Drugs
Rapt Therapeutics says it is terminating its zelnecirnon (RPT193) drug development program due to a serious safety issue. Zelnecirnon was being evaluated in two randomized, placebo-controlled Phase 2 clinical trials in asthma and atopic dermatitis (AD), and both trials were placed on clinical hold by FDA earlier this year
Drugs
FDA has granted Johnson & Johnson a breakthrough therapy designation for nipocalimab and its use for treating adults living with moderate-to-severe Sjögren's disease (SjD), a debilitating and chronic autoantibody disease. Nipocalimab is said to be the only investigational therapy to win breakthrough status for SjD. The
Drugs
AbbVie says its two Phase 2 EMPOWER trials investigating emraclidine as a once-daily, oral monotherapy treatment for adults with schizophrenia who are experiencing an acute exacerbation of psychotic symptoms did not meet their primary endpoint. Specifically, the trials did not show a statistically significant reduction (improvement) in the change
Drugs
FDA has released a recent untitled letter that was sent to Brookfield Medical/Surgical Supplies, an outsourcing facility in Brookfield, CT, due to GMP issues cited during a May inspection. The four inspection observations in the letter were: * failure to follow procedures designed to prevent microbiological contamination of drug products
Drugs
FDA has accepted for review an Unicycive Therapeutics 505 (b) (2) NDA for oxylanthanum carbonate (OLC) and its use in treating hyperphosphatemia patients with chronic kidney disease (CKD) on dialysis. The agency has set a 6/28/2025 user fee review action target date. Unicycive says OLC is designed to
Devices
FDA has cleared a Foundation Surgical 510(k) for its Vertiwedge Intraosseous, a motion-sparing partial vertebral body replacement device intended for use with the company’s Vertebral Body Osteotomy procedure. Vertiwedge is described as the “first intraosseous device designed to facilitate and maintain vertebral body correction of partially collapsed
Biologics
FDA has lifted a clinical hold on a Novavax IND for its Covid-19-influenza combination (CIC) and stand-alone influenza vaccine candidates. The agency said the company can begin enrolling patients in a planned Phase 3 trial following the determination that it satisfactorily addressed all clinical hold issues, according
Biologics
FDA has approved an Autolus Therapeutics BLA for Aucatzyl (obecabtagene autoleucel), a CAR-T therapy for treating adult patients with relapsed or refractory B-cell precursor acute lymphoblastic leukemia. The product was approved based on data from the company’s FELIX clinical trial. “In the efficacy evaluable patients (n=65)
Biologics
Johnson & Johnson has submitted a supplemental BLA for Darzalex Faspro (daratumumab and hyaluronidase-fihj) and its use as monotherapy for treating adult patients with high-risk smoldering multiple myeloma. The filing is based on data from the ongoing Phase 3 AQUILA study (NCT03301220). Smoldering multiple myeloma is described as
Devices
FDA has cleared a Bayer 510(k) for its Medrad Centargo CT Injection System, a multi-patient injector for use in computed tomography. The device is designed to minimize prep time as the daily set-up can be completed in under two minutes with the pre-assembled Day Set, the
Drugs
Allogene Therapeutics is reporting encouraging response rate data for its investigational ALLO-316 in advanced renal cell carcinoma (RCC), but the results also showed three patient deaths (Grade 5 adverse events). The results are from the Phase 1 TRAVERSE trial of ALLO-316’s potential as an “off-the-shelf”
Drugs
Advocacy group Public Citizen is criticizing FDA for dragging its feet in removing “ineffective, albeit safe, over-the-counter oral formulations of phenylephrine from the U.S. market.” On 11/7, FDA issued a proposed rule to remove the ingredient found in cough and cold medicines, either alone or in
Drugs
Dizal Pharmaceutical has submitted an NDA for sunvozertinib for treating locally advanced or metastatic non-small cell lung cancer patients with epidermal growth factor receptor (EGFR) exon 20 insertion mutations (exon20ins). The submission is based on data from the WU-KONG1 Part B study that demonstrated sunvozertinib had a statistically
Drugs
FDA has extended by three months its review of a Neurotech Pharmaceuticals BLA for NT-501 (revakinagene taroretcel) as a treatment for macular telangiectasia Type 2 (MacTel). The extension is needed to review additional data provided by the company in response to recent requests from the agency. The revised user
Drugs
Pharmaceutical Research and Manufacturers of America (PhRMA) says it appreciates FDA taking steps to increase the use of model-informed drug development (MIDD) approaches in regulatory decision-making, to understand how sponsors are using emerging MIDD approaches within medical product development, and to enhance interactions with sponsors regarding MIDD. Responding
Devices
Elekta Instrument AB has recalled (Class 1) its Elekta Disposable Biopsy Needle Kit for the Leksell Stereotactic System, which is used for localization and diagnosis of intracranial disorders and their surgical treatment, including radiosurgery and stereotactic radiation therapy. “Elekta has become aware that the Disposable Biopsy Needles can contain some
FDA Policy/General
[Commentary by David McFarland] Who will be the next FDA commissioner and what health role will Robert F. Kennedy Jr. play (see earlier story) in president-elect Donald Trump’s next administration? These are some of the top unanswered questions agency watchers are mulling just two days after his unprecedented
Drugs
Public Citizen says a potential legislative initiative expected to be advanced in 2025 would be dangerous in exempting rare disease drugs from the requirement to be subject to randomized trials. In a statement, Public Citizen says the proposal is being developed by retired FDA CDER director, acting commissioner, and principal
FDA Policy/General
Federal Register notice: FDA is making available a proposed administrative order entitled “Amending Over-the-Counter Monograph M012: Cold, Cough, Allergy, Bronchodilator, and Antiasthmatic Drug Products for Over-the-Counter Human Use.” If finalized, it would remove orally administered phenylephrine hydrochloride and phenylephrine bitartrate in an effervescent dosage as nasal
Devices
FDA says its analysis of form FDA-483s issued for Good Laboratory Practice inspection observations in FY 2023 found one overriding trend — inadequate training at multiple levels. The agency analysis says that 28 nonclinical inspections were conducted in FY 2023, with a form FDA-483 issued following 10 of them.
Devices
FDA has approved the Johnson & Johnson Varipulse pulse field ablation platform to treat atrial fibrillation. A company statement says Varipulse is the only pulse field ablation system in the U.S. fully integrated with the J&J CARTO 3 electro-anatomical mapping system, driving efficiency, reproducibility, and accuracy.
Drugs
Sarepta says it is discontinuing the development of SRP-5051 (vesleteplirsen), an investigational, next-generation peptide conjugated phosphorodiamidate morpholino oligomer for treating patients with Duchenne muscular dystrophy who are amenable to exon 51 skipping. The decision was based on FDA feedback about the therapy’s risk-benefit, according to the