Recon: FDA Panel Backs Akcea-Ionis Rare Disease Drug

Posted 11 May 2018 | By Ana Mulero 

Recon: FDA Panel Backs Akcea-Ionis Rare Disease Drug

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

In Focus: US
  • Akcea, Ionis win over most FDA experts to their rare disease drug volanesorsen, vastly improving odds of success (Endpoints) (Fierce) (PharmaTimes) (PMLiVE)
  • AstraZeneca's Fasenra Fails in Trial of Severe Lung Disease (Bloomberg) (Reuters) (BioCentury)
  • Akari CEO Horn Solomon ousted after allegedly abusing the company credit card (Endpoints) (BioSpace)
  • Faron fingers low bioactivity as possible cause of phase 3 flop (Fierce) (The Pharma Letter-$)
  • Trump touts plan to lower drug costs but rejects Medicare negotiations (Politico) (NYT)
  • HHS Secretary, FDA Commissioner, CMS Administrator: Help is on the way for Americans facing high drug prices (Fox)
Paid Advertisement]

Currently, approximately 70% of IVDs on the market are sold without the involvement of a Notified Body. The IVDR will change that while also making sweeping changes to medical device regulations, all aimed at protecting end users.

Join us on May 16th at 11:00am EDT for our complimentary webinar Overview of the New European IVDR and find out how you can prepare your business for the transition.

Register now!

