Regulatory Recon: Alexion Wins Priority Review Voucher for Lysosomal Acid Lipase Deficiency Treatment (8 December 2015)

Posted 08 December 2015 | By Michael Mezher 

Regulatory Recon: Alexion Wins Priority Review Voucher for Lysosomal Acid Lipase Deficiency Treatment (8 December 2015)

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

  • Tonix Pharma, US Defense Department partner on PTSD drug (Reuters) (Press)
  • Nearly half of eligible US adults don't take cholesterol drugs (Reuters)
  • FDA approves first drug to treat a rare enzyme disorder in pediatric and adult patients (FDA)
  • FDA clears military traumatic wound dressing for use in the civilian population (FDA) (MassDevice)
  • Wondering if you have the gene linked to Alzheimer's? You may have to leave the US to find out (Washington Post)
  • PDUFA VI: Breakthrough Issues Among FDA Priorities (Pink Sheet-$)

In Focus: International

  • China toughens drug quality standards, rejects 13 applications (Reuters)
  • What If The Pharmacist Just Dropped Pills Into Your Hand? (NPR)
  • Sandoz submits Enbrel biosimilar to European Medicines Agency (Biosimilar News)
  • What 2016 Might Bring for Chinese Medical Device Regulation (MDDI)

US: Pharmaceuticals and Biotechnology

  • Existing compound holds promise for reducing Huntington's disease progression (MNT)
  • All Camps Agree: Memorable Biosimilar Names Are A Must (Law360-$) (Focus)
  • Precision Medicine: Patient Engagement Extends Beyond Drug, Device Development (Thomson Reuters)
  • Teva's Reslizumab Faces FDA Questions On Breadth Of Asthma Indication (Pink Sheet-$)
  • Cystic Fibrosis: Keeping the Momentum Going (NIH)
  • Lack Of Preparedness By CMO's Imperils Pharma Manufacturers (RxTrace)
  • FDA Calls for More Efficient Communications with Sponsors (FDANews-$) (Focus)
  • Buying prescription drugs online is risky (Washington Post)
  • Using a deadly virus to kill cancer: Scientists experiment with new treatment (Washington Post)
  • Once-a-week diabetes meds are similar in safety and effectiveness (Reuters)
  • Chewable Stimulant OK'd for ADHD (MedpageToday) (Press)
  • bluebird outlines beta thalassemia regulatory plans (BioCentury)
  • Roche allies with cell engineering startup SQZ on $500M+ immuno-oncology pact (FierceBiotech)
  • FDA biosimilars labeling policies in flux (BioCentury)
  • New Approach to Predict Evolution of Influenza Viruses Can Enhance Vaccine Efficacy (ICT)
  • Architect of Valeant's Turnaround Is Under Fire (WSJ-$)
  • Study Warns of Alzheimer's Risk in Some Prostate-Cancer Drugs (WSJ-$)
  • Medical Affairs and Postmarketing Studies go Together Like Peas and Carrots (Cutting Edge Info)
  • Teva Pharmaceuticals and Eagle Pharmaceuticals Announce FDA Approval of BENDEKA (bendamustine hydrochloride) Injection (Press)

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

  • Wockhardt gets QIDP status for new antibiotic product from FDA (Economic Times)
  • Acetaminophen Provides No Benefits Against Influenza (ICT)
  • Marijuana Extract May Help Some Children With Epilepsy, Study Finds (NRP)
  • TherapeuticsMD says vaginal atrophy drug meets study goal (Reuters)
  • Novartis: Study shows copycat works just as well as Amgen drug (Reuters)
  • Real-World Data Confirms Safety and Efficacy Profile of XARELTO, With Shorter Hospital Stays, for the Treatment of Deep Vein Thrombosis (Press)
  • Responses Observed in Three-Quarters of Heavily Pre-Treated Multiple Myeloma Patients Receiving KEYTRUDA (pembrolizumab) Combined With Lenalidomide and Dexamethasone (Press)
  • Fate Therapeutics Provides Update on Adoptive Immunotherapy Programs for Hematopoietic Cell Transplantation (Press)
  • Kite Pharma Receives FDA Breakthrough Therapy Designation for KTE-C19 for the Treatment of Refractory, Aggressive Non Hodgkin Lymphoma (NHL) (Press)
  • Afferent Pharmaceuticals Advances Novel Molecule, AF-130, to Phase 1 Clinical Trial (Press)
  • First Patient Enrolled in Cerus' Phase IV PIPER Study (Press)
  • Bio-Path Holdings' Data from Phase I and Safety Segment of Phase II Clinical Trials in Blood Cancers Presented at 57th American Society of Hematology (ASH) Annual Meeting (Press)
  • New Phase III study of nintedanib in people with systemic sclerosis and lung fibrosis (Press)
  • Phase 3 Results for Zydelig With Bendamustine and Rituximab for Relapsed Chronic Lymphocytic Leukemia (CLL) Presented at American Society of Hematology Annual Meeting (Press)
  • Phase 1 results point to larger trial of enzalutamide and fulvestrant in breast cancer (MNT)

