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Nov 20, 2012

Shire Takes Another Step on Rare Diseases Path

  • Shire and Boston Children’s Hospital entered a three-year research collaboration in rare diseases. The goal of the collaboration is to develop novel therapies to treat rare pediatric diseases with high unmet medical need, leveraging the research expertise of The Manton Center for Orphan Disease Research at Boston Children’s and Shire’s development and commercialization capabilities.

    Shire will make an initial up-front payment to Boston Children’s and will have the opportunity to fund selected research programs. Following the completion of these programs, Shire will have an exclusive option to enter into a licensing agreement for each program.

    Upon entering into a licensing agreement, Boston Children’s will be eligible to receive certain development and commercial milestone payments, as well as royalties on product sales from Shire. Shire will have the primary responsibility for any further clinical development and commercialization of products arising from the collaboration.

    This collaboration marks another move forward in Shire’s focus on rare diseases. Just yesterday Shire reported a new initiative—the Shire Rare Disease Impact Report—to research the health, psycho-social, societal, and economic impact of rare diseases in patient and medical communities. The firm says surveys of patients, caregivers, physicians, payors, and thought leaders in the U.S. and the U.K. are under way. In addition, last month Shire reported providing $22 million in funding over five years to support a broad rare diseases collaboration with the Italian biomedical charity Fondazione Telethon’s Telethon Institute of Genetics and Medicine (TIGEM). The partnership will involve research on 13 rare disease indications that together encompass a number of lysosomal storage disorders and neurodegenerative diseases, which Shire says could provide a number of candidates for its early-stage pipeline.


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