Regulatory Recon: PTC to Buy Marathon's Emflaza; NICE Cleared to Fast Track More Approvals (16 March 2017)

Posted 16 March 2017 By Michael Mezher

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

In Focus: US

  • Trump FY 2018 Budget Blueprint: Hike in FDA User Fees? (Focus) (Reuters)
  • Trump's budget calls for seismic disruption in medical and science research (Washington Post) (Fierce) (Endpoints) (STAT)
  • Scott Gottlieb's fervor for deregulation could harm patients (STAT)
  • Middlemen's Secret Drug Rebates Targeted by Wyden's Bill (Bloomberg) (BioCentury)
  • 3 Women Blinded By Unproven Stem Cell Treatments (NPR) (Reuters) (NYTimes)  (NEJM)
  • Marathon Pharma to sell Duchenne drug to PTC Therapeutics (Reuters) (Endpoints)
  • Sanders, Cummings Press FDA On Marathon's Emflaza (BioCentury) (Fierce) (Endpoints)
  • Minnesota sees major medical device plant expansions (MassDevice)
  • House Committee Asks FDA for More Info on Contaminated Heparin Investigations (Focus)
  • BIO, Pew Weigh FDA's Revised Draft Guidance on Mixing, Diluting or Repackaging Biologics (Focus)
  • FDA Warns of Increased Risk of Serious Pancreatitis with Allergan's Viberzi (Focus)
  • With FDA's Blessing, Seres Preps New "Pivotal" Microbiome Drug Trial (Fierce) (Press)
  • The Hunt For Novel Treatments Against Deadly Bacteria:  Spero Therapeutics And Its Potentiators (LifeSciVC)
  • US government says 12.2 million consumers in Obamacare individual plans (Reuters)
  • One Certainty of GOP Health Plan: Tax Cuts for the Wealthy (NYTimes)
  • HHS Secretary vows GOP health care bill is 'so much better' than Obamacare (CNN)
  • Republicans to press ahead with plan to dismantle Obamacare health plan (Reuters)
  • Trump Urges Supporters to Unite Behind GOP Health Plan (NYTimes)
  • Pelosi Says Democrats Have A Responsibility To Look For Common Ground On Health Law (NPR)

In Focus: International

  • EU's Pediatric Regulation 'Not Fit For Purpose' Says UK Institute of Cancer Research (Pink Sheet-$)
  • NICE gets go-ahead to fast-track more drug approvals (NICE)
  • New plan to delay use of cost-effective medicines breaks Government's Manifesto promise and breaches patient rights under the NHS Constitution (ABPI)
  • EU Court of Justice Denies EMA Appeal to Release Documents on Two Medicines (Focus)
  • NICE backs use of Biogen's Zinbryta for MS (PharmaTimes)
  • Japan's drug price reforms spark opposing views (Bioworld)
  • Syria sanctions indirectly hit children's cancer treatment (Reuters)
  • Health care a casualty of 6 years of war in the Syrian Arab Republic (WHO)
  • Lilly Eyes Top 10 Japan Ranking Built On Stream Of Launches (SCRIP-$)
  • Foreign Drugs Welcome As China Reforms Progress: CFDA Commissioner (Pink Sheet-$)
  • New UK drug cost rules leave companies fuming (Reuters) (Pharmafile)
  • Pfizer Gets CFDA Approval for Xeljanz (Press)
  • Regulator grants license for Britain's first three-parent IVF babies (Reuters) (STAT)
  • Hikma Pharmaceuticals: Amman with a plan (Financial Times)

