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Product Reviews & Approvals

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Approvals

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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.

Drug Review Europe

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.

Europe International

EU Could Soon Have Three XBB.1.5 COVID-19 Vaccines For Winter Season

The head of the European Medicines Agency says a group of international regulators got together to ensure “global alignment” on the approach to be taken on COVID-19 vaccination. 

Europe Coronavirus COVID-19

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

AstraZeneca/Daiichi Sankyo’s Enhertu To Get Lung Cancer Label For EU

The European Medicines Agency has recommended new uses for 11 approved medicines, including an antibody drug conjugate for treating advanced non-small cell lung cancer.

Europe Approvals

Lebrikizumab Among Nine Drugs To Get EMA Nod; PTC Faces Disappointment For Translarna

The European Medicines Agency has OKd nine new medicines for EU-wide approval, but recommended against renewing the conditional marketing authorization of two drugs. 

Europe Drug Review

EU New Active Substance Approvals – A Comparison Of Three Halves

A comparison of EU approvals of medicines containing a new active substance in the first six months of 2021, 2022 and 2023 shows a sharp decline in the 2023 first-half numbers. Three of the products approved by the European Commission in the first six months of this year were for cancer, while two were for infectious diseases (including HIPRA’s COVID-19 vaccine Bimervax). Two products had orphan drug status.

Europe Approvals

Pediatric Data Concerns Can’t Derail US CDC’s Universal ’23-’24 COVID Vaccine Recommendation

FDA approval was swiftly followed by CDC advisory committee recommendation of the XBB.1.5. variant-based vaccines for all people aged 6 months and older.

Approvals Vaccines

Potential Blockbuster Lebrikizumab Among Latest Drugs Up For Decision On EU Approval

The European Medicines Agency  is set to decide whether to back EU marketing approval for eight new drugs.

Europe Drug Review

Keeping Track: Rybrevant Goes RTOR, Two-Part Review For Dasiglucagon, And CRL For Ultomiris

The latest drug development news and highlights from the Pink Sheet’s US FDA Performance Tracker

US FDA Performance Tracker Approvals

Coronavirus Notebook: UK Approves Comirnaty XBB.1.5, CEPI & Oxford Uni Strike ‘Disease X’ Vaccine Deal

The Swiss medicines regulator is reviewing an application for Novavax’s vaccine against the XBB.1.5 variant, while ACM Biolabs has announced positive results from a Phase I trial of its COVID-19 booster vaccine, ACM-001.

Europe Switzerland

New EU Approvals

The Pink Sheet's list of EU centralized approvals of new active substances has been updated to add four new products, including Abrysvo, Pfizer's RSV vaccine for use in older adults and for the immunization of pregnant women to help protect their infants immediately from birth through six months of age.

Europe Approvals
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Drug Reviews

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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.

Advisory Committees Drug Review

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.

Drug Review Europe

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.

Europe Drug Review

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

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

The $4 Million NDA: US FDA User Fees After 30 Years

The cost of filing a New Drug Application with the US FDA has pushed above the $4 million mark. That is a remarkable rate of increase from the original $100,000 fee set in 1992 – but it does buy a lot more.

User Fees Drug Review

Lack of Adequate Study Plan Prompts EMA Review Of Cardiovascular Risk With Mysimba

The European Medicines Agency is to recommend whether the marketing authorization for Currax’s weight management drug should be maintained, varied, suspended or revoked.

Europe Drug Safety

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.

Drug Review Profile Regenerative Medicine

Novavax’s ’23-’24 COVID Vaccine Can Bypass Another CDC Cmte Meeting – If FDA Authorizes It

CDC’s universal recommendation for XBB COVID vaccines means that Novavax’s protein-based vaccine can enter the market immediately upon FDA authorization, but some final questions from the agency are keeping the review going.

Vaccines Drug Approval Standards

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.

Drug Review Profile Complete Response Letters

Lebrikizumab Among Nine Drugs To Get EMA Nod; PTC Faces Disappointment For Translarna

The European Medicines Agency has OKd nine new medicines for EU-wide approval, but recommended against renewing the conditional marketing authorization of two drugs. 

Europe Drug Review

‘Poor Quality’ Submission: CellTrans’ Lantidra BLA Marked By Missing And Incongruent Data

FDA's review of documents and source data files to assess product efficacy and subject safety was complicated by missing and inconsistent data, the clinical reviewer said, concluding there was insufficient data monitoring during clinical studies or in preparing the documents for regulatory submission.

Drug Review Profile Regenerative Medicine
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Advisory Committees

Set Alert for Advisory Committees

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.

Advisory Committees Drug Review

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.

Advisory Committees Regenerative Medicine

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.

Advisory Committees Regenerative Medicine

CDC Panel Backs Pfizer’s Maternal RSV Vaccine But Leaves Room For AstraZeneca/Sanofi’s Beyfortus

Abrysvo is recommended for use in pregnant people 32-36 weeks gestation if administered from September to January. For infants whose mothers were not vaccinated in this window, nirsevimab is recommended for prevention of respiratory syncytial virus infection.

Advisory Committees Vaccines

Intarcia’s Implantable GLP-1 Gets Unanimous No Vote But Lots Of New Trial Design Advice

US FDA advisory panel feels there could be a place for Intarcia’s exenatide drug-device combo – if the company is willing to put in the work.

Advisory Committees Drug Safety

Delivery Device Continues To Be Intarcia’s Downfall Ahead Of FDA Panel On Nonapproval of Implantable GLP-1

CDER reviewers don’t buy that safety concerns with exenatide implant ITCA 650 are in line with the risks labeled for already approved GLP-1 agonists for type 2 diabetes – even other formulations of the same drug – potentially due to unique considerations associated with Intarcia’s delivery method.

United States Advisory Committees

Alnylam Onpattro’s Expanded Use Gets Weak Thumbs Up From US FDA Panel

A restrictive label could be the result of the lukewarm endorsement from the majority of the agency’s Cardiovascular and Renal Drugs Advisory Committee for use of Onpattro for the treatment of ATTR-associated cardiomyopathy.

Advisory Committees Clinical Trials

Alnylam's Onpattro: Not Following FDA Guidance Amplifies Clinical Meaningfulness Concerns

Even if Alnylam can get a favorable advisory committee vote and is able sneak out an approval for Onpattro for ATTR-associated cardiomyopathy, its lack of data on hard clinical outcomes makes it unlikely to unseat Pfizer’s tafamidis for the rare disease.

United States Advisory Committees

Recent And Upcoming FDA Advisory Committee Meetings

Recent and upcoming US FDA advisory committee meetings and a summary of the topics covered. 

Advisory Committees Drug Review

The US FDA Committee ‘Hearing’ Is Dead! Long Live The Committee Hearing!

The US FDA is free from the need to reconsider Accelerated Approvals in a formal hearing in front of an advisory committee. But now the agency is now trying that approach to hear an appeal of a rejected application.

Advisory Committees Drug Approval Standards

Intarcia Gets Its Hearing: FDA Panel To Weigh Nonapproval Of Diabetes Drug/Device Combo In September

Since the Center for Drug Evaluation and Research is conducting the single-day hearing on the type 2 diabetes treatment ITCA 650 under its usual process for advisory committee meetings, it denied Intarcia’s requests for input on adding ad hoc members and extended presentation time.

Advisory Committees Drug Review

Recent And Upcoming FDA Advisory Committee Meetings

Recent and upcoming US FDA advisory committee meetings and a summary of the topics covered. 

Advisory Committees Drug Review
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