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Regulatory Focus™ > News Articles > 2019 > 12 > Recon: Sarepta Wins Early Approval for DMD Drug After Earlier Rejection; PhRMA Sues Over Oregon Pric

Recon: Sarepta Wins Early Approval for DMD Drug After Earlier Rejection; PhRMA Sues Over Oregon Price Transparency Laws

Posted 13 December 2019 | By Michael Mezher 

Recon: Sarepta Wins Early Approval for DMD Drug After Earlier Rejection; PhRMA Sues Over Oregon Price Transparency Laws

Welcome to Regulatory Reconnaissance, your daily regulatory news and intelligence briefing.
In Focus: US
  • FDA gives early approval to Sarepta's DMD therapy  (Reuters) (STAT) (Endpoints) (Endpoints) (FDA)
  • What's next for Sarepta? A third DMD approval, analysts predict (Endpoints)
  • Biotech veteran Chris Garabedian on Sarepta’s legacy and Biogen’s future at the FDA (STAT)
  • Biogen scraps development of therapy for rare brain disease (Reuters)
  • Hahn Wins Senate Confirmation to be 24th FDA Commissioner (Focus) (NYTimes)
  • House Passes Pelosi Drug Pricing Bill on Party-Line Vote (Focus) (NYTimes)
  • Drug prices and health care are wild cards in the 2020 election (CNBC)
  • PhRMA sues Oregon over drug pricing transparency laws, calling them ‘unconstitutional’ (STAT)
  • Looming Medical Device Tax Has Makers Rushing to Capitol Hill (Bloomberg)
In Focus: International
  • GSK's ViiV seeks marketing license for baby-friendly HIV pill (Reuters)
  • Ebola spreads in Congo after militants attack treatment camp: WHO (Reuters)
  • Brexit nears as Conservatives sweep to victory in election (PMLive) (BBC)
  • Pfizer's rare disease drug gets EMA's positive recommendation (Reuters) (Press)
  • Novartis' Beovu scores CHMP recommendation after proving non-inferior to Eylea in wet age-related macular degeneration (Pharmafile)
  • Roche's Tecentriq cocktail scores trial success in melanoma (Reuters) (Press)
  • Scientists home in on potential treatments for deadly Nipah virus (Reuters)
Pharmaceuticals & Biotechnology
  • When FDA Requires Pediatric Studies for New Cancer Drug Submissions: New Draft Guidance (Focus)
  • A new HIV vaccine effort with a different kind of strategy (MPR)
  • Our 2019 Year In Review: Macro, Biotech, and Atlas (LifeSciVC)
  • Bristol-Myers' strategy, BD chief Paul Biondi exited the company — just ahead of the $74B Celgene deal close (Endpoints) (Fierce) (Press)
  • Paul Biondi's track record at Bristol-Myers covered billions in deals of every shape and size. Here's the complete breakdown (Endpoints)
  • Biopharma has abandoned antibiotic development. Here’s why we did, too. (Endpoints)
  • UK researchers make new Alzheimer’s disease discovery (PharmaTimes)
  • Therapies for rare diseases: therapeutic modalities, progress and challenges ahead (Nature)
  • Oncology drug shortages in the USA — business as usual (Nature)
  • FDA and Industry Quality Metrics Initiatives Are Paving the Way to Manufacturing and Regulatory Advancements (IPQ)
  • Gritstone posts 'one of the most potent immunogenic-responses for a neoantigen vaccine' (Fierce)
  • Anixa delays CAR-T trial in bid to boost efficacy (Fierce)
  • Intercept Provides Regulatory Update (Press)
Pharmaceutical & Biotechnology: Study Results, Filings & Designations
  • FDA Approves XELJANZ® XR (tofacitinib) Extended-Release Tablets for the Treatment of Ulcerative Colitis (Press)
  • OncoSec Presents Interim Data of 28.5% Objective Response Rate (ORR) from Ongoing KEYNOTE-890 Study Evaluating TAVO™ in Combination with KEYTRUDA® for Heavily Pretreated, Late-Stage,
