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Ophthalmic Drugs Already Lead Medicare Part B Cost Drivers: How Will Gene Therapy Fit?
Gene therapy for wet age-related macular degeneration is one of two later-stage gene therapies flagged in CVS Caremark’s latest pipeline report that are outliers because they target conditions afflicting millions of patients and could dramatically increase Part B spending.
DREAMM Comeback Prompts GSK To Seek Fast-Track EMA Review For Blenrep
The European Medicines Agency is this week deciding whether the planned EU filings for sipavibart, lifileucel and Blenrep – which was previously approved but withdrawn in the EU and the US – merit an accelerated assessment.
‘Threading The Needle:’ Finding A Regulatory Foundation For Ultra Rare Disease Development
Sometimes even the US FDA’s regulatory flexibility may not be enough to approve rare disease treatments in very small populations, but agency officials worry that creating a new pathway could be problematic.
Clinical Trial Reform
‘Antiquated’ Regulations Slowing Adoption of Innovative Clinical Trial Approaches
The current regulations were written when clinical trials involved far fewer entities, and a certain amount of ‘contortion’ is necessary when sponsors are trying to introduce decentralized, community-based and other modern elements, the FDA’s Kevin Bugin says in an interview with the Pink Sheet.
US FDA Looking To Modernize Clinical Trial Regulations To Spur ‘Evolution’ In Research
US FDA Focuses On Labs, Concomitant Medication To Avert Needless Clinical Trial Exclusions
Cancer Trials: FDA Wants Lower Performance Status Eligibility But Primary Analysis Exclusion OK
France Consults On Best Practice Guidelines For Decentralized Trials
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PTC’s Upstaza Takes Gene Therapy Straight To Brain (And US FDA); Translarna To Return Mid-Year
In a year full of regulatory milestones for the firm, PTC Therapeutics hopes to set some approval precedents – and basically hopes its candidates have an easier time of it than they have had before.
Global Pharma Guidance Tracker – April 2024
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.
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ANDA Suitability Petitions: The Timelines They Are A-Changin'
US suitability petition submissions see an uptick against the backdrop of GDUFA III new goals for the FDA's response to such petitions. The Pink Sheet tracks some of the activity as applicants seek to tap market opportunities without the need for new clinical data.
Accelerated Approval: Nippon Shinyaku’s Viltepso Fails DMD Confirmatory Trial, Raising Questions
The Duchenne muscular dystrophy treatment missed its primary endpoint of time to stand from supine in the RACER53 study, raising more questions about using dystrophin changes to support accelerated approval and the ability of functional endpoints to detect clinical change.
US FDA’s Novel Approvals Peak Near Year-End, Analysis Of Last Decade Suggests
The Center for Drug Evaluation and Research’s novel approvals concentrated in August, November, and December, while January and June were the slowest months, according to a Pink Sheet analysis of 2014-2023 data.
Weight Loss, Diabetes Treatments Focus Of FTC's Second Round Of Patent Listing Challenges
The Pink Sheet takes a deep dive into the Federal Trade Commission's second wave of challenges to patents it asserts are improperly listed in the FDA's Orange Book, including patents covering Novo Nordisk's self-injection pens for Saxenda (liraglutide) and Ozempic (semaglutide).
Regional Comparisons
Parallel Scientific Advice: Is The EMA User Fee Impacting Interest?
EMA charges participants to receive scientific advice through the fledgling program, unlike the US FDA, which may not fit the budgets of some complex generic sponsors. At the same time, sponsors also may simply not be aware the program exists yet.
For Generics, EU Reference Product Is Non-Negotiable – Even In Pilot On Parallel Advice
Acknowledging that it may be a barrier to some extent, EMA’s senior scientific specialist of clinical pharmacology Kevin Blake notes that EU reference medicinal products are a legal requirement for bioequivalence studies and cannot be circumvented.
