CF Foundation Supports Shire’s Cystic Fibrosis Technology Development

Specialty biopharmaceutical company Shire has just announced a new partnership with the Cystic Fibrosis Foundation to develop technology that can maintain and/or restore lung function, as well as protect against respiratory infections — two primary health concerns in people living with cystic fibrosis (CF). The announcement was made via webcast during yesterday’s company Research & Development day, which outlined Shire’s extensive pipeline of promising therapeutics that the company believes will generate sales of about $3 billion by the year 2020.

Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT) the non-profit drug discovery and development affiliate of the Cystic Fibrosis Foundation, agreed to invest up to $15 million in Shire’s messenger RNA (mRNA) platform for cystic fibrosis. mRNA works to transmit coded genetic information to cellular ribosomes, which then translate the information into functional protein. Several diseases, including cystic fibrosis, are rooted in this genetic lack of specific functional proteins. With CFFT’s assistance, Shire will be working on treatments that deliver mRNA directly into the lungs, where local cells can utilize them to produce the missing functional protein.

Shire announced that the US Food and Drug Administration has granted its SHP607 protein replacement therapy fast-track status as a preventive treatment for retinopathy in premature babies. The company also disclosed it has set a revenue increase goal of $10 billion by 2020, comprised of $7 billion from existing approved drugs in the market, and $3 billion from newly approved and launched products.

Flemming Ornskov, Shire’s Chief Executive Officer, said Wednesday that the group had a “laser focus” on today’s most pressing rare diseases and specialty disorders with unmet medical needs, and that its focused strategy will help them meet their new “tough” growth target.

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In other cystic fibrosis news, a new study titled, “Early Respiratory Infection Is Associated with Reduced Spirometry in Children with Cystic Fibrosis” suggests children diagnosed with CF and who acquire an early lung infection are more likely to manifest impaired lung function at school age. Lead researcher Dr. Kathryn A. Ramsey from the Telethon Kids Institute, University of Western Australia, said, “What this study suggests is that we should be doing routine [checks for] lower respiratory track infections in all 0-2 year-old infants because it may be able to tell you how they will develop in life.”