Regulatory Focus™ > News Articles > 4 > Recon: Bluebird's gene therapy eliminates blood transfusions for some beta-thalassemia patients

Recon: Bluebird's gene therapy eliminates blood transfusions for some beta-thalassemia patients

Posted 19 April 2018 | By Ana Mulero 

Recon: Bluebird's gene therapy eliminates blood transfusions for some beta-thalassemia patients

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

In Focus: US In Focus: International Pharmaceuticals & Biotechnology
  • Cancer and heart drugs boost Novartis sales (Financial Times)
  • Gates backs gene technologies in fight to end malaria (Endpoints) (The Guardian)
  • Amid tough respiratory competition, AstraZeneca inks Symbicort outcomes-based deals (Fierce)
  • Cosentyx jitters weigh on Novartis (BioPharma Dive)
  • GSK brings on Roche exec to lead pipeline growth (BioPharma Dive)
  • Eye Drop Suit Must Stay Tossed, Pharma Cos. Tell 1st Circ. (Law360-$)
Pharmaceutical and Biotechnology: Study Results, Filings and Designations
  • In knockout IPO offerings, MorphoSys and Surface Oncology haul combined $316M in upsized deals (Endpoints)
  • CNS Pharma turns to crowdfunding to advance glioblastoma drug (Fierce)
  • Argos abandons kidney cancer vaccine after phase 3 fail (Fierce)
  • 'Cell-free' CRISPR could improve cancer diagnostics, unravel how gene editing works (Fierce)
  • Three Steps Toward a More Sustainable Path for Targeted Cancer Drugs (JAMA)
  • GW’s Epidiolex takes a leap toward US approval (The Pharma Letter)
  • FDA clears first therapy for inherited rickets (The Pharma Letter)
  • FDA seeks info on DMD drug as hold remains (BioPharma Dive)
  • Landmark IMPACT study published in NEJM shows significant benefits of Trelegy Ellipta for patients with COPD (Press)
  • Biohaven Pharmaceuticals Announces Additional Phase 3 Rimegepant Data to be Released at Investor Event Held Concurrently with the 2018 American Academy of Neurology Meeting in Los Angeles (Press)
  • Additional Clinical Studies Supporting the Benefits of OMIDRIA Presented at the American Society of Cataract and Refractive Surgery and American Society of Ophthalmic Administrators Annual Meeting (Press)
  • Adamas Announces Final Results from the Two-Year Phase 3 Open-Label Study of GOCOVRI in Parkinson’s Disease Patients with Dyskinesia (Press)
  • Tocagen and ApolloBio Enter License Agreement to Develop and Commercialize Toca 511 & Toca FC in the Greater China Region (Press)
Medical Devices
  • Artificial intelligence will put a premium on physicians’ knowledge and decision-making skills (STAT)
  • FDA warns against endoscope connectors (MassDevice) (FDA)
  • Emboline raises $10m Series B for TAVR embolism filter (MassDevice) (Press)
  • Philips inks collab software integration deal with Sun Nuclear (MassDevice) (Press)
  • (Don’t) Take the Long Way Home: FDA Issues Draft Guidance on Expansion of the Abbreviated 510(k) Program (Arnall Golden Gregory LLP)
  • SpeeDx receives FDA clearance for Mycoplasma genitalium product (Press)
US: Assorted & Government
  • U.S. appeals court says Ohio cannot block Planned Parenthood funds (Reuters)
  • How doctors in the US prescribe opioids — four charts (CNBC)
  • NIH abruptly changes course on industry opioids partnership after ethics flags raised (STAT)
  • The FTC Appeals Shire ViroPharma Dismissal (FDA Law Blog)
  • EEOC Sues Another Health Care Provider For Alleged Pregnancy Discrimination (Lexology)
  • For Now, Hospitals Hang On Despite CVS, Optum And Walmart Threats (Forbes)
  • Does The U.S. Biosimilar Space Have A Case Of "Progressophobia?" (Biosimilar Development)
  • Federal Policy for the Protection of Human Subjects: Proposed Six Month Delay of the General Compliance Date While Allowing the Use of Three Burden-Reducing Provisions During the Delay Period (Federal Register)
Upcoming Meetings & Events Europe
  • MHRA Guidance: E-cigarettes: regulations for consumer products (MHRA)
  • New verification system will secure patient safety and support continuation of medicines supply (ABPI)
  • TAV, scientific advice and Brexit in the focus of the meeting between PEI and the BPI (Paul-Ehrlich-Institut)
  • Leading-edge healthcare: Industrial Strategy Challenge Fund (MHRA)
  • Submission of comments on 'Draft qualification opinion on Proactive in chronic obstructive pulmonary disease (COPD) (EMA)
  • Final NHS nod for Roche’s RoActemra (PharmaTimes)
  • MPs slate rate of progress on NHS cyber security (PharmaTimes)
  • Revisions of precautions: Omariglipin, etc. posted (PMDA)
  • China-Denmark Drug Regulatory Thematic Seminar held in Beijing (CFDA)
  • Chugai debuts Tecentriq in Japan for NSCLC (The Pharma Letter)
  • Tecentriq Now in PD-1/L1 NSCLC Showdown, Lynparza Also Hits Japan Market (Pharma Japan)
  • Biosimilar Use Essential to Sustain Universal Health Coverage: Society President (Pharma Japan)
  • This app can help determine the risk of diabetes for people in rural India (Mashable)
  • The Political 'Environment' of India's Mental Health (The Wire)
  • Health ministry to name hospitals which do not perform under Kayakalp (Times of India)
  • Foreign direct investment in Indian pharma sector causing concern (The Pharma Letter)
  • Pharma funds may not outperform diversified equity funds any time soon: Sonam Udasi of Tata MF (Economic Times)
  • Indian Pharma Inc shifts to high gear in filing patents (Pharmabiz)
  • Fluad trivalent influenza vaccine: Safety advisory - risk to patients with latex allergy (TGA)
  • Lives affected by motor neurone disease are changed by Australian medical technology (ABC News)
  • Replacement for outgoing SA Health chief announced (ABC News)
  • Final Notice: Classification of Dental Plaque-Disclosing Products as Drugs (Health Canada)
  • Notice - Update on Health Canada’s efforts to support innovative human therapeutic products to combat antimicrobial resistance (AMR) and consultation on a proposed Pathogens of Interest List (Health Canada)
General Health & Other Interesting Articles
  • Alzheimer’s disease starts in childhood, with symptoms found in babies less than a year old, study shows (Newsweek)
  • The Menopause-Alzheimer’s Connection (NYT)
  • Mirror Proteins Come Through (Science Translation Medicine)
  • More E. Coli Infections Tied to Romaine Lettuce Have Been Reported, CDC Says (Fortune)
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

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.