Regulatory Focus™ > News Articles > Regulatory Recon: FDA Lifts Hold on Alnylam Hemophilia Trials; EMA Recommends 7 New Medicines for EU

Regulatory Recon: FDA Lifts Hold on Alnylam Hemophilia Trials EMA Recommends 7 New Medicines for EU Approval (15 December 2017)

Posted 15 December 2017 | By Michael Mezher 

Regulatory Recon: FDA Lifts Hold on Alnylam Hemophilia Trials EMA Recommends 7 New Medicines for EU Approval (15 December 2017)

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

In Focus: US

  • Aclaris's drug to treat common skin growth gets FDA nod (Reuters)
  • FDA allows Alnylam to restart hemophilia treatment trials (Reuters) (Endpoints) (Press)
  • Pfizer's Xeljanz gets FDA nod as joint disease treatment (Reuters) (Press)
  • To Be (a Device) Or Not to Be? That Is the Question (Fortune)
  • CAR T cells — what have we learnt? (Nature)
  • What's Coming in 2018: FDA's Regulatory Agenda (Focus)
  • FDA Guidance: Regulations Under a Mask? (Bloomberg BNA)
  • Amicus CEO directly lobbied FDA's Gottlieb to reverse FDA's rejection of migalastat, claiming a delay of up to 7 years (Endpoints)
  • Grail taps Genentech vet Jennifer Cook as CEO, building expectations for 2018 (Endpoints)
  • Merck's Keytruda fails pivotal gastric cancer trial (Reuters) (Endpoints) (Press)
  • Galapagos snags co-promotion rights on filgotinib — just before Gilead's lock-up pact expires (Endpoints)
  • Biosimilar Act Trumps State Laws, Fed. Circ. Rules (Law360 1, 2-$) (Patent Docs) (STAT) (Pink Sheet-$) (Law360-$)
  • Americans Say They Are Suffering as Drug Costs Continue to Rise (Consumer Reports)
  • Follow the money to understand how drug profits flow (STAT)
  • Industry insiders are upbeat about 2018, but that doesn't mean more hiring (STAT)

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In Focus: International

  • EMA Recommends Seven New Medicines for EU Approval (EMA 1, 2, 3, 4)
  • Novo Nordisk wins EU panel's backing for key diabetes drug (Reuters)
  • Teva shares jump after Goldman upgrades embattled drugmaker (CNBC)
  • Can the Teva reorganization really achieve all its goals? Here's what the wags say (STAT)
  • Perrigo lines up bid for Merck's consumer health unit: sources (Reuters)
  • Germany's Merck in fresh bid to enter US MS pill market (Reuters)
  • Siemens CEO says company will not break up — but will become a 'fleet of ships' instead (CNBC)
  • Siemens buys Fast Track Diagnostics to boost molecular offering (Reuters)
  • EMA To Establish Minimum Requirements For CAR-T Cell Registries (Pink Sheet-$)
  • European Regulatory Roundup: Critical Moment for EMA (Focus)
  • NICE recommends Imbruvica for some MCL patients (PharmaTimes) (Fierce) (NICE)
  • Brexit Could Rob Industry Of Access To Markets Covered By 35 EU Free Trade Deals (Pink Sheet-$)
  • Novartis oncology CEO Bruno Strigini is calling it quits (Endpoints)
  • How Cancer Research UK looked to the cloud for improved clinical trials efficiency (MedCityNews)
  • As Zika Babies Become Toddlers, Some Can't See, Walk or Talk (NYTimes)

Pharmaceuticals & Biotechnology

  • Fighting the public health threat of counterfeit drugs (PBS)
  • The 50 most active biotech VC funds in 2017 (Endpoints)
  • FDA Investigators Want Top Management Involved In Manufacturing Inspections (Pink Sheet-$)
  • In the wake of #MeToo, a new spotlight on harassment in biomedical science (STAT)
  • Pfizer Not Planning US Launch of its Second Remicade Biosimilar Approved by FDA (Focus)
  • FDA to Begin Issuing Deficiency Review Letters to Generic Drugmakers (Focus)
  • Pharma Ups Its Game On Trial Transparency But There's 'Room For Improvement' (Forbes)
  • FDA to Pharma Firms: Update or Certify Drug Listings by End of Month (Focus)
  • Jnana launches with $50M to spotlight an unexplored protein territory (Endpoints)
  • Cannabis ingredient holds promise as antipsychotic medicine (Reuters)
  • Why Are Drug Prices So High? Let Us Count the Ways (Medpage)
  • Cancer: Strategies for mature T cell cancers (Nature)
  • Cancer Immunotherapy: The right shape (Nature)
  • Automating drug discovery (Nature)
  • Addressing challenges in the diagnosis and treatment of rare genetic diseases (Nature)
  • Petra Wicklandt to head up new Merck KGaA unit (PharmaLetter-$)
  • Bringing new hope to the fight against Alzheimer's (Financial Times)
  • Therapy focus – Antisense projects raise Huntington's hopes (EP Vantage)
  • Upcoming events – Approval decision for Aclaris and Elagolix's phase III data (EP Vantage)
  • Adapt Or Die: Sanofi CSO On R&D Challenges Within Unstable Industry (SCRIP-$)
  • Drugmakers Call for More Flexible Templates for REMS (FDANews-$)
  • Generic Labeling Rule Dies In Latest Reg Agenda, But Electronic Labeling Is Reborn (Pink Sheet-$)
  • The FDA's new regenerative medicine framework: What does it mean for product development? (PharmaLetter-$)
  • CMC Biologics to make Harpoon's cancer drugs in Seattle, US (BioPharmaReporter)
  • David Apelian jumps ship from Achillion (Fierce)
  • New Pfizer and Allergan rumors dismissed (PharmaLetter-$)
  • Celgene, CRT strike mRNA translation cancer R&D pact (Fierce) (PharmaTimes)
  • Rimidi and Lilly Collaborate to Personalize Solutions for People Using Insulin (Press)
  • FDA update: new 'patient medication document' and more competition in the works (PharmaLetter-$)
  • Issuance of Priority Review Voucher; Rare Pediatric Disease Product (FDA)

