Regulatory Recon: Vertex Stops Cystic Fibrosis Combination Trial After No Benefit Found Aurinia Lupus Drug Meets Endpoint Despite 13 Patient Deaths (16 August 2016)

Posted 16 August 2016 | By Michael Mezher 

Regulatory Recon: Vertex Stops Cystic Fibrosis Combination Trial After No Benefit Found Aurinia Lupus Drug Meets Endpoint Despite 13 Patient Deaths (16 August 2016)

Welcome to Regulatory Reconnaissance, your adaily regulatory news and intelligence briefing. Want to read Recon as soon as it's posted? Follow @Michael_Mezher, @Zachary Brennan and @RAPSorg on Twitter.

In Focus: US

  • Their Hair Fell Out. Should the F.D.A. Have the Power to Act? (NYTimes)
  • PDUFA VI: Highlights on What to Expect (Focus) (Pink Sheet-$)
  • Study Says FluMist Vaccine Does Indeed Work, Contradicting CDC (NPR)
  • Zika and the race to quell outbreaks: My talk with Anthony Fauci, NIH's top vaccine expert (Washington Post)
  • US Life Sciences CEOs Upbeat About Industry's Prospects (PharmTech) (Fierce) (Survey)
  • Aetna Will Abandon Obamacare Patients In More Than 500 U.S. Counties (Forbes) (WSJ) (Reuters)
  • Obamacare hikes: Average rate request of 24 percent(Politico)
  • Laws to shed light on drug pricing strategies probably won't work (STAT)
  • $81 Million in Zika Funding: Winners And Losers (Forbes)
  • Pain drugs in pregnancy tied to behavior issues in kids (Reuters)
  • Big price tags when orphan drugs treat common diseases(Politico)
  • 3D Medical Device Printing: Industry Seeks More Clarity From FDA (Focus)
  • Chance Collaboration Yields an Advance in Cancer Treatment (WSJ)
  • OncoGenex seeks strategic alternatives after drug study fails (Reuters)
  • Why CRISPR Is An Attractive Approach To Treat Sickle Cell Disease (Forbes) (PharmTech)
  • FDA Takes Unprecedented Steps In Bid To Push OTC Switch (Inside Health Policy)
  • A Wolf in Sheep's Clothing: When the Failure to Obtain a 510(k) for a Modification Is More Than a Regulatory Violation (FDA Law Blog) (Focus)
  • 13 deaths in Aurinia's lupus drug trial despite hitting Phase II target (Pharmafile) (EP Vantage) (BioCentury) (BioWorld)
  • Vertex ends trial of cystic fibrosis drug combination (Boston Globe) (Pharmafile) (Press) (Fierce)

In Focus: International

  • NHS paediatric workforce 'at breaking point' (PharmaTimes)
  • China Faces a Surge in Cardiovascular Disease, Study Finds (NYTimes)
  • EMA Adopts New Biologic, Biosimilar Pharmacovigilance Guidance (Focus)
  • BMS to supply Opdivo for Bavarian Nordic trial (PharmaTimes)
  • Owen Smith: Corbyn is exploiting my former work in pharmaceuticals (The Guardian)
  • EMA nails Artemis India plant over simvastatin API manufacturing issues (Fierce) (Focus)
  • Sanofi sends half a million dengue vaccines to high risk Brazilians (EPR)
  • Adapting for the real world (BioCentury)
  • True magnitude of stillbirths and maternal and neonatal deaths underreported (WHO)

US: Pharmaceuticals & Biotechnology

  • Playing the Long Game, Partnerships Required For Emerging Infectious Diseases (SCRIP-$)
  • Leaked pharma slide illustrates why investors care about clinical trial transparency (AllTrials)
  • Prescription drug abuse tied to increased risk of teen suicide (Reuters)
  • FDA Offers First Major Update to 'Orange Book' Website (Focus)
  • Emergency Response Protein against Influenza Identified for Innate Immune System (GEN)
  • The FDA Wants To Make It Harder To Buy And Sell Poop (BuzzFeed)
  • Yes, Medicine Can Use Virtual Reality, Emphasis on Reality (NYTimes)
  • Capsule Supplier Changes: FDA Offers an Updated Policy (Focus)
  • Purdue Pharma joins health policy research organization (Oursourcing-Pharma)
  • Researchers examine how Parkinson's disease alters brain activity over time (NIH)
  • Therapy focus – Hepatitis B developers try to repeat Gilead's hep C trick (EP Vantage)
  • Can 50 Million Blood Pressure Prescriptions Each Year Be Wrong? (CardioBrief)
  • Serialisation standard group makes progress towards uniform track & trace standard (Manufacturing Chemist)
  • Precision medicine's chicken and egg problem (MedCityNews)
  • Pathway To Novel Antibiotics May Be New Family Of Bacterial Cell Wall Builders (Forbes)
  • AdComm Recommendations – How Often FDA Does Not Follow Them? (Eye on FDA)
  • As Pfizer Buys, Closes Bind Therapeutics, CEO Hirsch Heads to Agios (Xconomy)
  • 2016 AAPS Election Results Announced (AAPS)
  • New Label for Chantix (FDA)
  • Novocure Converts More Than 500 U.S. Patients to Second Generation Optune within Four Weeks of FDA Approval on July 13 (Press)
  • Piramal Enterprises Enters into an Agreement to Acquire Ash Stevens Inc., a US based CDMO for High Potency APIs (HPAPIs) (Press)
  • No antigen left behind (BioCentury)
  • Sarepta's Duchenne drug trial hits 5-year mark: Parents look back (Boston Biz Journal)
  • Pfizer heads back to school with Trumenba vaccination campaign (Fierce)
  • Sorrento lures former Celgene exec as CMO (PharmaLetter-$)
  • With Zurampic, FDA Again Made Do With Sorry State Of Regulatory Science In Gout (Pink Sheet 1, 2-$)