US: Pharmaceuticals & Biotechnology

  • Fortress Adds New Rare Disease-Focused Subsidiary (SCRIP-$)
  • Express Scripts Expanding PCSK9 Coverage For FH Patients (BioCentury)
  • Biosimilars: The Pipeline Seams Seem To Be Bursting (Managed Care Mag)
  • Cancer Drug That Might Slow Parkinson's, Alzheimer's Headed For Bigger Tests (NPR)
  • Biosimilars: Next User Fee Cycle Will Be Judged On US FDA's Approvals, Rep. Burgess Says (Pink Sheet-$)
  • Pharma breathes sigh of relief at Trump's FDA pick Scott Gottlieb (Financial Times)
  • Keytruda's head-to-head against Opdivo rockets meds to Nos. 2 and 3 in ad spending (Fierce)
  • Medtronic's SynchroMed woes delay United Therapeutic's Remodulin (Drug Delivery)
  • CDC Foundation's New Business Pulse Focuses on Opioid Overdose Epidemic (CDC)
  • Higher rate of second heart attacks in patients who can't tolerate statins (Reuters) (NYTimes)
  • Many at risk for stroke don't get anti-clotting drugs (Reuters)
  • US FDA Strategy On Abuse-Deterrent Opioids Needs Rethinking, Panelists Say (Pink Sheet-$)
  • American-made biosimilars a differentiator in US market, Coherus (BioPharmaReporter)
  • Determination that Cyanocobalamin Injection, 1 Milligram per Milliliter in a 10 Milliliter Vial, Was Not Withdrawn From Sale for Reasons of Safety or Effectiveness (FDA)

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

  • Alkermes Initiates Phase 3 Gastrointestinal Tolerability Study of ALKS 8700 for Treatment of Multiple Sclerosis (Press)
  • Short-Course Radiation plus Temozolomide in Elderly Patients with Glioblastoma (NEJM)
  • Acceleron Announces Plans to Initiate a Phase 2 Trial of ACE-083 in Charcot-Marie-Tooth Neuromuscular Disease and Host Educational Webinar (Press)
  • MetrioPharm advances psoriasis drug after phase 2 readout (Fierce)

US: Medical Devices

  • Why surgical stapling is a hot medical device product (MassDevice)
  • An Unprecedented Lifetime Warranty in the Medical Device Sector – Medicrea (Forbes)
  • FDA Issues Proposed List of Class II Devices for 510(k) Exemption – Focus on IVDs, Including Drugs-of-Abuse Tests (FDA Law Blog)
  • Alere misses extended 10-K filing date (MassDevice)
  • DJO Global wins FDA 510(k) for Exprt revision hip portfolio (MassDevice)

US: Assorted & Government

  • FDA Faces Uncertainty Implementing 21st Century Cures Act (Law360-$)
  • USPTO rejects new reexamination request against Celyad's US Patent for Production of Allogeneic TCR-Deficient CAR-T Cells (Ceylad)
  • GAO: HHS Should Assess the Effectiveness of Its Efforts to Enhance Patient Access to and Use of Electronic Health Information (GAO)
  • Veteran's Health Care: Limited Progress Made to Address Concerns That Led to High-Risk Designation (GAO)
  • Solicitor General Urges Supreme Court to Reverse California's Ill-Conceived Version of "Specific Jurisdiction" (Drug & Device Law)
  • Trial concluding for pharmacist charged in deadly U.S. meningitis outbreak (Reuters)
  • FDA Rejected Paxil Label Changes, GSK Says In Suicide Trial (Law360-$)
  • Gilead Beats $50M Drug Bonus Call In Del. Chancery Ruling (Law360-$)
  • Judge Bryson Rules That Jury Need Not Be Apprised of Presumption of Validity Standard for Granted Claims (Patent Docs)

Upcoming Meetings & Events

Europe

  • Marketing authorisations granted in February 2017 (MHRA)
  • Parallel import licences granted in February 2017 (MHRA)
  • NHS prescription charges from April 2017 (UK DOH)
  • InVivo wins initial clearance to add UK trial sites to Inspire study (MassDevice)
  • Shire Receives European Approval for Label Extension of CINRYZE (C1 inhibitor [human]) to Prevent and Treat Hereditary Angioedema (HAE) Attacks in Paediatric Patients with HAE (Press)

India

  • DoP directs NPPA to refix/revise ceiling price of formulation "Pheniramine injection 22.75 mg/ml" (PharmaBiz)

Australia

  • Prescription medicines: new or extended uses, or new combinations of registered medicines (TGA)

General Health & Other Interesting Articles

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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