This Week at FDA: Lawmakers spar over LDT rule, new guidances, 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, US House lawmakers debated FDA’s rule to regulate laboratory developed tests (LDT), FDA offered a new path for reporting drug shortages, and the agency approved new drugs to treat a rare neuron disease and a muscle wasting disorder.

Lawmakers on the US House Energy and Commerce (E&C) health subcommittee sparred over whether FDA was within its right to develop a rule to regulate LDTs. While Republicans warned the rule would have devastating consequences for labs and patients, Democrats argued the agency was left with no alternative after Congress failed to pass the Verifying Accurate, Leading-edge IVCT Development (VALID) Act. The rule is likely about a month away from being finalized but as Axios reported all sides agreed the preference would be to pass diagnostic reform legislation.

Healthcare providers and consumers can now directly report potential drug shortage issues to the Center for Drug Evaluation and Research's (CDER) NextGen portal without having to create a NextGen account. Typically, providers and patients would have to email FDA about potential drug shortages, but now they can use the NextGen system, which is what drugmakers use to submit such information.

The Center for Biologics Evaluation and Research (CBER) published a website summarizing what it has done in the area of artificial intelligence/ machine learning (AI/ML). The page details meetings the center has participated in and how it is using AI/ML to regulate biological products.

The Center for Devices and Radiological Health warned US suppliers, consumers and healthcare providers to stop using plastic syringes made in China unless there were not alternatives. More specifically, it said it had sent warning letters to Jiangsu Shenli Medical, Sol-Millennium Medical, and Medline Industries for failing to meet quality system requirements.

FDA published a draft guidance on what sponsors should consider when submitting information from a non-interventional study also known as an observational study. The agency noted that the guidance was part of a series of guidances under its Real-World Evidence (RWE) Program.

Drugs & Biologics

Orchard Therapeutics' Lenmeldy (atidarsagene autotemcel) was approved by FDA as the first treatment for pre-symptomatic late infantile, pre-symptomatic early juvenile or early symptomatic early juvenile metachromatic leukodystrophy (MLD). The rare genetic disorder that affects the brain and nervous system is caused by an enzyme deficiency that leads to fatty substances build up in the patient's cells that ultimately can cause loss of motor and cognitive function and death in children.

Italfarmaco's Duvyzat (givinostat) was approved by FDA as the first non-steroidal drug to treat all genetic variants of the rare genetic disorder Duchenne Muscular Dystrophy (DMD) in patients six years or older. The drug is a histone deacetylase (HDAC) inhibitor that targets pathogenic processes in the patient's body to reduce inflammation and muscle loss.

Takeda's Iclusig (ponatinib) was granted accelerated approval to treat newly diagnosed Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL) with chemotherapy by FDA. The application for the drug was granted priority review and orphan drug designation, and the agency reviewed it under its Real-Time Oncology Review (RTOR) pilot program to streamline the submission process.

FDA said it will host a webinar on 25 April to educate prospective generic drug sponsors about how the agency develops, revises and publishes Product-Specific Guidances (PSG). The agency intends to cover general principles of PSGs, their lifecycle overview, utility, and updates to the PSG program.

Safety labeling changes for fluorouracil injection products were approved by FDA after its Office of Generic Drugs (OGD) and the Oncology Center of Excellence (OCE) addressed newer safety concerns about the risk of adverse events related to use of fluorouracil in patients with dihydropyrimidine dehydrogenase (DPD) deficiency. The revisions include changes to the Highlights of Prescribing Information, Warnings and Precautions, and Patient Counseling Information sections of the labeling, while also adding a Pharmacogenomics subsection to the Clinical Pharmacology section.

FDA proposed a rule that would establish criteria for the lists of drug products or categories of drug products that are considered hard to compound. Furthermore, the agency is already proposing to list the first three categories of drugs on the Demonstrable Difficulties for Compounding (DDC) Lists.

Medtech

The medtech lobby group AdvaMed and FDA are co-sponsoring a two-day conference on 2 April on medical device statistical methodology and practices. The co-sponsors say the aim of the meeting is to encourage clinical trial innovation and advance regulatory statistical science when evaluating medical devices and diagnostics.

External experts on FDA's Ophthalmic Devices Panel unanimously voted that Balance Ophthalmics' FSYX Ocular Pressure Adjusting Pump (OPAP) system's benefits outweighed its risks according to IEEE Spectrum. The device is intended to alleviate pressure in the eyes caused by glaucoma, which if left untreated can cause optic nerve damage.

FDA published a draft guidance asking medical device sponsors to test products that may cause changes in tissue temperatures, regardless of whether they heat or cool the tissue and whether the change in temperature is intentional. The agency provided details on how to conduct testing and gather data, and recommended sponsors talk to regulators early.

This Week at FDA: Lawmakers spar over LDT rule, new guidances, and more

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