What if Evie could meet the people who developed the drug for her ultra-rare disease?

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When Evie was born, she wasn't expected to survive due to the rare metabolic bone disease hypophosphotasia (HPP), which affects bone growth, breathing, and mobility. At the time of her birth, there was no treatment.

Strensiq® is an enzyme replacement therapy approved to treat patients like Evie with perinatal/infantile or juvenile-onset hypophosphatasia (HPP). It works by restoring the alkaline phosphatase (ALP) enzyme in order to promote and improve bone health.

Strensiq® was brought to market in October 2015 after being granted Breakthrough Therapy, Priority Review, and Orphan Drug designations by the FDA.

Asfotase alfa is an enzyme replacement therapy approved to treat patients like Evie with perinatal/infantile or juvenile-onset hypophosphatasia (HPP). It works by restoring the alkaline phosphatase (ALP) enzyme in order to promote and improve bone health.

Asfotase alfa was brought to market in October 2015 after being granted Breakthrough Therapy, Priority Review, and Orphan Drug designations by the U.S. Food and Drug Administration.

Safety Assessment is a critical step along the drug discovery and development continuum. Charles River performed toxicology studies, speciality musculoskeletal work, pathology, and imaging support for Strensiq®. Charles River's work in preclinical development and clinical support services was vital to ensuring the drug's dosage levels would be safe for patients like Evie.

Safety Assessment is a critical step along the drug discovery and development continuum. Charles River performed toxicology studies, speciality musculoskeletal work, pathology, and imaging support for asfotase alfa. Charles River's work in preclinical development and clinical support services was vital to ensuring the drug's dosage levels would be safe for patients like Evie.

Oncology is one of eight therapeutic areas that Charles River specializes in. The World Health Organization projects a 70% increase in cancer cases over the next 20 years. 4

Charles River’s work in oncology drug development spans from target identification to IND. Charles River oncology scientists worked on half of the novel oncology therapies approved by the FDA in 2016.

Alexion is a global biopharmaceutical company whose life-changing therapies serve patients and families affected by rare diseases. Specializing in innovation, development, and commercialization, Alexion partners with Charles River for safety assessment, as well as clinical laboratory and manufacturing support.

Driven by the pursuit of excellent science and a strong sense of purpose, Charles River is a leading global contract research organization (CRO), offering services that span the entire drug discovery and development pipeline. We celebrated our 70th anniversary in 2017.

Basic Research is the first step along the drug discovery and development continuum. With a global footprint of commercial breeding facilities, rigorous biosecurity and genetic standards, and research support services such as surgery and preconditioning, Charles River provides models to meet a variety of research needs.

Discovery is a critical phase in the life of any new drug. Our multidisciplinary teams use the latest technologies and platforms to advance our partners' discovery programs across therapeutic areas from hit ID to IND.

Clinical Support is a key part of the drug discovery and development continuum. It's essential that assays and testing methods used in the nonclinical lab transition easily to the clinical phase. As clinical trials begin, further in vitro and in vivo nonclinical studies performed at our global network of facilities provide additional information to support the continuation of clinical trials. Charles River performed sample analysis for Strensiq® clinical trials.

Clinical Support is a key part of the drug discovery and development continuum. It's essential that assays and testing methods used in the nonclinical lab transition easily to the clinical phase. As clinical trials begin, further in vitro and in vivo nonclinical studies performed at our global network of facilities provide additional information to support the continuation of clinical trials. Charles River performed sample analysis for alfotase alfa clinical trials.

Manufacturing is the last step along the drug discovery and development continuum. Charles River works to ensure the safe production and release of its clients' manufactured products.

From biologics to small molecule drugs, medical devices to personal care products, Charles River designs manufacturing and testing programs that promote the optimum use of resources.

It takes an average of 15 years of research and development for a new drug to reach the market. Charles River strives to provide the highest quality service, science, and regulatory support to accelerate the development of novel therapeutics to reach the patients who need them most.

Our Eureka blog allows scientists to share ideas on cutting-edge bench science driving drug discovery and development.

Cardiovascular is one of eight therapeutic areas that Charles River specializes in. Heart disease is the leading cause of death in the U.S. for both women and men. 5

We support our partners through the entire cardiovascular research process. We also offer preclinical cardiac risk assessment services—from GLP hERG and ADP assays to a comprehensive survey of cardiac ion channels and cardiomyocyte assays.

Neuroscience is one of eight therapeutic areas that Charles River specializes in. We offer end-to-end expertise in neurodegenerative diseases, pain, psychiatric disease, and other neurological indications.

In 2017, Charles River expanded the suite of services to include microdialysis, EEG, electrophysiology, and behavior testing for multiple species by acquiring Brains On-Line, a neuroscience-focused CRO with sites in North America and Europe.

Inflammation is one of eight therapeutic areas that Charles River specializes in. We offer a full portfolio of products and services in support of millions affected by conditions like rheumatoid arthritis and psoriasis.

We are proud to have worked on an arthritis biosimilar with Boehringer Ingelheim, which was accepted for regulatory review by the European Medicines Agency and FDA in January 2017.

Musculoskeletal Disease is one of eight therapeutic areas that Charles River specializes in. Millions of people are affected by osteoporosis, arthritis, and other musculoskeletal diseases.

Charles River offers services in the area of musculoskeletal research that combine established discovery and preclinical program development expertise with a host of novel bone research technologies.

Metabolic Disease is one of eight therapeutic areas that Charles River specializes in. Millions of people are at risk of increased heart disease, stroke, and diabetes.

Charles River's expertise includes the progression and analysis of compounds and agents targeting a range of conditions such as diabetes, obesity, bone disease, thyroid disease, and cholesterol and lipid disorders.