  • Metastatic Triple Negative Breast Cancer (mTNBC) at the 2019 San Antonio Breast Cancer Symposium (Press)
  • Boehringer Ingelheim and Lilly provide update on Jardiance® phase III exercise ability studies in chronic heart failure (Press)
  • Hansa Biopharma has Agreed With the FDA on a Regulatory Path Forward for Imlifidase in Kidney Transplantation of Highly Sensitized Patients in the US (Press)
  • Exelixis Announces Positive Results From IMspire150, the Phase 3 Trial of Atezolizumab Plus Cotellic and Vemurafenib in People With Previously Untreated BRAF V600 Mutation-Positive Advanced Melanoma (Press)
  • Significant Effect of Diamyd® in Type 1 Diabetes Shown in a New Comprehensive Analysis of Previous Phase III and Phase II Trials (Press)
  • BioLineRx Announces Updated Phase 2a Data From Triple Combination Arm of COMBAT/KEYNOTE-202 Study in Patients With Second-line Metastatic Pancreatic Cancer (Press)
  • Senhwa Biosciences Reports Positive Phase 1 Data of CX-5461 in Patients With Advanced Solid Tumors at 2019 SABCS (Press)
  • Transgene Reports on the Combination Trial of TG4010, Chemotherapy and Nivolumab in Non-Small Cell Lung Cancer (Press)
 Medical Devices
  • FDA qualifies tool for assessing safety of implanted devices (MedtechDive)
  • FDA authorizes first interoperable, automated insulin dosing controller designed to allow more choices for patients looking to customize their individual diabetes management device system (FDA)
  • Iradimed renews CE mark for MRI-safe patient monitor (MassDevice)
  • Livongo, Propeller, Omada tapped for Express Scripts digital health formulary (MedtechDive)
  • Class 1 Device Recall Medfusion Model 4000 Pump (FDA)
US: Assorted & Government
  • The ceasefire at the Humphrey Building (Politico)
  • Investor Wants To Block $9.7B Novartis-Medicines Co. Deal (Law360-$)
  • US Patent Office removes lawyer from high-profile case involving a Gilead HIV medicine (STAT) (Endpoints)
  • Federal Watchdog Questions Billions of Dollars Paid to Private Medicare Plans (NYTimes)
  • South Carolina Is the 10th State to Impose Medicaid Work Requirements (NYTimes)
  • U.S. vaping-related deaths rise to 52, hospitalizations to 2,409 (Reuters)
  • District of Nevada Dismisses Gadolinium Claim, Albeit Without Prejudice, on Personal Jurisdiction and Preemption Grounds (Drug & Device Law)
  • Berkeley Files Responsive Motion to Broad's Substantive Motion No. 2 in Interference (Patent Docs)
Upcoming Meetings & Events Europe
  • EU Preserves Status Quo On Drugs Under Additional Monitoring (Pink Sheet-$)
  • EU Regulatory Roundup: Finland Prepares for 2020 Introduction of New Clinical Trial Regulations (Focus)
  • CR UK research backs liquid biopsy for breast cancer (PharmaTimes)
India Australia
  • Advertising: Advertising Code and guidance (TGA)
  • IVD companion diagnostics: Guidance on proposed regulatory requirements (TGA)
  • TGA webinar presentation: Good Manufacturing Practice (GMP) clearance applications - common pitfalls, 6 December 2019 (TGA)
Other International
  • Costa Rica's president says therapeutic abortions will be allowed (Reuters)
  • New Zealand Seeks Human Skin to Treat Volcano Burn Victims (NYTimes)
General Health & Other Interesting Articles
  • How Prepared Are We For The Next Pandemic? Not Very, Experts Show (Forbes)
  • Flu Season Is Here, and It’s Targeting Your Kids (Bloomebrg)
  • How McKinsey infiltrated the world of global public health (Vox)
  • BMJ should retract flawed research paper on chronic fatigue syndrome (STAT)
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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