Regulatory Recon: Axovant Alzheimer's Drug Fails in Phase III; FDA Reviewers Say PTC's Duchenne Drug Data 'Not Persuasive' (26 September 2017)

Posted 26 September 2017 By Michael Mezher

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

In Focus: US

  • FDA pours cold water on PTC Therapeutics' Duchenne drug application (Reuters) (Endpoints)
  • Axovant's lead Alzheimer's drug flops in PhIII, ending an unlikely quest (Endpoints) (STAT) (Fierce) (Forbes) (Reuters)
  • Intercept does not expect restrictive boxed warning for liver drug (Reuters)
  • Genocea shelves its lead drug, slashes staff and switches focus to trendy neoantigens (Endpoints) (Xconomy)
  • How Patient Preferences Contribute to Regulatory Decisions for Medical Devices (FDAVoice)
  • FDA joins Interpol crackdown on illicit online pharmacies (Reuters) (Washington Post) (FDA)
  • The next big Alzheimer's trial? Not until 2019. Here's what in the works (STAT)
  • CVC to Explore Options for $4 Billion Drugmaker Alvogen (Bloomberg) (Reuters)
  • The Link Between SoftBank, Alzheimer's and Data (Bloomberg)
  • MedTech Industry Sees Challenges in Restarting Operations, Helping Puerto Rico (Focus)
  • FDA warns 'critical' drug shortages possible after Hurricane Maria battered Puerto Rico (USA Today) (FDA)
  • Health Bill Appears Dead as Pivotal GOP Senator Declares Opposition (NYTimes) (Reuters)
  • Did someone say pledge? AbbVie may reverse its promise on price hikes (STAT) (Fierce)
  • The drug-maker's guide to the galaxy (Nature)
  • Patient assistance charity says US contacted it in probe (Reuters)
  • Allergan's Executive VP and Chief Financial Officer steps down (Pharmafile) (WSJ)

In Focus: International

  • EMA's first public hearing: giving EU citizens a voice to help reduce the risk of valproate (EMA)
  • Cheap Drugs Pay for Aurobindo as U.S. FDA Approvals Surge (Bloomberg)
  • Novartis's new chief sets sights on 'productivity revolution (Financial Times)
  • UK, European Device Industries Urge Continued Regulatory Alignment Post-Brexit (Emergo)
  • New E&Y MedTech Report Highlights Growth, Regulatory Questions (Focus)
  • Saudi Arabian Regulators May Exempt Some Low-Risk Medical Devices from MDMA Registration (Emergo)
  • EU Legal Opinion In Roche/Novartis Case Has Competition And Licensing Implications (Pink Sheet-$)
  • EC approves Janssen's Symtuza (PharmaTimes) (PMLive) (Press)
  • Venezuela doctors in protest urge stronger WHO stance on health crisis (Reuters)
  • India Pricing regulator says Abbott can discontinue premium 'Xience Alpine' stent in a year (Economic Times)
  • Glythera teams with Cancer Research UK (PharmaTimes)
  • MSD launches once-daily Isentress in the UK (PharmaTimes)
  • Portugal: An Uptick In Life Science VC Funding (SCRIP-$)

Pharmaceuticals & Biotechnology

  • An ethical dilemma for doctors: When is it OK to prescribe opioids? (STAT)
  • Drug Wholesalers Struggle: Slower Revenue Growth and Lower Gross Margins for 2017 (Drug Channels)
  • FDA, HHS Finalize Guidance on Documenting IRB Activities (Focus)
  • New CMO hops onto Bicycle Therapeutics (Pharmafile) (Press)
  • Patient Experience Data May Require Separate Label, Genentech Suggests (Pink Sheet-$)
  • Allergan Urgently Seeks FDA Guidance To Unblock Vraylar sNDA Review (SCRIP-$)
  • Cancer trials underrepresent minorities and elderly, Mayo Clinic says (Pharmafile)
  • Merck's Keytruda resumes regulatory hot streak with stomach cancer approval (Fierce)
  • Bristol-Myers' I-O star Opdivo steps up to challenge Bayer's Stivarga in liver cancer (Fierce)
  • Set on challenging GlaxoSmithKline's hepatitis B shot, Dynavax gears up for Heplisav launch (Fierce)
  • Orphan Drugs Designations and Approvals have Something to Say about Risks (Harvard Bill of Health)
  • New FDA GMP Inspection Model (FDAZilla)
  • Fresenius Kabi starts work on $250m US sterile drug plant (InPharmaTechnologist)
  • (Clinical) Trials And Tribulations (Bloomberg)
  • You've heard about precision medicine. Now get ready for precision drug ads (STAT)
  • Expanding Our View of the Human Microbiome (NIH)
  • Aradigm's Linhaliq Gets PDUFA Date (BioCentury)
  • AstraZeneca, NewLink pen PD-L1 and IDO combination pact (Fierce)
  • Next-gen CAR-T, TCR player Autolus gains $80M for clinical trial work (Endpoints)
  • Two young scientists bag $20M to perfect their CRISPR attack on disease-causing bacteria at upstart Eligo (Endpoints)
  • ProQR spotlights the most encouraging results from a mixed snapshot of efficacy for cystic fibrosis (Endpoints)
  • Sensor captures bacteria's movements to measure antibiotic efficacy (Fierce)
  • Teva Announces Reintroduction of Generic Depo-Provera® in the United States (Press)

