This Week at FDA: FDA official criticizes UniQure, states eye cheaper drug imports, 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. An FDA official criticized a rare disease drug sponsor, state representatives met with the agency to import cheaper drugs, and regulators warned a slew of telehealth companies for marketing compounded GLP-1 drugs.STAT News reported that an anonymous senior FDA official accused UniQure of pushing “distorted” and “manipulated” clinical data for a “failed” gene therapy intended to treat Huntington’s disease during a media call set up by the agency. The article noted that it was highly unusual behavior by FDA to arrange the call to lambast UniQure, which could become another political headache for the Trump Administration.
Representatives from several states met with FDA to discuss the section 804 importation program (SIP), which allows states and Indian tribes to import certain cheaper prescription drugs from Canada. The agency said it allowed the states that have expressed interest in the program to discuss how they can obtain authorization to import the drugs as part of President Trump's executive order to lower drug prices.
FDA issued another barrage of warning letters to telehealth companies for what it says are false and misleading statements about the compounded GLP-1 products that they market on their websites. The agency issued 30 warning letters, arguing that the companies are trying to circumvent its traditional drug approval process.
The Health and Human Services (HHS) Office of Inspector General issued a statement that Sanja Ilic, former chief regulatory officer at ExThera Medical, has agreed to plead guilty to attempting to defraud and mislead the FDA. The agency said Ilic did not file adverse event reports related to the company's blood filtration devices used by cancer patients who traveled to Antigua for treatment.
FDA published a two of final guidances that conform to standards developed by the International Council for Harmonisation. The agency published an E2D(R1) post-approval safety data guidance and an M14 guidance on non-interventional studies that use real-world data.
FDA is hosting an annual hybrid public workshop on the state of generic drug science and research initiatives. The meeting is part of the agency's commitment under the Generic Drug User Fee Amendments (GDUFA III) and seeks stakeholder feedback on which research initiatives to prioritize.
FDA announced it will host a hybrid workshop on 9 April to discuss ways to advance pediatric cell and gene therapy (CGT) clinical trials. More specifically, regulators will discuss challenges in conducting CGT clinical trials, the data and information needed to enroll patients, the appropriate timing for enrolling children, and considerations for enrolling presymptomatic and early-stage disease children.
Drugs & biologics
The most recent over-the-counter (OTC) Monograph Drug User Fee Amendment (OMUFA) program arrears and paid facilities lists were published by FDA. The agency listed dozens of manufacturing facilities that it says have failed to meet their obligations under the program.
Janssen Biotech's Tecvayli (teclistamab) in combination with daratumumab hyaluronidase-fihj was approved by FDA to treat adult patients with relapsed or refractory multiple myeloma who have received at least one prior line of therapy, including a proteasome inhibitor and an immunomodulatory agent. The agency noted it was the third drug approved under the controversial new Commissioner’s National Priority Voucher (CNPV) pilot program, and the decision was issued only 55 days after the marketing application was filed.
Glenmark Specialty is the first company to receive approval to market a generic version of GlaxoSmithKline's Flovent HFA (fluticasone propionate) asthma inhalation aerosol, 44 micrograms per actuation, as a prophylactic therapy in patients 4 years and older. Tracy Beth Høeg, acting director of the Center for Drug Evaluation and Research (CDER), said the agency expects the approval to reduce costs and increase access to the treatment.
FDA's Oncologic Drugs Advisory Committee is scheduled to meet 30 April to discuss whether to recommend approval of two AstraZeneca cancer drugs. The committee will discuss the safety and efficacy of camizestrant in combination with a CDK4/6 inhibitor (palbociclib, ribociclib or abemaciclib) to treat adults with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative, locally advanced or metastatic breast cancer upon emergence of ESR1 mutation during first-line endocrine-based therapy, diagnosed with an FDA approved test, and Truqap (capivasertib) in combination with abiraterone to treat adults with metastatic hormone-sensitive prostate cancer (mHSPC) that is PTEN-deficient and diagnosed with an FDA-approved test.
Ascendis Pharma's one-weekly injection Yuviwel (navepegritide) was approved by FDA to improve growth in children two years of age and older with achondroplasia, the most common form of dwarfism, with open epiphyses. The agency noted the drug received accelerated approval on the condition that it continues a study to confirm that it improves final adult height.
Medtech
The Center for Devices and Radiological Health (CDRH) published meeting minutes from its 11 February meeting with industry to negotiate the next Medical Device User Fee Amendments (MDUFA VII) program. During the meeting, medtech industry representatives asked the agency for more clarity on the Total Product Life Cycle Advisory (TAP) program, stated that they oppose the agency's operational costs for the program, and asked for more metrics moving forward.
FDA issued a statement recommending that the public learn and use established choking rescue protocols, such as the Heimlich maneuver, before using anti-choking devices to save a person. Only if the protocols are unsuccessful should users consider using the anti-choking devices as a secondary option.
FDA updated several lists of new technologies it has approved, cleared or authorized for market. The lists cover medical devices with augmented reality and virtual reality (AR/VR), medical devices that incorporate sensor-based digital health technology (sDHT), and those that incorporate artificial intelligence (AI).
Medline has issued a class I recall for several of its reprocessed electrophysiology and ultrasound catheters, according to FDA. The company stated that the products may contain small residual particulates that may lead to inflammation, infection, cerebral or pulmonary embolism, and deep vein thrombosis.
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