Focus on the science

Drug research at HGT.

Turning to HGT, the picture looks quite different. Enzyme replacement therapy is still a relatively new area, but a very successful one in R&D terms. An extremely high proportion-around 75%-of development projects succeed in the clinic and HGT aims to run this process for three to five new products with the expectation that one product per year can move into development. The enzyme replacement therapy developed at Shire is unique in so far as they are almost all derived from human cells. This proprietary technology is ground breaking. As Sylvie Grégoire, the President of HGT says, "So far no drug using this technology has ever reached a peak level of sales, which shows how much potential there is for Shire in the future. However, it does make extra demands on us as a company. For example, while we would always prefer to outsource aspects of our business that others can do more cheaply, like manufacturing and some aspects of distribution, you can't always do that with orphan disease treatments. This means you have to manage more of the process yourself, but low volumes keep the costs under control. There are also special challenges in manufacturing enzyme replacement therapies. Specialty Pharma can do much of their manufacturing under license, but we need to invest in our own production facilities for HGT, so that we can retain full control of the quality and the impact this as on the efficacy of the final product."

HGT is also expanding its expertize into other new technologies. The agreement recently signed with Amicus doesn't only allow us the worldwide (excluding US) rights to three exciting new small molecule compounds for Fabry disease, Gaucher disease, and Pompe disease, but gives us access to their new 'chaperone technology' which could open up other possibilities in future.

The HGT business has changed out of all recognition since it was acquired by Shire in 2005. The investments we've made have accelerated its growth to a whole new level. HGT now has 43 approvals for ELAPRASE, our treatment for Hunter syndrome. Five new products were taken through the business development process, and there's the possibility of a further nine new products in the next seven years. At the same time, HGT now has the strength and breadth to take on joint venture partnerships, like the one with Amicus, which it would never have been able to contemplate before. As Sylvie Grégoire says, "HGT now has one of the strongest pipelines in the rare diseases sector. That's the beauty of this business. The challenge now is to execute it, and execute it well."

This brings new challenges for the HGT team. For the first time, HGT now has a number of different products at different stages of the development, approval and marketing process, and this demands new skills from the people who manage them. Having the US business on one site outside Boston, which will be completed by 2011 will be a significant advantage here, and in the meantime there's a new focus on developing people, and standardizing some of the management and sales processes, so that they can cope with a more complex business model. Looking forward, the scope for growth in HGT is substantial, with up to 300 genetic diseases that might be amenable to treatment some day.

Taking Specialty Pharma and HGT together, we're now seeing the results of a planned and considered period of pipeline growth. We now have a portfolio of products across all the phases of development, from lab, to trials, to marketing and sales. Some of these-like ELAPRASE and FIRAZYR-are providing hope for patients who previously had little to turn to; others-like VYVANSE and FOSRENOL-are offering effective treatments with clear benefits over previous alternatives. At Shire the objective is to seek global marketing approval whenever feasible.