Approval Standards

Another Regeneron CRL Prompts Regulatory Operations Questions

 

CEO Len Schleifer said during the company’s first quarter earnings call that most of the complete response letters the company has received related to third-party suppliers, not efficacy or safety.

Postmarketing Surprise Is Latest Plot Twist For Novavax’s COVID-19 Vaccine BLA

 

Three weeks after its user fee goal date, the FDA demanded a postmarketing commitment “to generate additional data,” Novavax said.

Gene Therapy Sponsor INTERACT, Pre-IND Meeting Confusion Lingers Despite FDA Guidance

 
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PhRMA questioned whether proof-of-concept studies are needed before an INTERACT meeting and said a draft guidance describing the timing of definitive toxicology studies creates confusion.

US FDA Shows Support For Generic Drug User Fee Goals Amid Cutbacks

 

The FDA struck an upbeat tone during its annual Generic Drugs Forum despite an unprecedented period of cutbacks and disruptions and reiterated a desire to continue meeting user fee goals.


US FDA’s Makary Floats Vague ‘Plausible Mechanism’ Approval Pathway For Rare Diseases

 

Ascertaining the novelty and benefits of Makary’s new drug approval idea – along with what it would take to implement it – requires more clarity on the types of studies he envisions being used to grant and confirm “plausible mechanism’ approvals.

New EU Approvals

 

The Pink Sheet's list of EU centralized approvals of new active substances has been updated to include four new products, one of which is Tivdak, Pfizer/Genmab’s treatment for previously treated recurrent or metastatic cervical cancer.

Industry Keen On ‘Stepwise’ PIPs, But Future Hinges On EU Pharma Legislation Reform

 

Industry feedback on a European Medicines Agency pilot that introduced a “stepwise” approach to pediatric investigation plans was positive, but the future of the new model will depend on the outcome of the pharmaceutical legislation negotiations, the agency says.

EU Regulators Push For Better Support For Sponsors On Using Animal Study Alternatives

 

The European Medicines Agency’s innovation network has recommended how the EU’s regulatory framework could be updated to support drug developers in using new approach methodologies, which are drug testing methods that reduce or replace animal studies.


Industry Urged To Respond To EU Expectations On Using Animal Testing Alternatives

 

The chair of a European Medicines Agency working group on reducing animal testing said that companies can provide “thoughts and suggestions” in relation to a review of its scientific guidelines on regulatory testing requirements and new approach methodologies.

Trump’s US FDA Takeover Similar To Flat Earth Proponents Running NASA, Woodcock Says

 
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The former principal deputy commissioner said the “decapitation” of senior leadership will make resolving internal disputes more difficult, which will slow application reviews. Woodcock also called the FDA layoffs a slow-moving catastrophe.

Device-Like System Proposed For Low-Risk Human Cell Therapies, Tissue-Based Products At US FDA

 
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A risk-based approach to human cell therapies and tissue-based products could incentivize development and prevent bad actors from taking advantage of the current FDA system.

US FDA Under Makary: MAHA With A Lighter Touch

 

US FDA Commissioner nominee Martin Makary is being embraced by industry, and Senate Democrats, as a more traditional pick than other Trump Administration nominees, but the Make America Healthy Again agenda still is clearly coming to the agency.


New EU Approvals

 

The Pink Sheet's list of EU centralized approvals of new active substances has been updated to include four new products, one of which is Vimkunya, Bavarian Nordic's chikungunya vaccine.

Unfreezing US FDA: Generic Drug Officials Make Plea For Public Workshops

 

The FDA generic drugs team’s first public workshop of the second Trump Administration ended with a request that industry amplify the value it finds from public engagement.

Almost Public: Interagency Meeting Replaces US FDA ‘25-’26 Flu Vaccine AdComm

 

Only government staff participated in the meeting defining the next season’s influenza vaccine composition after the annual public FDA vaccines advisory committee meeting was cancelled.

US FDA Spikes Vanda’s Hetlioz For Insomnia, Lack Of Well-Controlled Trial ‘Fatal’

 
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Acting Commissioner Sara Brenner rejected the company’s request for a hearing, saying the lack of an adequate and well-controlled trial showing tasimelteon's effectiveness in treating sleep-onset insomnia is “fatal to the application.”


Sponsors Increasingly Happy With CBER Written Responses To Formal Meeting Requests

 

The FY 2024 fourth quarter had the highest number of formal meetings cancelled because written responses were sufficient since the beginning of FY 2018.

Topical Gene Therapy Vyjuvek & Cancer Drug Lynozyfic Among Four New Products Set For EU Market

 

The European Medicines Agency has recommended in favor of approving four new products, and has also reaffirmed its positive opinion for the Alzheimer’s disease drug, Leqembi.

Turning Back The Clock At The US FDA

 

HHS Secretary Robert F. Kennedy Jr. is nostalgic for the 1960s. Will that include pushing the agency back to the pre-user-fee-era model of defining success based on blocking unsafe drugs, rather than accelerating access to effective ones?

Harmony Gets Dissonant US FDA Response For Wakix In Idiopathic Hypersomnia

 

The agency sent the neuroscience-focused company a refuse-to-file letter for Wakix as a treatment for Idiopathic Hypersomnia, a disease with only one approved treatment and few others in development.