How The EU & UK’s Contrasting Approaches To AI Regulation Could Impact Pharma
While EU preparations are underway to introduce strict legal requirements for all AI systems, the UK has doubled down on its flexible, non-regulatory framework. In this second of a two-part article, a lawyer explains the pros and cons of each for pharma and medtech firms.
For Psychedelics, US FDA Is Open To Creative Thinking But Firm On Approval Standards
Flexible thinking and rigorous standards will both be needed to develop psychedelics as drug therapies in order to surmount the many complicating factors, from unique ‘set and setting’ aspects to functional unblinding, speakers at Reagan-Udall Foundation meeting agree.
Recent Stories
Novo Nordisk Should Have Studied Insulin Icodec Twice-Weekly Dosing, US Panel Says
FDA had recommended studying a twice-weekly dose, but Novo declined because it would add complexity to treatment for type 1 diabetes. However, the firm’s proposed strategy for reducing hypoglycemia risk with once-weekly insulin icodec would create its own complexity, panel said.
Chikungunya Vaccine, Hemophilia B Gene Therapy Among 14 Products On Track For EU Approval
The EU is on track to get its first vaccine for preventing the disease caused by chikungunya virus. Thirteen other products have also been recommended for pan-EU approval.
England’s Genomics Service Working With Industry On Precision Medicine & Personalized Vaccines
England’s chief scientific officer explains how the NHS Genomic Medicine Service has led to faster access to targeted cancer drugs and outlines the organization’s ongoing work in pharmacogenomics, liquid biopsies and personalized RNA vaccines.
Gene Editing And Cell Therapy: US FDA Still Wants Careful Characterization
The US FDA does not seem ready to depart from its safety-focused emphasis on product characterization as cell therapies become more complex, despite calls for approaches to allow human studies that let different edits “compete” and “find the winners inside the human tumor.”
Feedback Sought On The Tricky Business Of Handling CoIs Under EU HTA Regulation
Representatives and individual experts taking part in joint assessments of new health technologies need to be free from conflicts of interest in relation to the industrial sector in which the health technology developer operates.
EU Member States Told To Step Up Efforts On Transitioning Trials To CTR
The European Commission wants member states to ramp up disseminating information on transitioning ongoing studies to the Clinical Trials Regulation and ensure sponsors have access to relevant information in their national language.
ANDA Suitability Petitions: The Timelines They Are A-Changin'
US suitability petition submissions see an uptick against the backdrop of GDUFA III new goals for the FDA's response to such petitions. The Pink Sheet tracks some of the activity as applicants seek to tap market opportunities without the need for new clinical data.
US FDA Oncology Team Says Sponsor Meetings Are Still Mostly Virtual, And That’s OK
The US FDA’s cancer drug review managers say most sponsors are happy to continue with virtual meetings, even though in-person opportunities are offered. Managers also see advantages to the virtual format for staff development.
Genome Editing: US FDA Eyes Platform-Based Approach To Development And Approval
CBER Director Peter Marks outlines a streamlined process to approval for treating different mutations of the same gene. NCATS’ Philip Brooks tells the Pink Sheet the approach avoids the need to “start from scratch for every new mutation.”
C3TI's Mission: Leverage FDA Trial Innovation Expertise, Educate Inspectors
The Center for Clinical Trial Innovation was created to be CDER’s hub for expertise and information on innovative clinical trial approaches. C3TI lead Kevin Bugin talks to the Pink Sheet about the program's genesis and what it hopes to accomplish.
EU Clinical Trials Discussion Platform Sets Off With Collaborative Tone
An eagerly-anticipated group has been set up in the EU to hold balanced discussions with multiple stakeholders on how to solve clinical trial-related issues.
Pandemic Treaty Talks Flounder But WHO Still Holds Out Hope For Eventual Agreement
At this week’s World Health Assembly, a working group will be set up to discuss the next steps for talks on a global pandemic treaty and try to reach a final agreement on amendments to the International Health Regulations.
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