This Week at FDA: Regulators pull Makena, publish guidances, preview more adcomms

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. The agency has been busy this week yanking a controversial pre-term birth prevention drug, publishing several guidances and more.

After years of wrangling with Covis Pharma, the US Food and Drug Administration (FDA) withdrew its approval of the premature birth prevention drug Makena which the agency said has not proven to work. The Swiss drugmaker made a final bid to keep the drug on the market, arguing it may have benefits for minority populations, but a panel of external experts disagreed and recommended nearly unanimously in October 2022 that the drug should be pulled from shelves.

While the year had started out slowly, FDA has been ramping up its efforts to publish guidances over the past few weeks. This week the agency published, among others, a guidance on how to include predetermined change control plans (PCCP) in artificial intelligence/ machine learning (AI/ML) device applications; the last in a series of guidances on patient-focused drug development (PFDD); and a guidance on data that can help address supply chain issues with active pharmaceutical ingredients (API).

FDA has also published its Resource Capacity Planning and Modernized Time Reporting Implementation Plan, which outlines how it will work on its resource capacity planning (RCP) capabilities under the latest product user fee agreements. The plan sets out how the agency intends to address the forecasted workload over the next five years and best use its user fee resources.

The agency has extended the comment period for a draft guidance on long-term clinical neurodevelopmental safety studies in neonatal product development. The comment period was slated to close 11 April but has been extended to 15 May.

As part of the Medical Device User Fees Amendments (MDUFA V) performance goals agreed to by FDA, the agency is considering revising a patient preference information (PPI) guidance and is asking stakeholders for early feedback on the use of PPI in regulatory submissions. Some of the issues it wants to address include important factors that the sponsor should consider when designing a patient preference study, appropriate PPI collection methods, and examples of PPI studies that could help support regulatory decisions.

Speaking during a webinar hosted by the Alliance for a Stronger FDA, Patrizia Cavazzoni, director of the Center for Drug Evaluation and Research (CDER), said there is likely to be a significant uptick in advisory committee meetings this year compared to recent years.

“We want more and therefore we’re doing work on our internal processes and thinking about the situation on when an adcomm is important and useful,” she said, and added FDA is working to ensure they have the right expertise for the panels.

Drugs & Biologics

According to federal officials who spoke with the Washington Post, FDA is gearing up to authorize a second omicron-specific coronavirus vaccine booster for people who are at least 65 or have weakened immune systems. The agency is expected to announce the step in the next few weeks, and the Centers for Disease Control and Prevention (CDC) is supposed to endorse the decision quickly soon after.

FDA has given emergency use authorization (EUA) for Gohibic (vilobelimab) injection for use in hospitalized COVID-19 patients to be treated within 48 hours of receiving artificial life support. The medicine from InflaRx GmbH targets a part of the immune system that is thought to play a role in the inflammation that leads to disease progression, according to FDA’s authorization letter.

Astellas Pharma's Padcev (enfortumab vedotin-ejfv) in combination with Merck's Keytruda (pembrolizumab) has received FDA accelerated approval to treat patients with locally advanced or metastatic urothelial carcinoma who are ineligible for cisplatin-containing chemotherapy. An initial study of the drug combination found that patients had better objective response rate (ORR) from the treatment.

FDA sent untitled letters to Fidia Pharma and Minneapolis Regenerative Medicine for marketing human cell, tissue, or cellular or tissue-based products (HCT/P) without biologics license applications (BLA).

FDA noted that Fidia has been marketing NuDYN, derived from amniotic membrane, to treat various diseases, including orthopedic diseases, based on the agency’s review of Fidia’s websites. Similarly, the agency wrote that based a review of web content from Minneapolis Regenerative Medicine, the company has been marketing a cell therapy from human umbilical cord, which the company represented as a regenerative therapy to treat diseases such as osteoarthritis and rheumatoid arthritis.

A study published recently in JAMA Health Forum found that more than half of confirmatory studies used to support accelerated approvals for drugs were not completed by their due date and that manufacturers faced few consequences for such delays. It also found that confirmatory trials for non-oncologic indications were more likely to be late than those for oncologic indications, and trials for small molecule drugs were more likely to be late than those for biologic drugs.

Medtech

Three former senior executives of Magellan Diagnostics were arrested this week and accused of knowingly selling faulty lead testing devices. The Justice Department (DOJ) said their actions caused tens of thousands of children and pregnant women living in public housing to receive inaccurate test results and endangered their lives.

FDA announced Abbott has issued a class I recall for its FreeStyle Libre, Libre 14 day, and Libre 2 Flash Glucose Management Systems. The rechargeable lithium-ion batteries used in the devices may get extremely hot and even catch fire if not used properly. The company reported 206 incidents of problems with the batteries, including at least seven fires, one injury, but no deaths.

FDA also said Philips has initiated a class I recall of certain Respironics DreamStation CPAP and BiPAP machines because they may deliver inaccurate or insufficient therapy. The agency said some of the devices were assigned incorrect or duplicate serial numbers during initial programming, which may lead to the wrong prescription or factory default settings and cause serious injuries or death.

Masimo has received a de novo authorization for its Opioid Halo device, which checks for drug-induced respiratory depression, a leading cause of overdose deaths. It is the first opioid overdose prevention monitor green-lit by FDA.

This Week at FDA: Regulators pull Makena, publish guidances, preview more adcomms

Related topics

Recommended content

Welcome to the new RAPS Digital Experience