Today's Rundown FDA surprises Sarepta by spurning its 2nd Duchenne drug Golodirsen snub highlights 'atypical communications,' heralds higher bar at FDA: analysts Synlogic scraps ammonia-lowering drug after early phase fail Pfizer's charity backs infectious disease projects Restoring neurons to preserve memory after heart attack or stroke Nabriva scores its first FDA nod with novel antibiotic Xenleta Featured Story | | | Monday, August 19, 2019 Sarepta pushed its first drug through approval, but its second drug is facing a different fate. The FDA rejected the New Drug Application for golodirsen, the follow-up to Exondys 51, Sarepta’s first treatment for Duchenne muscular dystrophy. |
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Top Stories Tuesday, August 20, 2019 Sarepta’s first move after its golodirsen rejection will be to negotiate a warning or other labeling for the drug rather than carry out more studies. But the implications of the rejection range beyond golodirsen. Tuesday, August 20, 2019 Synlogic has stopped development of SYNB1020 after the drug failed to lower blood ammonia levels in a phase 1b/2a trial. The engineered stain of Escherichia coli performed no better than placebo in the small trial. Tuesday, August 20, 2019 The Pfizer Foundation has awarded 20 grants to organizations involved in tackling infectious diseases in low- and middle-income countries. Monday, August 19, 2019 Injecting a microRNA-blocking drug into the hippocampus of rat models seemed to coax surrounding cells called astrocytes into becoming new neurons, Stanford researchers reported. Monday, August 19, 2019 With drug-resistant bacteria on the rise, antibiotics have struggled to keep up with the challenge. Now, with the FDA’s approval of a new antibiotic for bacterial pneumonia, some of that may change. Monday, the FDA approved Nabriva Therapeutics’ Xenleta (lefamulin) to treat community-acquired bacterial-pneumonia (CABP). Resources Sponsored by: Alexander Group From sales headcount and budgets to new product launches and saying “yes” to millennials, hear the top 7 topics that are trending in sales compensation. Sponsored by: Wesco Pharmaceutical Solutions and WBR Insights The regulations and managing processes for hazardous, controlled and rare chemicals and biologics necessary for drug discovery are a huge distraction for R&D labs. Learn from the perspectives of industry executives the pain points and improvements they want in their processes for chemicals and biologics in pharmaceuticals R&D. Sponsored by: Kinaxis Download this life sciences industry spotlight to discover how concurrent planning can help you overcome complex supply chain challenges to drive down costs and improve efficiency. Sponsored by: Patheon, by Thermo Fisher Scientific Patheon, part of Thermo Fisher Scientific, offers advanced API manufacturing technologies such as continuous processing and continuous analytics to deliver high-quality products with well-defined features to meet your timelines and volume requirements. Sponsored by: ICON Understanding the challenges for formulary decision makers and manufacturers. Sponsored by: Veeva This white paper details how leveraging new reporting capabilities, live UAT feedback sessions, and an agile design approach to study builds will save them time and money. Sponsored by: BBK Worldwide The Study Voices survey challenges the myth that physicians won’t refer patients to clinical trials, and it identifies opportunities to engage physicians as partners in the enrollment process. Sponsored by: Catalent Download the case study to learn about the clinical supply management tools that helped a small sized pharma company during phase III of a multi-arm oncology study. Sponsored by: Catalent Watch a short video on demand led supply model. It is designed to meet the needs of patients, clinical sites, clinical team and sponsors and results in shorter lead time, less waste, less stock out risk and no booklet labels. Sponsored by: Catalent Download the eBook to explore a proactive approach for clinical supply management. Latin America Healthcare Compliance Certificate Program September 23-26, 2019 BioBasics: Biotech For The Non-Scientist Oct 22-23, 2019 | San Diego, CA BioImmersion: Biotech for the Non-Scientist October 23-25, 2019 | Chicago, IL 4th FierceBiotech Drug Development Forum October 28-29, 2019 | Boston, MA Drug Development Boot Camp® 2019 November 20-21, 2019 | Boston, MA | Register NOW and start Pre-Boot Camp Preparation! | 