Biologics
FDA has placed a clinical hold on Regenxbio's investigational gene therapy RGX-121 after asymptomatic spine MRI findings were identified in five participants in a clinical trial evaluating the treatment for Hunter Syndrome or Mucopolysaccharidosis Type II (MPS II). In response, the company says it does not expect
Drugs
As Amgen continues its bid to prevent the withdrawal of Tavneos (avacopan) in the U.S., a new study using a U.S. rheumatology registry has found that while liver enzyme elevations were uncommon among patients treated with avacopan, it identified two cases of drug-induced liver injury (DILI), including
Devices
FDA has cleared Roche/Eli Lilly’s Elecsys Phospho-Tau (217P) Plasma (pTau217) blood test as a single blood-based option to aid in identifying amyloid pathology associated with Alzheimer's disease in individuals 55 years and older with signs, symptoms, or complaints of cognitive decline. The companies say
Devices
FDA has issued an early alert about a potentially high-risk problem involving certain CooperSurgical INCA neonatal and infant nasal continuous positive airway pressure (CPAP) sets that may develop a loose connection between the tubing and nasal cannula. Earlier this month, the company sent affected customers a letter recommending that
Biologics
FDA has extended by three months its review of a Capricor Therapeutics BLA for Deramiocel, an investigational cell therapy for Duchenne muscular dystrophy (DMD). The new user fee review action target date is 11/22. “As part of its ongoing discussions with the FDA following the July 2026 Advisory Committee
Biologics
CBER has updated its standard operating procedures for resolving scientific/procedural disputes between sponsors and the Center, establishing revised procedures and adding medical devices to the formal dispute resolution process. The updated SOPP 8005: Formal Dispute Resolution Process applies to disputes between sponsors and CBER review staff involving regulatory submissions.
Drugs
House Select Committee on China chairman John Moolenaar (R-MI) and Rep. Ben Cline (R-VA) are urging FDA intensify safeguards on clinical trial data generated in China, citing concerns about transparency and potential underreporting of serious adverse events and patient deaths. In an 8/20 letter to acting FDA
Drugs
Scholar Rock said Friday that it has made further regulatory progress for apitegromab, its investigational treatment for spinal muscular atrophy (SMA), removing a troubled Catalent (part of Novo Nordisk) facility from its BLA resubmission. The facility removal was recommended by FDA after Scholar added a second U.S.-based fill-
Devices
CDRH is issuing an Early Alert after becoming aware that GE HealthCare issued a letter alerting affected customers of updated use instructions for Portrait Mobile Monitoring Solutions devices operating on certain version of Portrait Core Services software. The alert recommends that users ensure “appropriate alternative monitoring is established for patients
Drugs
FDA has issued a final guidance describing how it evaluates therapeutic equivalence among prescription drug products and assigns therapeutic equivalence codes in the agency’s Approved Drug Products With Therapeutic Equivalence Evaluations (aka, the Orange Book). The document, “Evaluation of Therapeutic Equivalence,” describes the scientific and regulatory principles the agency
Devices
Boston Scientific has recalled its ENROUTE Transcarotid Neuroprotection System Plus, a single-use percutaneous catheter kit used during transcarotid artery revascularization. The device is intended to provide direct carotid access and dynamic blood flow reversal to capture loose plaque debris, protecting the brain from strokes during carotid stenting. An FDA
Devices
Medline has issued a device correction (Class 1 recall) for convenience kits containing B. Braun’s bupivacaine hydrochloride components. An FDA notice says Medline issued a letter to affected customers recommending that certain Medline convenience kits be corrected prior to continued use by removing components affected by the B. Braun
Devices
FDA Digital Health Center of Excellence director Rick Abramson says that digital health technologies (DHTs), especially those enabled by artificial intelligence, can continuously capture information about a person’s health in real time and in the context of their everyday life. Writing in an FDA Voices column, Abramson says DHTs
FDA Policy/General
Now that president Donald Trump has officially nominated White House health policy adviser Heidi Overton to become FDA commissioner, a quick Senate confirmation appears unlikely. In an 8/19 Truth Social post, Trump characterized Overton as a “rockstar” in the administration, “where she has worked directly with Secretary Kennedy, Dr.
Drugs
A 1/13-1/30 FDA inspection at the Safrel Pharmaceuticals drug manufacturing facility in Dayton, NJ, found significant violations of current good manufacturing practice (CGMP) regulations for finished drugs. An 8/7 Warning Letter says Safrel failed to establish an adequate quality unit and to have in writing and
Drugs
FDA has granted Ultragenyx Pharmaceutical accelerated approval for its Genglycos (pariglasgene brecaparvovec-opnr) for adults and children eight years and older with glycogen storage disease type Ia (GSDIa). The action marks the first gene therapy approval for Ultragenyx and the company’s fifth FDA approval overall. Ultragenyx also received an
Drugs
FDA has issued a complete response letter to Xspray Pharma for its NDA for Dasynoc (dasatinib), the company’s product candidate to treat chronic myeloid leukemia and acute lymphoblastic leukemia. A company statement says the FDA letter relates to the agency’s previously communicated observations about CGMP issues at NerPharMa,
Drugs
A 3/2-3/5 FDA inspection at the Suretec Innovations drug manufacturing facility in Gilbert, AZ, found significant violations of current good manufacturing practice (CGMP) regulations for finished drugs. An 8/13 Warning Letter says the four specific violations were: * failure to test samples of each component for identity
Drugs
FDA has accepted for review a Teva Pharmaceuticals NDA for ecopipam, a company-described first-in-class investigational therapy for treating pediatric patients with Tourette syndrome. If approved, the drug would become the first new treatment option indicated for pediatric patients with Tourette’s in more than 10 years and
Devices
FDA has granted Vitestro a de novo authorization for Aletta, the first standalone robotic device designed to draw blood from a patient’s arm without hands-on intervention by a phlebotomist. The device is authorized for use in adults in outpatient settings and must operate under the supervision of a
Biologics
FDA has published a guidance, Frequently Asked Questions—Developing Potential Cellular and Gene Therapy Products, to help facilitate the development of safe, effective, and high-quality cellular and gene therapy (CGT) products. The document contains common questions directed to FDA and spans multiple disciplines, including regulatory review; chemistry, manufacturing, and
Drugs
A 2/4-2/6 FDA inspection at the Auriga Research contract testing laboratory in Bengaluru, India, identified significant violations of current good manufacturing practice (CGMP) regulations for finished drugs. An 8/12 Warning Letter says the specific violations were: * failure to ensure that laboratory records included complete data derived