Regulatory Recon: Califf Speaks Out on Speeding Approvals, FDA Staffing US Panel Backs CRISPR Use in Human Embryos to Avoid Serious Disease (14 February 2017)

Posted 14 February 2017 | By Michael Mezher 

Regulatory Recon: Califf Speaks Out on Speeding Approvals, FDA Staffing US Panel Backs CRISPR Use in Human Embryos to Avoid Serious Disease (14 February 2017)

Welcome to Regulatory Reconnaissance, your daily regulatory news and intelligence briefing.

In Focus: US

  • Firm Delays Muscular Dystrophy Drug U.S. Launch Amid Criticism of $89,000 Price (WSJ) (Reuters) (Pharmafile)
  • Sanders, Cummings Slam Marathon's $89,000 Price Tag for Old Steroid (Focus)
  • The drug pricing debate gets more complex (Politico) (SNL) (BioWorld)
  • Former FDA Chief Cites 5 Things To Watch On Drug Approvals And Keeping Drugs Safe (KHN) (Pink Sheet-$)
  • 21st Century Cures Act Lowers Confidence In FDA-Approved Drugs And Devices (Health Affairs Blog)
  • The Soon-Shiong Hype Machine (In The Pipeline) (STAT)
  • Hurdles for Neulasta Biosimilars (BiologicsBlog)
  • 'Right to Try' is a sham (The Hill)
  • Scientists soften on DNA editing of human eggs, sperm, embryos: report (Reuters) (MIT Technology Review)
  • Evaluation of Evidence of Statistical Support and Corroboration of Subgroup Claims in Randomized Clinical Trials (JAMA)
  • Acknowledging and Overcoming Nonreproducibility in Basic and Preclinical Research (JAMA)
  • An endorsement of PCSK9 inhibitors funded by … their manufacturers (The BMJ)
  • CRISPR's breakthrough problem (C&EN)
  • The Rise and Fall of a K Street Renegade (WSJ) (Huffington Post)
  • Gilead challenges GSK with strong HIV drug data (Reuters) (Fierce) (The Street) (Endpoints) (Press)
  • GSK unveils strong showing for twin-drug HIV treatment (Pharmafile) (Financial Times)
  • Pharma cos await Trump decision on FDA Commissioner: Steve Usdin  (MoneyControl)
  • FDA Turmoil, Biology Mystery On List Of Hurdles For Microbiome Drugs (Xconomy)
  • Allergan to Buy Fat-Treatment Maker Zeltiq for $2.3 Billion (WSJ) (Reuters) (NYTimes) (Financial Times)
  • Lilly rejects reports US tax plan prompted Ireland investment rethink (BioPharma-Reporter)
  • GSK on America First: US will not be able to own every part of supply chain (InPharmaTechnologist)
  • EMA, Drugmakers Weigh FDA Draft Guidance on Physiologically Based PK Analyses (Focus)
  • Biotech IPOs Slow As Trump-Sown Uncertainty Rattles Nerves On Wall Street (Forbes)
  • How biomarkers cost Bristol-Myers the lung cancer market (BioPharmaDive)
  • This dentist broke his own opioid habit. Can the dental profession do the same? (STAT)

