Shire is 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. The bulk of the work will be carried out at TIGEM’s facility in Naples.

“Shire’s partnership with Fondazione Telethon is another way for us to ensure that we expand into new disease areas and enhance our collaborative relationships with academic institutions,” states Philip J. Vickers, svp R&D for Shire Human Genetic Therapies (HGT). “Shire’s developmental expertise combined with TIGEM’s early-stage research capabilities will enable us to accelerate our discovery and development efforts.”

U.K.-based Shire operates three primary divisions. Its specialty pharmaceuticals business is focused on developing small-molecule drugs for indications within the field of behavioral health (particularly attention deficit/hyperactivity disorder—ADHD), and gastrointestinal diseases, as well as other indications including major depressive disorder, schizophrenia, and essential thrombocythemia. Shire’s HGT business is dedicated to the research, development, and commercialization of products for patients with rare diseases, including Duchenne muscular dystrophy, Hunter syndrome, metachromatic leukodystrophy, and Sanfilippo A and B syndromes. The firm’s third business arm is centered on developing regenerative medicines, is headed by Dermagraft for diabetic foot ulcers, and Vascugel for hemodialysis access.

Shire’s specialty pharmaceuticals business reported sales of $1.44 billion for the first six months of 2012 (to June 30), up 12% on the same period in 2011. Major drivers of increased sales were the ADHD drug Vyvanse, which achieved sales of $526 million (up 36%), and Intuniv, for which sales were also up 36%, at $138 million. The firm’s marketed HGT products Replagal (Fabry disease), Elaprase (Hunter syndrome), Vpriv (Gaucher’s disease) and Firazyr (acute attacks of hereditary angioedema), achieved combined sales of $711 million, up 21% on the first six months of 2011.

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