Regulatory Recon: FDA Questions Sarepta DMD Drug in Briefing Documents (15 January 2016)

Posted 15 January 2016 | By Michael Mezher 

Regulatory Recon: FDA Questions Sarepta DMD Drug in Briefing Documents (15 January 2016)

Welcome to Regulatory Reconnaissance, your daily regulatory news and intelligence briefing. Want to read Recon as soon as it's posted? Follow @Michael_Mezher, @Zachary Brennan and @RAPSorg on Twitter.

In Focus: US

  • Patient Groups Respond to BioMarin DMD Drug Rejection (CureDuchenne) (Parent Project MD)
  • FDA delays decision on Heron's nausea drug (Reuters) (Press)
  • NIH launches sequencing center (BioCentury) (STAT) (NIH)
  • FDA Makes 9 Observations over Wockhardt's Shendra Plant, Company Recalls 62k Bottles of Antibiotics (Economic Times) (Reuters)
  • FDA staff unconvinced by Sarepta's DMD drug, shares tank (Reuters) (FDA Briefing Documents)
  • House Committee Requests Pricing Info From Shkreli Ahead of 26 January Hearing (Letter)
  • Days After Senate Report, FDA clears Olympus Duodenoscope with Design Changes  to Reduce Infection Risk (FDA) (Focus)
  • CBER's Planned Guidance Documents for 2016 (FDA)

In Focus: International

  • ANVISA Reports Improved Timeframes for Some Medical Device Market Applications (Emergo)
  • Dengue vaccine enters phase 3 trial in Brazil (NIH)
  • Dozens feared exposed as Sierra Leone confirms new Ebola case (Reuters) (WHO)
  • When New Cancer Treatments Fail, Italy Wants Its Money Back (Bloomberg)

Sponsored Content: The Regulatory Affairs Landscape has Evolved. Will You?

US: Pharmaceuticals and Biotechnology

  • Barriers to biologicals competition (GaBI)
  • Delayed deaths declining among pediatric cancer survivors (Reuters)
  • NIH's big cancer database coming soon (Washington Post)
  • HPV Vaccine Uptake is Highest Among Girls in High Poverty and Hispanic Communities (ICT)
  • FDA's Post-Approval Drug Tracking Not Reliable, GAO Says (Law360-$) (NYTimes) (Focus)
  • Pre-exposure Prophylaxis Found to be as Safe as Aspirin (ICT)
  • Living With Cancer: A Broken Covenant With Patients (NYTimes)
  • FDA to discuss drug-induced liver injury (BioCentury)
  • NIH asked to march in on Xtandi's price (BioCentury) (STAT) (March-in Letter)
  • FDA Rejects New Drug Application for BioMarin's Duchenne Treatment (WSJ) (Reuters)

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

  • Astellas, Medivation report successful enzalutamide mCRPC PhII data in Lancet (FiercePharmaAsia)
  • Grazax misses Phase III asthma prevention endpoint (BioCentury)
  • Merck KGaA, Darmstadt, Germany, Pfizer and Syndax Announce Collaboration to Evaluate Combination of Avelumab and Entinostat in Ovarian Cancer (Press)
  • Results published from the Phase 2 TERRAIN trial of Enzalutamide compared to Bicalutamide in metastatic castration-resistant prostate cancer (MNT)

US: Medical Devices

  • Industry Attorneys Criticize FDA's Emerging Signals Draft Guidance (Gray Sheet-$)
  • Insiders' Guide To Winning In the Drug Delivery Device Market — Part 1: Design (MedDeviceOnline)
  • FDA Hits User-Fee-Era Record For 'Novel' Devices: A New Normal? (Gray Sheet-$)
  • Cyber attacks on industrial control systems mount (MassDevice)
  • Edwards Lifesciences wins FDA nod for new Sapien 3 trial (MassDevice)
  • FDA makes increasing use of 'real-world' device data a strategic priority (FierceMedicalDevice)
  • Medtronic Launches OsteoCool RF Ablation System Following 510(k) Clearance (MedDeviceOnline)
  • Conavi wins 510(k) for cardiac ultrasound imager (MassDevice)

US: Assorted and Government

  • Boston Scientific Used Counterfeit Materials, RICO Suit Says (Law360-$) (MassDevice)
  • U.S. patent office rules against Amgen Humira challenge (Reuters)
  • First Amendment Follow-Up (Drug and Device Law)
  • FTC Report: Pay-for-Delay Cases Dropped in Fiscal 2014 (FDANews-$) (Focus)
  • Michigan introduces new biosimilars substitution bill (GaBI)
  • No Need To Split Provigil Claims Before Trial, Cephalon Says (Law360-$)
  • AngioDynamics must face Bard patent infringement lawsuit (MassDevice)
  • High Court Won't Hear InFuse Off-Label Preemption Case (Gray Sheet-$)
  • Dietary supplement sellers could face fresh challenges in 2016 (Reuters)

Upcoming Meetings and Events              


  • Lorraine Nolan Appointed Chief Executive Of Irish Drug, Device Agency (SCRIP-$)
  • Commission clears EU health directorate reshuffle (ScienceBusiness)
  • Patient Engagement Helps Nexavar Secure Scottish Reimbursement For Liver Cancer (Pink Sheet-$)
  • Meeting highlights from the Pharmacovigilance Risk Assessment Committee (PRAC) 11-14 January 2016 (EMA)
  • Imricor wins CE Mark for MR-conditional recording system (MassDevice)
  • Loxo Oncology Announces EMA Orphan Drug Designation Granted to LOXO-101 for Treatment of Soft Tissue Sarcoma (Press)


  • CDSCO unveils online portal 'Sugam' to strengthen e-governance initiatives (PharmaBiz)
  • ISCR asks govt to amend norms to empower IEC to take final call on number of trials an investigator can undertake (PharmaBiz)
  • Government launches four mobile health services (Economic Times)
  • Task Force asks govt to enhance access to safe, affordable drugs to address burden of communicable & non communicable diseases (PharmaBiz)
  • NPPA Reverses Course, Withdraws Pricing Order For FDC Moxifloxacin + Prednisolone Acetate (24 Insight)
  • Low costs and less stringent regulatory requirements in India (GaBI)




  • Health Canada issues draft revised SEB guideline (GaBI)


  •  Chi-Med initiates HMPL-523 Phase I clinical trial in hematological cancer in Australia (Press)

Other International

  • Low-cost mosquito netting flies high in test to repair groin hernias (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

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

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