Pink Sheet: 世界の薬事規制と承認審査に関するニュース

Zolgensma Data Integrity Fallout

20 Nov 2019

Novartis finds itself facing possible criminal or civil action for admitted data manipulation on its application for Zolgensma, a gene therapy used to treat children with spinal muscular atrophy (SMA) – manipulation disclosed only after FDA approval. Pink Sheet was the first to report on the drug’s loss of EU accelerated assessment, nearly 3 weeks before the pharma giant admitted to submitting falsified data to FDA. Follow the scandal and anticipate its implications on the industry with Pink Sheet’s coverage and timeline.

Topic cell-and-gene-therapy fda

Datamonitor Healthcare: 医療用医薬品の市場調査レポート

オンラインセミナー：NASH治療薬の展望と市場動向

05 Nov 2019

NASH（非アルコール性脂肪性肝炎）はさらに大きな流行病となりつつあり、あらゆる規模の企業が、業界初のNASH治療薬を市場に出すためにしのぎを削っています。新薬および転用された新薬探索、NASHの治療に関する詳細なディスカッション、およびこの分野のディールメイキング、ライセンスシング、提携戦略についてお聞きください。

Topic cell-and-gene-therapy drug-development-landscape drug-approval

Scrip: 世界の医療用医薬品＆製薬産業に関するニュース

Progress Being Made With Cell And Gene Therapy Market Access, But Challenges Remain

18 Oct 2019

Drug developers and payers are coming together long before product approvals to work out payment options for high-cost, high-impact cell and gene therapies, but challenges like portability of commercially insured patients and Medicaid best price requirements remain.

Topic cell-and-gene-therapy drug-development-landscape

Scrip: 世界の医療用医薬品＆製薬産業に関するニュース

Payers Must Brace For Coming Wave Of Cell And Gene Therapies

18 Oct 2019

One regenerative medicine for an ultra-rare disease may have a small impact, but payers will be flooded with dozens of new cell and gene therapies over the next few years. Enzyvant, Novartis and Spark share how they are positioning their complex products.

Topic Cell and gene therapy

Topic cell-and-gene-therapy

Scrip: 世界の医療用医薬品＆製薬産業に関するニュース

J.P. Morgan Notebook Day 1: Pfizer, Gilead, Alnylam, Novartis, Sarepta, Deal Trends And Cell Therapy Challenges

11 Jun 2019

News and notes from the 2019 J.P. Morgan Healthcare Conference. Pfizer CEO Bourla underscores need for scientific and commercial innovation, Gilead makes M&A a priority, Novartis' new R&D chief reflects on first meeting with CEO Narasimhan, Sarepta CEO Ingram clarifies regulatory path for micro-dystrophin, J.P. Morgan outlines deal and macro biopharma trends in 2019, and cell therapy panel discusses manufacturing challenges.

Topic BioPharmaceutical Cell and gene therapy

Datamonitor Healthcare: 医療用医薬品の市場調査レポート

Gene Therapy: A Paradigm Shift in Medicine

10 Apr 2019

The following analysis reviews several aspects of the gene therapy market. The history of gene therapy is briefly discussed, alongside a look at the trend in the volume of candidates in development over the past 20+ years, and how the regulatory landscape in the US and EU has recently adapted to help move the field forward. A deep dive into the pipeline with  breakdowns by delivery method and therapy area is provided, as are the key companies involved and the major partnership deals that have made gene therapy a hot commodity. Manufacturing, pricing, and reimbursement issues are also addressed.

Topic cell-and-gene-therapy

Scrip: 世界の医療用医薬品＆製薬産業に関するニュース

Bayer Bosses Beg Risk-Averse Europe To Rethink

By Kevin Grogan 07 Mar 2019

The German group believes it is going to be at the forefront of development in the cell and gene therapy space but is worried that Europe is failing to recognize the value that the pharma industry and its new technologies can create for society.

Topic cell-and-gene-therapy

Scrip: 世界の医療用医薬品＆製薬産業に関するニュース

Competitive Strategies Critical As EMA Decides On Kymriah And Yescarta

22 Aug 2018

With Novartis’s and Kite’s CAR T-cell therapies both expected to gain recommendations for pan-EU approval this week, discounts will need to be negotiated for these new treatments to reach the markets in Europe.

Topic cell-and-gene-therapy