Regulatory Focus™ > News Articles > Regulatory Recon: Spark Prices Gene Therapy at $850,000; WHO Prequalifies Typhoid Vaccine (3 January

Regulatory Recon: Spark Prices Gene Therapy at $850,000 WHO Prequalifies Typhoid Vaccine (3 January 2018)

Posted 03 January 2018 | By Michael Mezher 

Regulatory Recon: Spark Prices Gene Therapy at $850,000 WHO Prequalifies Typhoid Vaccine (3 January 2018)

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

In Focus: US

  • Spark to charge $850,000 per patient for blindness gene therapy (Reuters) (Endpoints) (STAT) (CNBC) (Washington Post) (Forbes) (WSJ)
  • Drugmakers raise 2018 US prices, stick to self-imposed limits (Reuters) (STAT) (Fierce)
  • 2017 Was the Year of Gene-Therapy Breakthroughs (MIT Technology Review)
  • Scott Gottlieb issues a lot of statements. Quite a lot. So we counted his words (STAT)
  • Gottlieb: We're working to protect consumers (USA Today)
  • In Orrin Hatch's retirement, pharma loses chief congressional ally (STAT) (BIO) (Senate Finance)
  • Confirmation hearing for Trump's health secretary pick scheduled for next week (The Hill) (Senate Finance)
  • Trump's Tax Law Could Lead To Loss Of Biopharma Jobs (Forbes)
  • Novartis breast cancer therapy gets FDA breakthrough designation (Reuters)
  • FDA issues recall on J&J heart device (Reuters)
  • Drug companies may have shorted government more than $1 billion in Medicaid rebates (CNBC)
  • The NIH is in danger of losing its edge in creating biomedical innovations (STAT)
  • Pfizer doubles down on Sangamo's zinc finger tech, adding ALS to a mix of gene therapy alliances (Endpoints) (Reuters)
  • FDA Leaves Tainted Foods on Shelves Too Long, Report Finds (NYTimes) (The Hill)
  • The Health 202: Generic drugs had a great 2017 (Washington Post)
  • Biosimilar Legal Landscape: Lessons From 2017 (Pink Sheet-$)

In Focus: International

  • Made in China: New and Potentially Lifesaving Drugs (NYTimes)
  • WHO prequalifies breakthrough vaccine for typhoid (WHO)
  • In Israel, Teva has become more than just a drug company. But its future is now in question (STAT)
  • All in: Boehringer Ingelheim more than doubles its venture fund to €250M (Endpoints)
  • Israel's Protalix claims early success with oral anti-TNF candidate (Fierce)
  • Hetero launches HumiraTM biosimilar in India (Economic Times)
  • MedTech Groups Phase Out Direct Sponsorship of HCP Training and Education Events (Statement)
  • Report slams European Union's high-risk medical device approvals rate (Medical Design & Outsourcing)
  • HSA Accepted to ICH as New Regulatory Member (HSA)
  • NICE publishes final draft guidelines backing GSK's Strimvelis (PharmaTimes) (NICE)
  • NICE to look again at Pfizer's Besponsa after appeal (PMLive)
  • French Govt Plans To Boost Access To Innovation & Increase Use Of Generics & Biosimilars (Pink Sheet-$)
  • MSD's Keytruda wins bladder cancer indication in Japan (PMLive)
  • Flu vaccine efficacy problems prompt switch in UK to Seqirus' Fluad for over-75s (Fierce)

Pharmaceuticals & Biotechnology

  • Breakthrough drugs offer hope for the 360 million people with hearing loss (CNBC)
  • Which biopharmas are best positioned for 2018? Leerink pegs Vertex and Alexion (Fierce)
  • FDA Achieves Historically Low Eight-Month Median Drug Review Time In 2017 (Pink Sheet-$)
  • Putting More Value Into Biopharmaceutical Value Assessments (Health Affairs)
  • John Mendlein steps in to help focus on the big picture at a fast-growing Moderna (Endpoints)
  • Abbott shares gain after JP Morgan upgrades them to overweight (CNBC)
  • AbbVie tops the TV ad-spend pile one more time to close out 2017 (Fierce)
  • Interview with Nina Hunter, Molecular Biologist at FDA (FDA)
  • New cancer treatments have perplexing side effects (Washington Post)
  • FDA 2017 First Generics Approvals Retain India Imprint (Pink Sheet-$)
  • Testosterone Products Need Clinical Endpoints, Not Just Lab Measurements – FDA (Pink Sheet-$)          
  • Pharmacists Slow To Dispense Lifesaving Overdose Drug (KHN)
  • VirionHealth receives $4.2 m from DARPA (Manufacturing Chemist)
  • Neuroscientist Predicts 'Much Better Treatment' For Alzheimer's Is 10 Years Away (NPR)
  • 2018 is here. Now get ready for 'market-defining' I-O readouts (Fierce)
  • Upstart Expansion bags a $55M launch round to join the hunt for RNA-targeting small molecules (Endpoints)
  • Kindler's Centrexion raises $67M to fund phase 3 pain trial (Fierce)
  • Immune cells linked to very early spread of breast cancer (Reuters)
  • Pfizer, Valneva stand to gain from Sanofi's C. diff vaccine flop: report (Fierce)
  • Starting new COPD inhaler tied to heart attack risk (Reuters)
  • Biggest FDA Developments for 2017 (Eye on FDA)
  • Biogen vet Jean-Paul Kress takes the reins at NYC's Syntimmune (Endpoints) (Fierce)
  • A better approach to growth factors? Scholar Rock raises $47M on SMA promise (Endpoints) (Fierce)
  • Thermo Fisher and Juno sign seven-year CAR-T tech deal (BioPharmaReporter)
  • MilliporeSigma and bluebird bio ink multi-year viral vector deal (BioPharmaReporter)
  • Bioinformatics Company Dnanexus Raises $58M (BioCentury)
  • Big Pharma vets bag $39M to drive I/O firm Elstar forward (Fierce)