Respiratory Disease is one of eight therapeutic areas that Charles River specializes in. Asthma, chronic obstructive pulmonary disease (COPD), cystic fibrosis, and tuberculosis affect millions of people around the world.

Charles River recently served as a $25,000 sponsor to Great Strides®, a walk supporting the Cystic Fibrosis Foundation. More than 150 participants (staff and family) from our Cleveland and Ashland, Ohio sites participated in the walk.

Ophthalmology is one of eight therapeutic areas that Charles River specializes in. We offer an integrated approach to ophthalmology research.

Compounds or implantable devices intended for ocular treatment can be assessed for efficacy, safety, and toxicity in multiple species. Ocular effects of compounds administered by other routes, such as oral, can also be characterized.

Charles River strives to immerse itself in niche markets, stay abreast of regulatory changes, and keep up with new technologies in order to support the specific testing and manufacturing requirements of all product types including biosimilars, vaccines, and cell and gene therapies.

As the world's population rises, so does the need for innovative drugs and therapies to help the millions of people fighting diseases and chronic conditions. Charles River and its industry partners are committed to improving the health and well-being of patients everywhere by continuing to push drug discovery and development advancements forward.

Charles River is proud to work with the world's leading pharmaceutical companies by providing expertise along the entire drug discovery and development continuum. Charles River is a customer-focused organization committed to advancing science and helping clients reach their goals.

More than 30% of Charles River's total work is represented by strategic relationships where we work closely with clients.

Between 25 and 35 million Americans have a rare disease and 50% are pediatric patients. That's why Charles River partners with nonprofits, academia, and industry to discover novel drugs and methods of study for diseases like Huntington's and muscular dystrophy. We also validate mouse models to reveal new functional and behavioral endpoints in preclinical animal models.

Every Step Of The Way

Interactive Experience

This network diagram starts with Evie's story then expands to reveal how Charles River is working in every area along the drug discovery and development continuum toward bringing life-saving drugs to patients.To learn more and explore connections, start by clicking on any node below. Click in the white space to return to the diagram in full view.

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References

  1. “Key Statistics for Lung Cancer,” American Cancer Society, accessed March 2, 2017, https://www.cancer.org/cancer/lungcancer-non-smallcell/detailedguide/non-small-cell-lung-cancer-key-statistics
  2. Julian R. Molina, MD, PhD, Ping Yang, MD, PhD, Stephen D. Cassivi, MD, Steven E. Schild, MD, and Alex A. Adjei, MD, PhD, "Non–Small Cell Lung Cancer: Epidemiology, Risk Factors, Treatment, and Survivorship," Mayo Clinic Proceedings 83(5) (2008): 584–594, accessed March 3, 2017, doi: 10.4065/83.5.584
  3. "FDA approves first blood test to detect gene mutation associated with non-small cell lung cancer," U.S. Food & Drug Administration, accessed June 1, 2016, https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm504488.htm
  4. “Cancer,” World Health Organization, accessed March 2, 2017, https://www.who.int/mediacentre/factsheets/fs297/en/
  5. "Heart Disease Facts," Centers for Disease Control and Prevention," accessed March 3, 2017, https://www.cdc.gov/heartdisease/facts.htm
  6. "U.S. and World Population Clock," United States Census Bureau, accessed March 3, 2017, https://www.census.gov/popclock/
  7. "Medicines in Development," PhRMA, accessed March 3, 2017, https://phrma.org/science/in-the-pipeline/medicines-in-development
  8. "Chronic diseases and health promotion," World Health Organization, accessed March 3, 2017, https://www.who.int/chp/en/
  9. "Hypophosphatasia," National Institutes of Health, accessed February 6, 2018, https://ghr.nlm.nih.gov/condition/hypophosphatasia#statistics
  10. "ALPL Gene," National Institutes of Health, accessed February 6, 2018, https://ghr.nlm.nih.gov/gene/ALPL#conditions
  11. "Developing Products for Rare Diseases & Conditions," Food and Drug Administration, accessed February 6, 2018, https://www.fda.gov/ForIndustry/DevelopingProductsforRareDiseasesConditions/ucm2005525.htm
  12. "FDA Approves Strensiq™ (Asfotase Alfa) For Treatment Of Patients With Perinatal-, Infantile- And Juvenile-Onset Hypophosphatasia (HPP)," Alexion, accessed February 6, 2018. https://news.alexionpharma.com/press-release/product-news/fda-approves-strensiq-asfotase-alfa-treatment-patients-perinatal-infantil
  13. “Cancer,” World Health Organization, accessed February 6, 2018 https://www.who.int/mediacentre/factsheets/fs297/en/
  14. "Heart Disease Facts," Centers for Disease Control and Prevention, accessed February 6, 2018, https://www.cdc.gov/heartdisease/facts.htm
  15. "U.S. and World Population Clock," United States Census Bureau, accessed February 6, 2018, https://www.census.gov/popclock/
  16. "Medicines in Development," PhRMA, accessed February 6, 2018, https://phrma.org/science/in-the-pipeline/medicines-in-development
  17. "Chronic diseases and health promotion," World Health Organization, accessed February 6 2018, https://www.who.int/chp/en/
  18. "Rare Diseases, Genomics and Public Health: An Expanding Intersection," Centers for Disease Control and Prevention, accessed February 6, 2018, https://blogs.cdc.gov/genomics/2016/02/17/rare-diseases/
  19. "About Rare Diseases," Orphanet, accessed February 6, 2018, https://www.orpha.net/consor/cgi-bin/Education_AboutRareDiseases.php?lng=EN
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