Regulatory Focus™ > News Articles > 2019 > 2 > Recon: Sangamo Tanks on Weak Genome Editing Data; FDA Approves Sanofi Rare Blood-Clotting Disorder D

Recon: Sangamo Tanks on Weak Genome Editing Data; FDA Approves Sanofi Rare Blood-Clotting Disorder Drug

Posted 07 February 2019 | By Michael Mezher 

Recon: Sangamo Tanks on Weak Genome Editing Data; FDA Approves Sanofi Rare Blood-Clotting Disorder Drug

Welcome to Regulatory Reconnaissance, your daily regulatory news and intelligence briefing.
In Focus: US
  • Sanofi blood disorder drug wins FDA approval (Reuters) (FDA) (Press)
  • Sangamo Therapeutics shares dive after poor early-stage trial data (Reuters) (Bloomberg) (STAT) (Endpoints) (Press)
  • White House shakes up drug policy office (Politico)
  • Trump Administration Salutes Parade Of Generic Drug Approvals, But Hundreds Aren’t For Sale (KHN)
  • Congress invites 7 drugmakers to testify at potentially hostile hearing on drug prices. Here's what they're saying (CNBC)
  • Eli Lilly, Novartis back US proposal on drug rebates to lower costs (Reuters 1, 2)
  • Democrats Unite to Begin Push to Protect Pre-Existing Condition Coverage (NYTimes)
  • Medicare Part D no match for runaway specialty drug costs: study (Reuters)
  • Alphabet is taking on health care with a bunch of projects (CNBC)
  • Solid Biosciences to soldier on as initial muscular dystrophy gene therapy results disappoint (STAT) (Endpoints)
  • As magnitude and frequency of drug price hikes thaw, Express Scripts reports lowest increase in spending by commercial plans in 25 years (Endpoints) (BioCentury)
  • Lawmakers revive Creates Act opposed by Big Pharma (Modern Healthcare-$)
  • US Prosecutors Sue To Stop Nation's First Supervised Injection Site For Opioids (NPR)
  • Stanford will investigate its role in the Chinese CRISPR baby debacle (MIT Technology Review)
In Focus: International
  • Europe’s vast new anti-bogus-drugs system will not find many (The Economist) (Pink Sheet-$)
  • France's Sanofi targets growth from new drugs after stronger quarter (Reuters) (Financial Times) (Press)
  • Sanofi punts 38 R&D projects to narrow pipeline focus (Fierce)
  • ICH Seeks to Harmonize Generic Drug Standards (Focus)
  • Vertex fears its triplet will also face UK reimbursement battle (BioPharmaDive)
  • Pfizer ignores strike in Ireland as 900 employees' pensions affected (Pharmafile) (Fierce)
  • Mundipharma launches Pelmeg biosimilar in Europe (PharmaTimes)
  • Pharmacy stocks run low as Turkey's drug price policy hits supplies (Reuters)
  • PhRMA Wants US-UK Trade Deal To Cover Regulatory Alignment (Pink Sheet-$)
  • China says tests of possibly tainted medical product show no HIV (Reuters) (NPR)
  • Measles Outbreak in Philippines Spreads Beyond Capital (NYTimes)
  • Tens of thousands infected in measles outbreak in Madagascar (CNN)
Pharmaceuticals & Biotechnology
  • Pouring Billions Of Dollars Into Marketing Of Drugs (Forbes)
  • Diabetes Moves Down the List of Priorities for Big Pharma (Bloomberg)
  • Mega docking library poised to speed drug discovery (NIH)
  • New York biotech debuts, targeting dormant metastatic cancer cells (STAT) (Endpoints)
  • Biotech Insider Stock Sales: Peer Benchmarks And Guidelines (LifeSciVC)
  • The Prescription Drugs That Rich People Buy (NYTimes)
  • Precision medicine: Opening the aperture (McKinsey)
  • Rare Pediatric PRV Program Not Linked to Uptick in New Drugs Starting Trials, Study Finds (Focus)
  • FDA Drafts Guidance on Developing Drugs for Rare Chronic Digestive Disorder (Focus) (FDANews-$)
