Regulatory Focus™ > News Articles > Regulatory Recon: Bluebird Planning for First Three Approvals by 2019; EU Approves Vertex' Orkambi f

Regulatory Recon: Bluebird Planning for First Three Approvals by 2019 EU Approves Vertex' Orkambi for Younger Patients (10 January 2018)

Posted 10 January 2018 | By Michael Mezher 

Regulatory Recon: Bluebird Planning for First Three Approvals by 2019 EU Approves Vertex' Orkambi for Younger Patients (10 January 2018)

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

In Focus: US

  • Bayer, J&J win reversal of $28 million verdict in Xarelto lawsuit (Reuters)
  • HHS Secretary Nominee: 'No Silver Bullet' to Bring Down Drug Prices (Focus) (NYTimes) (STAT) (Fierce) (Axios)
  • Bluebird to seek approvals for three treatments by end of 2019 (Reuters) (Endpoints)
  • Trial Transparency: NIH Says Final Rule From 2017 has Helped (Focus)
  • Faced with public pressure, research institutions step up reporting of clinical trial results (STAT)
  • J&J chief's pharma-tech vision is pretty, but it's certainly not reality: experts (Fierce)
  • Health Care Just Became the US's Largest Employer (The Atlantic)
  • Health care's 'upstream' conundrum (Politico)
  • A Seven-Step Plan for Ending the Opioid Crisis (Bloomberg)
  • IV bag shortage has hospitals scrambling to treat flu (AP)
  • Clarus' Jatenzo Suffers From Off-Label Concerns At US Advisory Committee (Pink Sheet-$)
  • Biotech Fumbles the Start of Its Biggest Showcase (Bloomberg)
  • How pharma can accelerate business impact from advanced analytics (McKinsey)
  • Meet the Woman Using CRISPR to Breed All-Male "Terminator Cattle" (MIT Technology Review)
  • A group of disruptive upstarts on a do-or-die mission to launch new meds (Endpoints)

In Focus: International

  • Foreign funds add billions to market cap of little known Celltrion (Financial Times) (Bloomberg)
  • Europe Has Completely Turned the Tables on Brexit (Bloomberg)
  • What a 'no deal' Brexit scenario would mean for key UK industries (The Guardian)
  • Commission reassures 'worried' member states on Health Technology Assessment (EurActiv)
  • NICE backs wider use of MSD's Simponi (PharmaTimes)
  • Vertex' CF therapy Orkambi cleared for younger patients (PharmaTimes) (Press)
  • Aspen CEO Defends Earnings Amid Post-Steinhoff Share Jitters (Bloomberg)
  • Glaxo Shifts Gears to Fire Up Growth With Rival Astra Closing In (Bloomberg)
  • Novo Nordisk's once-weekly diabetes injection cleared in Canada (Drug Delivery)
  • Valeant CEO looks to boost performance with seven key products (CNBC)
  • GSK's long-quiet cancer R&D group is ready to make some noise as it mounts a breakout late-stage effort (Endpoints)
  • Roche, Syapse Explore Ai-Based Tech For Cancer Precision Medicine (BioCentury)
  • Tanzania Consults On East African Community Guidelines, Including For Biosimilars (Pink Sheet-$)

Pharmaceuticals & Biotechnology

  • Will Life Sciences Companies Face More Scrutiny In 2018? (Law360-$)
  • Fallout From 'Nuclear Button' Tweets: Jump In Sales Of Radiation Drug (NPR)
  • In Defense of Less-Is-More in Medicine (MedScape)
  • Swallowing A 'Mini-Pillbox' Could Change The Way HIV Drugs Are Delivered (NPR)
  • Bristol Investors Just Need to Broaden Their Minds (Bloomberg)
  • Genentech adds a new program for Syndax's lead cancer drug to its Tecentriq combo platform (Endpoints) (Fierce)
  • New Study Looks at FDA's Use of Social Media to Communicate on Drug Safety (Focus)
  • FDA Denies Amneal Petition Seeking to Block Generic Versions of Osteoporosis Treatment (Focus)
  • Human antibodies made in cows could be developed to treat MERS (Reuters) (NIH)
  • Pfizer, pocketing a big tax cut from Trump, will end investment in Alzheimer's and Parkinson's research (LA Times)
  • How Allergan Continues to Make Drug Prices Insane (Bloomberg)
  • Truffle roots for new biotechs after first close of a planned $240M fund (Endpoints)
  • J.P. Morgan Notebook Day 2: Walmsley On Diversity, Amgen's Overseas Cash, Growing Opdivo, Mylan On US Generic Pricing, Boston's Pipeline And J&J In Alzheimer's (Pink Sheet-$)
  • Tufts: Growing Volumes of Clinical Data are Slowing Drug Development (FDANews-$)
  • Payers' Latest Attack On Copay Coupons: 'Accumulator' Programs (Pink Sheet-$)
  • New guidance: When can the FDA refuse to file NDAs and BLAs? (PharmaLetter)
  • New Data: Pharmacy Owners' Profits Fall As Industry Competition Rises (Drug Channels)
  • Novartis to test Intec's Accordion controlled-release delivery tech (InPharmaTechnologist)
  • BioClin Therapeutics hires Gilead blood cancer R&D exec (Fierce)
  • Not all elderly lung cancer patients get recommended care (Reuters)
  • Advisory Committee Activity for 2017 (Eye on FDA)
  • Few differences observed in safety information for biosimilars and related originators (Pharmaceutical Journal)
  • After $270M round, BioNTech pens TCR pact with Scancell (Fierce)
  • Evolus plans $75M IPO to bring Botox biosimilar to market (Fierce)
  • Datavant integrates 150 data sets, teams with Duke, G3, Verge Genomics (Fierce)
  • Pain Therapeutics Gains On Abuse-Deterrence Data (BioCentury)
  • Spark To Develop Gene Therapy For Pompe's (BioCentury)
  • Chinese Manufacturer Cited for Sanitation, Sterility Issues (FDANews-$)

