Regulatory Recon: FDA Reviewers Still Skeptical on Sarepta DMD Drug, Slow Sales for PCSK9s Amid Pricing Debate (21 April 2016)

Posted 21 April 2016 | By Michael Mezher 

Regulatory Recon: FDA Reviewers Still Skeptical on Sarepta DMD Drug, Slow Sales for PCSK9s Amid Pricing Debate (21 April 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

  • Sarepta shares sink as FDA staff stay sour on muscle disorder drug (Reuters) (The Street)
  • An Open Letter To FDA Regarding Eteplirsen (Forbes)
  • UDI Rumbles On (Politico) (Focus)
  • FDA: TransEnterix Surgibot 510(k) does not meet criteria for "substantial equivalence" (MassDevice) (Press)
  • Breakthrough cholesterol drugs fizzle amid price pushback (AP) (Reuters) (DD&D)
  • Biden to cancer researchers: How can we move faster on this? (Washington Post)
  • GW Pharma's cannabis drug gets orphan drug status (Reuters)
  • Aggressive 'mini-stroke' therapy may cut serious stroke risk by half (Reuters) (NEJM)
  • Fighting Cancer With Killer T-Cells: 5 Developments To Watch (Forbes)
  • FDA Official Testifies on Lab Safety After Smallpox, Anthrax Incidents (Focus)
  • CRISPR/Cpf1 Can Cleave RNA, Says Study From Charpentier Lab (GenomeWeb)
  • Strengthening Partnerships: FDA's China Office Engages in Key Outreach with Chinese Provincial FDA, Academia, and Industry (FDA Voice)

In Focus: International

  • Europe is malaria free, 214 million cases in rest of world in 2015: WHO (Reuters)
  • FDA to Pharma Companies: Indian CRO's Clinical and Bioanalytical Studies are Unacceptable (Focus)
  • Glenmark receives 3 observations from USFDA for Indore plant; company says issue resolved (Economic Times)
  • Taking advantage of FDC ban, traders in Kerala return even non-banned drugs resulting in big loss to companies (PharmaBiz)
  • Roche details hit to Avastin price in Japan (Fierce)
  • European Regulatory Roundup: Germany Joins EU Medical Countermeasure Procurement Alliance  (Focus)
  • Billboards in Brazil have been rigged to lure and kill Zika-carrying mosquitoes (STAT)
  • Europe Warms to New Novartis Heart Drug Amid Glacial US Sales (Bloomberg) (Financial Times)

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US: Pharmaceuticals & Biotechnology

  • No Evidence To Support Screening Young Athletes (CardioBrief)
  • Do ethicists hinder HIV prevention research? (Reuters)
  • Promoting Healthcare Innovation on the Demand Side (SSRN)
  • Pfizer, Roche join $42.6M round to fund microbiome player Second Genome (MedCityNews-$)
  • Targeted therapy: Generic imatinib — impact on frontline and salvage therapy for CML (Nature)
  • NASH Drug Cost Concerns On Level With HCV Treatments For Payers (Pink Sheet-$)
  • Fixed-Combination Drug Products: Are Phase 2 and 3 Studies Really Necessary? (The 505(b)(2) Blog)
  • Biogen profit beats on higher Tecfidera sales (Reuters)
  • BioCellChallenge granted US patent for antibody delivery tech (BioPharma-Reporter)
  • Drug-induced Raynaud's phenomenon 'probably underestimated' (Pharmaceutical Journal-$)
  • Single-use and Stainless steel: Hybrid technologies the future of drug production says expert (BioPharma-Reporter)
  • AbbVie gets option on early Argenx cancer drug for $40 million (Reuters)
  • OTC antihistamine shows potential to repair MS nerve damage (Pharmaceutical Journal-$)
  • Existing drugs are effective against human parainfluenza virus (Pharmaceutical Journal-$)
  • Basilea lands BARDA contract for ceftobiprole (BioCentury)
  • Hummingbird, Sanofi to develop cancer antibodies (BioCentury)
  • Industry's Rebate Arms Race: Securing Market Access At A Cost (SCRIP-$)
  • US Foreign Bribery Investigations Ramp Up, With 'Carrot' For Cooperation (PharmasiaNews-$)
  • PCI Synthesis sets up polymers development lab (In-PharmaTechnologist)
  • BioClinica partnership to support 'continuum of pharmacovigilance services' (Outsourcing-Pharma)
  • J&J success will spur pharma's shift to continuous manufacturing, Rutgers (In-PharmaTechnologist)
  • Exco InTouch partners with Regulatory Strategies to meet data regulations (Outsourcing-Pharma)
  • Scuttled IPOs cast pall on struggling biotech sector (EP Vantage)
  • Economics Of Harm From Biologic-Biosimilar Competition (Law360-$)