  • FDA Finalizes Buprenorphine Drug Development Guidance (Focus)
  • Study of PrEP and vaginal ring for HIV prevention begins in girls and young women (NIH)
  • Azar: International Regulatory Harmonization Will Create More Stable Generic Markets (Pink Sheet-$)
  • Pennsylvania hospital patient tests negative for Ebola (Reuters)
  • Galapagos continues fibrosis pipeline buildout with Evotec deal (Fierce)
  • Precision medicine’s rosy predictions haven’t come true. We need fewer promises and more debate (STAT)
  • Antidepressants Can Interfere With Pain Relief Of Common Opioids (NPR)
  • Doctors debate whether to stop calling low-risk tumors 'cancer' (Reuters)
  • Dermatology patient advocacy groups may not disclose conflicts (Reuters)
  • Tightening its wallet for a clinical comeback, Seres lets go of CSO and 30% of workforce (Endpoints)
  • The biotech unicorns are back: Alector burnishes its $1.3B valuation in an IPO that generates $176M in cash (Endpoints) (Fierce)
  • CRISPR restores expression in models of vision loss (Nature)
Pharmaceuticals & Biotechnology: Study Results, Filings & Designations
  • Alnylam shores up enthusiasm for givosiran with early dataset ahead of crucial PhIII readout (Endpoints) (Press)
  • Zogenix submits new drug application to FDA for Fintepla (PharmaTimes)
  • Fasenra granted Orphan Drug Designation by FDA (PharmaTimes)
  • Incyte Announces US FDA Has Extended the Review Period for Ruxolitinib (Jakafi®) in Acute Graft-Versus-Host Disease (Press)
  • Altan Pharma Submits New Drug Application for Acetaminophen Solution for Infusion for Treatment of Pain and Reduction of Fever (Press)
  • Arch Oncology Announces First Patient Dosed in Phase 1 Clinical Trial of AO-176, an Anti-CD47 Antibody with a Best-in-Class Profile (Press)
Medical Devices
  • FDA reports new cases of cancer linked to textured breast implants (NBC) (FDA)
  • FDA's new draft guidance could hinder applications for digital combination products (mobihealthnews)
  • Global Kinetics touts movement-monitoring study results (MassDevice)
  • Smith & Nephew shares up on Q4, FY2018 results (MassDevice)
  • FDA clears Current Health’s patient monitoring system (MassDevice)
  • Orthofix Announces FDA Approval of the M6-C Artificial Cervical Disc to Treat Patients with Cervical Disc Degeneration (Press)
  • Medtronic Receives FDA Approval on Expanded Indication for Pipeline Flex Embolization Device (Press)
US: Assorted & Government
  • Vanda Pharmaceuticals Sues FDA Over Partial Clinical Hold (Focus)
  • Federal court enters consent decree against Ranier’s Rx Laboratory and owner for manufacturing purportedly sterile drug products in insanitary conditions (FDA)
  • Biogen faces multibillion-dollar Tecfidera loss if Mylan wins latest patent threat (Fierce)
  • The First Circuit Creates Havoc By Ignoring Facts And An Elephant In The Room (Drug & Device Law)
  • USPTO Releases Performance and Accountability Report for FY 2018 (Patent Docs)
  • Drugmaker Files Ch. 11, Blames Low-Selling Urination Drug (Law360-$)
Upcoming Meetings & Events Europe
  • EU Regulatory Roundup: Ireland Asks Companies to Proactively Discuss Possible Effects of Brexit (Focus)
Asia Australia
  • Australia Considers Changes To Bioequivalence Requirements For Foreign Comparators (Pink Sheet-$)
  • Consultation: Reforms to the generic medicine market authorisation process (TGA)
  • Publication of interim decisions amending, or not amending, the current Poisons Standard, February 2019 (TGA)
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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