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Featured Stories



FTC Merger Guidelines Cast ‘Perilous Shadow’ Over Biopharma

BIO objects to guidelines seeking to block mergers that would eliminate a potential entrant in a concentrated market and give greater scrutiny to transactions that are part of a series of company deals. APhA and NCPA call for FTC to consider more factors in PBM mergers.

M & A Enforcement Policy

New Pink Sheet PRIME Tracker Reveals The Companies With The Most Entries On EMA Scheme

Securing a place on the European Medicines Agency’s priority medicines scheme is not easy, but some companies have managed to make the grade more than once.

Europe Regulation Research & Development

EU Bodies Agree On Ways To Future-Proof EMA Funding System

EU ministers say that implementing an appropriate fee structure that is more in line with actual costs will promote innovation in the pharmaceutical sector while ensuring “fair access” to safe and effective medicines for patients.

Europe User Fees Regulation

Lantidra Drug Review Profile

CellTrans’ Lantidra: Type 1 Diabetes Cell Therapy Overcame The Odds On Nontraditional Path To Approval

Despite a host of challenges with donislecel’s development and BLA, the US FDA approved based on clinical data from only 30 patients, using a clinically meaningful insulin independence endpoint and in a more carefully tailored population. Pink Sheet’s Drug Review Profile takes a deep dive into the FDA review of the first approved allogenic pancreas islet cell product.

Drug Review Profile Regenerative Medicine

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Pink Sheet Podcast

Keep up with latest biopharma regulatory and policy developments with Pink Sheet podcasts.

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Performance Trackers

Keeping Track: Return & Renewal At US FDA

Takeda’s TAK-721 is back, ARS Pharma will be back soon to appeal neffy complete response letter, US FDA’s Project Renewal brings Temodar labeling in current day, and more highlights from the Pink Sheet’s US FDA Performance Tracker.

US FDA Performance Tracker Drug Review Drug Approval Standards

Global Pharma Guidance Tracker – August 2023

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.

Guidance Documents Regulation International


Multimedia

NurOwn Adcomm Is 20 Members Strong Despite US FDA’s ‘Struggle’ To Find Unconflicted Neurology Experts

Seven panelists are regular members of the Cellular, Tissue and Gene Therapies advisory committee, while many of the 13 temporary voting members bring neurology and neuroscience expertise. In a conflict-of-interest waiver, the agency noted a struggle to find experts in clinical neurology and neurodegenerative diseases who lack disqualifying financial interests.

Gene And Cell Therapy Clinical Holds Decline While Those For Drugs Hit 12-Year High

FDA’s Center for Biologics Evaluation and Research saw IND clinical holds for gene and cell therapies dropped to 70 in calendar year 2022, a 52% decline from 2018. Center for Drug Evaluation and Research’s total clinical holds in fiscal year 2022 were 380, the highest number in the past 12 years.

Lantidra Chronology: 19 Years From IND Submission To Approval For The Cellular Therapy

Pink Sheet’s Drug Review Profile looks at the timeline behind the development and FDA review of CellTrans’ treatment for type 1 diabetes.

Lantidra’s First-Cycle Complete Response Letter Centered On Inspection And Product Quality Issues

Although the CRL delayed the cell therapy’s approval by almost two years, additional CMC-related testing requested by the FDA ultimately came to be seen as a good thing by sponsor CellTrans because it provided assurance of product consistency.

Regional Comparisons

Gene Therapy HTA: How Do The European, Australian And Canadian Systems Shape Up?

A report comparing the health technology assessment methods used in nine European countries, Australia and Canada found that England has the most favorable reimbursement landscape for gene therapies – but outlined several areas for improvement across the board.

Swiss New Drug Approval Times On A Par With EMA, But Longer Than US FDA

The median approval time for new drugs in Switzerland lengthened in 2022, mainly because of applications filed during the peak of the COVID-19 pandemic, says the country’s regulator.

How Do Japan’s Clinical Trial eConsent Rules Compare With The EU And US?

A newly-translated guidance document from Japan’s drug and device regulator outlines the points that clinical trial sponsors should consider when using electronic methods to collect informed consent from participants.

