Drugs
uniQure says its investigational gene therapy AMT-130 significantly slowed disease progression in Huntington’s disease patients, which the company calls the most convincing evidence to date of a disease-modifying effect in the fatal neurodegenerative disorder. In a pivotal Phase 1-2 study, patients who received a high dose
Biologics
An 11/11-11/19/2024 FDA inspection at the Janssen Vaccines drug manufacturing facility in Incheon, South Korea, found significant violations of current good manufacturing practice (CGMP) regulations for finished drugs. A just-released 7/18 Warning Letter says the specific violations were: * failure to thoroughly investigate any unexplained
Drugs
An FDA review of records submitted by Chengdu Brilliant Biopharmaceutical Co. in Shuangliu District, Chengdu, Sichuan, China, in response to an agency request, found significant deviations from current good manufacturing practice (CGMP) regulations for active pharmaceutical ingredients (APIs). A 9/11 Warning Letter says the specific deviations were: * failure to
Drugs
FDA has issued Scholar Rock a complete response letter for its BLA seeking approval of apitegromab, a first-in-class muscle-targeted therapy for spinal muscular atrophy (SMA). The letter cited observations made during an FDA inspection of Catalent Indiana, a third-party fill-finish facility acquired by Novo Nordisk
Devices
FDA has published a guidance, Computer Software Assurance for Production and Quality System Software, with recommendations on computer software assurance for computers and automated data processing systems used as part of medical device production or the quality system. The document says it describes: * computer software assurance as a risk-based
Drugs
FDA has released the form FDA-483 issued following a 1/9-1/17 inspection at the Genzyme Ireland drug manufacturing facility in Waterford, Ireland. The two inspection observations were: * failure to establish procedures, processes, and testing designed to prevent microbial contamination of the drug product purporting to be sterile;
Drugs
FDA has released the form FDA-483 issued following an 8/4-8/12 inspection at the Cohance Lifesciences drug manufacturing facility in Secunderabad, Telangana, India. The inspection observations were: * failure to thoroughly review any unexplained discrepancy and the failure of a batch or any of its components to meet
Drugs
Sens. Rick Scott (R-FL) and Kirsten Gillibrand (D-NY), the chairman and ranking member of the Senate Aging Committee, have asked FDA commissioner Martin Makary for information about the agency’s oversight of foreign drug manufacturing and how it may threaten the quality and safety of drugs marketed and
Devices
Olympus has recalled (Class 1) its ViziShot 2 FLEX (19G) EBUS-TBNA needles manufactured prior to 5/12 after receiving reports of device components detaching during procedures. The ViziShot 2 FLEX is protected by a hypotube and sheath, a recall notice says, adding that the “hypotube protects the sheath from
Devices
FDA has issued an early alert after becoming aware that Abiomed had issued a letter to affected customers recommending certain Automated Impella Controllers be removed from where they are used or sold. “Abiomed identified Automated Impella Controller purge pressure issues due to purge retainer failures, such as cracks,” the alert
Drugs
FDA has issued Biogen a complete response letter on a supplemental NDA for a high-dose regimen of Spinraza (nusinersen) for treating spinal muscular atrophy (SMA). The letter requested an update to the technical information included in the Chemistry Manufacturing and Controls module of the submission, according to the company.
Drugs
FDA has extended by three months its review of Sanofi’s multiple sclerosis drug tolebrutinib in response to additional analyses submitted during the ongoing NDA review. The agency determined that the new data constituted a “major amendment,” triggering the extension. The revised user fee review action target date is 12/
Drugs
Roche says its investigational breast cancer drug giredestrant met key goals in a pivotal Phase 3 study, showing significant benefits for patients with advanced estrogen receptor (ER)-positive disease. The evERA trial evaluated giredestrant in combination with everolimus against standard-of-care endocrine therapy plus everolimus in patients with ER-
Drugs
FDA has released a draft guidance entitled Malaria: Developing Drugs for Treatment that outlines key considerations for companies developing drugs to treat the tropical disease. The document is intended to help drugmakers design clinical trials for therapies targeting malaria caused by a range of Plasmodium species, including P. falciparum, the
Drugs
Lexicon Pharmaceuticals says FDA is requiring additional time to review its new clinical data submitted earlier this month in an effort to advance its stalled NDA for its diabetes drug Zynquist (sotagliflozin). The company is seeking to address concerns raised in a 12/2024 complete response letter that cited an
Drugs
FDA has accepted for priority review a Chiesi NDA for idebenone and its use in treating Leber hereditary optic neuropathy (LHON), a rare inherited condition that causes sudden and severe central vision loss. LHON is a mitochondrial disorder that damages retinal ganglion cells, leading to rapid vision decline, typically beginning
Devices
FDA has released a final guidance outlining how the agency will decide when to temporarily allow use of unapproved diagnostic tests during a declared public health emergency. The guidance, Consideration of Enforcement Policies for In Vitro Diagnostic (IVD) Tests During a Section 564 Declared Emergency, explains the factors FDA will
Drugs
FDA has granted accelerated approval to a Stealth Biotherapeutics NDA for Forzinity (elamipretide) injection for treating Barth syndrome, a rare and often fatal mitochondrial disease that primarily affects boys. The disease typically begins with severe heart failure in infancy, significantly reducing life expectancy and impairing quality of life, an agency
Drugs
FDA has rejected Otsuka Pharmaceutical and H. Lundbeck’s bid to expand the use of Rexulti (brexpiprazole) for treating post-traumatic stress disorder in adults. An agency-issued complete response letter said the application, which sought approval for Rexulti in combination with the antidepressant sertraline (Zoloft), did not provide “substantial
Drugs
FDA late 9/22 took two actions against autism, first ordering a label change for acetaminophen (Tylenol) to reflect evidence suggesting that its use by pregnant women may be associated with an increased risk of neurological conditions such as autism and attention deficit in children, and second approving leucovorin calcium
FDA Policy/General
Rep. Frank Pallone Jr. (D-NJ) has sharply criticized Health and Human Services Secretary Robert F. Kennedy Jr., accusing him of undermining vaccine access and endangering public health. Pallone’s comments followed a vote by the CDC’s Advisory Committee on Immunization Practices (ACIP), which recently recommended changes that could
Devices
FDA has approved Medtronic’s Altaviva implantable tibial neuromodulation device, described by the company as a minimally invasive therapy for treating urge urinary incontinence. The device, inserted near the ankle, works by sending mild electrical impulses to the tibial nerve to help restore communication between the bladder and brain, according