Pharmaceutical and Biotechnology: Study Results, Filings and Designations

  • Sanofi And Regeneron Announce Positive Topline Pivotal Results For PD-1 Antibody Cemiplimab In Advanced Cutaneous Squamous Cell Carcinoma (Press)
  • Egalet Receives FDA Tentative Approval for Expanded Label for ARYMO® ER (morphine sulfate) C-II, an Extended-Release Morphine Product Formulated with Abuse-Deterrent Properties for Treatment of Chronic Pain (Press)

Medical Devices

  • FDA Discusses Device Least Burdensome Provisions in New Draft Guidance (Focus)
  • Usage Of Uterine Device Down Since Warning, FDA Says (Law360-$) (FDA)
  • Risk-based IVD regulations: What do they mean for the global IVD industry? (MedCityNews)
  • CDRH Issues Draft CLIA Waiver Guidances (FDA Law Blog)
  • Cybersecurity: Email is largest source of healthcare data breaches for 2017 (MassDevice)
  • pSivida touts pilot osteoarthritis trial for Durasert implant (Drug Delivery)
  • Genomic Screening: What's Age Got To Do With It? (Harvard Bill of Health)
  • Why an FDA Panel Shot Down Intrinsic's Spinal Implant (MDDI)
  • Estimated clinical impact of the Xpert MTB/RIF Ultra cartridge for diagnosis of pulmonary tuberculosis: A modeling study (PLOS)
  • Chemence Medical wins FDA nod for Exofin Fusion skin closure system (MassDevice)

US: Assorted & Government

  • White House tamps down expectations of additional opioid funding this year (STAT)
  • Arizonans get $4.6 million in refund checks from blood test company Theranos (AZCentral)
  • Lab bankruptcies thwart Medicare efforts to recover millions in testing overcharges (STAT)
  • Healthcare.done: ACA open enrollment comes to an end in 39 states (Politico)
  • Updates on Modernizing the Electronic Patent Application Process (USPTO)
  • DaVita Rx Agrees to Pay $63.7 Million to Resolve False Claims Act Allegations (DoJ)
  • Mass. City Latest To Sue Pharma Cos. Over Opioid Crisis (Law360-$)
  • Xarelto Bellwether Loser's New Trial Bid Rejected By Judge (Law360-$)
  • Boston Scientific Settles Nearly 350 Pelvic Mesh Suits (Law360-$)
  • J&J Unit Hit With $15M Pelvic Mesh Verdict In NJ (Law360-$)
  • A Review Of Massachusetts' Marijuana Industry In 2017 (Law360-$)
  • Michigan Strikes Back . . . In Pennsylvania (Drug & Device Law)

Upcoming Meetings & Events


  • Mycophenolate: updated recommendations for contraception for men and women (EMA)
  • Italy to Allow Living Wills and the Refusal of End-of-Life Care (NYTimes)


  • WuXi Biologics bets on China's MAH scheme with new facility (PharmaLetter-$)
  • Tagrisso Reimbursed In Korea, After Difficult Price Negotiations (Pink Sheet-$)


  • Supreme Court asks drugs advisory body to decide fate of over 334 banned combination medicines (Economic Times)
  • Govt fixes ceiling prices of 849 formulations till Nov (Economic Times)


  • Notice Administrative Processing of Submissions and Applications: Human or Disinfectant Drugs (Health Canada)

Other International

  • Thailand battles drug-resistant malaria strains that imperil global campaign (Reuters)
  • Infectious disease: Blocking malaria parasite invasion and egress (Nature)
  • Biocad set to begin selling biosimilars in North Africa (BioPharmaDive)

General Health

  • Families of dementia patients see positive effect of social robot seal (Reuters)
  • Baby Born With Cystic Fibrosis After IVF Screening Blunder (Forbes)
  • Brain cells develop more mutations as we age (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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