US: Medical Devices

  • Google Life Sciences has a new name: Verily (MedCityNews)
  • Medtronic CEO Rejects Notion that Company Only Innovates Through Acquisitions (MDDI)
  • The combination product development 3-step: Considerations for clinical trials (MassDevice)
  • Postmarket Surveillance Reform Board Gets Injection Of Device Industry Experience (Gray Sheet-$)
  • Inspiration LS, 5i and 7i Ventilator Systems by eVent Medical: Class I Recall - May Shut Down without Alarm (FDA)
  • DSM Receives FDA 510k Clearance for Dyneema Purity® Radiopaque Cerclage Cable for Use in Orthopedic Trauma (Press)
  • New technology may standardize sickle cell disease screening for infants (MNT)
  • Most men with erectile dysfunction are satisfied with penile prostheses (MNT)
  • Autonomous Robotic Intubation Device from Ohio State (medGadget)
  • Transdermal Delivery Solutions launches Testagen pivotal trial (MassDevice)

US: Assorted and Government

  • VMS industry requests FDA dedicate greater resources to regulating industry (DSN)
  • HP&M Asks FDA Not to Narrowly Constrict the Formal Dispute Resolution Process (FDA Law Blog)
  • Testing labs agree to pay $8.5 million to settle U.S. false billing claims (NECIR)
  • Concerns Over Valeant Lead to Troubles for Other Companies (Policy and Medicine)
  • Pharma companies 'among happiest places to work' (Pharmafile)
  • Patent board hands St. Jude Medical a win over patent troll (MassDevice)
  • Supreme Court Won't Hear Biolitec-AngioDynamics Case (Gray Sheet-$)

Upcoming Meetings and Events              


  • UK Lung Cancer Patients Get Early Access To AZ's Osimertinib (SCRIP-$) (FDANews-$) (Focus)
  • ExThera Medical wins approval for a CE Mark trial for its Seraph blood filter (MedCityNews)
  • Slovenia and Netherlands lead way in cholesterol treatment (EurActiv)
  • British review calls for urgent cuts to antibiotic use in livestock (Reuters)
  • Five medicines cleared for use by NHS Scotland (PharmaTimes)
  • Ignyta Receives Orphan Drug Designation in the European Union for Entrectinib for the Treatment of Neuroblastoma (Press)
  • EUCAST researches new critical breakpoints for antimicrobial agents (EPR)
  • Ex-NMT Medical PI faces suspended med license (MassDevice)
  • EU Validates Gilead's Four-in-One Hepatitis C Treatment (DD&D)
  • AstraZeneca, NCI tap French company to develop companion mobile app for cancer drugs (FierceMedicalDevices)


  • Govt to introduce injectable contraceptives for women (Economic Times)
  • Hetero gets DCGI approval to market Hepatitis C drug (Economic Times)
  • Seven-member committee to examine online drug sale issue (Economic Times)
  • Unichem Lab gets USFDA nod for hypertension drug (Economic Times)
  • Keytruda, Ibrance On Course To India Debut But What About Pricing? (SCRIP-$)
  • Access to essential medicines in India boosted by drug price caps (PharmaLetter-$)
  • India a potential center for orphan drug development and demand (FiercePharmaAsia)
  • Al Shifa College of Pharmacy in Kerala develops software to support pharmacovigilance programme (PharmaBiz)
  • Health ministry to set up testing lab for infectious diseases in Dehradun (PharmaBiz)
  • Centre notifies phytopharmaceuticals in D&C (Eighth Amendment) Rules, 2015 in Rule 2 (PharmaBiz)


  • Latest Agreements Could Improve Chinese Markets For US Devices (Gray Sheet-$)


  • Japan's Kaketsuken at center of major vaccine, blood product records fraud (FiercePharmaAsia)


    • Reasons for scheduling delegates' final decisions (Joint ACMS-ACCS), December 2015 (TGA)

    Other International

    • Teva gets FDA approval for cancer drug (Reuters)
    • Turkey Struggles To Curb Excessive Antibiotic Use (PharmAsiaNews-$)

    General Health & Interesting Articles

    • New Vaccine Strategy Better Protects High-Risk Cancer Patients From Flu (ICT)

    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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