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Drug Review Pathways & Approval Standards

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Review Pathways

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New EU Filings

Sepiapterin, PTC Therapeutics' treatment for hyperphenylalaninemia in adult and pediatric patients with phenylketonuria, is among the latest products that have been filed for review by the European Medicines Agency for potential EU marketing approval.

Drug Review Europe

PDUFA VIII: Continuous Fee Increases May Be Wearing On Sponsors

A Pfizer exec says that the old prescription drug user fee formulas that usually increase funding and staffing for many FDA programs no longer deliver the same return on investment.

User Fees Drug Review

First Drug Treatment For Myopia Among 14 New EU Filings

Vyluma’s eyedrop for treating myopia in children and Vertex’s next-generation triple combination treatment for cystic fibrosis are among the latest drugs that the European Medicines Agency has begun reviewing for potential EU marketing approval.

Europe Drug Review

US FDA User Fee Conundrum: Collections Increase After 25% PDUFA Rate Hike

With likely more fee-paying applications arriving at the agency, sponsors may be wondering whether user fees should have been increased as much as they were at the start of FY 2024.

User Fees Drug Review

US FDA’s ‘Platform’ Pathway Fits For LNPs And siRNA But Not Delivery Devices

The US FDA’s new guidance defining a “Platform Technology Designation” makes clear that what counts as a “platform” may not match industry’s use of the term.

Guidance Documents Review Pathway

Oh NOOH! Vanda’s Multi-Front Battle With US FDA Extends To Insomnia Drug Hetlioz

The notice of opportunity for hearing on the US FDA’s proposal not to approve an insomnia sNDA for Vanda’s Hetlioz opens another front in the company’s aggressive campaign against complete response letters and generic competition to the sleep disorder drug.

Drug Approval Standards Review Pathway

US FDA’s Yim Hints At Revisiting Biosimilar Suffix Naming Convention, Reassures On Immunogenicity

In an ‘ask me anything’ Q&A on Reddit, Sarah Yim, director of US FDA’s Office of Therapeutic Biologics and Biosimilars, offered some insights on the US biologics naming convention, biosimilar interchangeability, and the possibility of future competition on cell and gene therapies.

Biosimilars Review Pathway

EU Launches New Mechanisms To Boost Quality Of Clinical Trial And Marketing Applications

As of today, drug developers can apply to take part in two pilot programs that will involve regulators across the EU strengthening their coordination when it comes to providing companies with advice on their applications for clinical trials and marketing authorization.

Europe Clinical Trials

Three Gene Therapies, Moderna Metabolic Drug In Inaugural START Class; CBER Oversubscribes

Seven rare disease programs make up the first round of participants in the US FDA’s ‘Operation Warp Speed’-style program. Sponsors will benefit from ‘rapid, ad hoc’ communication with the agency.

Gene Therapy Rare Diseases

Quotable: Top Experts On Policy Hot Topics

The Pink Sheet highlights recent comments and insights from pharma officials and executives on key issues we are covering.

International Europe

EU CHMP Opinions And MAA Updates

This is an update of recommendations from the European Medicines Agency's Committee for Medicinal Products for Human Use on the authorization of new medicines in the EU, and updates on EU marketing authorization changes recommended by the CHMP.

Drug Review Europe

Project Orbis Led To 80% Cut In Submission Gap For Cancer Drugs In Switzerland

The international Project Orbis regulatory collaboration initiative reduced the submission gap between filings made in the US and Switzerland even more than expected, marking a step towards faster access to innovative cancer drugs, regulatory experts have told the Pink Sheet.

BioPharmaceutical Drug Review
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Drug Approval Standards

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CAR-T Therapies: Access Considerations May Influence US FDA's Efficacy Evaluation

CBER's Lola Fashoyin-Aje offers some thoughts on when and how the FDA might consider the accessibility of a product in its approval decisions at BIO 2024.

Drug Approval Standards Clinical Trials

Oh NOOH! Vanda’s Multi-Front Battle With US FDA Extends To Insomnia Drug Hetlioz

The notice of opportunity for hearing on the US FDA’s proposal not to approve an insomnia sNDA for Vanda’s Hetlioz opens another front in the company’s aggressive campaign against complete response letters and generic competition to the sleep disorder drug.

Drug Approval Standards Review Pathway

Project Orbis, 'Cluster' Calls Validate Global Regulators’ Drug Application Concerns

Regulators from the US, Canada, European Union, Switzerland and Japan talk about the benefits of global collaboration, and reasons why they might reach different decisions on the same drug, at the American Society of Clinical Oncology annual meeting.

Drug Review Regional Comparisons

Lykos’ MDMA Heads To AdComm With US FDA Concerns About Adverse Event Reporting

‘Short-term effects of midomafetamine were not captured’ in the clinical program for post-traumatic stress disorder, FDA notes. Proposed REMS for the psychedelic therapy to be discussed at 4 June would limit dispensing setting, restrict post-treatment driving.

Advisory Committees Drug Safety

US FDA’s June Approvals Forecast: Nearly 30 Goal Dates Suggest Hot Start To Summer

June goal dates include RSV and pneumococcal vaccines, two COPD therapies, Rocket and Sarepta gene therapies, and lots of oncology.

US FDA Performance Tracker Drug Review

‘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.

Rare Diseases Drug Approval Standards

Joint Clinical Assessments Of ATMPs Add To Ethical Concerns Over Need For Randomized Trials

Advances in cell and gene therapy, together with the forthcoming EU joint clinical assessments, have raised questions about the ethics of having to use randomized controlled trials to test new advanced therapies.

Europe Drug Approval Standards

European Pharmacopoeia Gets To Grips With Quality Standards for mRNA Vaccines

Stakeholders have until 30 June to comment on a set of common quality standards for mRNA vaccines that are said to be needed in light of rapid developments in this field and the emergence of novel vaccines coding for a range of specific antigens.

Vaccines Quality

Overall Survival Data Collection Can Help Mitigate Bias In Open-Label Studies, US FDA Says

FDA oncology review staff discuss cases studies involving Amgen’s Lumakras and Novartis’ Pluvicto, where there was a perceived loss of equipoise, and how mitigation strategies helped save the latter’s pivotal trial.

Clinical Trials Drug Approval Standards

Beyond CAR-Ts: Cell-Based Gene Therapy Sponsors Are ‘Branching Out,’ CBER’s Marks Says

As sponsors explore development in autoimmune and infectious diseases, plus a range of hematologic and solid tumors, manufacturing processes can be centralized or decentralized, but Marks says the FDA is ‘neutral’ on that decision.

Drug Approval Standards ImmunoOncology

Overall Survival Is Not A Required Primary Endpoint In All Cancer Trials, US FDA Says

Seeking to clear up a ‘misconception,’ Oncology Center of Excellence officials say earlier endpoints, such as progression-free survival and overall response, continue to be useful in getting therapies to market quicker, but sponsors still should plan to systematically collect OS data to ensure there is no detriment to survival.

Clinical Trials Drug Approval Standards

Preterm Birth Prevention Studies Need To Look Beyond Makena Endpoints, Experts Say

Gestational age and a binary, neonatal composite endpoint are insufficient to assess outcomes that are important to babies and families, clinicians and researchers say, suggesting development of continuous variables or scales with weighted components and a comparison of outcomes among newborns in gestational age ‘buckets.’

Drug Approval Standards Pediatrics
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