United States

Califf: Sponsors Should Go Beyond Letter Of Law With Trial Diversity Plans

 

The FDA Commissioner wants sponsors to incorporate diversity into their development programs prior to Phase III.

Marks Believes Trump Team Will Not Threaten US FDA’s Medicines Expertise

 

The US FDA’s Peter Marks told the Jefferies meeting that scientific leadership will be protected and that the agency wants to do more to help the sector accelerate innovation.

Diabetes Endpoints: A1C Reduction Is ‘Still Enough,’ Just Not For Lexicon’s Sotagliflozin, US FDA Says

 
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The agency reaffirmed support for a hemoglobin A1C endpoint for glycemic control in type 1 diabetes, but the diabetic ketoacidosis risk with sotagliflozin is so concerning that it is looking for additional benefits in patients with chronic kidney disease, a targeted subpopulation.

Pink Sheet Podcast: Trump’s Potential Impact On FDA User Fees, Dr. Oz’s Potential Impact On CMS

Pink Sheet reporter and editors discuss concerns that the incoming administration could try to influence the upcoming FDA user fee reauthorization, as well as the changes Dr. Mehmet Oz, President-elect Donald Trump’s controversial selection to run the US Centers for Medicare and Medicaid Services, could impose if confirmed.


AstraZeneca’s Andexxa May Continue Dangling In Accelerated Approval After Mixed Advisory Panel

 

The Cellular, Tissue and Gene Therapies Advisory Committee appeared unsatisfied with the risk-benefit balance of the anticoagulant reversal agent in the confirmatory trial, but was not ready to throw out the product, suggesting new trials may be needed.

Dose Optimization And Rare Diseases: Doing More With Less

 

US FDA officials said dose optimization for rare diseases should not be sacrificed in the name of speeding access to medicines, but is the oncology approach spreading to rare diseases?

Medicare Price Negotiation Under CMS Chief Oz May See Early 2025 Test

 
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The next round of the negotiation program is scheduled to begin in February unless the new administration delays the start of the process.

EMA: Regulatory Decisions On Pediatric Cancer Drugs ‘Cannot Happen In Isolation’

 

Stakeholders must work together to “identify the most promising products based on evidence” to treat childhood cancers, as drugs developed for adults do not always work, the scientific officer for the pediatric medicines office at the European Medicines Agency says.


US FDA’s Drug Center Losing Shortages, Controlled Substance Leadership With Throckmorton Retirement

 

Throckmorton is known for enhancing the FDA’s drug shortage work by building bridges between the compliance and quality teams, as well as opioid oversight.

Skinny Label Limits: Supreme Court Denies Norwich Bid For US Xifaxan Reprieve

 
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Removing the patent-protected indication from the label of Norwich Pharmaceuticals's proposed generic for Xifaxan did not convince the US Supreme Court to hear its petition to review a case from Bausch Health that blocked approval until 2029.

US FDA Biosimilar Standards Likely Sealed Interchangeability Designation’s Fate

 

When the agency decided biosimilarity standards would be high, it likely ensured the interchangeability designation eventually would become unnecessary.

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.

CMS Administrator Nominee Oz Is ‘Communicator’ In Sync With RFK’s Healthy Living Focus

 
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But the expected nominee brings little experience running a large government organization or with the policy and budget challenges confronting Medicare and Medicaid.

Gene/Cell Therapies: US FDA Clarifies INTERACT, Pre-IND Meeting Timing In FAQ Guidance

 
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New US FDA draft guidance attempts to address sponsor confusion about the different types of regulatory meetings under PDUFA, as well as frequently asked questions on nonclinical testing, CMC, pharm/tox and clinical studies.

Robert F. Kennedy Jr. As HHS Secretary: Prepare For The Worst, But Hope For The Best

 

Kennedy’s nomination for HHS secretary already is drawing concerns, which could mean a more conventional pick emerges, but industry should prepare for Kennedy to control the US Health and Human Services Department for at least some time.


AstraZeneca Tries Plan B For Dato-DXd After Regulator Feedback

 

The FDA said the antibody-drug conjugate looks more promising in a subgroup of EGFR mutated lung cancer patients, which caused AstraZeneca to refile the application.

Trump’s US FDA User Fee Cycle: ‘An Underappreciated Threat’

 

Expected HHS Secretary nominee Robert F. Kennedy Jr.'s dislike of user fees and interest in major FDA reforms could come to a head in the 2027 PDUFA reauthorization.

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.

MedPAC Explores Drug Coverage Plan Problems As Part D Stand-Alone Plan Access Declines

 
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Commission members say the standalone prescription drug plan market needs to be bolstered, given that more than 40% of Medicare beneficiaries in fee-for-service Medicare are enrolled in PDPs.