In Focus: International

  • Pakistan's biopharma sector struggles to establish regulations (BioWorld)
  • Teva's Paranoid Downfall (Haaretz)
  • 2017 Outlook: Medical Device Companies See Better Growth Prospects in US, Europe (Emergo)
  • Why Lupin fails to earn a premium (Economic Times)
  • MHLW Mulls Administrative Penalties, Tighter Regulations after Counterfeit Harvoni Case (PharmaJapanWeb)
  • Indian Pharmaceutical Alliance demands exclusion of India from USTR Priority Watch List (PharmaBiz) (Economic Times)
  • Sourcing Medical Devices in China: Phase 6 – Long-term Supplier Relations (Pacific Bridge Medical)
  • EMA Identifies Steps To Foster ATMP Development (Pink Sheet-$)
  • EMA's Pediatric Committee Seeks to Tweak ICH Q&A on Nonclinical Evaluation of Cancer Drugs (Focus)
  • EMA Touts Growth of Post-Authorisation Study Database (Focus)
  • EC approves Olumiant for RA (BioCentury) (Press)
  • Should Teva join the split-up club? With CEO gone and options open, analysts press for answers (Fierce)
  • Portugal Becomes EMA's Latest Suitor As Agency Cost Pressures Grow (Pink Sheet-$)
  • CDSCO to augment manpower with infusion of 31 technical experts armed with bio-med tech qualification (PharmaBiz)
  • Cinven's M&A Ambitions Over Stada Under Scrutiny, Advent and Others Circle (SCRIP-$) (Financial Times)
  • In West Africa, clinics confront suspicion, and husbands, one IUD at a time (STAT)
  • IFPMA launches its 2017 facts & figures report (IFPMA)
  • Twelfth meeting of the Emergency Committee under the International Health Regulations (2015) regarding the international spread of poliovirus (WHO)

US: Pharmaceuticals & Biotechnology

  • GPhA Rebrands itself AAM, Association for Accessible Medicines (AAM)
  • No Drugs for Back Pain, New Guidelines Say (WSJ)
  • May Deadline for FDA's eCTD Transition Approaches (Focus)
  • Value-Based Contracts: Relief From Regulatory Barriers In Sight? (Pink Sheet-$)
  • Sharp Rise Reported in Older Americans' Use of Multiple Psychotropic Drugs (NYTimes)
  • Liquid biopsies: The next frontier in cancer? (BioPharmaDive)
  • Step Therapy—Clinical Algorithms, Legislation, and Optimal Prescribing (JAMA)
  • Researchers testing remyelination method using Schwann cells (BioCentury)
  • Hurry Up! Program Fee Clean-up Reports Slow to Come into FDA (Lachman Consultants)
  • FDA's Draft Guidance on Listeria monocytogenes (In Case You Missed It) (FDA Law Blog)
  • FDA Places Indian Co Ultratech India LTD on Import Alert (FDA)
  • Watch: Biomarkers in drug development with Dr. Bernie Zeiher (PhRMA)
  • Fireside Chat with Dr. Kim of Inovio Pharmaceuticals: Immunotherapies and Vaccines (BIO)
  • How the language we use to communicate about disease matters (AHCJ)
  • Can Roche's Herceptin-Perjeta combo expand yet again? Check the impending data (Fierce)
  • Diabetes drugs pop on January's TV ad spending list, but AbbVie's Humira stays at the top (Fierce)
  • Incyte takes over antibody work from Agenus as pair rejig pact (Fierce)
  • Ionis earns $75 million for advancing IONIS-FXI Rx (PharmaLetter-$)
  • Cleveland Clinic's new $276 million cancer hub boasts a 'pod' approach to care (Modern Healthcare)
  • Plastic electronics to bring "mind-blowing" developments for pharma (Pharmafile)

US: Pharmaceuticals and Biotechnology: Clinical Study Results, Filings & Designations

  • Axsome Therapeutics Receives FDA Fast Track Designation for AXS-05 for Treatment Resistant Depression (Press
  • CSL says FDA accepts CSL Behring's biologics license application supplement for using Privigen to treat CIDP (Reuters)
  • Sage climbs on Phase II MDD readout (BioCentury) (The Street)
  • Acacia to file NDA for PONV drug within months (PharmaLetter-$)
  • Axovant's nelotanserin heading to Phase III (BioCentury) (Fierce)
  • Early study prompts hope for new approach to type 2 diabetes (PharmaLetter-$)
  • Transgene and Léon Bérard Cancer Center Announce Dosing of the First Patient in a Phase 1 Immunotherapy Clinical Trial Evaluating the Intra-Tumoral Co-Administration of Pexa-Vec plus Ipilimumab (Yervoy®) in Solid Cancers (Press)
  • Vaxart Announces Positive Phase 1 Study of Oral Norovirus Vaccine (Press)

