Regulatory Focus™ > News Articles > Regulatory Recon: FDA Approves Roche Hemophilia A Drug Hemlibra; Ultragenyx Gets Priority Review Vou

Regulatory Recon: FDA Approves Roche Hemophilia A Drug Hemlibra Ultragenyx Gets Priority Review Voucher With Enzyme Replacement Therapy Approval (16 November 2017)

Posted 16 November 2017 | By Michael Mezher 

Regulatory Recon: FDA Approves Roche Hemophilia A Drug Hemlibra Ultragenyx Gets Priority Review Voucher With Enzyme Replacement Therapy Approval (16 November 2017)

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

In Focus: US

  • Billionaire founder of Insys to plead not guilty to opioid bribe scheme (Reuters) (Forbes)
  • Old Habits Die Hard: Newer Diabetes Drugs Slow to be Adopted (Medscape)
  • Powerful new cancer drugs are saving lives, but can also ignite diabetes or other autoimmune conditions (Science)
  • Astellas faces US probe over patient assistance charity support (Reuters)
  • FDA To Begin Hiring Pilot In Early 2018 (BioCentury) (FDA Voice)
  • FDA Approves Roche Hemophilia A Drug Hemlibra (Reuters) (Endpoints)
  • FDA Approves Ultragenyx Enzyme Replacement Therapy for MPS VII, Awards Priority Review Voucher (GEN) (Fierce) (Endpoints)  (Reuters) (FDA)
  • $1M For Luxturna? ICER Says Big Discount Needed To Be Cost Effective (SCRIP-$) (Endpoints)
  • FDA, EMA put a star CAR-T from bluebird and Celgene on the VIP list (Endpoints) (Press)
  • George Church: Compelling Reasons for Repairing Human Germlines (NEJM)
  • Emergency Legal Authority and the Opioid Crisis (NEJM)
  • Getting Doctors To Stop Prescribing Codeine To Kids Has Taken Years (NPR)
  • Nearly 1.5 million people signed up for Obamacare plans so far: officials (Reuters)
  • Alnylam starts rolling FDA filing for patisiran (Fierce) (Press)
  • Novel Biologics Will Get Suffixes (Or At Least One Did) (Pink Sheet-$)
  • Public Citizen Asks US FDA To Ban Olmesartan Hypertension Medicines (Pink Sheet-$)
  • US FDA Drug Office Reform: 'Everything' Is On The Table (Pink Sheet-$)
  • He rails against the drug industry. But Trump is turning to its ranks to fill his administration (STAT)

In Focus: International

  • EMA Maps Steps for Improving Product Information for Patients (FDANews-$) (EMA)
  • Pfizer, Novartis agree UK price cuts for new breast cancer drugs (Reuters) (BBC) (PharmaTimes) (NICE)
  • Europe approves GlaxoSmithKline's new triple lung drug (Reuters) (Pharmafile)
  • First WHO Global Ministerial Conference: Ending TB in the SDG Era (WHO)
  • Fake drug siege: Interpol finds 300kg of counterfeit meds in West Africa (InPharma)
  • From 'chaos' to the second largest pharma market in the world, China aims to be first (Outsourcing Pharma)
  • Philippines reallows contraceptive devices after certifying them safe (Reuters)
  • India government need not consult statutory board to ban combo drugs: Supreme Court (Economic Times)
  • Merck KGaA Says Outcomes-Based Pact With NHS England 'Precedent-Setting' (Pink Sheet-$)
  • Rare strain of gonorrhea identified in Canada, compounding fears of drug resistance (STAT)