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

  • MedDay Reports Full Data from Pivotal Phase IIb/III MS-SPI / MS-ON Studies with MD1003 in Multiple Sclerosis at AAN (Press)
  • Cara Resuming Phase III Trial In Post-Op Pain After Clinical Hold Lifts (Pink Sheet-$)
  • Micro heart muscle created from stem cells (MNT)
  • Eisai Announces Non-Clinical Research Findings At AACR 107th Annual Meeting Regarding Combination Of Anticancer Agent Lenvatinib With Everolimus (Press)
  • Positive top-line results from the Phase III PALOMA-2 trial (EPR)
  • Cartiva, Inc. Announces Positive FDA Advisory Committee Review of Cartiva SCI (Press)
  • Inotek Pharmaceuticals Corporation Announces Publication of Phase 1 Data for Trabodenoson in Healthy Adult Volunteers in the Journal of Ocular Pharmacology and Therapeutics (Press)

US: Medical Devices

  • Don't Finalize LDT Guidance, House Appropriators Tell FDA (Gray Sheet-$)
  • Speedy eye-tracking device seeks to detect concussions (Reuters)
  • Insiders' Guide To Winning In the Drug Delivery Device Market — Part 5: The Internet of Things
  •  (MedDeviceOnline)
  • Hologic Announces FDA Clearance and Commercial Availability of the Affirm Prone Biopsy System, the First 2D/3D™ Imaging-Guided Dedicated Prone Biopsy System (Press)
  • VolitionRx announces study results showing NuQ blood test detects prostate cancer early with significantly higher accuracy than PSA test (MNT)

US: Assorted & Government

  • Pot legalization group says to meet White House officials (Reuters)
  • One-Two Punch On Supplements By Former FDA Investigator, Harvard Researchers (Tan Sheet-$)
  • US to Delay Release of New Hospital Ratings (WSJ-$) (KHN)
  • NIH Reluctant To "March In": Collins Suggests Authority Not Intended To Address Pricing Concerns (RPM Report-$)
  • Healthcare No. 1 target for cyberattacks in 2015 (Fierce)
  • Illinois says five more people with bacterial infection have died (Reuters)
  • Drug Labels Must Reflect Risk Sooner, Tylenol Court Told (Law360-$)
  • Maine follows N.Y. in mandating e-prescribing of opioids (MedCityNews)
  • Patent Invalidation Faces Its Biggest Test: Supreme Court To Decide IPR Standard (Pink Sheet-$)
  • How Things Get Done In Washington: Insights From 23 Former CMS Leaders (Health Affairs Blog)
  • Sandoz Loses Bid To Invalidate IP In Toviaz Row With Pfizer (Law360-$)
  • Variability of US State Workplace Wellness Program Laws (Harvard Bill of Health)
  • VA Changes Course to Require Covered Drugs that are TAA Non-Compliant to be Available on the Federal Supply Schedule (FDA Law Blog)
  • Biotech Entrepreneur, Others Want OxyContin Case Reheard (Law360-$)

Upcoming Meetings & Events


  • Germany and the pharma industry: seeing eye to eye at last? (PharmaPhorum)
  • Draft concept paper on an addendum to the guideline on the evaluation of medicinal products indicated for treatment of bacterial infections (CPMP/EWP/558/95 Rev. 2) to address paediatric-specific clinical data requirements (EMA)
  • EU member states urged to support the Dutch Presidency's vision on access to affordable medicines (PharmaLetter-$)
  • NHS England unveils multi-billion-pound plan for general practice (PharmaTimes)
  • Boston Scientific wins CE Mark for MR-safe Emblem S-ICDs (MassDevice)
  • EU physicians prefer greater detail in the labeling of biosimilars (PharmaLetter-$)
  • Is Compliance With MDD Really No Defence In Product Liability/Negligence Claims? (Clinica-$)


  • Contentious Indian Pricing Orders Now In Court (SCRIP-$)
  • Anti-dumping duty likely on a chemical used by pharma industry (Economic Times)
  • Government plans implementing National Framework for Malaria Elimination with Sun Pharma (Economic Times)
  • Avon Lifesciences clarifies stand in drug seizure case as FDA investigation continues (PharmaBiz)
  • Pharmexcil to explore Korean markets through India Pavilion at 'Korea Pharm' (PharmaBiz)


  • Merck's Keytruda now available for Canadians with metastatic NSCLC (PharmaLetter-$)
  • Canada aims for marijuana legalization in 2017 (Reuters)

Other International

  • Angola yellow fever outbreak exposes vaccine supply dilemma (Reuters)

General Health & Other Interesting Articles

  • Sewer Robots Sift Data From Raw Human Waste (IEEE Spectrum)
  • Common medicines tied to changes in the brain (Reuters)
  • Drones could speed up HIV tests in remote areas (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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