Regulatory Focus™ > News Articles > 9 > Recon: Alnylam Reports Positive Ph. III for Second Gene-Silencing Drug; GW Gets DEA Nod for Cannabis

Recon: Alnylam Reports Positive Ph. III for Second Gene-Silencing Drug; GW Gets DEA Nod for Cannabis-Based Epidiolex

Posted 27 September 2018 | By Michael Mezher 

Recon: Alnylam Reports Positive Ph. III for Second Gene-Silencing Drug; GW Gets DEA Nod for Cannabis-Based Epidiolex

Welcome to Regulatory Reconnaissance, your daily regulatory news and intelligence briefing.
In Focus: US
  • Early approval of Alnylam's gene-silencing drug in question (Reuters) (Forbes) (STAT) (Endpoints) (Press)
  • GW Pharma cannabis drug set to hit market following DEA nod (Reuters)
  • Congress may soon require drug, device companies to disclose payments to nurse practitioners and other clinicians (STAT)
  • J&J dumps its development deal on Geron’s imetelstat, shattering shares (Endpoints) (Xconomy)
  • Nasdaq triple IPO play brings in $345M as the big biotech party rocks on (Endpoints)
  • U.S.-Cuban biotech venture created to bring new cancer therapies to USA (Reuters) (Endpoints)
  • Merck asks SCOTUS for its $200M in Gilead patent dues, never mind the misconduct (Fierce)
  • Big Pharma's antibody drugs for asthma show benefits but cost 50% too much: ICER (Fierce)
  • The murky world of disclosures (Nature)
  • At least 80,000 people died of flu last winter in US, CDC says (NBC)
  • Aetna, seeking antitrust nod, sells Medicare drug business to WellCare (Reuters) (CNBC)
  • Defibrillator vest fails test because many heart attack survivors wouldn't wear it (Reuters)
  • On the Trail of a Deadly Disease That Cuts Down Farmworkers in Their Prime (Bloomberg)
  • HHS funding package en route to Trump's desk (Politico)
  • Hologic to buy Focal Therapeutics for $125m (MassDevice)
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In Focus: International
  • Countries pledge billions to fight tuberculosis worldwide (AP) (WHO) (WHO Director-General)
  • Michael Bloomberg Reappointed as WHO Global Ambassador for NCDs and Injuries (WHO)
  • Indivior cuts sales guidance sending shares down 15% (Financial Times)
  • Draft NICE decision rejects Respreeza for incurable, genetic disorder (PharmaTimes)
  • BIA and MHRA publish report ‘Collaborative Working in the UK, Driving Innovation Forward’ following their eighth annual joint conference (MHRA)
  • French 2019 Budget A Mix Of Good And Bad News For Pharma (Pink Sheet-$)
  • Norwegian-Danish Procurement Deal Targets Biogen’s Spinraza (Pink Sheet-$)
  • Scathing short attack from an obscure source accuses Nanjing Legend of manipulating CAR-T data — Genscript shares nosedive (Endpoints)
  • Novartis joins forces with GARDP for better, accessible infectious disease treatments in the developing world (Pharmafile)
  • Lilly’s snaps up Chugai’s experimental diabetes drug (PharmaTimes)
  • Call for genetic tests to bring personalised medicine to children with cancer (PMLive)
  • Rare Diseases: What Makes China A Rising Star? (Pink Sheet-$)
  • Fund gives J&J, Oxford experts $19 million to fight deadly viruses (Reuters) (Fierce)
  • China issues guidance for use of new cancer medicines (PharmaLetter-$)
  • Mandatory Drug Shortage Reporting In Australia Starts In January 2019 (Pink Sheet-$)
Pharmaceuticals & Biotechnology
  • Abolishing Drug Rebates May Push Consumer Drug Costs Higher (Forbes)
  • Epidiolex Almost Didn't Get Proprietary Name Because Of Confusion With OTC Pedia-Lax (Pink Sheet-$)
  • US FDA Gets Earful On Biologic ‘Umbrella’ Exclusivity, Patent Listings, Biosimilar Bridging Study Waivers (Pink Sheet-$)
  • FDA announces new and expanded compounding research projects (FDA)
  • Biotechnology Company Maps The Genome To Find The Dark Matter Of Infection (Forbes)
