Biologics
FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee supported Vertex Pharmaceuticals and CRISPR Therapeutics safety assessments on their sickle cell disease (SCD) gene therapy exagamglogene autotemcel (exa-cel). This moves the therapy one step closer to FDA approval which would make it the first CRISPR gene-edited therapy for
Drugs
FDA expects to see the increase in cannabis research that has occurred in recent years to continue. Writing in an online post, CDER Office of the Center Director public health advisor Cassandra Taylor and CDER Office of Executive Programs project manager Schuyler Pruyn say that as the agency sees more
Drugs
A Florida federal judge has sentenced Marina Sievert to two years in federal prison for mail fraud and introducing into interstate commerce a foreign unapproved new drug. As part of her sentence, the court entered an order of forfeiture in the amount of $1.5 million, the proceeds of the
Drugs
Tonix Pharmaceuticals is discontinuing the development program for major depressive disorder candidate TNX-601 ER based on disappointing efficacy results from the company’s Phase 2 UPLIFT study. “The primary efficacy endpoint of change from baseline in depression severity, as measured by the Montgomery-Åsberg Depression Rating Scale (MADRS) total
Drugs
Five Sidley Austin attorneys say a 10/24 FDA draft guidance on communications from firms to healthcare providers on unapproved uses of approved or cleared medical products raises many questions. Writing in an online post, the attorneys say the revision of a 2009 final guidance on off-label reprints reflects
Drugs
FDA has issued NATCO Pharma an eight-item Form FDA-483 after concluding an inspection of the firm’s Telangana, India, manufacturing facility 10/18 and citing significant GMP deficiencies. For example, agency investigators said the firm’s equipment and utensils are not cleaned and maintained at appropriate intervals. The
Devices
A 3/23-3/29 FDA inspection at WAVi Co. in Denver, CO, found that the firm manufactures and distributes the “WAVi Desktop,” intended to collect, analyze, and clinically interpret data to aid in the assessment or diagnosis of patients with a variety of potential neurological conditions. A 10/20
Drugs
Stakeholders have provided comments to FDA through a 10/14/22 discussion paper and 11/14-16/22 public workshop on the agency’s Framework for Regulatory Advanced Manufacturing Evaluation (FRAME) initiative to foster a regulatory framework to support the adoption of advanced manufacturing technologies that could benefit patients. An
Drugs
FDA has lifted a clinical hold against Mersana Therapeutics’ Phase 1 clinical trial of XMT-2056, a systemically administered Immunosynthen STING-agonist antibody drug conjugate that is designed to target a novel human epidermal growth factor receptor 2 (HER2) epitope and locally activate STING signaling in both tumor-resident immune
Drugs
FDA has granted Sage Therapeutics an orphan drug designation for SAGE-718 and its use in treating Huntington’s disease (HD). SAGE-718 is described as a first-in-class investigational NMDA receptor-positive allosteric modulator (PAM) being developed as a potential oral therapy for cognitive disorders associated with NMDA
FDA Policy/General
Federal Register notice: FDA has determined for patent extension purposes the regulatory review period for Fresenius Kabi’s Omegaven (fish oil triglycerides) indicated as a source of calories and fatty acids in pediatric patients with parenteral nutrition-associated cholestasis. The regulatory review period for Omegaven is 4,246 days, of
Devices
Pharmaceutical Research and Manufacturers of America and the Biotechnology Innovation Association say they appreciate FDA’s timely publication of its information technology (IT) strategy and its efforts to meet its commitments under the most recent PDUFA and BsUFA agreements, as well as requirements under the FDA Omnibus Reform Act of
Drugs
A 9/26-10/3/22 FDA inspection at Ali Pharmaceutical Manufacturing in Omaha, NE, found significant deviations from current good manufacturing practice (CGMP) regulations for active pharmaceutical ingredients (API). A just-released 3/30 Warning Letter says specific deviations were: * failing to demonstrate that the firm’s manufacturing process
Drugs
A 6/27-7/8/22 FDA Bioresearch Monitoring Program inspection at Samm Solutions, doing business as BTS Research in San Diego, CA, identified objectionable conditions in the company’s role as the testing facility in two nonclinical laboratory studies. A 10/20 Warning Letter says FDA emphasized: * the study
Drugs
FDA has accepted for priority review an X4 Pharmaceuticals NDA for once-daily, oral mavorixafor to treat individuals aged 12 and older with WHIM (Warts, Hypogammaglobulinemia, Infections, and Myelokathexis) syndrome, a rare immunodeficiency. The agency has set a review action target date of 4/30/2024. The NDA is based
Biologics
FDA has approved a Tenaya Therapeutics IND for the company to begin a Phase 1b trial of TN-401, an adeno-associated virus serotype 9 (AAV9)-based investigational gene therapy product candidate for the treatment of arrhythmogenic right ventricular cardiomyopathy (ARVC) caused by mutations in the plakophilin-2 (PKP2) gene.
Devices
Responding to an Office of the National Cyber Director request for information on opportunities for and obstacles to harmonization of cybersecurity regulations, AdvaMed points out that FDA “is the primary authority in regulating medical devices, including establishing that they are cyber secure. FDA administers comprehensive regulations and implementing guidance that
Devices
Asensus Surgical has recalled (Class 1) its Senhance Surgical System due to the potential for the devices to malfunction. The company has identified a malfunction that has occurred and may recur involving “uncontrolled arm motion of the Laparoscope Instrument Actuator (LIA) where the LIA rotated continuously in one direction after
FDA Policy/General
Federal Register notice: FDA has determined that 17 listed drug products were not withdrawn from sale for safety or efficacy reasons. The determination means that the agency will approve ANDAs that reference the drug products as long as they meet relevant legal and regulatory requirements. To view the notice, click
Biologics
FDA has approved an Amgen BLA for Wezlana (ustekinumab-auub), an interchangeable biosimilar to Janssen’s Stelara for multiple inflammatory diseases. Both products are indicated for adults and pediatric patients with moderate to severe plaque psoriasis who are candidates for phototherapy or systemic therapy, and active psoriatic arthritis. They are
Devices
The Advanced Medical Technology Association (AdvaMed) is urging FDA to proceed with stakeholder recommendations to develop a “central item bank or horizontal PRO [patient-reported outcomes]” tool for all vision disease states in clinical trials. AdvaMed’s comments were submitted to the docket of a 9/29 FDA virtual public
Drugs
FDA has issued a warning on the use of 26 over-the-counter eye drop products due to the potential risk of eye infections that could result in partial vision loss or blindness. “Patients who have signs or symptoms of an eye infection after using these products should talk to