This Week at FDA: Another budget cliffhanger, new adcomm, and a missed EtO deadline

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, the House passed an FDA appropriations bill, FDA created a new advisory committee for genetic metabolic diseases, and the Environmental Protection Agency (EPA) missed a deadline to publish final ethylene oxide (EtO) rules.

Last week, stakeholders waited with bated breath for EPA to finalize rules restricting the use of the EtO, which is commonly used to sterilize medical devices. The agency signed a consent decree in 2023 that required it to finalize its EtO rules by 1 March 2024, though it has yet to do so as of this writing.

After averting a government shutdown last week, the US House passed a Fiscal Year 2024 appropriations bill on Wednesday that would provide full-year funding to FDA. The Senate has advanced the bill for a vote and is expected to sign it, though Reuters reports that it is uncertain whether the bill will pass before funding expires at midnight Friday. Steve Grossman at the Alliance for a Stronger FDA notes the House bill includes $3.52B for FDA salaries and expenses, which is $8M below the FY2023 budget.

This week, FDA adopted two International Council for Harmonisation (ICH) guidances. The Q2(R2) guidance creates a framework on principles of analytical procedure validation that can be used to validate and analyze spectroscopic data, while the Q14 document addresses scientific procedures that can be used to address analytical procedure development.

The Office of Management and Budget (OMB) has received two notices from FDA for review concerning enforcement policies for diagnostic tests during a declared public health emergency (PHE) or tests used in the immediate public health response in the absence of a declared PHE.

FDA has added the Genetic Metabolic Diseases Advisory Committee (GeMDAC) to its list of standing advisory committees to help evaluate drugs and biologics to treat genetic metabolic diseases. The agency said it will consist of nine core voting members, including a chairperson.

The Center for Drug Evaluation and Research (CDER) this week released a revised version of its Manual of Policies and Procedures (MAPP) governing good review practice for breakthrough therapy-designated drugs and biologics. The document has been revised to reflect changes to the Office of New Drugs’ organizational structure, recent user fee agreement commitments, and updated CDER workflow procedures.

Drugs & Biologics

CDER denied Vanda Pharmaceuticals' request for a hearing to discuss the agency’s refusal to approve its supplemental new drug application for its drug Hetlioz (tasimelteon) to treat jet lag. The drug was initially approved in 2014 to treat non-24-hour sleep-wake disorder, which primarily affects people who are totally blind.

FDA's Vaccines and Related Biological Products Advisory Committee (VRBPAC) met this week and recommended FDA approve a trivalent influenza virus vaccine that would include two influenza A viruses (H1N1 and H3N2) and one influenza B virus. More specifically, the committee recommended the egg-based influenza vaccines for the US 2024-2025 influenza season include an A/Victoria/4897/2022 (H1N1)pdm09-like virus, an A/Thailand/8/2022 (H3N2)-like virus, and a B/Austria/1359417/2021 (B/Victoria lineage)-like virus. For the cell- or recombinant-based influenza vaccines it recommended an A/Wisconsin/67/2022 (H1N1)pdm09-like virus, an A/Massachusetts/18/2022 (H3N2)-like virus, and a B/Austria/1359417/2021 (B/Victoria lineage)-like virus.

Eli Lily said that FDA has delayed a decision on approving its Alzheimer's drug donanemab, and plans to convene its Peripheral and Central Nervous System Drugs Advisory Committee (PCNS) to get external expert feedback on the Phase 3 TRAILBLAZER-ALZ 2 trial, which was used to evaluate the drug in patients with early symptomatic Alzheimer's disease. The company says it doesn't expect any action from FDA until at least the second quarter of 2024, and Reuters reported this is the second time the agency has delayed its decision on the drug.

Reuters also reported that Eli Lily announced it had found bacteria and high levels of impurities in compounded versions of tirzepatide, the active ingredient in Lily's branded drugs Mounjaro and Zepbound, which are used to treat diabetes and for weight loss, respectively. The company claims some of the compounded versions of the drug have different chemical structures than the approved drugs, and in one case, the sample was found to be sugar alcohol.

FDA approved several cancer treatments this week, including Pfizer's Besponsa (inotuzumab ozogamicin) to treat children one year and older with relapsed or refractory CD22-positive B-cell precursor acute lymphoblastic leukemia (ALL). The drug was approved after receiving priority review and orphan drug designation.

FDA also approved Sandoz's Jubbonti (denosumab-bbdz) to treat osteoporosis and Wyost (denosumab-bbdz) to treat certain cancers, including multiple myeloma. They are the first approved interchangeable biosimilars to Amgen's Prolia (denosumab) and Xgeva (denosumab). The agency also approved Bristol-Myers Squibb's Opdivo (nivolumab) in combination with cisplatin and gemcitabine as a first-line treatment for patients with unresectable or metastatic urothelial carcinoma (UC).

FDA also expanded the indication for Novo Nordisk's Wegovy (semaglutide) to be the first weight-loss drug that is also approved to reduce the risk of cardiovascular death, heart attack, and stroke in patients with cardiovascular disease who are either obese or overweight. The agency said the injection should be administered in addition to a reduced-calorie diet and exercise.

FDA granted BeiGene USA's cancer drug Brukinsa (zanubrutinib) accelerated approval to treat relapsed or refractory follicular lymphoma (FL) after two or more lines of systemic therapy in conjunction with obinutuzumab.

FDA posted several recent warning letters sent to drug manufacturers and other firms this week, including warning letters to Bell International Laboratories, Colgin, Inc., Cosmetic Specialty Labs, Inc., and ivsupplyclinic.com.

FDA also announced that its Blood Products Advisory Committee will meet on 9 May 2024 to review strategies to reduce the risk of transfusion-transmitted malaria by testing donations by blood donors who have a risk of malaria exposure.

The New York Times reports that Amylyx Pharmaceuticals’ amyotrophic lateral sclerosis (ALS) drug Relyvrio (sodium phenylbutyrate and taurursodiol) failed a confirmatory Phase 3 trial and may be withdrawn from the market. FDA approved the drug in September 2022 after convening two advisory committee meetings earlier that year to review it. During the second advisory committee meeting, former Office of Neuroscience Director Billy Dunn asked Amylyx executives if the company would withdraw the drug if it were found to be ineffective in a confirmatory study, to which Amylyx co-CEO Justin Klee said the company would voluntarily withdraw the drug. Now that it has failed the confirmatory study, Amylyx said it will work with regulators over the next eight weeks to determine a path forward, “which may include voluntarily withdrawing RELYVRIO/ALBRIOZA from the market.”

Medtech

Dexcom’s Dexcom Stelo Glucose Biosensor System was cleared by FDA as the first over-the-counter (OTC) continuous glucose monitor (CGM). The agency, however, cautioned the device is only intended for adults who do not use insulin and is not meant for people with problematic hypoglycemia (low blood sugar) because it is not meant to alert users about dangerous blood glucose levels.

In a warning letter posted to FDA’s website this week, the agency warned Fresenius Kabi AG concerning its procedures around corrective and preventive action (CAPA), design controls, and design validation related to its Ivenix Infusion System.

FDA also announced several Class I medical device recalls, including for Medtronic Neurosurgery’s Duet External Drainage and Monitoring System Catheter Tubing, Ventec Life Systems’ VOCSN Patient Breathing Package, Cardinal Health’s Sterile Monoject Luer-Lock and Enteral Syringes, Baxter Healthcare’s ExactaMix Pro 1200 and Pro 2400, and Smiths Medical’s Medfusion Model 3500 Syringe Pump.

This Week at FDA: Another budget cliffhanger, new adcomm, and a missed EtO deadline

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