Pharmaceutical and Biotechnology: Study Results, Filings and Designations

  • Vivet's First Gene Therapy Product, VTX 801 for Wilson's Disease, Receives European and US Orphan Drug Designation (Press)
  • Intercept Spikes; Novocure Jumps on Phase 3 Data -Biotech Movers (The Street)
  • SYNERGYS Announces Phase 1 SBIR Grant Award From National Institutes of Health (Press)
  • DFB Soria Gives Update on Enrollment in Phase 2 Clinical Trial of SOR007 (Nanoparticle Paclitaxel) Ointment for Actinic Keratosis (AK) (Press)
  • Akebia Announces Positive Top-Line Results from Phase 2 Study of Vadadustat in Japanese Patients with Non-Dialysis-Dependent Chronic Kidney Disease (Press)
  • Alnylam Initiates Phase 2 Clinical Study of Cemdisiran (ALN-CC5) in Patients with Atypical Hemolytic-Uremic Syndrome (aHUS) (Press)

Medical Devices

  • Top 20 medtechs jockey for position, but their overall share will drop (EP Vantage)
  • Update Regarding Mortality and Neurological Adverse Events with SynCardia Systems TAH-t Companion 2 Driver System and Freedom Driver System (FDA)
  • Baxter Launches First 3-In-1 Set For Use in Continuous Renal Replacement Therapy and Sepsis Management Protocols (Baxter)
  • LivaNova nabs 510(k) clearance for Optiflow Arterial cannulae (MassDevice)
  • TherOx Announces Its PMA Application for AMI Therapy System is Accepted for Filing by FDA (Press)
  • Mortise Medical Receives FDA Clearance for LigaMetrics™ Suture Anchor System (Press)

US: Assorted & Government

  • FDA halts monkey nicotine research denounced by Jane Goodall as 'shameful' (Chicago Tribune) (Washington Post)
  • CDC launches campaign to help states fight prescription opioid epidemic (CDC)
  • Graham-Cassidy Health Care Hearing Starts With Eruption Of Protests (NPR)
  • CBO: Graham-Cassidy Would Add 'Millions' to Uninsured Rolls (Medpage) (CBO)
  • Movie's Ads Protest Rules Restricting Gay Men From Donating Blood (NYTimes) (Jezebel)
  • US immigration restrictions may slow medical research (Reuters)
  • Report Finds Gaps in Access to Opioid Addiction Help on Staten Island (NYTimes)
  • Reluctance to Learn Genetic Risk for Cancer Grows With Affordable Care Act Repeal Fears (GenomeWeb)
  • Attorney General Jeff Sessions targets medical professionals in 12 U.S. opioid epidemic 'hot spots' (Fierce)
  • Mohawk Nation Exercises Sovereign Immunity in Inter Partes Review (Patent Docs)
  • No Causation, No "Parallel Claim" (Drug & Device Law)

Upcoming Meetings & Events

Europe

  • Russian government to simplify launch of new drugs (PharmaLetter-$)
  • Europe-wide cases brought to EMA in valproate hearing (PharmaLetter-$)
  • Freshers warned against self-prescribing: you're not doctors yet (MHRA)
  • Siemens Healthineers lands CE Mark for Atellica advanced diagnostics system (Drug Delivery)

Asia

  • Domestic firms predict a 16% growth in the South Korean pharma market (PharmaLetter-$)

India

  • Mankind Pharma looking to sell 15 per cent stake; PE funds make a bee-line (Economic Times)
  • Pharma majors knock at Niti Aayog's doors with concerns over new drug policy (Economic Times)
  • Supply all knee implant brands, government tells companies (Economic Times)
  • Currency fluctuation in semi regulated markets to hit Indian pharma: Fitch (Economic Times)
  • Health ministry to constitute special task force in each district to restrict availability of prohibited and regulated drugs like oxytocin (PharmaBiz)
  • Gujarat FDCA writes to health ministry to accelerate process of setting up much awaited medical device testing lab at Vadodara (PharmaBiz)

Australia

  • Updated GMP Clearance application forms (TGA)
  • TGA presentation: RACI and CAPSIG societies, 16 August 2017 (TGA)

Other International

  • Merck names Paolo Carli as the head of MEA (PharmaLetter-$)
  • Sixty-ninth Session of the WHO Regional Committee for Americas (WHO)

General Health & Other Interesting Articles

  • Building Community: One Stride At A Time (LifeSciVC)
  • Breast surgeons exert 'enormous influence' on patients' choices (Reuters)

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 news@raps.org.

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

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