he was a leading part of chemistry teams that helped to take the antihypertensive alpha blocker CarduraTM to market and 6 drugs to the clinic, including the once daily PDE5 agent PF-489791, currently in phase II for Raynaud's disease. Mike has over 40 patents and publications, and has been recognised by the American Chemical Society with a TAOC award for his contributions to the chemistry community. He recently co-edited Neglected Diseases and Drug Discovery for the Royal Society of Chemistry.
Inhaltsangabe
Introduction Foreword on Rare Diseases Definitions, History and Regulatory Framework for Rare Diseases and Orphan Drugs Diagnosis of Rare Inherited Diseases Treating Rare Diseases: Business Model For Orphan Drug Development Disease Advocacy Organizations Lysosomal Storage Diseases Discovery and Clinical Development of Idursulfase (Elaprase®) for the Treatment of Mucopolysaccharidosis II (Hunter Syndrome) Discovery and Development of ILARIS® for the Treatment of Cryopyrin-Associated Periodic Syndromes Discovery and Development of Tafamidis for the Treatment of TTR Familial Amyloid Polyneuropathy Small Molecules that Rescue F508del CFTR as Cystic Fibrosis Therapies Rare Neuromuscular Diseases Unleashing the Power of Semi-synthesis: The Discovery of Torisel® Treatments for Pulmonary Arterial Hypertension Soliris (Eculizumab): Discovery and Development The Discovery and Development of Ruxolitinib for the Treatment of Myelofibrosis Possible Solutions to Accelerate Access to Rare Disease Treatments
Introduction Foreword on Rare Diseases Definitions, History and Regulatory Framework for Rare Diseases and Orphan Drugs Diagnosis of Rare Inherited Diseases Treating Rare Diseases: Business Model For Orphan Drug Development Disease Advocacy Organizations Lysosomal Storage Diseases Discovery and Clinical Development of Idursulfase (Elaprase®) for the Treatment of Mucopolysaccharidosis II (Hunter Syndrome) Discovery and Development of ILARIS® for the Treatment of Cryopyrin-Associated Periodic Syndromes Discovery and Development of Tafamidis for the Treatment of TTR Familial Amyloid Polyneuropathy Small Molecules that Rescue F508del CFTR as Cystic Fibrosis Therapies Rare Neuromuscular Diseases Unleashing the Power of Semi-synthesis: The Discovery of Torisel® Treatments for Pulmonary Arterial Hypertension Soliris (Eculizumab): Discovery and Development The Discovery and Development of Ruxolitinib for the Treatment of Myelofibrosis Possible Solutions to Accelerate Access to Rare Disease Treatments
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