US Advisory Committees


US FDA Advisory Committee Freeze Raises Concerns Of Delayed, More Controversial Approvals

 

HHS communications restrictions are keeping the FDA from publicly scheduling expected advisory committee meetings. The lack of upcoming meetings is raising concern about missed user fee dates or lower-quality agency decision-making.

US FDA Opioids Adcomm Postponed; Are Communications Freeze, Travel Ban To Blame?

 
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A Trump Administration communications freeze and travel ban may be complicating efforts to convene FDA advisory committees. A 5 February meeting on postmarketing requirements for extended-release/long-acting opioids was postponed, while a 24 February meeting on a Novartis rare disease drug was cancelled.

Seikagaku’s Pain Drug Gets Advisory Panel OK, But With Limited Indication

 

The injection for radicular leg pain appears likely to be approved, but with a very prescriptive label to help balance safety concerns and a lack of long-term outcomes data.

Seikagaku’s Pain Drug May Need More Tailored Indication, REMS For Approval

 

An FDA advisory committee will consider modifications to the indicated patient population, as well as the appropriate health professionals and settings to administer the proposed treatment for radicular leg pain.

EU CHMP


New EU Filings

 

Mavorixafor, X4 Pharmaceuticals/Norgine's treatment for WHIM syndrome, is among the latest products that have been filed for review by the European Medicines Agency for potential EU marketing approval.

Depemokimab & Tzield Among 10 New EU Filings

 

GSK’s depemokimab, if approved, could become the first ultra-long-acting drug for treating asthma and chronic rhinosinusitis with nasal polyps. Meanwhile, Sanofi’s teplizumab, which the US approved as the first disease-modifying therapy for type 1 diabetes in 2022, has now been filed for review by the European Medicines Agency.

EMA’s Tougher Stance On Substandard Filings Shows Early Success

 

Insight obtained by the Pink Sheet relating to the European Medicines Agency’s stricter approach to requests for clock-stop extension shows that refusals have not led to any companies withdrawing their marketing applications. On the contrary, companies were able to provide responses within the agreed timeframe after all.

New EU Filings

 

Etuvetidigene autotemcel, Fondazione Telethon’s investigational gene therapy for Wiskott-Aldrich syndrome, is among the latest drugs that have been filed for review by the European Medicines Agency for potential EU marketing approval.

Approvals


New EU Filings

 

Mavorixafor, X4 Pharmaceuticals/Norgine's treatment for WHIM syndrome, is among the latest products that have been filed for review by the European Medicines Agency for potential EU marketing approval.

Depemokimab & Tzield Among 10 New EU Filings

 

GSK’s depemokimab, if approved, could become the first ultra-long-acting drug for treating asthma and chronic rhinosinusitis with nasal polyps. Meanwhile, Sanofi’s teplizumab, which the US approved as the first disease-modifying therapy for type 1 diabetes in 2022, has now been filed for review by the European Medicines Agency.

Precision Underlines US FDA’s 2025 Cancer, Neuroscience Candidates

 

Top areas for potential 2025 approvals were shaped by R&D focused on increasingly tightly targeted therapies, including the eight new candidates to join the still-burgeoning kinase inhibitor class and RNA interference, as well as many varieties of antibodies.

Novel Agents Up For US FDA Approval In 2025

 

CBER has at least 14 and CDER another 10 novel biologics among the more than 60 candidates with a user fee goal in 2025.

Complete Response Letters


Better Luck Next Year: US FDA CRLs May Be Rising, But Are Not The End Of The Story

 

Prospects are promising for many of the 16 novel candidates that received complete response letters in 2024 after half of the 2023 CRL class was approved the following year.

Pugilistic Vanda Collects Another NOOH As US FDA Defends Tradipitant Turndown

 

Vanda’s fight against a complete response letter for its gastroparesis drug results in a notice of opportunity for hearing on a formal FDA proposal to refuse to approve the NDA.

AstraZeneca Says Andexxa Will Not Convert To Full Approval, Quietly Reveals Complete Response

 

Whether the FDA will seek withdrawal of the anticoagulant-reversal agent or request additional confirmatory data is unclear.

Deleted Clinical Trial Data Derailed Applied Therapeutics’ Govorestat NDA

 

An FDA complete response letter followed a bioresearch warning letter citing the integrity of electronic data capture and handling of dosing errors in a pivotal trial of govorestat for galactosemia.

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


US FDA In ‘First Stages’ Of Reconsidering Black Box For Antipsychotics

 

Patient advocates and sponsors want the US to reconsider a 20-year-old class warning against the use of antipsychotics in patients with dementia. The agency is weighing the issue, but also defending the current labels of newer agents.

Shorter Gene Therapy Postmarket Studies ‘On The Table’

 

Amid complaints about the challenges of following gene therapy patients for up to 15 years, OTP Director Nicole Verdun said the FDA is considering how to conduct long-term postmarket studies more efficiently.

Accelerated Approval: US FDA Explains When A Confirmatory Trial Is ‘Underway’

 
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The agency will consider study enrollment, accrual rates and the number of clinical site activations, among other factors, in determining whether a confirmatory trial is underway prior to accelerated approval, but the new draft guidance does not specify the proportion of patients that must be enrolled at the time of approval.

Australia Unveils Final Product Recall Reforms Ahead Of March Implementation

 

The Therapeutic Goods Administration is advising companies to get up to speed with its new recall procedure, which is designed to improve the timeliness of recalls, alerts and corrections, and reduce regulatory burden for sponsors.