Pharmaceutical and Biotechnology: Study Results, Filings and Designations

  • J&J's predicted blockbuster depression posts P2 data (Pharmafile)
  • Perrigo Announces Tentative FDA Approval For The Generic Version Of Prolensa Ophthalmic Solution 0.07% (Press)
  • Palatin Technologies, Inc. Announces FDA Clearance of Investigational New Drug (IND) Application for PL-8177 For Ulcerative Colitis (Press)
  • Omeros Corporation Reaches Agreement with FDA on OMS721 Phase 3 Trial Protocol for IgA Nephropathy (Press)
  • Liquidia Initiates Phase 3 Clinical Trial of LIQ861 in Patients with Pulmonary Arterial Hypertension (Press)
  • BK170114: ASI Automated RPR test for Syphilis for use on the ASI Evolution (FDA)

Medical Devices

  • Sterilmed Reprocessed Agilis Steerable Introducer Sheath recalled due to improper seal of the sheath hub (FDA)
  • Abbott wins CE Mark for Alinity h-series hematology system (MassDevice)
  • Masimo wins CE Mark for RD Rainbow Lite SET sensors (MassDevice)
  • RenalGuard Solutions touts first-in-man heart failure device study results (MassDevice)
  • Medical device startups: Here's how you successfully plan (MassDevice)
  • First Patients Implanted with Axonics Sacral Neuromodulation System in U.S. FDA Pivotal Study (Press)
  • Glaukos Submits IDE Application to FDA to Study iStent infinite Trabecular Micro-Bypass System (Press)
  • FDA Issues Final Guidance on Additive Manufactured ("3D-Printed") Devices (FDA Law Blog)
  • Problem with Quality of Mammograms at Palm Beach Broward Medical Imaging Center in Deerfield Beach, Florida: FDA Safety Communication (FDA)

US: Assorted & Government

  • FDA to Shift Tobacco Regulatory Focus (Medpage)
  • We Wrote About CMS' Data, and It Blew Up (Medpage)
  • The Opioid Crisis: An Examination of How We Got Here and How We Move Forward (Senate HELP)
  • Judge Tosses 'Inconsistent' AbbVie Bellwether Verdict, Orders New Trial (FDA News-$)
  • Lupin, Sandoz Settle With Retailers In Solodyn Suit (Law360-$)
  • Funds Say Valeant Price-Gouging Probe Cost Them $80B (Law360-$)
  • Copay Accumulators: Costly Consequences of a New Cost-Shifting Pharmacy Benefit (Drug Channels)
  • Ackman's Pershing Square, Valeant to appear in court to discuss an insider trading lawsuit settlement (CNBC)
  • Avandia Case Flunks Warning Causation (Drug & Device Law)

Upcoming Meetings & Events


  • Open Public Consultation on "Strengthened cooperation against vaccine preventable diseases" (EC)


  • CDRH Launches Manufacturing and Product Quality Pilot Program (MedTechIntelligence)
  • Angiochem, Xinogen In China Deal (BioCentury)
  • Korean Manufacturer Warned for Adulterated Products (FDA News-$)


  • Indian pharma will continue to stare at single digit growth: ICRA (Economic Times)
  • Fresenius Kabi Hit With Two Warning Letters At Sites In India In December (Pink Sheet-$)
  • Dip in pharma exports due to shifting of manufacturing bases, market consolidation: Government (Economic Times)
  • India only accepting requests to trial and sell vaccines online (InPharmaTechnologist)


  • Notice: Introduction of the Regulatory Enrolment Process (REP) and the Use of the Common Electronic Submission Gateway (CESG) for Medical Devices (Health Canada)


  • TGA Issues New Criteria for Comparable Overseas Regulators (FDA News-$)

General Health

  • Exercise Alters Our Microbiome. Is That One Reason It's So Good for Us? (NYTimes)
  • Obesity Is the Main Contributor to Diabetes in Blacks and Whites (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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