US: Medical Devices

  • AdvaMed Lobbies For Repeal Of Medical Device Tax In 1st Reconciliation Bill (Inside Health Policy)
  • Siemens mulls US IPO for healthcare biz (MassDevice)
  • FDA Devices Center Issues Multiple Guidances in the Obama Administration's Final Months (National Law Review)
  • Here Is What Donald Trump's Policies May Mean for Medical Equipment Companies (The Street)
  • Centric Medical™ Announces FDA Clearance of Metatarsal Hemi Implant System (Press)
  • Medtronic launches DxTerity, TRA-line of radial access devices for PCIs (MassDevice)
  • AcelRx touts Dsuvia data for burn victims (MassDevice)
  • Intact Vascular launches BTK balloon angioplasty trial of its Tack endovascular system (MassDevice)

US: Assorted & Government

  • Will Obamacare Really go Under the Knife? (NYTimes)
  • Angry Town Hall Meetings on Health Care Law, and Few Answers (NYTimes)
  • Fourth Circuit Vigorously Applies Daubert (Drug & Device Law)
  • CMS Nominee Seema Verma Visits Senate Finance (National Law Review)
  • How Can We Increase The Use Of Palliative Care In Medicare? (Health Affairs Blog)
  • U.S. Representatives vote against D.C. assisted suicide law (Reuters)
  • Sandoz to Supreme Court: Circuit Erred in Ruling 180-Day Notification Required After Biosimilar Approval (FDANews-$)
  • What To Know About HHS Revised 'Common Rule' (Law360-$)
  • Illinois lawmakers propose biotechnology tax credits (Law360-$)
  • Public Hearing on Partnerships to Enhance the Safety of Imported Foods (FDA)

Upcoming Meetings & Events


  • Researchers find metal impurities in vaccines, but European regulator challenges study (Pharmaceutical Journal)
  • Revised document published on Management of applications for new CEPs and revisions/renewals of CEPs (EDQM)
  • New initiative may significantly increase drug prices for state tenders in Russia (PharmaLetter-$)
  • HeartFlow wins nod from U.K. healthcare board (MassDevice)
  • Dutch Biopharma launches Phase III trial of Leukemia donor cell therapy (BioPharma-Reporter)
  • Micro Medical Solutions wins CE Mark for MicroStent BTK vascular stent (MassDevice)


  • China, India account for half world's pollution deaths in 2015: study (Reuters)
  • Inovio grants ApolloBio China rights to HPV candidate (BioCentury)
  • Aurinia Announces Results from Japanese Phase I Ethnic Bridging Study for Voclosporin (Press)


  • Commerce ministry to device action plan to reduce dependence on China for API imports (PharmaBiz)
  • Formation of separate ministry for pharma and medical device under active consideration of PM: Ananth Kumar (PharmaBiz)
  • Sun Pharma Q3 profit falls 5% to Rs1,471.82 crore on higher tax outgo (LiveMint)
  • Govt slashes cardiac stent prices by over 75 per cent (Economic Times)


  • Consultation: Reforms to the regulatory framework for complementary medicines: Assessment pathways (TGA)
  • Conference participation by the TGA (TGA)


  • Summary Safety Review - Over-the-Counter Topical Pain Relievers Containing Menthol, Methyl Salicylate or Capsaicin - Assessing the Risk of Serious Skin Burns (Health Canada)

Other International

  • Hikma to market new Easyhaler product in MENA (PharmaLetter-$)
  • Teva to Work With Heidrick & Struggles on Global Search for CEO (Bloomberg)

General Health & Other Interesting Articles

  • Heavy snowfall tied to higher heart attack risk for men (Reuters)
  • From Vector To Zoonotic: A Glossary For Infectious Diseases (NPR)
  • Study Finds New Bacterial Strain Can Contaminate Shellfish (NYTimes)

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.

Need to contact the editor of Regulatory Reconnaissance? Find him on Twitter at @Michael_Mezher or send him an email at

A story's inclusion in Regulatory Reconnaissance does not imply endorsement by Regulatory Focus or RAPS.

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