Rare and Niche Diseases Catalyst Monitor - Key Event Analysis H1 2012

Introduction

GlobalData’s new report, "Rare and Niche Diseases Catalyst Monitor - Key Event Analysis H1 2012" takes a look at rare and niche diseases, which are an area of intense interest in the global biopharmaceutical industry today. The passage of the Orphan Drug Act in the United States in 1983 was the initial impetus behind the development of products for these diseases. Since that time, similar legislation has been enacted in the UK, Europe, Japan, Singapore, and Australia. As a result of this global legislation, numerous products have been brought to market to treat diseases for which there were no existing therapies, saving countless lives worldwide. As biotechnology and pharmaceutical companies look for ways to reinvigorate their product pipelines, they are increasingly focusing on developing products for rare and niche diseases. The market for many of these diseases is virtually wide open and will continue to be a major driving force in the industry.

Scope

- AVI BioPharma’s eteplirsen, for Duchenne muscular dystrophy: expected release of unblinded data from a Phase IIb trial.
- Elelyso (taliglucerase alfa) for Gaucher disease, developed by Protalix in partnership with Pfizer: FDA decision on New Drug Application (NDA) resulted in approval.
- Talon Therapeutics’ Marqibo (Optisome-encapsulated formulation of vincristine), for adult Philadelphia chromosome negative (Ph-) acute lymphoblastic leukemia in second or greater relapse or that has progressed following two or more lines of anti-leukemia therapy: expected FDA decision on NDA.
- Amicus Therapeutics is developing Amigal (migalastat HCl) for Fabry disease in partnership with GlaxoSmithKline: expected completion of Phase II study (Study 013) in H1.
- Intermune’s Esbriet (pirfenidone), for idiopathic pulmonary fibrosis (IPF): additional launches in France, Spain, and Italy in H1.

Highlights

Reasons to buy

- Understand the key therapeutic areas that are driving the rare and niche disease markets, which will enable you to strategically focus your product development.
- Develop business strategies by understanding the trends shaping and driving the global rare and niche diseases market.
- Drive revenues by understanding the key trends, innovative products and technologies, market segments and companies likely to impact the global rare and niche diseases market in the future.
- Formulate effective sales and marketing strategies by understanding the competitive landscape and by analyzing the performance of various competitors.
- Identify emerging players with potentially strong product portfolios and create effective counter-strategies to maintain or gain a competitive advantage.