FDA Policy/General
Sarepta Therapeutics says it will file a supplemental BLA for Elevidys (delandistrogene moxeparvovec-rokl) to expand the therapy’s use in all patients with Duchenne muscular dystrophy (DMD). In June, FDA granted the gene therapy accelerated approval to treat pediatric patients aged four and five years of age who have
Drugs
GSK says a Phase 3 trial evaluating its Jemperli (dostarlimab) in adult patients with primary advanced or recurrent endometrial cancer met its primary endpoint of overall survival (OS). The trial (RUBY/ENGOT-EN6/GOG3031/NSGO) is investigating dostarlimab plus standard-of-care chemotherapy (carboplatin and paclitaxel), followed by dostarlimab as
Drugs
Novartis says it will seek FDA accelerated approval after reporting favorable data from an interim analysis of the ongoing pivotal Phase 3 ALIGN study (NCT04573478) of atrasentan, an oral endothelin A receptor antagonist (ERA) for treating patients with IgA nephropathy (IgAN). The interim data show that the study met its
Devices
FDA has approved Impulse Dynamics’ Optimizer Smart Mini system for whole-body MRI-conditional use. Impulse Dynamics says the device is used to deliver the company’s proprietary cardiac contractility modulation (CCM) therapy. The approval expands the product labeling to allow for full-body magnetic resonance diagnostic imaging with 1.
Drugs
Deciphera Pharmaceuticals says it will file an NDA in the second quarter of 2024 for vimseltinib for treating patients with tenosynovial giant cell tumor (TGCT) based on just-announced “positive top-line results” from the MOTION pivotal Phase 3 study of the drug in patients with TGCT not amenable to
Drugs
FDA has approved an Eli Lilly BLA for Omvoh (mirikizumab-mrkz) infusion (300 mg/15 mL)/injection (100 mg/mL), described as the first interleukin-23p19 (IL-23p19) antagonist for treating moderately to severely active ulcerative colitis in adults. Approval was based on data from two Phase 3 trials (LUCENT)
Drugs
A New Braunfels, TX man, Evan Asher Field, was sentenced in federal court in San Antonio to five years in prison for selling unapproved drugs through the mail. According to a Justice Department release, Field and codefendant Michael Dominic Diaz purchased bulk quantities of various drugs, including synthetic opioids and
Drugs
FDA has approved Genentech’s Vabysmo (faricimab-svoa) for treating macular edema following retinal vein occlusion (RVO). This is the third indication for Vabysmo; it was previously approved for treating wet, or neovascular, age-related macular degeneration (AMD) and diabetic macular edema (DME). The bispecific antibody’s latest approval was
Drugs
FDA reviewers will ask an advisory committee 10/31 whether Vertex Pharmaceuticals and CRISPR Therapeutics need to conduct additional studies on their sickle cell disease (SCD) gene therapy exagamglogene autotemcel (exa-cel) to obtain a better safety assessment. According to briefing documents released 10/27 in advance of the meeting,
Drugs
Senator Marco Rubio (R-FL) has introduced a bill entitled Further Strengthening America’s Supply Chains and National Security Act to reduce America’s dependence on China for pharmaceuticals and to address related supply chain risks that lead to shortages of critical drugs. “The COVID-19 pandemic highlighted how vulnerable
Drugs
FDA has approved a Coherus BioSciences and Junshi Biosciences BLA for Loqtorzi (toripalimab-tpzi) in combination with cisplatin and gemcitabine for the first-line treatment of adults with metastatic or recurrent locally advanced nasopharyngeal carcinoma (NPC), and as monotherapy for treating adults with recurrent, unresectable, or metastatic NPC with disease
Devices
Cardinal Health has recalled (Class 1) certain lots of its Monoject luer-lock tip syringes (1, 6, 12, 20, 35 and 60 mL) after the company documented recognition and compatibility issues with certain syringe infusion pumps. As a result, Cardinal Health recommends that the affected devices not be used with
Devices
FDA has granted Paige a breakthrough device designation for its Paige Lymph Node, an artificial intelligence (AI) application used to detect breast cancer metastases in lymph node tissue. The company says the device is the first AI application of its kind to receive a breakthrough designation from the agency. “Pathologic
Drugs
FDA is seeking public comment on the usefulness of biosimilarity statements in the product labeling. In an online post, CDER Office of Therapeutic Biologics and Biosimilars director Sarah Yim says FDA is recommending that all labeling for biosimilars, including interchangeable biosimilars, include a biosimilarity statement, rather than both a biosimilarity
Drugs
FDA is advising healthcare providers and the public about the possible risks that probiotic products containing live bacteria or yeast pose to hospitalized preterm infants. An agency news release (contains links to related materials) says the agency is concerned that such products are being illegally sold to treat or prevent
Biologics
Pfizer and BioNTech have announced “positive topline results” from their Phase 1/2 study (NCT05596734) that is evaluating the safety, tolerability and immunogenicity of mRNA-based combination vaccine candidates for influenza and Covid-19 among healthy adults 18 to 64 years of age. “The data from the trial showed that
Devices
Olympus has recalled (Class 1) its high-flow insufflation units (model UHI-4) that are used in laparoscopic surgeries after receiving reports about patients suffering arrhythmias, described as short cardiac arrests, during surgical procedures where the devices were used. “These events may have been due to an over-insufflation of
Devices
Researchers from several academic medical centers say FDA should consider making some changes to its Breakthrough Devices Program (BDP) to prevent it from triggering Centers for Medicare and Medicaid Services (CMS) supplemental payments for many costly, new devices. Writing in a JAMA Cardiology Special Communication, the researchers say (article purchase
FDA Policy/General
Federal Register notice: FDA is making available a draft guidance entitled “Remote Interactive Evaluations of Drug Manufacturing and Bioresearch Monitoring Facilities.” The document describes how the agency requests and conducts voluntary remote interactive evaluations at facilities where drugs are manufactured, processed, packed, compounded, or held, and at facilities covered under
Devices
Qiagen and Myriad Genetics say they have a master collaboration agreement to develop cancer companion diagnostic tests. A Myriad statement says the partnership aims to deliver innovative services and products to pharmaceutical companies, enabling the development and commercialization of proprietary cancer tests for the U.S. clinical market, and providing
Drugs
CDER’s Office of Prescription Drug Promotion (OPDP) has launched a new Web page to display untitled letters issued for drug advertising and promotion violations. The Web page consolidates all OPDP untitled letters issued since 2017 onto a single page. Previously, they were displayed on separate Web pages based on
Devices
Sens. Charles Grassley (R-IA) and Elizabeth Warren (D-MA) have written to FDA commissioner Robert Califf urging that the device identifier (DI) portion of a medical device’s unique device identifier be included on Medicare claim forms. “Given the decades-long effort to include DI information on Medicare claims