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Recon: FDA Approves First Blood Thinner for Pediatric Use

Posted 16 May 2019 | By Michael Mezher 

Recon: FDA Approves First Blood Thinner for Pediatric Use

Welcome to Regulatory Reconnaissance, your daily regulatory news and intelligence briefing.
In Focus: US
  • FDA approves first anticoagulant (blood thinner) for pediatric patients to treat potentially life-threatening blood clots (FDA)
  • Roche: FDA approves drug combo Venclexta plus Gazyva for previously untreated chronic lymphocytic leukemia (Reuters) (Press)
  • Gilead CEO to testify before a House committee about pricing for its HIV prevention pill (STAT)
  • Novartis may be close to settling a kickback case on the eve of a trial (STAT)
  • Analysts spotlight the winners — and losers — on ASCO preview night (Endpoints)
  • Biotech in the spotlight as pharma takes back seat at ASCO (BioPharmaDive)
  • Genentech lines up a new autoimmune R&D alliance on a ‘potentially transformative’ tech (Endpoints)
  • A Rival to Botox Invites Doctors to Party in Cancun, With Fireworks, Confetti and Social Media Posts (NYTimes)
  • FDA schedules Aimmune's peanut allergy drug for September expert review (BioPharmaDive) (Press)
  • The Met Will Turn Down Sackler Money Amid Fury Over the Opioid Crisis (NYTimes)
  • Swarms of Bugs, Missing Data Plague Firms Now Making Heart Drug (Bloomberg)
  • US drug pricing is in need of an overhaul (Financial Times)
  • House Democrats ready to jam GOP on drug pricing vote (Politico)
In Focus: International
  • UK cancer programme to target treatment resistance (Financial Times) (BBC) (Endpoints) (Reuters)
  • Pharma is making progress addressing global health, but it’s still a mixed bag (STAT) (Financial Times)
  • Opioids crisis has spread beyond United States: OECD (Reuters)
  • Nestlé Enters Exclusive Talks to Sell Skin-Health Business (WSJ) (Fierce)
  • Will drugs companies follow EU agency from London to Amsterdam? (Financial Times)
  • EMA Undertook 14 RWE Studies In 2018 To Complement Company Data (Pink Sheet-$)
  • Brexit Relief For Pharma As EMA Grants Most Batch Testing Exemptions (Pink Sheet-$)
  • EC Approves Celgene’s Revlimid and Imnovid Regimens for Multiple Myeloma (Press)
  • European Commission Confirms Quality of South Korean Active Substances (Focus)
  • J&J to invest €100 million into Limerick manufacturing facilities creating 100 new jobs (Pharmafile)
  • Thermo Fisher snaps up GSK site in Ireland as part of its expansion campaign (Fierce)
  • New HIV Map Offers Most Detailed Look Yet At The Epidemic (NPR)
  • 'Hidden health crisis' of snakebites gets $100 million funding injection (Reuters)
Pharmaceuticals & Biotechnology
  • People should be able to get health care without going bankrupt, GoodRx co-CEO says (CNBC)
  • Shaped like a tennis ball, this cancer protein was thought ‘undruggable.’ Amgen found a way to target it (STAT) (Fierce)
  • FDA to Follow DOJ Memo on Limiting its Regulation of Execution Drugs (Focus)
  • FDA Unveils 34 New and Revised Product-Specific Draft Guidances (Focus)
  • Risk-Based Monitoring: Pfizer and BMS Seek Clarity on FDA Draft Guidance (Focus)
  • Gottlieb: Lack Of Compromise On Right-To-Try Bill Is 'Interesting Lesson In Politics' For Stakeholders (Pink Sheet-$)
  • Gilead seeks to keep the CAR-T ball rolling at ASCO (PharmaLetter-$)
  • Defining REMS Success An Unanswered Question For Daiichi's Pexidartinib (Pink Sheet-$)
  • Anything You Can Do I Can Do (Better?): Demonstrating Biosimilar Interchangeability (FDA Law Blog)
  • ASCO data won't help AZ's Calquence rival Imbruvica in CLL: analyst (Fierce)
  • Drug Prices In TV Ads: How Transparent Are They? (Forbes)
  • Why a pharma investor quit his day job to follow a vision (Guest interview) (EFPIA)
  • Best Pathway To Interchangeable Insulins Is In The Eye Of The Beholder (Pink Sheet-$)
  • Insulin Biosimilars: US FDA Hears Wide Range Of Views From Small Set Of Sponsors (Pink Sheet-$)
  • Installment payments could be used across gene therapy space, Spark CEO says (MedCity)
  • Xellia to produce antibiotics for hospital-backed Civica Rx (Fierce)
  • As Daiichi Sankyo-partnered breast cancer drug marches toward finish line, AstraZeneca brings in Canadian scientist Sunil Verma to rev up R&D (Endpoints)
  • Essa strikes deal to buy Realm, gaining cash to fund cancer R&D (Fierce)
  • Can a little known drug in AstraZeneca’s pipeline make a comeback as a PARP add-on? Yale scientists explain how (Endpoints)
  • After a long march with Cabo, Exelixis is expanding its focus with an antibody-drug conjugate deal (Endpoints)
  • Benzodiazepines in early pregnancy tied to heightened risk of miscarriage (Reuters)
Pharmaceuticals & Biotechnology: Study Results, Filings & Designations