Rare Diseases: European Market Access ‘Better Organized’ Than US, Says Rhythm

Rhythm is working with country-level authorities and centers of excellence in Europe to gain reimbursement and access for its obesity treatment Imcivree.

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Recent Stories

Republican Lawmaker Takes Unusual Step Of Attacking FDA Salaries Over Abortion Pill Decisions

A move to cut senior FDA positions including CDER Director through the appropriations process is rare and almost certain to fail, but experts say the attempt is indicative of an environment that seems to be encouraging these personal amendments.

NurOwn Adcomm Is 20 Members Strong Despite US FDA’s ‘Struggle’ To Find Unconflicted Neurology Experts

Seven panelists are regular members of the Cellular, Tissue and Gene Therapies advisory committee, while many of the 13 temporary voting members bring neurology and neuroscience expertise. In a conflict-of-interest waiver, the agency noted a struggle to find experts in clinical neurology and neurodegenerative diseases who lack disqualifying financial interests.

Belgium Aims To Drive Biologic Competition Through Tendering

New tendering rules in Belgium aim to improve competition between biosimilars and originators, for example by setting contract durations for tendering contracts and limiting the criteria for such contracts.

UK Phases In New Standards On Information Materials For Potential Study Participants

The Health Research Authority has developed UK-wide Quality Standards and Design and Review Principles to improve the information given to people who are invited to take part in research. Their use will become mandatory in December.

BrainStorm’s NurOwn: Cell Therapy Lacks Substantial Evidence Of Efficacy In ALS, US FDA Says

Sponsor’s assertion of a ‘floor effect’ cannot explain the lack of difference versus placebo on clinical endpoints, and there is no evidence that assessed biomarkers are reasonably likely to predict clinical benefit, agency says. Although advisory committee will focus primarily on efficacy, FDA also cites concerns about ‘grossly deficient’ product quality information.

Gene And Cell Therapy Clinical Holds Decline While Those For Drugs Hit 12-Year High

FDA’s Center for Biologics Evaluation and Research saw IND clinical holds for gene and cell therapies dropped to 70 in calendar year 2022, a 52% decline from 2018. Center for Drug Evaluation and Research’s total clinical holds in fiscal year 2022 were 380, the highest number in the past 12 years.

Takeda Pens Mid-Trial Settlement To Dismiss US Colcrys Antitrust Suit

Japanese originator Takeda has agreed to settle two-year-old anticompetitive claims lodged by wholesalers in the US that it conspired with several generics manufacturers to maintain supracompetitive pricing for its Colcrys branded treatment for gout.

Yet Another Bad Day For PBMs On Capitol Hill

Democrats and Republicans in the US Congress are not getting along on most topics these days. But when it comes to criticizing the pharmacy benefit management sector, they see eye-to-eye.

Products Targeting Alzheimer's, Duchenne & COVID-19 Among New EU Filings

EU marketing authorization applications have been submitted for a number of new drugs, including Eli Lilly’s donanemab, Italfarmaco's givinostat and Arcturus Therapeutics/CSL’s ARCT-154.

Gene Therapy HTA: How Do The European, Australian And Canadian Systems Shape Up?

A report comparing the health technology assessment methods used in nine European countries, Australia and Canada found that England has the most favorable reimbursement landscape for gene therapies – but outlined several areas for improvement across the board.

Coronavirus Notebook: UN Pandemic Declaration Disappoints, Focus Turns To Draft WHO Treaty

The World Health Organization has published a “step-by-step guide” for countries wishing to increase their capacity for monitoring pathogens with pandemic and epidemic potential. The EU and the UK have cleared an updated version of Moderna’s COVID-19 vaccine Spikevax targeting the XBB.1.5 variant, while the EU has followed the US in approving Gilead’s Veklury as the first treatment for COVID-19 patients with mild-to-severe liver damage.

Finding The Sweet Spot For Real-World Evidence: Aetion Stresses Early Feasibility Assessment

Robust natural history data sets with well-chosen outcomes are fundamental in Aetion’s regulatory feasibility tool for real-world evidence, built through analysis of US FDA case studies and guidance.

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