Drugs
The ACLU has filed a lawsuit seeking to force FDA to release documents related to its ongoing safety review of mifepristone, accusing the agency of withholding information requested under a Freedom of Information Act filing submitted in August. The FOIA request seeks details on the scope of the agency’s
Drugs
FDA says it is approving new labeling submitted by Sarepta Therapeutics’ for its gene therapy Elevidys (delandistrogene moxeparvovec-rokl) that includes the addition of a Boxed Warning describing the risk of serious liver injury and acute liver failure, including fatal outcomes based on such reports in non-ambulatory patients treated
Drugs
FDA has granted Parabilis Medicines a fast track designation for FOG-001, a first-in-class inhibitor targeting the β-catenin:TCF interaction, for treating desmoid tumors. The action is based on the company’s early clinical data showing signs of meaningful antitumor activity. In an ongoing Phase 1-2
Drugs
FDA has released a final guidance entitled “Considerations for Waiver Requests for pH Adjusters in Generic Drug Products Intended for Parenteral, Ophthalmic, or Otic Use.” The document describes how the agency intends to evaluate a request for a waiver, concerning a pH adjuster, of the regulatory requirement that a drug
Drugs
Gilead Sciences says its investigational single-tablet regimen combining bictegravir and lenacapavir met its primary endpoint in a pivotal Phase 3 trial, marking a potential advance for people with HIV who rely on complex, multi-pill treatments. The open-label ARTISTRY-1 study tested the once-daily tablet, which combines
Biologics
FDA has accepted for review an Outlook Therapeutics BLA resubmission for ONS-5010 (bevacizumab-vikg), an investigational ophthalmic formulation of bevacizumab for treating wet age-related macular degeneration (wet AMD). The agency reportedly told the company that the submission is a complete, Class 1 response to an 8/27 complete
Drugs
NYU Grossman School of Medicine ethicist Arthur Caplan says he is concerned that reductions in force at FDA and HHS can adversely affect clinical trial oversight and participant protections. In a Medscape video, Caplan says, “I am extremely worried about what’s been taking place at FDA and at HHS
Drugs
An 11/7 Wiley Rein petition submitted on behalf of a client asks FDA to order the complete and immediate withdrawal of the Wells Pharma compounded 100 mg indomethacin suppository. The petition also calls on the agency to require Wells to cease manufacturing and distribution of the suppository until it
Biologics
FDA has approved Shanghai Henlius Biologics’ Poherdy (pertuzumab-dpzb), the first interchangeable biosimilar to Perjeta (pertuzumab), Genentech’s HER2-targeted therapy for breast cancer. Poherdy is described as a HER2/neu receptor antagonist indicated for use in combination with trastuzumab and chemotherapy in several settings, including: * Metastatic breast cancer (MBC)
FDA Policy/General
University of Chicago researchers say that while the federal government has proposed expanding direct-to-consumer ad oversight to include influencer partnerships and sponsored content, “effective oversight will be challenging because promotion is driven by thousands of individual creators rather than a few dozen companies.” Writing in a JAMA Research
Drugs
CDER has qualified glutamate dehydrogenase (GLDH) as a safety biomarker to detect possible drug-induced liver injury in conjunction with alanine aminotransferase (ALT) and other standard liver injury markers in clinical trials, particularly in participants with elevated transaminases due to muscle injury. An agency notice says innovation in biomarker development
Drugs
FDA has accepted for review an ITM Isotope Technologies NDA for 177Lu-edotreotide (ITM-11), a targeted radiopharmaceutical therapy for gastroenteropancreatic neuroendocrine tumors (GEP-NETs). 177Lu-edotreotide is described by the company as a no-carrier-added lutetium-177–based radiotherapeutic designed to deliver targeted radiation to somatostatin receptor–positive
FDA Policy/General
FDA has introduced a new regulatory framework — the “plausible mechanism pathway” — designed to speed approval of highly personalized therapies for rare genetic diseases, according to an 11/12 article published in The New England Journal of Medicine by CBER director Vinay Prasad and commissioner Marty Makary. The accelerated pathway aims
Drugs
The CDER Office of Generic Drugs (OGD) has published a MAPP, Using Four-Part Harmony in OGD-Related Assessment Communications, that describes how OGD assessors can apply the principles of four-part harmony to enhance the clarity of OGD-related communications. (In this context, four-part harmony refers to the
Drugs
Alkermes says that its investigational once-daily therapy alixorexton significantly improved wakefulness and reduced daytime sleepiness in patients with narcolepsy Type 2 (NT2), meeting both primary endpoints in the company’s Vibrance-2 Phase 2 clinical trial. The company says the data highlight alixorexton — the first oral orexin 2 receptor
FDA Policy/General
An 11/19 MAHA Summit in Washington will bring together leaders and influencers of the Make America Health Again movement, according to an event schedule obtained by Politico. An online post says the gathering will hear from vice president JD Vance and HHS secretary Robert F. Kennedy, Jr. The summit
Biologics
A biotechnology company specializing in adult-derived stem cells is urging FDA to loosen federal regulation of some regenerative therapies, arguing that products meeting strict manufacturing and facility standards should be governed under the agency’s lower-risk framework for human cell and tissue products. A just-filed citizen petition
Drugs
Cumberland Pharmaceuticals has petitioned FDA to decline to accept or approve any ANDAs or 505(b)(2) applications that cite its Acetadote (acetyleysteine) as the reference-listed drug with labeling that omits the branded prescribing information relating to a two-bag dosing regimen. The company’s petition also asks that
Drugs
FDA is joining the United States Pharmacopeia (USP) and the Association for Accessible Medicines (AAM) on 12/11 for a public workshop focused on how compendial standards can strengthen drug quality, streamline development, and improve regulatory predictability across the pharmaceutical industry. According to an FDA notice, the workshop aims to
Devices
Based on the discussion at an FDA Digital Health Advisory Committee public meeting on “Generative Artificial Intelligence-Enabled Digital Mental Health Medical Devices,” five Orrick attorneys say that manufacturers developing large language model-based prescription therapies for major depressive disorder “should anticipate a risk-based, lifecycle regulatory approach.” In an
Devices
FDA has approved Promega’s OncoMate MSI Dx Analysis System as a companion diagnostic for identifying patients with microsatellite stable endometrial carcinoma who may benefit from treatment with Merck’s Keytruda (pembrolizumab) in combination with Eisai’s Lenvima (lenvatinib). The polymerase chain reaction-based assay is designed to determine microsatellite
Drugs
A 5/19-5/21 FDA inspection at the Miers Laboratories drug manufacturing facility in Wellington, New Zealand, found repeat significant violations of current good manufacturing practice (CGMP) regulations for finished drugs. An 11/3 Warning Letter says the two specific violations were: * failure to conduct at least one test