Drugs
Neurocrine Biosciences is reporting that its Phase 2 study of NBI-1070770, an investigational NMDA receptor modulator for major depressive disorder (MDD), failed to meet its primary endpoint. The 73-patient, placebo-controlled trial tested three dose levels of the oral NR2B-selective negative allosteric modulator as adjunctive treatment. Neurocrine
Devices
Three Alston & Bird attorneys say that complying with FDA’s forthcoming Quality Management System Regulation (QMSR) is not just a regulatory obligation but a critical business strategy. Writing in a detailed Drug & Device Advisory, the attorneys discuss FDA’s 10/27 draft guidance describing the Quality Management System
Biologics
enGene says its investigational gene therapy detalimogene voraplasmid produced a 62% complete response rate at six months in patients with high-risk, BCG-unresponsive non–muscle invasive bladder cancer (NMIBC), according to new preliminary data from the company’s Phase 2 LEGEND trial. The company’s analysis included patients treated
FDA Policy/General
FDA Matters president Steven Grossman says strong morale will be built at FDA based on a foundation of trust. After reviewing all that has battered the agency in the first year of President Trump’s second term, Grossman raises the question of whether FDA staff morale is primed to rebound
Drugs
A 3/10-3/14 FDA inspection at the Taizhou Kangping Medical Science and Technology Co. drug manufacturing facility in Jiangsu, China, found significant violations of current good manufacturing practice (CGMP) regulations for finished over-the-counter drugs. A just-released 10/9 Warning Letter says the specific violations were:
Drugs
FDA has appointed longtime oncology regulator Richard Pazdur as the new CDER director, replacing George Tidmarsh after he stepped down earlier this month amid an internal ethics investigation and a defamation lawsuit tied to a public post criticizing a company’s drug (see story). Tidmarsh’s abrupt exit came just
Drugs
FDA has granted Avenzo Therapeutics a fast track designation for AVZO-1418, the company’s investigational EGFR/HER3 bispecific antibody-drug conjugate being developed for certain patients with non-small cell lung cancer (NSCLC). The designation covers adults with unresectable, locally advanced or metastatic NSCLC harboring common EGFR-activating mutations
Drugs
In what it describes as one of the most sweeping policy shifts in women’s health in decades, FDA says it will begin removing broad boxed warnings from menopause-related hormone replacement therapy (HRT) products. HHS secretary Robert F. Kennedy Jr. and FDA commissioner Marty Makary unveiled the changes at
Drugs
Roche says its investigational Bruton’s tyrosine kinase (BTK) inhibitor fenebrutinib showed the strongest late-stage results yet reported for the drug class in multiple sclerosis, meeting primary endpoints in two pivotal Phase 3 studies and positioning the oral therapy as a potential competitor — and possible successor — to some of
Drugs
FDA has cited Alvotech’s Reykjavik, Iceland, manufacturing site for widespread sterility assurance, environmental monitoring, and quality-system failures following a June–July inspection, according to a 13-page Form 483. Investigators from CDER’s Office of Pharmaceutical Quality identified fundamental lapses in aseptic processing controls, cleaning programs, water-system
Drugs
Merck is reporting positive Phase 3 results for its oral PCSK9 inhibitor enlicitide, showing LDL-cholesterol reductions approaching 60% with a safety profile comparable to placebo. In the 2,900-patient study, adults with or at risk for atherosclerotic cardiovascular disease (ASCVD) taking once-daily enlicitide saw a 55.8%
Drugs
Fresenius Kabi has recalled (Class 1) three lots of its famotidine injection after reserve samples from one lot tested out of specification for endotoxin levels. According to the company, elevated endotoxin levels in injectable drugs can trigger serious systemic reactions, including sepsis, septic shock, and life-threatening inflammatory responses. One
Drugs
A group of leaders from government, academia, and the pharmaceutical industry is calling for sweeping federal action to stabilize the U.S. drug supply, warning that persistent shortages now pose risks not just to public health but to national security. At a National Academies workshop held last month at the
Drugs
Cogent Biosciences is reporting positive data from a Phase 3 study of its KIT inhibitor bezuclastinib, saying that the drug combined with standard therapy sunitinib significantly extended progression-free survival in patients with imatinib-resistant or intolerant gastrointestinal stromal tumors (GIST). It is the first successful Phase 3 study in
Drugs
FDA has cited Medispray Laboratories, a finished drug product manufacturer based in Ponda, Goa, India, for significant lapses in its quality control (QC) procedures following a January inspection at its facility. According to a just-posted Form FDA 483, investigators found that the company’s quality control responsibilities and procedures
Drugs
Intellia Therapeutics is reporting that a patient who experienced liver toxicity after being administered the company’s CRISPR-based therapy nexiguran ziclumeran (nex-z) has died. Late last month, FDA issued a clinical hold on Intellia’s MAGNITUDE and MAGNITUDE-2 Phase 3 trials for nex-z after the company
Biologics
FDA has approved Janssen’s Darzalex Faspro (daratumumab and hyaluronidase-fihj, Janssen Biotech) for adults with high-risk smoldering multiple myeloma (SMM), marking the first approved therapy for this early stage of the blood cancer. The decision is based on data from the Phase 3 AQUILA trial (NCT03301220), which compared
Drugs
Senate Democrats says they are worried that FDA and HHS may rely on partisan “junk science” during its recently announced review of the existing safety and effectiveness data of the abortion drug mifepristone. In a 11/6 letter to FDA and HHS, the Democrats expressed concern that the administration might
Drugs
FDA has publicly released an 11/4 complete response letter issued to Biohaven on its NDA seeking approval of troriluzole to treat spinocerebellar ataxia (SCA). The quick posting comes just three days after the company received the rejection letter and thoroughly complained about the agency action in an 11/5
Drugs
FDA has extended by three months its review of Rhythm Pharmaceuticals’ supplemental NDA for Imcivree (setmelanotide) and its use in treating acquired hypothalamic obesity. The revised user fee review action target date is 3/20/2026. The review extension is related to an agency request for additional sensitivity analyses of
Drugs
FDA has released the form FDA-483 issued following an 8/19-8/27 inspection at the Baxter Oncology drug manufacturing facility in Halle, Westfalen, Germany. The inspection observations were: * failure to establish process, procedural controls, and testing designed to assure that manufactured drug products have the identity, strength, quality,
Drugs
FDA has published a batch of 32 new and 44 revised draft product-specific guidances (PSGs) with recommendations for developing generic drugs and generating evidence to support ANDA approvals. An agency post has links to each of the new or revised guidances. The batch contains 64 PSGs for products with