This Week at FDA: Trump nominates O’Neill for HHS deputy, CDRH presents MDUFA data
Welcome to another installment of This Week at FDA, your weekly source for updates – big and small – on FDA, drug, and medical device regulation, and what we’re reading from around the web. This week President-elect Donald Trump nominated Jim O'Neill as second-in-command at HHS, FDA published a report on its medical device user fee program, and more.Over the past few weeks, President-elect Trump has nominated Robert F. Kennedy Jr. to lead the US Department of Health and Human Services (HHS) and Martin “Marty” Makary to lead the Food and Drug Administration (FDA). This week, he tapped Jim O'Neill, former CEO of the Thiel Foundation and a close associate of Trump funder Peter Thiel, to be deputy secretary of HHS. O'Neill, who has argued that FDA should rely on safety data but not efficacy data when granting marketing authorization medical products, was considered for FDA commissioner for the first Trump administration.
The Center for Devices and Radiological Health (CDRH) published its 2024 fourth-quarter Medical Device User Fee Amendment (MDUFA V) performance report and details from a 22 November meeting where those results were presented. The report notes an uptick in first-cycle major deficiency letters for premarket approval (PMA) applications, PMA original applications, and panel track supplements. It also shows a drop in average total days for MDUFA decisions.
The Oncologic Drugs Advisory Committee (ODAC) has been renewed for another two years until 1 September 2026. FDA said it has determined the committee's work continues to serve the public interest.
FDA published several guidances this week including a final guidance for sponsors who intend to use circulating cell-free plasma derived tumor DNA (ctDNA) as a biomarker in cancer clinical trials conducted under an investigational new drug application (IND) and/or to support marketing approval of drugs and biological products for treating solid tumor malignancies in the early-stage (curative-intent) setting. The guidance also discusses standardization and harmonization of ctDNA assays and methodologies.
Drugs & Biologics
The Center for Drug Evaluation and Research (CDER), the Center for Biologics Evaluation and Research (CBER), and outside researchers presented results from a study exploring whether adding a concise summary of drug risk information affecting pregnant and lactating patients and including a lactation subsection in the prescribing information (PI) affects healthcare providers’ decision-making. The researchers concluded improved communication about the safety of drugs for breastfeeding patients would better suit the needs of time-constrained healthcare providers.
The Anesthetic and Analgesic Drug Products Advisory Committee will meet virtually on 10 January to discuss Seikagaku Corp.'s biologics licensing application (BLA) for its condoliase injection to treat radicular leg pain associated with lumbar disc herniation in adults. Earlier this summer, the company presented positive results from a phase 3 trial of the drug at the annual meeting of the American Society of Pain and Neuroscience.
PTC Therapeutics's drug Kebilidi (eladocagene exuparvovec-tneq) for the treatment of aromatic L-amino acid decarboxylase deficiency has been awarded a priority review voucher due to its qualification as a rare pediatric disease product application. The drug received the priority review voucher on 13 November, according to FDA.
BridgeBio Pharma's heart drug Attruby (acoramidis), intended to treat adults with cardiomyopathy of wild-type or variant transthyretin-mediated amyloidosis (ATTR-CM) to reduce death and hospitalization related to heart problems, was approved by FDA. The company said the drug is the first and only product approved with a label specifying near-complete stabilization of transthyretin (TTR) and was designed to mimic a naturally occurring rescue mutation of the TTR gene (T119M).
FDA recommended that healthcare providers consider alternative therapies to bluebird bio's gene therapy drug Skysona (elivaldogene autotemcel) after receiving reports of hematologic malignancies, including life-threatening cases of myelodysplastic syndrome and acute myeloid leukemia, in patients treated with the drug. The agency is investigating the risk of hematologic malignancies and said patients on the drug should continue to be monitored throughout their lives.
FDA's Oncology Center of Excellence, in collaboration with the American Association for Cancer Research (AACR), will hold a hybrid workshop on 16 January to discuss dihydropyrimidine dehydrogenase (DPD) deficiency testing for patients receiving fluoropyrimidine drugs as part of their cancer treatment regimen. The agency said that the DPD enzyme is vital in breaking down certain cancer drugs into non-toxic products. However, no tests have received marketing authorizations so far to help identify patients with DPD deficiency.
FDA warned consumers not to use LipoFit Turbo, which is sold online as a weight loss drug. The agency found it contains sibutramine, a controlled substance that was removed from the market in October 2010 for safety reasons; metformin; fluoxetine; and furosemide, none of which are listed on the label and could cause life-threatening side effects.
Medtech
CDRH announced it plans to hold the last in its series of town hall meetings to discuss medical device sterilization intended to encourage manufacturers to find alternative methods to ethylene oxide (EtO) to sterilize their products. The agency said it plans to discuss its transitional enforcement policy for EtO sterilization facility changes for class III devices guidance that was published this week, and research and modeling on diffusion of vaporized hydrogen peroxide (VH202) through select polymeric materials.
Elekta Instrument has issued a class I recall for some of its Disposable Biopsy Needle Kit used with the Leksell Stereotactic System for neurosurgery after reports that found microscopic steel debris on the inside of the biopsy needle, according to FDA. The agency said needles from affected lots should be discarded and warned use of the needles could allow debris to enter the brain and cause death.
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