Clinical OMICS

NOV-DEC 2017

Healthcare magazine for research scientists, labs, pathologists, hospitals, cancer centers, physicians and biopharma companies providing news articles, expert interviews and videos about molecular diagnostics in precision medicine

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Page 12 of 47 November/December 2017 Clinical OMICs 11 T wo FDA drug approvals earlier this year point to an encouraging future for "precision medicine" — an approach for disease treatment that tailors medical therapies, including medications, to the needs of individ- ual patients. These approvals involve diseases resulting from particular genetic characteristics identified by laboratory testing. • In mid-May, FDA announced that we expanded the approval of Kalydeco (ivacaftor), enabling a larger number of patients with cystic fibrosis (CF) to benefit from the drug. The expanded approval includes CF patients with one of 23 additional rare mutations. Kalydeco is now indicated for 33 CF mutations, up from 10 previously. • Also in May, we announced expanded approval for Keytruda (pembrolizumab) to treat patients whose cancers have a specific genetic feature. This is the first time FDA has approved a cancer treatment based on a genetic feature, rather than the location in the body where the cancer originated. FDA has approved many more advances in precision medicines, also called "targeted therapies." In the past three years alone, our Center for Drug Evaluation and Research has approved more than 25 new drugs that benefit patients with specific genetic char- acteristics. And we have approved many more new uses — also based on specific genetic characteristics — for drugs already on the market. Some of these drug approvals are for patients with rare genetic disorders. Others are new targeted therapies to treat cancer, hepatitis C, or HIV. Medication dosing for specific diseases may also be tai- lored to the individual. Precision medicine holds great promise, but to continue developing targeted therapies, we will need sci- entific advances in the use and devel- opment of biomarkers. Biomarkers are indicators in the body that can be measured—like blood pressure, blood sugar, and tumor size. Tests to identify genetic variants are another form of biomarker. Biomarkers can enable health care professionals and researchers to identify patients at risk of disease, determine the stage of a disease, and predict the likeli- hood that a patient will benefit from a drug. They also play a role in drug development. A particular biomarker, for example, can be used to identify appropriate candidates for a clinical trial, such as those patients likely to respond to treatment. This can make it easier and faster to recruit patients and may result in a shorter time for drug approval. In a similar way, biomarkers can sometimes identify positive treatment effects before tra- ditional clinical endpoints would. For instance, biomarkers might show a tumor shrinking before improvement in a patient's condition is detected. So, using biomarkers in clinical trials can speed up the time it takes for an inves- tigative drug to reach a patient. The ability to identify useful bio- markers depends on how well scien- tists understand the disease they are seeking to treat. In some areas, such as cancer and infectious diseases, we have made real progress in under- standing how these diseases develop and how to treat them with drug ther- apy. FDA continues to encourage drug developers to use strategies based on biomarkers. One way we do that is by ensuring that a given biomarker is really able to single out those patients who are likely to respond to a spe- cific drug. Another way is using bio- markers to identify people whose disease is progressing rapidly. Beyond working on biomarkers for individ- ual products, FDA also works with stakeholders and scientific consor- tia in qualifying biomarkers that can be used in the development of many drugs. Once qualified, these biomark- ers may be used in the specified man- ner by any drug sponsor. New provisions under the recently passed 21st Century Cures Act provide direction and opportunity for FDA to strengthen the science of biomarkers and to advance precision medicine. We believe it is important to make drugs such as Kalydeco and Keytruda avail- able to as many patients as can benefit from them. FDA is actively pursuing more advances in targeted therapies. Errata: On page 29 of the October- November issue, Eric Ostertag was incor- rectly identified as the CEO of Caribou Biosciences. He is the CEO of Poseida Therapeutics, and we incorrectly stated that Jennifer Doudna left Editas to found Caribou Biosciences, when the company was already operating when Doudna joined. We regret these errors. Recent FDA Approvals Demonstrate Promise of Precision Medicine OP-ED Janet Woodcock, M.D. Director, FDA Center for Drug Evaluation and Research

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