  • Roche’s personalised medicine entrectinib shrank tumours harbouring NTRK, ROS1 or ALK gene fusions in children and adolescents (Press)
  • AZ confirms cardiovascular safety with roxadustat results (PharmaTimes)
  • Pfizer Oncology to Showcase New Data from Innovative Science That Address Patient Needs at ASCO 2019 Annual Meeting (Press)
  • Sanofi oncology pipeline targeting various cancers highlighted at ASCO 2019 (Press)
  • Bayer presents new analyses for Vitrakvi® (larotrectinib) in adult and pediatric patients with TRK fusion cancer with solid tumors, and new data in primary central nervous system tumors and brain metastases (Press)
  • Updated Libtayo® (cemiplimab-rwlc) Results Reinforce Durable and Substantial Response Rates in Advanced Cutaneous Squamous Cell Carcinoma (Press)
  • Phase III Study Results for HP-3070 (Asenapine) Transdermal Patch for the Treatment of Schizophrenia to be Presented at the Society of Biological Psychiatry 74th Annual Meeting (Press)
  • Tempest Therapeutics Starts Phase I/Ib Clinical Trial with First-in-Class PPAR Alpha Antagonist (Press)
  • Verastem Oncology to Present New COPIKTRA™ (Duvelisib) Dose Modification Data from Patients Treated in the Phase 3 DUO Study (Press)
  • ALX Oncology to Present ALX148 Clinical Data at the 2019 American Society of Clinical Oncology (ASCO) Annual Meeting (Press)
  • New Data From the Phase I Study of Birinapant in Combination With Keytruda® Will be Presented at the ASCO 2019 (Press)
  • Alligator Bioscience: ADC-1013 Clinical Phase I Data to be Presented at ASCO (Press)
  • IMV to Present New Data at 2019 ASCO Annual Meeting From Its Phase 2 Clinical Trial Evaluating DPX-Survivac in Ovarian Cancer (Press)
  • ImaginAb Enrolls First Patient in Phase II Clinical Trial at a World-Leading Cancer Center in Los Angeles County (Press)
  • Dicerna Announces Dosing of First Patient in Phase 1 Clinical Trial of DCR-HBVS for the Treatment of Chronic Hepatitis B Virus (Press)
Medical Devices
  • FDA Updates on Essure Postmarketing Study (Focus)
  • Experimental brain-controlled hearing aid can pick out voices in a crowd (STAT)
  • DiaSorin Molecular Gets CE Mark for Herpes Virus Assay (GenomeWeb)
  • Gene Sequencing Co. ArcherDX Scores $60M Funding Round (Law360-$)
  • Medtronic Launches Telescope(TM) Guide Extension Catheter to Support Complex Coronary Cases (Press)
  • Masimo Announces FDA Clearance of Radius PPG™, the First Tetherless SET® Pulse Oximetry Sensor Solution (Press)
US: Assorted & Government
  • In Washington, a partisan approach to lowering drug costs leaves Democrats doubting their own party leadership (STAT)
  • BD’s CareFusion Agrees to $3.3M DOJ Settlement Over Unapproved Devices (Focus)
  • Five more US states sue Purdue Pharma over its role in opioid crisis (The Guardian)
  • Federal judge orders FDA to start regulating e-cigarettes (The Hill)
  • Letter to the House Committee on Oversight and Reform (Yale School of Medicine)
  • Submarine Exclusivity: Unseen Risk For 505(B)(2) Applications (Lassman Law+Policy)
  • Senate working group releases surprise medical bills legislation (Politico) (Cassidy)
  • Trump’s Talk On Preexisting Conditions Doesn’t Match His Administration’s Actions (KHN)
  • CVS Health to Require Third-Party Testing of Vitamins, Supplements (WSJ)
  • Public Health Law Watch Files Amicus Brief in Opiate MDL (Harvard Bill of Health)
  • Another Blow Against “Loose And Spurious” Personal Jurisdiction In Missouri (Drug & Device Law)
Upcoming Meetings & Events Asia
  • Genus shares surge on deal to market gene-edited pigs in China (Reuters)
  • Indian pharma at the crossroads as US cracks the whip (livemint)
  • Maha FDA proposes to DCGI for inclusion of color coding to track medicines for govt healthcare institutions (PharmaBiz)
  • Final Report: Fees for Drugs and Medical Devices (Health Canada)
  • Performance Standards for the Fees in Respect of Drugs and Medical Devices Order (Health Canada)
  • Webinar recording: SME Assist - Permitted indications for listed medicines, March 2019 (TGA)
General Health & Other Interesting Articles
  • Brain training shows promise as a treatment for veterans’ cognitive problems after TBI (STAT)
  • New Organ Transplant Rules Mean Livers Ship To Sickest Patients Not Nearest (NPR)
Regulatory Reconnaissance is our daily intelligence briefing for the regulatory affairs space, bringing you the top regulatory news stories from around the globe. Each weekday morning, we aim to bring you the latest highlights of new approvals, meetings, legal and political developments, regulations and guidance, and the latest trends with the potential to impact regulatory affairs professionals and the industry in which they work.
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