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Recon: BioMarin Eyes $2-3M Price for Hemophilia Gene Therapy as Other Gene Therapies Test Europe’s Willingness to Cover Them

Posted 16 January 2020 | By Michael Mezher 

Recon: BioMarin Eyes $2-3M Price for Hemophilia Gene Therapy as Other Gene Therapies Test Europe’s Willingness to Cover Them

Welcome to Regulatory Reconnaissance, your daily regulatory news and intelligence briefing.
In Focus: US
  • BioMarin Explores Pricing Experimental Gene Therapy at $2 Million to $3 Million (WSJ)
  • Neon Therapeutics makes one last retreat, selling itself cheap in a bargain basement M&A deal (Endpoints) (BioPharmaDive)
  • Hedge fund veteran looks to cash in on biotech boom (Financial Times)
  • Pharma execs pitch ideas at JPM to lower drug costs. None include dropping their own prices (CNBC)
  • A court struck down a ban on pharma ‘price gouging.’ One state wants to revive it — and others could follow (STAT)
  • Verily tangos at JPM with a health tech partner — and steps on a few toes (STAT)
  • FDA panel says ’no’ to another new opioid (Politico) (Press)
  • The kill-switch for CRISPR that could make gene-editing safer (Nature)
In Focus: International
  • Gene therapies test Europe’s willingness to pay (Financial Times)
  • Biopharma's China plan gets a boost from patent protections sealed in now official trade deal (Endpoints) (Pharmafile) (Law360-$)
  • Three African nations roll out first malaria vaccine in closely watched trial (STAT)
  • Celltrion to build plant in China (Yonhap)
  • NICE ‘OK’ for Lynparza (PharmaTimes)
  • HIV infections among gay and bi men fall by 71% in UK (BBC)
  • Japan confirms first case of infection with new China coronavirus (Reuters) (Japan Times)
  • Comparator Report on Cancer in Europe 2019 (EFPIA)
  • Policy Proposals to Minimize Medicine Supply Shortages in Europe (EFPIA)
  • Upward trend in market entry of high-cost drugs for rare diseases shows no sign of abating (Health Canada)
Pharmaceuticals & Biotechnology
  • GSK, Pfizer contradict on the future of consumer health JV — report (Endpoints)
  • New AMR Report Is ‘A Wake-Up Call’, Says Industry Body (Pink Sheet-$)
  • Richard Pazdur: 20th Anniversary Edition (Pink Sheet-$)
  • US generic drug supply chain continues to shrink (Pharmacompass)
  • Lilly ends $1.4bn deal with immuno-oncology biotech NextCure (PMLive)
  • How Moderna is using Amazon cloud to produce personalized cancer vaccines (CNBC)
  • Analysts tip Biogen to file for aducanumab approval within weeks (Fierce)
  • Astellas Completes Acquisition of Audentes Therapeutics (Press)
  • An Overview of FDA OPDP Enforcement for 2019 (Eye on FDA)
  • Kite Pharma adds year before commercial production begins at Urbana site (Frederick News-Post)
  • Biosimilar and Biologic Business Developments Update – Collaborations and Licensing Deals (Big Molecule Watch)
  • Express Scripts Could Negotiate Drug Rebates For 100m Lives With Prime Therapeutics Deal (Scrip-$)
  • JPM20: Orchard isn't for the picking, Biohaven's Allergan headache, and who's on Vascepa? (BioPharmaDive)
  • JPM: A Humira-beating $20B in sales from Skyrizi and Rinvoq? It's possible, AbbVie CEO says (Fierce)
  • Novartis' David Lennon on next steps for AveXis, selling Zolgensma's price to the public (BioPharmaDive)
  • #JPM20: Five years after first PCSK9, Novartis goes for win as Sanofi moves on, Amgen salvages what's left (Endpoints)
  • Frazier closes 10th fund in 30 years, with $617M to bet on cell/gene therapy, Big Pharma spinoffs and more (Endpoints)