  • NIH to build a detailed map of cells within the human body (NIH)
  • AMAG exercises option on orphan drug candidate (PharmaLetter-$)
  • Gene Therapy Play Logicbio Proposes IPO (BioCentury)
  • Pharma execs talk challenges, opportunities of partnering with digital therapeutics (mobihealthnews)
  • F-Prime, Atlas lead cancer startup Akrevia to $30M A round (Fierce)
  • Antibody combo controls HIV for months (Fierce) (Reuters)
  • Pharma advertising in 2018: TV, midterms and specialty drugs (BioPharmaDive)
  • Eli Lilly picks up an oral GLP-1 diabetes drug from a big Roche sub for $50M in cash-plus deal — and it’s not a peptide (Endpoints)
  • Vivus, diving into the cystic fibrosis sphere, backs stadium step-climbing fundraiser (Fierce)
  • Atlas, F-Prime provide launch money for Akrevia — a little biotech with big plans to design better drugs (Endpoints)
  • Goldacre Clashes With Pharma Over Trial Data Transparency (Pink Sheet-$)
  • Fee for Using a Rare Pediatric Disease Priority Review Voucher in Fiscal Year 2019 (Federal Register)
Pharmaceuticals & Biotechnology: Study Results, Filings & Designations
  • FDA accepts Allergan’s Vraylar for review (PharmaTimes)
  • CytoSen Therapeutics Receives FDA’s Favorable Response to Pre-IND Meeting Package for Phase 2 Study in AML (Press)
  • Cidara Therapeutics Initiates Phase 3 Trial of Lead Antifungal Rezafungin for Treatment of Invasive Candida Infections (Press)
  • ImmusanT Announces First Patient Dosed in Phase 2 Trial of Therapeutic Vaccine for Celiac Disease (Press)
  • Hansa Medical Reports Positive Final Top Line Results from Two Phase 2 Studies of Imlifidase for Kidney Transplantation (Press)
  • Decibel Therapeutics and Oricula Therapeutics Announce Exclusive License for ORC-13661 in Phase 1 Development for Hearing and Balance Protection (Press)
  • Targovax Announces Interim Results From Phase I Trial of ONCOS-102 in Checkpoint Inhibitor Refractory Melanoma (Press)
Medical Devices
  • FDA clears Baxter’s Altapore bone graft for spinal indications (MassDevice)
  • Device Cybersecurity: For Better — and Worse? The Very Connected World of Medical Devices (MDDI)
  • 2/3s of commercially available blood glucometers don't meet FDA accuracy standards, study finds (mobihealthnews)
  • Effects of “No Deal” Brexit for Medical Device Sector (Emergo)
US: Assorted & Government
  • Dozens of Doctors Who Screen Immigrants Have Record of ‘Egregious Infractions,’ Report Says (NYTimes)
  • VA Adding Opioid Antidote To Defibrillator Cabinets For Quicker Overdose Response (NPR)
  • Federal Courts Diverge On Bristol-Myers And Class Actions (Law360-$)
  • Expert's Analyses Questioned In AbbVie Merger Cert. Row (Law360-$)
  • Allergan Medicaid Rebate Suit Redo Denied At 3rd Circ. (Law360-$)
  • McKesson Is Roped Into Generic-Drug Price-Fixing Row (Law360-$)
  • J&J Fights $245M Faulty Hip Implant Judgment (Law360-$)
  • 3rd Circ. Won't Revive Chinese Co.'s GSK Probe Suit (Law360-$)
  • Plaintiffs’ Lead Counsel Off The Hook In Yaz MDL (Drug & Device Law)
  • FDA Adopts New Policy on Release of Retailer Lists During Human and Animal Food Recalls (FDA Law Blog)
  • GAO clears HHS of wrongdoing in ACA social media dispute (Modern Healthcare)
  • New York regulator warns CVS-Aetna merger could raise prices, reduce consumer choice (HealthcareDive)
Upcoming Meetings & Events Europe Canada
  • Health Canada approves subcutaneous treatment for breast cancer (BioPharmaReporter)
  • Avita Medical lands tax credit Down Under (MassDevice)
Other International
  • Amazon basin DNA project aims to bolster indigenous people (Financial Times)
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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