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The US Zolgensma Review: Full Speed Ahead Despite Dose, Stability, Method Uncertainties

Normally, toddlers with infantile-onset spinal muscular atrophy would be in bed on ventilators. But most of these were sitting up on their own. Two were even standing and walking. The question was how to get the manufacturing process for the gene therapy that worked this magic ready for prime time.

 

Gene Therapy Quality Manufacturing

Cell/Gene Therapy Manufacturing Readiness Urged As A Condition For US Expedited Designation

Requiring that certain chemistry, manufacturing and controls conditions be satisfied before awarding breakthrough or regenerative medicine advanced therapy status would help ensure that the quality side of product development keeps pace with the clinical side, says Parexel's Mo Heidaran, a former CMC reviewer in the FDA’s biologics center.

Regenerative Medicine Gene Therapy Review Pathway

UK MHRA Starts Internal Review Over Stolen Drug Revelations

The UK MHRA has reacted to a television program on the sale in the UK of medicines stolen in Italy in 2014 by launching an internal review of actions taken at the time. It remains to be seen how effective the EU's new system for detecting falsified medicines will be in preventing the trafficking of stolen drugs. 
Distribution United Kingdom Italy

Sharpless Wonders Why Cancer Success Doesn't Translate To Other Diseases

Acting US FDA commissioner also said he wants to be nominated to the permanent position.

Cancer Research & Development Regulation

Postmarketing Commitments: Sponsors Often Do Not Publish Results Of New Clinical Trials

Only 15% of postmarketing commitments for drugs approved in 2009-2012 were for clinical trials and only half of these were published, study finds; authors call for US FDA to provide more trial information.

Post Market Regulation & Studies Clinical Trials FDA
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US FDA Performance Tracker

European Performance Tracker

Global Pharma Guidance Tracker – May 2019

Stay up to date on regulatory guidelines from around the world with the Pink Sheet's Guidance Tracker. The complete Global Pharma Guidance Tracker, with sortable and searchable listings going back to 2014, is available online.
International Europe Australia

NEW from CMIC: Tailoring Bioanalytical Testing In A New Era Of Drug Development

With unrelenting pressure to enhance the speed, efficiency and cost-effectiveness of drug discovery and development, companies need to be confident their Bioanalytical testing is not only tailored to these translational goals, but delivers added value in an environment in which scientific innovation, commercial imperatives and unmet patient needs are rapidly transforming the drug-development paradigm.

Read the Whitepaper

Embedding Time-Critical Scheduling in Drug Production

Manufacturing is the engine room of the pharmaceutical industry. Ultimately, a medicine has little value if it does not reach patients on schedule, in the required quantity and quality. Download this whitepaper which discusses time-critical scheduling in drug production.

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Policy & Regulation Explore this Topic

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Price Arbitration In Medicare: MedPAC Supportive But Congress Running Out Of Time

In its June report to Congress, the Medicare Payment Advisory Commission expands upon previous recommendations for establishing a binding arbitration process to control drug prices in Medicare.

Reimbursement Pricing Debate Medicare

Latest From Regulation

New Filings At The EMA

New medicines under evaluation at the European Medicines Agency.
Drug Review European Performance Tracker

US FDA Seeks Ways To Prevent Non-Responders From Sinking Tissue-Agnostic Indications

Agency officials and oncologists debate considerations where a data package overwhelms non-responders in favor of a tissue-agnostic indication.

Cancer Clinical Trials

Will US FDA Remove Higher Strength Opioids From The Market?

Advisory committee members are divided on whether agency should restrict access to higher dose opioids, but most want option kept on the table.

Advisory Committees FDA

Latest From Policy

Medicare Part B International Price Index Could Result In 25% R&D Cuts – Study

Reductions in research and development spending will be much greater than the 1% average often quoted by HHS, consultant tells the Biotechnology Innovation Organization annual convention.

Medicare Reimbursement

The Future Of US Biosimilars Litigation: Method-Of-Use Patents And ‘Dance’ Obligations

Expect plenty of litigation over what constitutes infringement of a method-of-use patent even when the protected indication is not found on a biosimilar’s labeling, a panel of legal experts said at the recent BIO meeting; another area of unsettled law is the extent to which a biosimilar sponsor has satisfied the information-exchange provisions of the BPCIA ‘patent dance.’

Biosimilars Intellectual Property

Antibiotic Incentives: Advocates Sounding The Alarm For DISARM

Antibiotic developers have a blunt message for Congress: the time for action is now or never. They see a targeted reimbursement change (The DISARM Act) as vital to send a signal to investors that the sector can be saved – and don’t want to delay to debate other ideas like “subscription” models.

Infectious Diseases Legislation

Clinical / R&D Explore this Topic

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Latest From Research & Development

Now It Really Is Personal: US FDA 'Rushing' To Develop Policy For 'Bespoke Therapies'

Recent cases of individual designed treatments have FDA playing a bit of catch up. The concept has been on the horizon, but now “bespoke therapies” are here.

Drug Approval Standards Gene Therapy

Roche Outlines Use Of Real-World Evidence In Entrectinib NDA

Roche used its Flatiron Health oncology EHR database to generate an external control arm for pivotal single-arm data supporting the personalized medicine candidate, which has an 18 August user fee goal.
ASCO Innovation

EMA Enlists GPs’ Help To Generate More Real-World Evidence

The generation of more real world evidence on medicines is one of the aims of a newly announced collaboration that will see family doctors playing a bigger role in providing data for the European Medicines Agency to use in its drug evaluation activities.

Europe Drug Review

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Latest From Commercial

BIO 2019 Notebook: Lartruvo Lookback, M&A Soars, BIO Vs. ASCO

News and views from day four of the BIO International Convention: Former FDA official Amy McKee said "everything worked right" with accelerated approval and removal of Lilly's Lartruvo; emerging company M&A deals nearly double in 2018; and the ASCO effect on the 3-6 June BIO meeting.

Drug Review Review Pathway

A New Drug Price Milestone

Much like Genzyme’s Ceredase did two decades ago, Novartis’ Zolgensma is setting a new pricing benchmark. The first million-dollar product is launching in the US and it will actually cost more than $2 million.

Pricing Debate Rare Diseases

BIO 2019 Notebook: Merck; RMAT; Out-Licensing Deals

News and views from day one of the BIO International Convention: Merck reaffirms commitment to antibiotic space the same day Zerbaxa picks up a new indication; US FDA regenerative medicine advanced therapy designations now outnumber breakthrough designations for cell/gene therapies; BIO survey finds out-licensing cash for emerging firms more than doubled in 2018.

Regenerative Medicine Financing

Manufacturing Explore this Topic

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Latest From Manufacturing

US FDA Pressing Industry To Use Standards For Cell and Gene Therapies

A US FDA official is exhorting the biopharmaceutical industry to adopt standards to help in developing new cell and gene therapy products, yet this may pose difficulties as there is a lack of standards in this space, particularly in areas such as cell viability, chain of identity and viral vector gene quantification.

Manufacturing Quality

USP Standards For Biological Drugs Would Restrict Innovation, US FDA Says

Top officials at the US FDA explain why the agency opposes USP monographs for biological products as a Senate committee proposes to disallow them and the USP launches an advertising campaign in their favor.

Biologics Manufacturing

US FDA Puts Compounding Office Under CDER Compliance To 'Institutionalize' Maturing Program

The US FDA plans to give its young drug compounding oversight program new leadership and move it from the drug center director's office down to the center's compliance office. Whether this move translates into heightened enforcement of drug compounders remains to be seen.

Manufacturing Quality
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