US Advisory Committees


Clozapine REMS A Barrier To Treatment And Unnecessary For Safe Use, US FDA Adcomms Say

 
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The atypical antipsychotic’s Risk Evaluation and Mitigation Strategy should be retired because the requirements for prescriber education and documentation on absolute neutrophil count monitoring are hindering access, panelists said.

AstraZeneca’s Andexxa Could Be Headed For Withdrawal After US FDA’s Negative Advisory Committee Preview

 

The confirmatory trial for the anticoagulant reversal agent raised safety and efficacy concerns from the agency.

Is A Clozapine REMS Still Needed? Two US FDA Adcomms Will Weigh Neutropenia Risk Mitigation

 
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The antipsychotic’s Risk Evaluation and Mitigation Strategy has never been fully implemented or enforced due to operational and technical challenges. The FDA now is re-evaluating the need for the program given changes in the health care landscape and medical training since the drug’s 1989 approval.

RSV Vaccines: ACIP Recommendations For Adults Younger Than Age 60 Await More Data

 
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GSK’s Arexvy and Pfizer’s Abrysvo are FDA-approved for individuals younger than 60 years old, but CDC’s Advisory Committee on Immunization Practices wants more data on Guillain-Barré syndrome cases in older adults and efficacy in severely immune-compromised persons before making a recommendation.

EU CHMP


Why The EMA Said No To Kizfizo And Cinainu

 

The European Medicines Agency has recommended against EU marketing approval for two drugs targeting children.

Gohibic For SARS-CoV-2-Induced ARDS Among Eight New Drugs To Win EMA Nod

Patients in the ICU continue to die from SARS-CoV-2-induced acute respiratory distress syndrome, says InflaRx, which is one of companies whose product the European Medicines Agency today announced should be approved for marketing in the EU.

Leqembi Secures EMA Thumbs Up For Narrower Indication

 

After an initial rejection, the European Medicines Agency now says that Eisai/Biogen’s Alzheimer’s disease drug should be approved, albeit with a recommendation for initial generic screening and ongoing safety scans.

EU Crunch Time For Kizfizo, Sipavibart, Coacillium And Eight Other Products

 

The European Medicines Agency’s human medicines committee is meeting this week due to decide whether or not to back pan-EU marketing approval for a number of new drugs.

Approvals


New EU Approvals

 

The Pink Sheet's list of EU centralized approvals of new active substances has been updated to include Hympavzi, Pfizer's treatment for adults and adolescents with severe hemophilia A or B without inhibitors.

Why The EMA Said No To Kizfizo And Cinainu

 

The European Medicines Agency has recommended against EU marketing approval for two drugs targeting children.

Gohibic For SARS-CoV-2-Induced ARDS Among Eight New Drugs To Win EMA Nod

Patients in the ICU continue to die from SARS-CoV-2-induced acute respiratory distress syndrome, says InflaRx, which is one of companies whose product the European Medicines Agency today announced should be approved for marketing in the EU.

Leqembi Secures EMA Thumbs Up For Narrower Indication

 

After an initial rejection, the European Medicines Agency now says that Eisai/Biogen’s Alzheimer’s disease drug should be approved, albeit with a recommendation for initial generic screening and ongoing safety scans.

Complete Response Letters


Dark Skies For Ocaliva: US FDA Sides With AdComm In Complete Response Letter

 

The US FDA rejected Intercept’s bid to convert Ocaliva’s accelerated approval in primary biliary cholangitis, meaning withdrawal prospects loom.

Vanda’s Tradipitant Gets CRL In Gastroparesis But Another NDA Filing Is Still On

 

The company will file a new drug application with the US FDA by the end of the year for tradipitant in a different indication, motion sickness.

US FDA Advisory Committee Conduct Could Take Center Stage in Lykos Appeal of MDMA Rejection

 

Sponsor of psychedelic PTSD treatment will try rarely successful formal dispute resolution process after receiving a complete response letter. 

Steady On For US FDA’s Novel Approvals At Mid-Year, But Another 2023 Is Unlikely

 

US FDA would need to approve 44 novel agents by year-end to match 2023’s big total, but only 40 candidates are known to have user fee goals in the second half of 2024.

Drug Review Profiles


US FDA Review Of Akebia’s Vafseo Informed By Roxadustat, Daprodustat Safety Concerns

 
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Vadadustat's review overlapped with two other oral HIF-PH class drugs. Safety issues with FibroGen/AstraZeneca’s roxadustat drew attention during vadadustat's first-cycle review, while GSK’s Jesduvroq labeling and postmarketing requirements informed the second-cycle approval.

Akebia’s Vafseo: Japanese Postmarketing Data, Narrowed Indication Eased Liver Toxicity Worries

 
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The Pink Sheet Drug Review Profile explores the US FDA’s approval of vadadustat to treat anemia in chronic kidney disease patients on dialysis. A complete response letter cited the risk of drug-induced liver injury, but postmarketing data from Japan reassured reviewers.

Vafseo Chronology: Complete Response Letter, Dispute Resolution And A Second-Cycle Approval

 
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The Pink Sheet’s Drug Review Profile looks at the timeline behind the development and US FDA approval of Akebia’s Vafseo, a treatment for anemia in chronic kidney disease patients on dialysis.

Biosimilars: US FDA’s Updated Scientific Thinking Led To Interchangeability For Amgen's Wezlana

 
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Pink Sheet’s Drug Review Profile explores Amgen’s Wezlana, an interchangeable biosimilar to Janssen’s Stelara. FDA determined late in the review that switching studies generally would not be needed for interchangeable ustekinumab products, prompting Amgen to seek the designation.

Post-Marketing Regulation & Studies


AstraZeneca’s Andexxa Could Be Headed For Withdrawal After US FDA’s Negative Advisory Committee Preview

 

The confirmatory trial for the anticoagulant reversal agent raised safety and efficacy concerns from the agency.

EMA Urges Variation Submissions By 30 November Before Holiday Closure

 

The European Medicines Agency is encouraging companies to submit the type I variations they want to make to their marketing authorizations by the end of this month.

‘Unforeseen’ Variations & ‘Super-Grouping’ Addressed In New EU Guidance

 
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Updated guidance issued by the EU regulator explains how to comply with the provisions of the amended Variations Regulation, which will apply to new submissions from January 2025 and is intended to make lifecycle management “more efficient and future-proof.”

Growth Spurt: Pediatric Labeling Is On The Rise, Taxing US FDA Safety Monitoring

 

The US FDA Pediatric Advisory Committee uses web-posted reviews of “low safety risk” products to keep up with postmarketing monitoring requirements amid rising interest in pediatric development.