  • #JPM20 exclusive: Olivier Brandicourt follows the Big Pharma CEO path to private equity, joining Blackstone ahead of a mammoth fund debut (Endpoints)
  • #JPM20: After Year of NASH collapses, all eyes on two biotechs (Endpoints)
  • US FDA's Decade In Review: From Biosimilars To Real-World Evidence (Pink Sheet-$)
  • Enhancing the pharmaceutical supply chain with big data (Pharmaceutical Manufacturing)
Pharmaceutical & Biotechnology: Study Results, Filings & Designations
  • Neuroptika Announces Enrollment Of First Patient In Phase 2 Clinical Trial For Dry Eye Disease (Press)
  • First Patient Dosed with Cellectis’ New Allogeneic UCART123 Product Candidate for Relapsed/Refractory Acute Myeloid Leukemia (Press)
Medical Devices
  • Health IT to lead 2020 deals, KPMG predicts (MedtechDive)
  • Foundation Medicine touts concordance ahead of 1H FDA ruling on liquid biopsy test (MedCityNews)
  • ResMed pays $37.5M to settle false claims allegations (MedtechDive)
  • REVA files for bankruptcy after bioresorbable scaffold flops (MedtechDive)
  • Request for Nominations for Voting Members on a Public Advisory Committee; Technical Electronic Product Radiation Safety Standards Committee (FDA)
US: Assorted & Government
  • How Anti-Vaccine Activists Doomed a Bill in New Jersey (NYTimes)
  • FDA Says That Theranos Discovery Strain Is Causing Other FDA Enforcement Efforts to Take a Backseat (FDA Law Blog)
  • Genentech, Inc. v. Hospira, Inc. (Fed. Cir. 2020) (Patent Docs)
  • Another Good Pradaxa Preemption Ruling—This One In California (Drug & Device Law)
  • Gov't Initiatives May Boost Health Coverage For Genetic Tests (Law360-$)
  • GSK Seeks To Forgo Trial In First Zofran MDL Bellwether (Law360-$)
  • Bayer Says 27K Essure Suits Can't Head 'Full-Steam' To Trial (Law360-$)
  • UnitedHealth bets on government health plan growth in 2020 (Reuters)
Upcoming Meetings & Events Europe
  • Germany joins the 1+Million Genomes Initiative (EC)
  • EMA welcomes new Head of Information Management Division (EMA)
  • LC and GC columns in monographs (Council of Europe)
  • Breast cancer patients to have further NICE-approved drug combination option on Cancer Drugs Fund (NICE)
  • Janssen submits Spravato expansion in EU (PharmaTimes)
  • Convex two-piece skin barriers (Natura /Surfit/Combihesive Wafers) for use with ostomy bags – recall due to risk of stoma injury, bleeding and leakage under the skin barrier (MDA/2020/002) (MHRA)
  • Aiming for an IPO, Antengene wants to be China’s Celgene (PharmaLetter-$)
  • Wockhardt gets DGCI approval for 2 new antibiotics (Economic Times)
  • PMP set to promote domestic production of medical devices (Economic Times)
  • Strides gets USFDA nod for anti-allergic drug (Economic Times)
  • A Decade’s Deals In Indian Pharma Point To 2020 Trends (Scrip-$)
  • Gujarat leads in collection and analysis of drug samples with 13,616 samples tested last year (Pharmabiz)
  • Health Products Regulation Group: Regulatory Science Strategy, 2020-2025 (TGA)
  • TGA presentations: The 2nd Industry Forum on Good Manufacturing Practice (GMP), 21 November 2019 (TGA)
Other International
  • Hanan Sboul takes over the position of IGBA Chair (IGBA)
  • Public-Private Collaboration Commits to Accelerate Access to Health Services in Africa, Reaching 1.7 Million People (Press)
General Health & Other Interesting Articles
  • Blood Pressure Patterns Are Different for Women (NYTimes)
Regulatory Reconnaissance is our daily intelligence briefing for the regulatory affairs space, bringing you the top regulatory, biopharma and medtech news stories from around the globe.
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