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

  • New real world evidence validates pivotal clinical trial outcomes for Brilinta in MI patients (PharmaLetter-$)
  • Troubled tivozanib wedded to Opdivo in new kidney cancer trial (Fierce)
  • Tivorsan Pharmaceuticals Granted FDA Orphan Drug Designation for Human Recombinant Biglycan in the Treatment of Duchenne Muscular Dystrophy (Press)
  • Results from Phase 1 Trials with Ligand's LGD-6972 in Type 2 Diabetes Mellitus Published in Diabetes, Obesity and Metabolism (Press)
  • Positive top-line results from first Phase III trial of pSivida's Medidur (PharmaLetter-$)
  • vTv Therapeutics Completes Enrollment of Phase 2 Trial Evaluating TTP273 for the Treatment of Type 2 Diabetes (Press)
  • Millendo Therapeutics Announces Initiation of Phase 2b Clinical Trial of MLE4901 in Patients with Polycystic Ovary Syndrome (Press)
  • Aratana Therapeutics Granted FDA Approval of NOCITA® (bupivacaine liposome injectable suspension) (Press)

US: Medical Devices

  • FDA Seeks Input on OTC Infectious Disease Testing (Medpage)
  • FDA approves BioSense Webster's Thermocool SmartTouch catheter (MassDevice)
  • Cook Medical Issues Global Recall of Roadrunner® UniGlide® Hydrophilic Wire Guides due to raw materials issue (FDA)
  • Zimmer Biomet acquires CD Diagnostics (MassDevice)
  • Interview – CVRx holds its nerve (EP Vantage)
  • Physicians and medical device reps: Too close for comfort? (MassDevice)
  • MITA Signs On To Coverage Request For CTC Colonography (MITA)
  • FDA approves Intuity Elite rapid valve from Edwards Lifesciences (MassDevice) (Press)
  • Sanuwave files for de novo FDA nod for DermaPace wound care device (MassDevice)
  • Centric Medical™ Announces FDA Clearance of TARSA-LINK™ Bunion System (Press)
  • Bovie Medical Receives FDA 510(k) Clearance for Its 'PlazXact™' Ablator (Press)
  • Massachusetts woman left behind at Fresenius dialysis clinic after it closed (Modern Healthcare)

US: Assorted & Government

  • Express Scripts Investor Demands Look At Books, Records (Law360-$)
  • FDA's Abilify Logic Invites 'Gamesmanship,' Otsuka Says (Law360-$)
  • 5 ways drug makers and the health care industry are shaping campaigns (STAT)
  • Fed. Circ. Finds Pill-Dispensing Patent Valid Again (Law360-$)
  • Agency Information Collection Activities; Submission for Office of Management and Budget Review Comment Request; Voluntary Qualified Importer Program Guidance for Industry (FDA)
  • FDA Issues Final Rule on Generally Recognized as Safe Substances (FDA)
  • Miami Man Pleads Guilty to Fraud Charges for Role in $4.2 Million Home Health Care Scheme (DoJ)

Upcoming Meetings & Events


  • NHS cancer patients still denied treatments available in other countries (OnMedica)
  • List of products to be stocked as mandatory reserve supplies (Fimea)
  • Marken opens new GDP-compliant facility (Outsourcing-Pharma)
  • EMA Begins Consultation on Revised Crohn's Disease Guidance (EMA)
  • Blaming UK cancer drugs woe on failed pharma negotiations is 'too simplistic', says ABPI (PharmaLetter-$) (APBI)



  • The MIT Startup Giving Indian Women Access To Biodegradable Sanitary Pads (Forbes)
  • Natco Pharma gets EIR from USFDA for Kothur facility (Economic Times)
  • Cadila gets EIR from USFDA for Changodar facility (Economic Times)


  • TGC meeting statement, Meeting 42, 13 May 2016 (TGA)


  • Summary Safety Review - INTUNIV XR (guanfacine hydrochloride) - Assessing the Potential Risk of Raynaud's Phenomenon (Health Canada)
  • ADEMPAS (riociguat) - New Contraindication for Patients with Pulmonary Hypertension Associated with Idiopathic Interstitial Pneumonias (PH-IIP) (Health Canada)
  • Summary Basis of Decision (SBD) – Darzalex (Health Canada)
  • Summary Basis of Decision (SBD) for Praxbind (Health Canada)
  • Post-Authorization Activity Table (PAAT) for Ibrance (Health Canada)


  • Zika Spreads From Florida to Texas (NBC)

Other International

  • Outbreak of yellow fever in DR Congo could go global, children's charity warns (Reuters)
  • Mass vaccination campaign to protect millions against yellow fever in Angola and Democratic Republic of the Congo (WHO)

General Health & Other Interesting Articles

  • Lack of stores with fresh foods linked to signs of early heart disease (Reuters)
  • Mmmm, Hydrogel Donuts. Is There Anything They Can't Do? (GEN)

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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