This Week at FDA: Appropriations update, new guidance and marketing decisions

Corrected 27 June 2023*

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, we saw movement on FDA’s budget on the Hill, continued political battles, and a slew of products that have received a regulatory decision.

Budget season is well under way as Congress works on funding packages for various federal agencies, including the Food and Drug Administration (FDA). This week the Senate Appropriations Committee unanimously approved a FY 2024 budget that includes $3.55B for FDA. If passed as is, it would give the agency an additional $20M over this year’s agency budget. The move comes a week after the House Appropriations committee advanced its FY 2024 spending bill for FDA.

As Democrats and Republicans continue to wrestle over reproductive health in the aftermath of the US Supreme Court’s decision overturning Roe v. Wade, President Joe Biden signed an executive order intended to increase access contraceptives and lower out-of-pocket costs. Commemorating the supreme decision, Senate Democrats have also proposed several bills meant to protect access to reproductive health and ensure continued access to contraception.

House Republicans have raised concerns that an idea to investigate the virulent nature of the mpox virus using a so-called gain-of-function study could be dangerous, STAT reports. The research was proposed by Bernard Moss, a scientist at the National Institute of Allergy and Infectious Diseases (NIAID), and echoes concerns they’ve raised about an unproven theory the coronavirus is linked to research funded by NIAID at a lab in Wuhan, China.

Amid the ongoing shortage of Teva’s attention-deficit/hyperactivity disorder (ADHD) drug Adderall and its generics, Takeda has announced that its ADHD drug Vyanese (lisdexamfetamine dimesylate) is also "currently experiencing low inventory" due to a manufacturing delay and increased demand, according to Fierce.

On the issue of drug shortages, the Healthcare Distribution Alliance (HDA) has written to FDA asking it to use a phased approach when implementing the Drug Supply Chain Security Act (DSCSA), which is supposed to go into full effect on 27 November. The group argues that a phased approach will minimize the potential for supply disruptions and interruptions to patient care.

After years of back-and-forth with the medtech industry, the Centers for Medicare & Medicaid Services (CMS) has released its latest proposal for covering breakthrough medical devices more quickly after failing to implement the 2021 Medicare Coverage of Innovative Technologies (MCIT) rule. The new proposed Transitional Coverage for Emerging Technologies (TCET) voluntary pathway for certain breakthrough devices would use current national coverage determination (NCD) and coverage with evidence development (CED) processes to expedite Medicare coverage of certain breakthrough devices.

CMS also announced a plan for how people can get coverage for Alzheimer's drugs. The agency says Medicare will reimburse Alzheimer's drugs with traditional FDA approval if a physician and clinical team collects data on the drug in a registry to understand how it works in the real world.

Drugs & biologics

A critical 27 June deadline is approaching for biologics manufacturers to comply with provisions of the 2023 Consolidated Appropriations Act. Under the law, biologics license application (BLA) holders have until Tuesday to review information in the Purple Book and submit a report to FDA stating that their product with an “Rx” in the book is available for sale or has been withdrawn or was never available for sale.

FDA published several guidances including a draft guidance on how to conduct formal dispute resolutions and administrative hearings to resolve scientific and/or medical disputes over final orders between the Center for Drug Evaluation and Research (CDER) and sponsors of over-the-counter drugs. The agency notes the guidance applies to nonprescription drugs without approved new drug applications.

The agency also published a draft guidance on developing psychedelic drugs for treatment of medical conditions such as psychiatric disorders and substance use disorders, and it issued a final guidance to provide a notice of exceptions and alternatives regarding blood and blood components. The latter guidance is meant to address the urgent and immediate need for platelets to treat active bleeding when conventional platelets are not available, or their use is not practical.

FDA approved several new drugs this week including Agepha Pharma's Lodoco (colchicine) with statins to reduce the risk of stroke, coronary revascularization, myocardial infarction and cardiovascular death in patients with atherosclerotic disease or with multiple risk factors for cardiovascular disease.

FDA also approved Sarepta Therapeutics' Elevidys (delandistrogene moxeparvovec-rokl), the first gene therapy to treat children four- to five-years-old with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene who do not have a pre-existing medical reason preventing treatment with this therapy.

Eli Lilly and Boehringer's Jardiance (empagliflozin) and Synjardy (empagliflozin and metformin hydrochloride) received approval to improve blood sugar control in children 10 years and older with type 2 diabetes. The combination is meant to be taken in addition to diet and exercise and marks the first new class of drugs approved to treat pediatric type 2 diabetes in more than two decades.

On the other hand, FDA also rejected several drugs and paused clinical trials, including rejecting F2G's antifungal drug olorofim, Endpoints reports. The UK-based company said it has received a complete response letter (CRL) stating that FDA could not approve the product at this time and needs additional data and analysis.

Similarly, FDA turned down Aldeyra Therapeutics’ application for ADX-2191 (methotrexate injection, USP), which the Massachusetts-based company was developing to treat primary vitreoretinal lymphoma (PVRL), a rare eye cancer, BioSpace writes. The company said the CRL attributed the denial to a “lack of substantial evidence of effectiveness” and a “lack of adequate and well-controlled investigations.”

FDA has also put the brakes on a phase 2 trial of Arcellx’s CAR-T cell therapy, an anti-BCMA drug, after a patient died. Gilead recently invested in the company, but the drug was already behind in development compared to its rivals.

Medtech

The Center for Devices and Radiological Health (CDRH) has strongly advocated for early conversations between medtech sponsors and insurers, which is a key component of its pilot Total Product Lifecycle Advisory Program (TPLC). Now, the center has updated its Payor Communication Task Force web site to connect device manufacturers and insurers in conversations that it hopes will shorten the time between when FDA gives marketing authorization for a product and when it gets a coverage decision. FDA said the task force's activities include the Early Payor Feedback Program (EPFP) and Parallel Review with the Centers for Medicare & Medicaid Services.

FDA's Circulatory System Devices Panel of the Medical Devices Advisory Committee is set to meet virtually for two days in August to discuss and vote on devices indicated to reduce blood pressure in patients with hypertension. On 22 August, external experts are scheduled to discuss the premarket approval application (PMA) for the ReCor Paradise Ultrasound Renal Denervation System by ReCor, Inc. The following day, the panel is scheduled to do the same for Medtronic's PMA for the Medtronic Symplicity Spyral Renal Denervation System.

FDA published a warning letter it sent to Vitang Technology, maker of UniSmile Clear Aligner, a class II dental aligner. In its 12 June letter, the agency said the company made changes to its product without submitting a new 510(k) and failed to conform to current good manufacturing practice (CGMP) requirements.

*A previous version of this story incorrectly stated that FDA recently launched its Payor Communication Task Force. Regulatory Focus regrets the error.

This Week at FDA: Appropriations update, new guidance and marketing decisions

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