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Preparing For A Shutdown: US FDA To Retain 81% Of Workforce, Thanks Mostly To User Fees
However, user fee carryover balances will not last forever and service cuts are inevitable if a shutdown lingers for several weeks.

BrainStorm’s NurOwn Gets Many, Many Thumbs Down From US FDA Panel
In overwhelmingly concluding that efficacy has not been demonstrated for the mesenchymal stromal cell therapy, panelists went beyond just the failed Phase III trial and cited BrainStorm’s ‘statistical magic’ and failure to adequately describe the mechanism of action, as well as safety and product quality concerns.

Austria & Germany Call For ‘Competitiveness Check’ On EU Pharma Revision Package
The two countries want the European Commission’s proposals to be more closely examined for their possible negative effect on pharmaceutical investment decisions. The industry federation EFPIA says it is surprised more EU member states aren’t taking the same stance.
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.
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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.

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.
US FDA Performance Tracker
European Performance Tracker
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.

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.

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.
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.
Recent Stories
Gene Therapy: Nonclinical Questions Do Not Seem Ideal For The US FDA’s New Type D Meeting
Nonclinical testing questions should be answered in a pre-IND meeting, rather than the new Type D session and indicated support for alternatives to animal testing when possible, a US FDA Center for Biologics Evaluation and Research official suggests.
Europe’s Proposed Chemical Ban Could Hit Many Thousands Of Global Marketing Authorizations
Drug manufacturing in the EU would grind to a halt in under three years if a proposal to restrict per- and polyfluoroalkyl substances in the European Economic Area is implemented in its current form, says industry group EFPIA.
EU Advances Plans For New Catalogs Of RWD Sources & Non-Interventional Studies
Users of the new catalogs will benefit from a modern technology with enhanced view, search, export and data submission functionalities, says the European Commission.
Orchard On The Trials, Tribulations And Triumphs Of Gene Therapy HTA
Convincing payers to fund a $3m therapy is no mean feat, even for innovations with curative potential. Orchard Therapeutics, a company with much experience in this field, tells the Pink Sheet why it values open dialogues, and outlines how health technology assessment methods could be improved.
Imbruvica, Enbrel, Stelara Offer Best Chance Of Savings From Medicare Negotiation In First Round
The other seven drugs on the initial list for negotiation are already heavily rebated and CMS could choose not to go much lower than current net prices, SSR Health report points out.
FTC Commissioner Recusal Decisions Should Be Public, US Chamber Of Commerce Says
Chamber files petition to have FTC amend its disqualification rules to have commissioners explain their rejection of recusal requests. FTC is seeking public comments on the petition.
New EU Filings
Givinostat, Italfarmaco’s investigational treatment for Duchenne muscular dystrophy, is among the latest products that have been filed for review by the European Medicines Agency for potential EU marketing approval.
WHO Seeks Feedback On Gaps In Current QA & GMP Guidelines
The World Health Organization has shortlisted over 30 quality assurance and good manufacturing practice guidelines that are over seven years old or need a major review.
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.
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.
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.
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