Drugs
FDA has made available a draft guide entitled “Rare Diseases: Natural History Studies for Drug Development.” The document is intended to help inform the design and implementation of natural history studies that can be used to support safe and effective drug and biological product development for rare diseases. Specifically, it
Devices
FDA has cleared a Genetesis 510(k) for its cardiac imaging platform, which pairs its CardioFlux Magnetocardiograph with the integrated Faraday Analytical Cloud (FAC). The company says the new technology provides emergency room physicians and cardiologists with access to measure and visualize magnetic fields produced by the heart’s natural
Drugs
CDER’s Office of Clinical Pharmacology (OCP) says that in 2019 it will focus on current health issues related to the opioid epidemic, antimicrobial resistance, and rare diseases. In its latest annual report, OCP says the application of pharmacogenomic information will continue to play an important role in clinical trial
FDA Policy/General
Federal Register notice: FDA has announced a 4/26 Antimicrobial Drugs Advisory Committee meeting. Panel members are scheduled to discuss the safety and effectiveness of bacitracin for intramuscular injection for treating infants with pneumonia and empyema caused by staphylococci shown to be susceptible to the drug, which is the only
Drugs
FDA has issued a safety alert on the investigational use of AbbVie/Genentech’s Venclexta (venetoclax) for treating patients with multiple myeloma based on data showing an increased death risk for patients on the therapy compared to those on an active control. Earlier this month, the agency placed a partial
Drugs
FDA has issued Lexicon Pharmaceuticals and Sanofi a complete response letter on their NDA for investigational Zynquista (sotagliflozin), a dual SGLT1 and SGLT2 inhibitor for adults with Type 1 diabetes in combination with insulin. Without disclosing FDA’s concerns, the companies say they will work closely with the agency to
Drugs
FDA has issued Recro Pharma a complete response letter for intravenous (IV) meloxicam for managing moderate to severe pain. The letter focused on onset and duration of IV meloxicam, noting that the delayed onset fails to meet the prescriber expectations for IV drugs, according to the company. It also cited
FDA Policy/General
Federal Register notice: FDA has announced a 4/8 public workshop entitled “Development of Antibacterial Drugs for the Treatment of Nontuberculous Mycobacterial Disease.” FDA officials and attendees are expected to discuss clinical trial design considerations, including endpoints, related to developing antibacterial drug products for treating the disease. To view the
Drugs
Indiana University School of Medicine researcher Xiongbin Lu is reporting several important findings related to triple negative breast cancer and its treatment. Unlike the three most common forms of breast cancer, he says, triple negative breast cancer has no approved targeted therapies for treatment. In a paper, Lu and University
Drugs
Biogen and Eisai have discontinued global Phase 3 trials, ENGAGE and EMERGE, designed to evaluate the efficacy and safety of aducanumab in patients with mild cognitive impairment due to Alzheimer’s disease and mild Alzheimer’s disease dementia. Their decision is based on a futility analysis conducted by an independent
Devices
FDA has cleared a GenePOC 510(k) for the GenePOC Strep A assay for use on its revogene device. The qualitative in vitro diagnostic test is intended for detecting Streptococcus pyogenes nucleic acids from throat swab specimens obtained from patients with signs and symptoms of pharyngitis. The assay can provide
Drugs
As part of the FDA program to use real-world evidence based on real-world data for safety and efficacy decisions involving drugs, demonstration projects will cover data, real-world evidence tools, and real-world evidence study design. CDER Office of Medical Policy director Jacqueline Corrigan-Curray reviewed in a
Devices
FDA has approved an Impulse Dynamics PMA for the Optimizer Smart system for treating patients with chronic, moderate-to-severe heart failure who are not suited for treatment with other heart failure devices such as cardiac resynchronization therapy to restore a normal timing pattern of the heartbeat. The device comprises
Drugs
FDA has granted Enzyvant rare pediatric disease and fast track designations for RVT-801, an investigational enzyme replacement therapy. RVT-801 was previously granted an orphan drug designation and is currently in preclinical development for Farber disease, a rare condition for which there are no approved treatments. RVT-801 is
Drugs
Novo Nordisk has submitted two NDAs to FDA for oral semaglutide, the first once-daily glucagon-like peptide-1 receptor agonist in a pill. The company says one NDA was submitted seeking approval as an adjunct to diet and exercise to improve blood sugar in adults with Type 2 diabetes.
Drugs
FDA has approved a Jazz Pharmaceuticals NDA for Sunosi (solriamfetol) to improve wakefulness in adult patients with excessive daytime sleepiness associated with narcolepsy or obstructive sleep apnea (OSA). Once-daily Sunosi is approved with doses of 75 mg and 150 mg for patients with narcolepsy and doses of 37.5
Drugs
FDA Anesthetic and Analgesic Drug Products Advisory Committee chair Raeford Brown, a University of Kentucky professor, and Public Citizen Health Research Group founder and senior advisor Sidney Wolfe are calling on HHS and FDA to immediately impose a moratorium on FDA approval of any new or reformulated opioids. The two
Drugs
CDER director Janet Woodcock says the Center reorganization first announced 7/2018 has been approved. “The reorganization aims to transform and strengthen the way FDA fulfills its public health and regulatory role,” she says in a staff memo. The organizational changes take effect 3/31. The changes involve the Office
Devices
Soliton has submitted a 510(k) for the Rapid Acoustic Pulse (RAP) tattoo removal device. The device is indicated as an accessory to the 1064 nm Q-Switched laser for black ink tattoo removal on the arms, legs and torso in Fitzpatrick Skin Type I-III individuals. The company says
Drugs
FDA imposed its most restrictive distribution program on the nation’s first postpartum depression (PPD) drug for adult women, Sage Therapeutics’ intravenous Zulresso (brexanolone). The severe restrictions in its Risk Evaluation and Mitigation Strategy (REMS) is due to concerns about serious risks, including excessive sedation or sudden loss of consciousness
Drugs
The DC federal court has granted an FDA motion for a voluntary remand of Vanda Pharmaceuticals’ suit challenging the agency requirement to perform nine-month large animal studies of its tradipitant before beginning studies in humans. (Tradipitant is a potential treatment for several human conditions including gastroparesis). The court declined
FDA Policy/General
Oregon attorney general Ellen Rosenblum says Pfizer will pay $975,000 to settle state charges that it distributed misleading marketing materials and coupons to Oregon consumers. Rosenblum says the coupons were designed to help consumers afford copays or deductibles associated with branded drugs. Some $620,000 of the settlement money