This Week at FDA: FDA’s budget justification, Nurtec ODT recall, and more

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, FDA released more details on its FY2024 budget request, Pfizer announced it is recalling millions of packages of a migraine drug over concerns about its packaging and FDA advisors back the approval of Pfizer’s COVID-19 drug Paxlovid.
 
FDA’s nearly 300-page FY 2024 budget justification contains a more in-depth rundown of the agency’s asks for the coming year. As previously reported, the White House is asking Congress to allocate $7.2B, of which about half would be from appropriations while the other half would be from user fees. In all, the agency is asking for $372M more in budget authority. FDA Commissioner Califf said the increase is necessary to help the agency modernize and to enable it to bolster its food safety program.
 
The Senate Appropriations Committee has released a schedule of upcoming meetings to discuss the FY 2024 budget, including a hearing on 19 April to hear from FDA on its request.
 
On Thursday, Pfizer and the US Consumer Product Safety Commission announced that the company is recalling more than 4 million units of its migraine drug, Nurtec ODT (Rimegepant), due to concerns that the packaging of its 75mg 8-unit dose blister pack presentation is not child resistant as required under the Poison Prevention Packaging Act.
 
Earlier this week, FDA authorized Pfizer and BioNTech’s bivalent COVID-19 vaccine as a booster for children between six months and four years of age who have completed a three dose primary series of the monovalent vaccine. FDA initially authorized the bivalent vaccine for children in this age group last December but limited its authorization to children who had not yet received a third shot of the monovalent vaccine.
 
An FDA advisory committee on Thursday voted 16-1 in favor of the agency approving Pfizer’s Paxlovid to treat mild to moderate COVID-19 in adults who are at high risk for severe outcomes from the disease, Reuters reports. The drug is currently available under an emergency use authorization (EUA). The agency is expected to make a final decision on whether to fully approve the drug in May. FDA has said the EUA will remain in effect for children even if the drug is approved for adults.
 
We also read this assessment of FDA’s lack of communication on another round of COVID-19 boosters this spring in STAT. While regulators in other countries, including the UK and Canada have offered such recommendations, FDA has not indicated whether it will recommend additional boosters before the fall.
 
Plus, STAT reports that FDA Commissioner Robert Califf thinks insurance companies should play a larger role in conducting postapproval studies, based on his remarks at the America’s Health Insurance Plans (AHIP) conference this week.
 
This week, Trump-appointed US District Judge Matthew Kacsmaryk heard arguments in a case seeking to overturn FDA’s approval of the abortion drug mifepristone, Reuters reports. The case has wide-ranging implications for FDA if the approval is overturned, according to The Washington Post, though the case is likely to be appealed regardless of the decision.
 
The Government Accountability Office (GAO) has issued a report compiling stakeholder views on how to improve patent information for drugs listed in FDA’s Orange Book. The report cites concerns over patent listing practices that may inhibit generic competition, including for drug-device combination products, which can be particularly challenging for generic drugmakers to develop cheaper alternatives to. The stakeholders GAO spoke to were split on whether FDA should take a more active role in evaluating the patents listed in the Orange Book (7 of 13), or whether the agency’s current role in overseeing the publication is sufficient (6 of 13). Most of the stakeholders said FDA should provide greater clarity on which device-related patients should be listed, though their views diverged on what criteria the agency should use.
 
Drugs & biologics
 
FDA this week announced it is continuing its Regulatory Project Management Site Tours and Regulatory Interaction Program and is inviting interested drugmakers to participate. Under the program, FDA’s Center for Drug Evaluation and Research (CDER) sends small groups of its regulatory project managers to visit drugmakers to observe their manufacturing, packaging, pathology/toxicology laboratory and regulatory affairs operations. Don’t think of this as an inspection or review, FDA stresses that the visits are intended “to improve mutual understanding and to provide an avenue for open dialogue” with drugmakers. The program also features daily workshops on various issues relevant to industry and CDER staff, including drug discovery, nonclinical and clinical evaluation, postmarketing activities and regulatory submissions.
 
On Monday, FDA announced the approval of Acadia’s Daybue (trofinetide) as the first drug to treat Rett syndrome. The rare genetic neurological disorder causes the progressive loss of motor skills and language in children. FDA said the drug was approved based on the results of a randomized, double-blinded placebo-controlled trial that measured changes from baseline in the Rett Syndrome Behavior Questionnaire (RSBQ) and the Clinical Global Impression-Improvement (CGI-I) after 12 weeks of treatment or placebo. FDA said the drug demonstrated a statistically significant improvement on both co-primary endpoints. The most common side effects were diarrhea (81%) and vomiting (27%).
 
FDA also approved Novartis’ Trafinlar (dabrafenib) in combination with Mekinist (trametinib) to treat children ages one and older with low-grade glioma with a BRAF V600E mutation who need systemic therapy. FDA said the approval is the first first-line systemic therapy for the indication and that it has approved new oral formulations of both drugs for patients who cannot take them in pill form.
 
This week, FDA issued draft guidance on pharmacogenomic data submissions in investigational new drug applications (INDs), new drug applications (NDAs) and Biologics License Applications (BLAs). FDA said the aim of the guidance is to “clarify the contexts in which pharmacogenomic study findings and data must be included” in such submissions, as well as recommendations on the content and format of pharmacogenomic data in those applications. The guidance will supersede the agency’s 2005 guidance on the same topic.
 
The Biden administration made two moves with implications for drug pricing this week. The Department of Health and Human Services (HHS) announced Wednesday that 27 drugs will be subject to penalties for price increases that go above inflation in the first round of such penalties under the Inflation Reduction Act (IRA). HHS also released its initial guidance on Medicare drug price negotiation under the IRA for public comment.
 
We also learned this week that Novo Nordisk and Sanofi will cut the price of their insulin products in the US, following a move by Eli Lilly earlier this month to cut its insulin prices by 70% and limit out-of-pocket costs for patients to $35 per month. Reuters reports that Sanofi will cut the price of Lantus (insulin glargine) by 78%, while Novo Nordisk will reduce the price of multiple insulin products by up to 75%. The price cuts could potentially save drugmakers money but reducing rebates the companies pay to Medicaid, Bloomberg reports.
 
Medtech
 
Kaiser Health News and CBS recently reported on lawsuits concerning the implantation of Anterior Growth Guidance Appliances (AGGA). The lawsuits allege that while the products are meant to change a patient's jaw structure, many patients have been seriously harmed. This week, KHN reported that former FDA senior policy advisor Cara Tanenbaum said the agency is “looking into” the product, and whether it can regulate it as a medical device.
 
Baxter International has issued a safety communication for its Life2000 Ventilation System due to the potential for patients to suffer from low blood oxygen under certain conditions when the device is connected to a third-party oxygen concentrator, Medtech Insight reports. The company warned that the issue might cause the most vulnerable patients to suffer life-threatening events or permanent impairment, and in some cases, may lead to death.
 
FDA announced this week it has authorized the first medical device with a Safer Technologies Program (STeP) designation. The agency has given the green light to Endolumik’s FG Bougie, which is intended to be used during gastric and bariatric surgical procedures.