In Focus: International Pharmaceuticals & Biotechnology
  • La Jolla grabs $125M royalty deal to back drug launch, fund R&D (Endpoints)
  • Rest easy, pharma. Word is, Trump's drug pricing plan won't be a tough pill to swallow (Fierce)
  • Getting contraceptives for men to the market will take pharma’s help (STAT)
  • White House trumpeting drug plan as most far-reaching ‘of any president’ (STAT)
  • In taking on high drug prices, Trump faces a complex nemesis (AP News)
  • HHS Secretary Azar says everybody is 'wetting their beak ' on high drug list prices (CNBC)
  • Cramer vets biotech giants Biogen, Celgene, Gilead and Regeneron for positive prospects (CNBC)
  • Our new drug could drastically change hemophilia patients' lives, Biomarin CEO says (CNBC)
  • Trump plan for drug prices seen largely sparing industry (Reuters)
  • The 5 most expensive drugs in the United States (CNN)
  • Why VC Tim Draper Keeps Defending Theranos CEO Elizabeth Holmes (Fortune)
  • That $1.2 Million Payment to Trump's Lawyer Wasn't Even a Drop in the Bucket for Novartis (Fortune)
Pharmaceutical and Biotechnology: Study Results, Filings and Designations
  • Celldex plans life after glembatumumab, cutting 2 more drugs from pipeline (Fierce)
  • Puma shares plummet as results from key trial delayed (BioPharma Dive)
  • Will Real World Performance Replace RCTs As Healthcare's Most Important Standard? (Forbes)
  • CABANA: No Outcomes Benefit In First Big Trial Of AF Ablation (Forbes)
  • Ablation groups scramble to cope with Cabana (EP Vantage)
  • PharmaCyte completes pore size studies in Cell-in-a-Box capsules used in pancreatic cancer therapy (Pharmabiz)
  • PARP-1 mutations could drive PARP inhibitor resistance (BioCentury)
Medical Devices
  • This tiny chip uses A.I. to control blood sugar levels for critical diabetes patients (CNBC)
  • Instead of Giving Her a Prosthetic Ear, Army Doctors Grew a New One in This Soldier's Arm (Fortune)
  • FDA clears PeraHealth’s lifesaving clinical surveillance tech (pharmaphorum)
  • CARB-X backs Talis to develop point-of-care gonorrhea tests (Fierce)
  • Mayo Clinic and AliveCor use AI to detect ‘invisible’ heart condition (Fierce)
  • AI predicted a patient’s odds of dying with 90% accuracy (Axios)
  • BioElectronics Receives FDA Pre-Submission Approval for its Relief of Musculoskeletal Pain Market Clearance Application (Press)
US: Assorted & Government
  • FDA Publishes Long-Awaited Draft Guidance on Waivers, Exceptions, and Exemptions from the DSCSA (FDC Act Section 582) (FDA Law Blog)
  • Stop the Presses: FDA Publishes Policy for Granting Requests to Conduct Expedited Reviews of NDA and BLA CMC Prior Approval Supplements (FDA Law Blog)
  • Connecticut legislature passes pricing transparency bill (BioCentury)
  • Amazon is building a 'health & wellness’ team within Alexa as it aims to upend health care (CNBC)
  • How Humana And Walmart Can Overcome Organizational Autoimmune Disease (Forbes)
  • GOP senator calls for mandatory Medicaid work requirements (The Hill)
  • Nurse charged with neglect, manslaughter in death of former Trump adviser H.R. McMaster's father (ABC News)
  • Senators to Azar: Why aren't you fixing Medicare wage index? (Modern Healthcare)
  • Anthem PBM CEO Brian Griffin leaving after a few months in the role (Modern Healthcare)
  • Who should lead Veterans Affairs? Five veterans explain what they’re looking for in a leader. (The Washington Post)
  • June 12, 2018: Circulatory System Devices Panel of the Medical Devices Advisory Committee Meeting Announcement (FDA)
  • Medicare Program; Meeting of the Medicare Evidence Development and Coverage Advisory Committee—July 25, 2018 (CMS)
Upcoming Meetings & Events Europe
  • EMA’s PRIME driving innovation to where it is most needed (The Pharma Letter-$)
  • Janssen’s Tremfya backed for NHS use (PharmaTimes)
  • Updated: Good clinical practice inspection metrics (MHRA)
  • Marketing authorisations granted in April 2018 (MHRA)
  • Parallel import licenses granted in April 2018 (MHRA)
  • Brainomix Announces CE Mark Certification for New e-CTA Software (Press)
Asia India
  • Epygen looking to raise Rs.300 cr to propel biosimilars in India (Pharmabiz)
  • Can Modicare cure India's healthcare woes? (Financial Times)
  • Clinical trials by pharmaceutical companies still gasping for transparency (Economic Times)
  • Novartis India appoints Milan Paleja as vice chairman and MD (Economic Times)
  • FDA warning letter tells India's Goran Pharma to get professional help (Fierce)
  • Why Pharma Doesn't Always Keep Phase IV Promises In India (Scrip-$)
Australia
  • Fitness apps found to make almost no difference to users' health (The Guardian)
  • Australian scientists welcome boosts in new federal budget (Science Magazine)
  • NSW mental health units face overhaul (The Australian)
  • Philips, Emory Healthcare and Royal Perth Hospital in Australia partner to launch new remote intensive care monitoring program (Press)
Canada
  • Why Canada hasn’t issued a recall on romaine lettuce after an E. coli outbreak (Global News)
  • ReShape Lifesciences Announces Submission for Approval of ReShape Balloon  in Canada (Press)
General Health & Other Interesting Articles
  • To make medicines of the future, hospitals build their own cell factories (STAT-$)
  • Some opioid-hit areas left off rural response grants priority list (Modern Healthcare)
  • Medicare covers less than you might think. Here's how to avoid surprises (CNBC)
  • The Most Potent Prescription Ever: Take Time For Yourself, As Directed By Your Doctor (Forbes)
  • Hangover Pill Shows Promise in Lab Tests (Fortune)
  • The shrinking health spending gap (Axios)
Regulatory Reconnaissance is our daily intelligence briefing for the regulatory affairs space, bringing you the top 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? Email us at news@raps.org.

A story's inclusion in Regulatory Reconnaissance does not imply endorsement by Regulatory Focus or RAPS.

Categories: Recon

Regulatory Focus newsletters

All the biggest regulatory news and happenings.

Subscribe