Pharmaceuticals & Biotechnology

  • FDA Releases Draft Guidance on Assessing Biosimilar User Fees (FDA)
  • Which top cancer R&D companies are looking to make a splash at ASH? (Endpoints)
  • Johns Hopkins has $65M to spend on taking their drugs right up to human studies (Endpoints) (Press)
  • Arsanis prices $40M IPO for infectious disease work (Endpoints)
  • J&J goes back to Ionis for its second experimental pill for a GI autoimmune disease (Endpoints)
  • Google-backed Evelo teams up with Mayo Clinic on microbiome drugs (Endpoints)
  • X4, nearing late-phase trials of ex-Sanofi drug, raises $27M (Fierce) (Endpoints)
  • Do Accelerated Approval Pathways Mean Earlier Patient Access? (Pink Sheet-$)
  • Industrial Cybersecurity Defenses Essential for Pharma Companies (Pharmaceutical Manufacturing)
  • Fusion Genomics Turns to IBM Cloud to Help Support Advances Designed to Conquer Global Pandemics (IBM)
  • Avid letting Peregrine fly off as it focuses on bio-CDMO business (BioPharmaReporter)
  • Evelo taps Mayo Clinic expertise for therapeutic bacteria programs (Fierce)
  • In conversation with: Alice Zhang, CEO of Verge Genomics (Fierce)
  • AstraZeneca, Merck, Clovis and Tesaro, all vying for share, may all win on faster PARP growth (Fierce)
  • AstraZeneca goes to battle with GSK with nod for Nucala challenger Fasenra (Fierce)
  • Stem cell therapy fails to improve symptoms of peripheral artery disease (Pharmafile)
  • Forget what drugs were approved. What drugs performed well? (MedCityNews)
  • FDA Drug Safety Communication: FDA to evaluate increased risk of heart-related death and death from all causes with the gout medicine febuxostat (Uloric) (FDA)
  • FDA Announces Minor Use/Minor Species (MUMS) Grant Application Period Due January 12, 2018 (FDA)
  • Mepergan Fortis Capsules; Final Decision on Proposal To Refuse Approval of Supplemental New Drug Application; Availability of Final Decision (FDA)
  • Sunscreen: FDA Reviewed Applications for Additional Active Ingredients and Determined More Data Needed (GAO)

Pharmaceutical and Biotechnology: Study Results, Filings and Designations

  • bluebird bio Announces First Patient Treated in Northstar-3 (HGB-212), Phase 3 Study of LentiGlobin in Patients with Transfusion-Dependent β-Thalassemia (TDT) and β0/β0 Genotype (Press)
  • Alkermes' New Drug Application for Investigational Product Designed for Initiation Onto ARISTADA Accepted for Filing by FDA (Press)
  • FDA Grants Qualified Infectious Disease Product Designation for RECCE 327 (PharmaLetter-$)
  • PIN Pharma Announces Initiation of its First Phase 1 Study (Press)
  • Ascentage Pharma Announces FDA Acceptance of IND Application for Clinical Study of Novel IAP Inhibitor APG-1387 to Treat Advanced Solid Tumors and Blood Cancers (Press)

Medical Devices

  • Express Scripts inks deal with Propeller Health to provide members access to inhaler monitoring tools (MedCityNews) (CNBC)
  • How a Tiny Breast Cancer Device Carries Big Value (MDDI)
  • Lumendi Reports Successful Completion of DiLumen™ EIP First-in-Human Trials (Press)
  • Insulia receives FDA clearance and CE mark to integrate Basaglar and Tresiba (Press)
  • Smith & Nephew touts reduced pressure ulcers in Allevyn Life dressing trial (MassDevice)

US: Assorted & Government

  • Internet Marketers of Dietary Supplement and Skincare Products Banned from Deceptive Advertising and Billing Practices (FTC)
  • C.D. Cal. Excludes Three Plaintiff Experts in Breast Implant Case (Drug & Device Law)
  • What to Do When You Receive a DEA Order to Show Cause (FDA Law Blog)
  • J&J, Bayer Fight Ex-FDA Head's Xarelto Warning Testimony (Law360-$)
  • Abbott Won't Face Autism Claim In Depakote Birth Defect Trial (Law360-$)

Upcoming Meetings & Events


  • Improving the availability of vaccines for animals within the EU (EMA)
  • GSK: Survey of Antibiotic Resistance (SOAR) (ABPI)
  • Marketing authorisations granted in October 2017 (MHRA)
  • Parallel import licenses granted in October 2017 (MHRA)
  • Nevro wins MR-conditional CE Mark for Senza spinal cord stim (MassDevice)
  • British Biosimilars Association appoints new chair (EPR)


  • Daiichi Sankyo files fresh plea against Singh brothers (Economic Times)
  • Govt to issue uniform code for medical device marketing practices soon (PharmaBiz)
  • Medical device park at Sanand in Gujarat yet to start even after 2 years (PharmaBiz)


  • Medicines Safety Update, Volume 8, Number 5, October-November 2017 (TGA)

General Health & Other Interesting Articles

  • Tom Marsilje, a relentless advocate for cancer patients, dies at 45 (STAT)
  • Uwe Reinhardt, 80, Dies; a Listened-to Voice on Health Care Policy (NYTimes)
  • Heart Attack Survivors at Risk of Later Dementia (NYTimes)
  • A Baby Exposed To Zika Virus Is Doing Well, One Year Later (NPR)

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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