Pharmaceutical and Biotechnology: Study Results, Filings and Designations

  • BerGenBio's AXL inhibitor clears early efficacy test in NSCLC (Fierce)
  • Breakthrough Designation For Global Blood's Sickle Cell Therapy (BioCentury)
  • GenSight Biologics Receives MHRA Approval to Initiate Phase I/II PIONEER Clinical Trial of GS030 Gene Therapy in Retinitis Pigmentosa (Press)

Medical Devices

  • Barriers to FDA and CMS Parallel Reviews: Fear and Bandwidth, Shuren Says (Focus)
  • Wanted: 'Breathalyzer' for Cannabis Use (Medpage)
  • How Stressful Are Jobs in Medtech? (MDDI)
  • How Edwards is Winning With Focus, Not Diversification (MDDI)
  • 3 Reasons TAVR Will Continue to Prosper (MDDI)
  • MITA Urges House And Senate Leadership To Support Full Repeal Of The Medical Device Tax (MITA)
  • CDRH Issues New Draft Least Burdensome Guidance (FDA Law Blog)
  • FDA Approves Innovative Clinician Programmer for the Medtronic SynchroMed II Intrathecal Drug Delivery System for Chronic Pain and Severe Spasticity (Press)
  • Baxter to Host Fourth Quarter 2017 Financial Results Conference Call for Investors (Press)
  • Nevro wins FDA nod for next-gen Senza II SCS (MassDevice)
  • Gynesonics touts pivotal IDE Sonata trial results (MassDevice)
  • BioProtect Launches an International Multi-Center Clinical Study Following FDA investigational Device Exemption (IDE) (Press)

US: Assorted & Government

  • Medicaid work requirements are coming (Politico)
  • Nevada's law on diabetes drug-pricing transparency may gain 'trade secret' option (STAT)
  • Federal Judge Seeks Speedy Resolution of Opioid Lawsuits (WSJ) (Law360-$)
  • Hatch Closing Act: OTC Monograph Reform Legislation? (Pink Sheet-$)
  • Allergan Wants Out Of Restasis Patent Challenges (Law360-$)
  • Amgen Moves to Dismiss Genentech's Misrepresentation Claim (National Law Review)
  • Apotex Gets PTAB To Trim OSI's Cancer Drug Patent (Law360-$)
  • Hagens Berman, Others Want $31M In Pfizer Celebrex Deal (Law360-$)
  • Third Circuit Requires But-for Causation for False Claims Act/Off Label Retaliatory Claim (Drug & Device Law)
  • OPIOIDS: Sen. Murray Highlights Need for More Resources to Help Communities Respond, Prevent Opioid Misuse (Senate HELP)
  • Senate Dems seek $25B in opioid funding (The Hill)
  • Senate health committee hosts opioid hearing with a single witness: a journalist (STAT)
  • Jump in HIV Cases Among Drug Users Seen in Northern Kentucky (WSJ)
  • Native American tribes sue opioid manufacturers, distributors (CBS)
  • Trump orders mental health aid for military veterans to prevent suicide (Reuters)

Upcoming Meetings & Events


  • Public consultation on EU funds in the area of investment, research & innovation, SMEs and single market (EC)
  • Every £1 invested in medical research delivers a return equivalent to 25p every year, forever (MRC)
  • MSD launches Clostridium difficile antitoxin in the UK (PharmaTimes)
  • A study on biosimilars begins (Fimea)
  • IPR-Enhancing EU-Mercosur Trade Talks Set To Resume After Stalling (Pink Sheet-$)
  • Guideline on clinical investigation of medicinal products for the treatment of rheumatoid arthritis (EMA)
  • How pharma operations in Europe have changed in the last 25 years (EPR)
  • Change Your RMS In Just Days – And For Free, Ireland's Brexit Guidance Says (Pink Sheet-$)
  • EU Filings For Amgen/UCB's Evenity, iv Relenza From GSK, And A New PRIME Product (Pink Sheet-$)
  • Infliximab monograph adopted by European Pharmacopoeia Commission (InPharmaTechnologist)


  • Japan reports first suspected bird flu case in poultry this winter (Reuters)


  • Eyeing over 80% sales from US, India, Europe by 2021: Glenmark (Economic Times)
  • Decisions That Made A Difference: Wockhardt Chief On A Life In Business (SCRIP-$)


  • Ontario has added etanercept biosimilar Erelzi to public drug plan (BiosimilarNews)
  • Advanced Bionics Announces Health Canada Approval for the New HiFocus™ SlimJ Electrode (Press)

Other International

  • WHO supports the immunization of 1 million people against cholera in Zambia (WHO)

General Health

  • RSV? She Hadn't Heard of It. Then Her Child Was Hospitalized. (NYTimes)
  • More than 10,000 donated organs in 2017, making a record (NBC)

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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