This Week at FDA: User fee reauthorization, Amylyx ALS drug approved, and more

This Week at FDAThis Week at FDA | 30 September 2022 |  By 

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. Just hours ago, the House voted 230-201 to approve a stopgap funding bill that will reauthorize FDA’s user fee programs for the next five years.
The news comes as a relief to FDA and the medical product manufacturers who spent months negotiating the user fee deals. The user fee programs hit a snag this summer when Republicans objected to several riders attached to the bill. Ultimately, Democrats pushed forth with a “clean” user fee reauthorization to avoid FDA from laying off user fee-funded staff and tabled the riders, which they plan to reintroduce later in the year when Congress must pass a new budget.
In other major news, FDA on Thursday approved Amylyx Pharmaceuticals’ Relyvrio (sodium phenylbutyrate/taurursodiol) to treat patients with amyotrophic lateral sclerosis (ALS) after convening two advisory committee panels to review the drug. After initially voting 6-4 against the drug’s approval last march, FDA’s Peripheral and Central Nervous System Drugs Advisory Committee reversed course and endorsed the drug earlier this month, after reviewing new analyses from the company and hearing from Amylyx’s co-CEO Justin Klee that the company would withdraw the drug if a confirmatory trial found it to be ineffective. Now, that pledge is in question, as Endpoints reports, with Klee telling investors on Friday that the PHOENIX trial is “not a formal commitment.”
Drugs & biologics
FDA on 28 September published a draft guidance outlining how over-the-counter (OTC) drugmakers can submit OTC monographs electronically. Examples of OTC monograph submissions include monograph order requests (OMORs), public comments to a proposed administrative order, format meeting requests, formal dispute resolution requests and administrative hearing requests.
Submissions should be sent to either the CDER NextGen portal or the OTC monographs portal. OMORs, formal meeting requests and meeting packages, formal dispute resolution requests, administrative hearing requests, and responses to record requests by FDA relating to minor changes should be submitted to CDER NextGen portal, while data, information, and public comments to a proposed order or interim final order should be submitted to the OTC monographs portal.
Another big story was biopharma firm Biocon was hit with three FDA Form 483s containing inspection observations about manufacturing problems at the two of the company’s sites in Bengaluru, India and one in Johor, Malaysia.
During one inspection at the Bengaluru site conducted in early August, investigators issued a 483 for a multitude of GMP problems, including lax procedures for preventing microbiological contamination, and a failure to investigate discrepancies. An inspection conducted later in the month found similar problems at a nearby site, yielding another 483 for inadequate microbiological controls, lack of oversight over laboratory and manufacturing systems and inadequate control over their electronic systems.
During the inspection at the Malaysian site, investigators hit the company with a 483 for inadequate microbiological safeguards and inadequate failure investigations.
When it rains it pours, and this week FDA updated a slew of digital health guidances, including one on device software functions and mobile medical applications (MMAs) intended for use on mobile platforms or on general-purpose computing platforms, and another much-anticipated guidance on clinical decision support (CDS) software. It also published a number of radiology digital health guidances including a guidance on computer-assisted detection devices applied to radiology images and radiology device data in 510(k) submissions, one clinical performance assessments of such devices, and another on display devices for diagnostic radiology.
To help sponsors figure out of their digital health products will be regulated by the agency, FDA also published a number of web sites, including one specific to CDS software and a digital health policy navigator.
FDA also announced it will continue to use its virtual COVID-19 test development town halls to also address questions about monkeypox test development. The next town halls on the matter will be held on 5 and 12 October.
In its ongoing effort to address the national opioid crisis, FDA also announced it is hosting two public workshops with the National Institutes of Health (NIH) titled, “Diagnostic and Monitoring Medical Devices for Opioid Use” and “Risk Prediction Devices of Opioid Use and Opioid Use Disorder – Opportunities and Challenges.” The agency says the purpose of the workshops is to have discussions about how to properly conduct clinical studies for medical devices that can diagnose, monitor and manage people who use opioids and improve safer use of the drugs.
FDA also announced a class I recall of LivaNova's (TandemLife) LifeSPARC Controller due to a software malfunction. The device is part of the LifeSPARC System used to pump blood through the circulatory system. The agency said the "controller’s monitoring feature may erroneously detect frozen or unresponsive software and trigger the device to enter Critical Failure mode—clearing the controller screen and issuing an alarm that cannot be muted or turned off." If the pump stops for an extended period of time, it could cause serious injury or death.
FDA said that LivaNova has reported 66 complaints related to the controlled, and it has received reports of two injuries and no deaths.


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