This Week at FDA: FDA’s major reorg takes effect, rare disease grants, and a TAP update

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 announced it had instituted a massive internal reorganization, launching a revamped human foods program and a new inspections office. The agency also awarded several rare disease research grants and expanded the list of devices qualified to be in its total product lifecycle advisory program (TAP) pilot.

FDA leadership announced this week that the agency has finally undergone a major reorganization meant to strengthen its human foods and inspectional oversight following the infant formula crisis of 2022. The reorganization involves the creation of a unified Human Foods Program and a new field operations office called the Office of Inspections and Investigations (OII) in place of the former Office of Regulatory Affairs (ORA). The agency also said it will launch a pilot program to get consumer complaints through an online form, which is intended to allow it to address complaints more efficiently and effectively internally and better respond to public health risks.

FDA announced it has awarded seven new clinical trial grants worth $17.2 million for FY 2024 under its Orphan Products Grants Program. The grants will fund clinical trials of treatments meant to treat various rare diseases, including pediatric aplastic anemia, microcystic lymphatic malformations, and Cushing disease. The agency also announced it had awarded three new natural history study grants worth $4.7M under the same program to study how rare diseases progress.

Furthermore, FDA said it had awarded $5.4 million to support clinical research under the Rare Neurodegenerative Disease (RNDD) Grants Program in FY 2024. The agency received 15 applications but ultimately funded a four-year project to develop retinal imaging biomarkers in people affected by cerebral autosomal dominant arteriopathy with subcortical infarcts and leukoencephalopathy (CADASIL).

Now that Eli Lilly has resolved the shortages of its GLP-1 weight-loss and diabetes drugs Mounjaro (tirzepatide) and Zepbound (tirzepatide), FDA has issued a statement to compounding pharmacies reminding them of the legal restrictions of making compounded versions of FDA-approved drugs. The agency advised compounders that when a drug shortage is resolved, they are only allowed to make the drugs in limited quantities and their drug cannot be identical or nearly identical to the approved drug.

Drugs & biologics

The Cellular, Tissue, and Gene Therapies Advisory Committee is scheduled to meet virtually on 21 November to discuss AstraZeneca's supplemental biologics license application (sBLA) for its drug Andexxa (coagulation factor Xa (recombinant), inactivated-zhzo). The drug is meant to be used in patients treated with rivaroxaban or apixaban when reversal of anticoagulation is needed due to life-threatening or uncontrolled bleeding.

FDA will host a webinar on 21 November to discuss the International Council for Harmonisation's (ICH) M13A guideline, which the agency recently adopted. The guideline details a harmonized framework for assessing the bioequivalence (BE) of immediate-release (IR) solid oral dosage forms and assessing BE after postapproval changes with other regulatory bodies.

FDA published a web page with results from studies on how generic drugs improve access to affordable medications. For instance, in one study, the agency found that in 2022, 742 generics yielded almost $19 billion in savings in the 12 months after the drug was approved.

Bristol Myers Squibb's cancer drug Opdivo (nivolumab) was approved for use with platinum-doublet chemotherapy as neoadjuvant treatment and single-agent nivolumab after surgery as adjuvant treatment for patients with non-small cell lung cancer (NSCLC) and no known epidermal growth factor receptor (EGFR) mutations or anaplastic lymphoma kinase (ALK) rearrangements. The drug was reviewed under the Project Orbis program, which allows it to be reviewed by multiple international regulators.

FDA approved Grifols' sBLA for Vistaseal (fibrin sealant), which is used as an adjunct to hemostasis during surgery to expand the indication to patients younger than 18. The company, however, is required to conduct a postmarketing study on pediatric patients, including conducting a human factors assessment as part of the trial.

Medtech

As of 1 October, the Center for Devices and Radiological Health (CDRH) announced it has again expanded the list of devices that can enroll in its Total Product Life Cycle Advisory Program (TAP) pilot. The pilot was already open to cardiovascular, neurological, and physical medicine devices but now includes ophthalmic and radiological devices. On 1 January, it will also open up to orthopedic devices.

CDRH has updated its Customer Collaboration Portal, which allows it to accept Small Business Determination (SBD) requests, and track the progress of Premarket Approval (PMA) applications and PMA Panel-Track supplements. The agency noted the portal update is part of CDRH's commitment under the Medical Device User Fee Amendments (MDUFA V) to be more transparent and efficient in its review process using technology.

FDA published a web page about its Safety and Performance-Based Pathway, which expands on its Abbreviated 510(k) pathway for certain well-understood device types. The web page details what the pathway is, what devices are appropriate for the pathway, and how to submit an application for a device to be considered under the pathway.

On 3 December, FDA plans to host a webinar to provide information on how manufacturers can comply with registration and listing requirements for in vitro diagnostic devices (IVDs), including laboratory-developed tests (LDT). Earlier this year the agency finalized a long-anticipated rule that phases out its regulatory discretion of LDT oversight.

FDA will host a webinar on 12 November to discuss its recent final guidance on clinical pharmacology considerations for human radiolabeled mass balance studies. The guidance details when sponsors should consider conducting radiolabeled mass balance studies, how they should be designed, and how to report the results.

Smiths Medical has issued a class I recall correcting issues with its paraPAC Plus P300 and P310 Ventilators due to problems with the volume knob, according to FDA. The faulty knobs could move from their intended position, potentially delaying therapy, causing too much tidal volume, or not enough tidal volume. Those who find devices with the problem should stop using them.

Philips has issued a mandatory software update for its Trilogy Evo, EV300, EvoO2, and Evo Universal ventilators as part of an ongoing class I recall, according to FDA. Software problems on the devices could lead to displaying faulty oxygen delivery, false battery depletion alarms, and other critical problems that could cause serious adverse events, including death.

Fresenius Kabi USA also asked users to update the software on its Ivenix Infusion System Large Volume Pumps (LVP) as part of a class I recall due to several problems that could delay treatment or lead to underdosing patients, according to FDA. The company has provided temporary risk mitigation steps until users update the software.

Sentec/Percussionaire has updated its instructions as part of a class I recall for its Phasitron 5 In-Line Valve to prevent users from failing to remove the blue plug from the expiratory port, according to FDA. The company has received reports that patients have been injured after users failed to remove the plug during Intrapulmonary Percussion (IPV) therapy.

This Week at FDA: FDA’s major reorg takes effect, rare disease grants, and a TAP update

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