Regulatory Recon: FDA Reverses on Amicus’ Fabry Disease Treatment Sanofi Acquires Protein Sciences (11 July 2017)

Posted 11 July 2017 | By Zachary Brennan 

Regulatory Recon: FDA Reverses on Amicus’ Fabry Disease Treatment Sanofi Acquires Protein Sciences (11 July 2017)

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

In Focus: US

  • FDA orders a rare retreat on Amicus’ migalastat, dropping PhIII demand and signaling a major shift for drug developers (Endpoints) (Stat) (Fierce) (Forbes)
  • FDA Adds Immediate-Release Opioids to REMS Program (MedPage Today) (Bloomberg) (WSJ-$)
  • Two Recent Scientific Advances Underscore an Encouraging Future for Precision Medicine at FDA (FDA Voice)
  • Novartis readies for FDA review of $1 billion CAR-T child cancer hope (Reuters) (EP Vantage)
  • Call it a comeback: Arena Pharma shares soar on positive lung disease study results (Stat-$) (CNBC)
  • Value-Based Pricing and State Reform of Prescription Drug Costs (JAMA)
  • White House’s dwindling science office leaves major research programmes in limbo (Nature)
  • Briefing Information for the July 13, 2017 Meeting of the Oncologic Drugs Advisory Committee (FDA)
  • FDA Finalizes List of 1,003 Class II Device Types Exempt From 510(k) Requirements (Focus)
  • FDA Speeding Generic Drug Approvals: Not Just Lip Service (Focus)
  • FDA Looks to Standardize PQ/CMC Data and Terminologies (Focus)

In Focus: International

  • Measles continues to spread and take lives in Europe (WHO)
  • IMDRF meeting in Canada in September 2017 open for registration (IMDRF)
  • Sanofi to acquire Protein Sciences for $650m Upfront (Sanofi) (TheStreet) (Reuters)
  • UK pharma launches legal challenge to NICE plans (PMLive) (Pharmafile)
  • Vaccine booster for France, big pharma prepared (In-PharmaTechnologist)
  • Cancer in Asia-Pacific, a CRO perspective (Outsourcing-Pharma)
  • India aims for $100 billion biotech industry by 2025 (PharmaLetter-$)
  • EMA seeks views of public during its safety review of valproate (EMA)
  • Submissions received and TGA response: Reforms to the regulatory framework for complementary medicines (TGA)

Pharmaceuticals & Biotechnology

  • Pfizer seeks second chance for leukemia drug Mylotarg, but FDA raises concerns (SNL)
  • Teamsters criticize McKesson for ‘excessive’ CEO pay and its role in the opioid crisis (Stat-$)
  • After raising $100M, Google-backed Evelo looks to blaze a new trail with monoclonal microbials (Endpoints)
  • Generic Drug Trade Association Sues to Enjoin Maryland Price Gouging Law (FDA Law Blog)
  • Allergan’s Social Contract, One Year Later (Timmerman Report-$)
  • This drug reversed memory loss in mice. Could Calico use it in humans? (SF Business Times)
  • Genetic Testing Underutilized in Early Breast Cancer (MedPage Today)
  • Teva accuses former executive of siphoning trade secrets to her rival CEO boyfriend (Fierce)

Pharmaceuticals and Biotechnology: Clinical Study Results, Filings & Designations

  • Gene therapy in hemophilia advances with big drops in patient bleeding rates (Stat-$) (Endpoints)
  • Ocular seeks to extend regulatory timeline for Dextenza due to manufacturing concerns (Mass Device)
  • BioMarin's Investigational Gene Therapy for Hemophilia A at 6e13 vg/kg Dose Maintains Average Factor VIII Levels within Normal Range for over One Year (Press)
  • Principia Biopharma Announces PRN1008 Receives Orphan Drug Designation From FDA for Treatment of Pemphigus Vulgaris (Press)
  • Priority review for Lilly’s breast cancer drug (PharmaTimes)

Medical Devices

  • FDA clears Medtronic’s CoreValve Evolut TAVR in intermediate risk patients (Mass Device)
  • Defymed reveals development of novel insulin delivery device (Mass Device)
  • pSivida licenses European rights for Durasert to Alimera (Mass Device)
  • Amendia rebrands under acquired company moniker Spinal Elements (Mass Device)
  • FDA Clears Pesticide-Free Lice & Egg Elimination Kit (Press)

US: Assorted & Government

  • Walden and Murphy Urge NIH to Improve Reporting and Investigation of Pathogen Incidents (E&C Committee)
  • Two off-label bills on tap at E&C (Politico)

Upcoming Meetings & Events


  • Britain to hold inquiry into contaminated blood scandal which killed 2,400 (Reuters)
  • Commission welcomes agreement by Parliament and Council to protect workers better against cancer-causing chemicals (EC)

General Health & Other Interesting Articles

  • Drink coffee? It won’t hurt you, and may reduce your risk of an early death (Stat)
  • Accidental effect of fewer Pap screenings: more cases of undiagnosed chlamydia (Stat)
  • Can Patients Make Recordings of Medical Encounters? (JAMA)

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.

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