This Week at FDA: OMUFA II, FDA names new Rare Disease Innovation Hub leader, 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, FDA named the leader of its new Rare Disease Innovation Hub, an advisory committee voted against Lexicon Pharmaceuticals' diabetes and kidney disease drug Zynquista, and the agency is kicking off discussions to renew its Over-the-Counter Monograph Drug User Fee Act (OMUFA) Program.

In a blog post discussing progress made toward its recently announced Rare Disease Innovation Hub, Patrizia Cavazzoni, director of the Center for Drug Evaluation and Research (CDER), and Peter Marks, director of the Center for Biologics Research and Evaluation (CBER), announced that Amy Comstock Rick will be joining the agency to serve as the hub’s Director of Strategic Coalitions.

Rick previously led the Food and Drug Law Institute (FDLI) from 2014 to 2022, and most recently worked as a principal consultant at Leavitt Partners. In an email to CDER and CBER staff, Cavazzoni and Marks explained that Rick will be accountable to both center directors, though her formal position will be Associate Director for Rare Disease Strategy within CDER’s Office of the Center Director.

“With her advocacy experience across constituencies, including patient groups, Ms. Rick is excited to begin creating important partnerships needed for FDA’s outreach throughout the rare disease community,” Cavazzoni and Marks wrote.

This week, FDA announced the first meeting in its negotiations for the second iteration of OMUFA, before the current program expires at the end of FY 2025. The meeting will take place virtually on 20 November.

The Office of Management and Budget (OMB) is reviewing a draft FDA guidance on validating certain in vitro diagnostics for emerging pathogens during a public health emergency. Under Section 564 of the Food, Drug and Cosmetics (FD&C) Act, the agency is allowed to grant emergency use authorization (EUA) to products circumventing the traditional premarket pathways if the Secretary of Health and Human Services (HHS) has declared an emergency.

On 31 October, the Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) voted 11-3 that the benefits of Lexicon Pharmaceuticals' Zynquista (sotagliflozin) as an adjunct treatment to insulin therapy in adults with type 1 diabetes and chronic kidney disease did not outweigh its risks. MarketWatch notes that FDA turned down a similar premarket application from the company in 2019. The company, however noted that some panel members expressed support for the drug in alternative subpopulations.

The International Medical Device Regulators Forum (IMDRF), which is currently chaired by FDA, published a white paper summarizing its recent meeting in Seattle. The paper noted that attendees representing various regulatory bodies discussed issues such as international reliance (IR), legal challenges, and how IMDRF can support development of regulatory systems.

Drugs & biologics

FDA published a final guidance that aligns it with the International Council for Harmonization of Technical Requirements for Pharmaceuticals for Human Use M13A guideline. In conjunction, the agency has published draft product-specific guidances that align with the technical requirements and make recommendations for generic drug sponsors, including conducting one bioequivalence study for products with a non-high risk of bioequivalence due to food effect under either fasting or fed condition rather than conducting two bioequivalence studies.

FDA issued a consumer alert to the public, including health care professionals, that human immunodeficiency virus (HIV) blood sample self-collection kits are on the market and have not been reviewed by regulators for safety and efficacy. The agency warned that without proper marketing authorization, it cannot guarantee that the kits give valid and accurate results.

FDA issued a statement that manufacturers of the HIV drug ritonavir should test their product for nitrosamine drug substance-related impurity, (N-((2-isopropylthiazol-4yl)methyl)-N-methylnitrous amide (NITMA), also known as N-nitroso-2,4-thiazoleamine (NNTA). The agency said it knows the impurity may be found in finished drug products that contain ritonavir and has determined that a daily limit of 26.5 ng is recommended for NITMA, unless the manufacturer can justify a higher amount.

Novartis’ cancer drug Scemblix (asciminib) has been granted accelerated approval by FDA to treat adults diagnosed with Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML) in chronic phase (CP).

Chemigroup France's Skin-Cap aerosol sprays may contain undisclosed glucocorticosteroid, according to FDA, after the agency tested the products. The sprays are sold by major online retailers, including Amazon.com and Walmart.com, to treat psoriasis, seborrheic, dermatitis, and other skin conditions, but the steroids could cause adverse reactions such as skin thinning, increased blood glucose levels, bone damage, and more.

Medtech

The Center for Devices and Radiological Health (CDRH) has published two training modules for small business qualification and certification under the Medical Device User Fee Amendments (MDUFA V). The modules describe how to complete Form FDA 3602 for small businesses in the US and Form FDA 3602A for small businesses outside the US.

FDA’s Office of In Vitro Diagnostics in the Office of Product Evaluation and Quality (OPEQ) is hosting a hybrid roundtable on 12 November to discuss in vitro diagnostics (IVD). The agency says the meeting will be an opportunity for IVD manufacturers to talk to the agency.

The Digital Health Center of Excellence (DHCoE) has updated its cybersecurity page with recent news and resources. The agency has published several new reports on medical device cybersecurity that may be useful to industry.

FDA held another medical device sterilization town hall on 30 October. During the meeting, the agency discussed applying for Small Business Innovation Research (SBIR) grants to develop new medical device sterilization methods and using bundling for sterility-related submissions. The agency plans to publish the transcript of the meeting, but stakeholders can already download printable slides that were used during the meeting.

GE HealthCare has updated instructions for its Class I recall of EVair Compressors after final testing found that formaldehyde emissions are below the safety threshold. The devices provide airflow to patients in clinical scenarios when wall air is not available, and faulty devices could cause airway irritation or inflammation that could lead to airway hyperresponsiveness in newborns or infants. FDA also issued a statement alongside the updated recall notice stating that it is continuing to monitor the postmarket performance of the products.

GE Healthcare also updated its use instructions for the Class I recall of its Giraffe OmniBed and Giraffe Omnibed CareStation devices due to the risk of heater doors falling and hurting infants. The combination incubator and warmers are used to help neonates who can't regulate their body temperatures yet, but the company has received reports the screw securing the warmer heater doors may not be properly tightened, which may cause them to fall off on the patient.

This Week at FDA: OMUFA II, FDA names new Rare Disease Innovation Hub leader, and more

Related topics

Recommended content

Welcome to the new RAPS Digital Experience