Drugs
FDA has accepted for review an Orphalan NDA for trientine tetrahydrochloride (TETA 4HCl) for the first-line treatment of Wilson’s Disease. The rare inherited disorder of copper transport primarily affects the liver and brain, according to the company. If approved, trientine tetrahydrochloride would be an alternative copper chelating agent
Drugs
CDER Office of Generic Drugs (OGD) has reached the milestone of approving more than 100 generic drug applications with a Competitive Generic Therapy designation. The Competitive Generic Therapy program encourages the development and marketing of generic drugs for products with little to no competition. “Additionally, more than 80% of the
Drugs
FDA has approved Janssen’s Invega Hafyera (paliperidone palmitate) extended-release injectable suspension to treat schizophrenia in some adults. An agency statement says the atypical antipsychotic is the first long-acting schizophrenia medication to be injected once every six months. Other long-acting injectable antipsychotics are administered every few weeks
Drugs
An 8/18 petition submitted to FDA by attorney Jordan Thomas (Labaton Sucharow) asks FDA to halt two ongoing Cassava Sciences trials of its novel Alzheimer’s drug simufilam pending an agency audit of concerns raised in the petition. The document says Cassava has publicly announced the completion of a
Drugs
FDA has ordered a drug class warning for Janus kinase (JAK) inhibitors after reviewing a recent clinical trial that showed an increased risk of serious heart-related events such as heart attack or stroke, cancer, blood clots, and death with Pfizer’s arthritis and ulcerative colitis medicines Xeljanz and Xeljanz
Drugs
FDA says new analytical methods for detecting the carcinogen NDMA (N-nitrosodimethylamine) developed by CDER scientists have shown that actual levels of NDMA in ranitidine drug products were much lower than previously reported. In the “What is New in Regulatory Science” online summer issue, CDER says that to further address
Drugs
A 9/9-9/19/19 FDA inspection at AnazaoHealth, Las Vegas, NV, found that drug products compounded at the facility don’t meet legal requirements for exemptions from the Federal Food, Drug, and Cosmetic Act for drugs produced by an outsourcing facility. An 8/18 Warning Letter says the
Drugs
Astellas Pharma says it has paused screening and dosing of additional participants in its ASPIRO clinical trial evaluating AT132 in patients with X-linked myotubular myopathy. A company statement says the pause follows reporting of a serious adverse event in a study participant due to abnormal liver function tests observed
Drugs
FDA has converted Merck’s accelerated approval to full approval for its anti-PD-1 Keytruda (pembrolizumab) and its use in first-line treatment of advanced urothelial carcinoma (bladder cancer). In addition, the indication has been revised to be for the treatment of patients with locally advanced or metastatic urothelial
Drugs
FDA says its review of requested records supplied by Gulsah Uretim Kozmetik Sanayi Anonim Sirketi, Istanbul, Turkey, found significant violations of current good manufacturing practice (CGMP) regulations for finished drugs. A just-released 5/13 Warning Letter says specific violations were: * failing to have for each batch of drug product
Drugs
A 5/18-5/19 public meeting on potential medication error risks with IND drug container labels gathered information from national and international stakeholders for FDA to consider and determine if regulations are needed. The meeting was sponsored by FDA and the Reagan-Udall Foundation for FDA. CDER Office of
Drugs
FDA has released a guidance entitled Technical Specifications for Submitting Clinical Trial Data Sets for Treatment of Noncirrhotic Nonalcoholic Steatohepatitis (NASH). The document provides detailed information and specifications for the content of the tabulated domains and analysis data sets submitted to FDA as part of a sponsor’s application for
Drugs
Former FDA commissioner Scott Gottlieb says the U.S. needs a “coordinated national clinical trial infrastructure in the setting of a public health crisis.” Writing online in JAMA Health Forum, Gottlieb says the need for a coordinated infrastructure was demonstrated during the Covid-19 pandemic when U.S. investigators conducted
Drugs
FDA has added information in a revised guidance about remote monitoring of clinical trials conducted during the Covid emergency and whether onsite monitoring should audit the remote monitoring once Covid restrictions are lifted. The re-monitoring discussion is contained in the appendix and the just-revised guidance entitled Conduct of
Drugs
FDA has released two new CDER Manuals of Policies and Procedures (MAPPs). The first, Scientific Interest Groups: Criteria and Policies, outlines the process and requirements for creating a scientific interest group (SIG) in the CDER Office of Clinical Pharmacology (OCP). The MAPP says a SIG is a group of volunteer
Drugs
FDA has approved a UCB expanded indication for Briviact (brivaracetam) tablets, oral solution, and injection to treat partial-onset seizures in patients as young as one month of age. The company says its data in pediatric patients includes long-term retention rates over two years. “In an open label follow-
Drugs
FDA has approved an Anixa Biosciences IND for its CAR-T (Chimeric Antigen Receptor-T cell) therapy, which is being developed in partnership with Moffitt Cancer Center, and its use in treating ovarian cancer. The autologous cell therapy comprises engineered T-cells that target the follicle stimulating hormone receptor, which
Drugs
FDA has revised its guidance entitled Conduct of Clinical Trials of Medical Products During the Covid-19 Public Health Emergency. The document provides general considerations to assist sponsors in assuring the safety of trial participants, maintaining compliance with good clinical practice, and minimizing risks to trial integrity for the duration
Drugs
FDA has granted Lantern Pharma an orphan drug designation for LP-184 and its use in treating glioblastoma multiforme (GBM) and other malignant gliomas. LP-184 is a small molecule drug candidate and next generation alkylating agent that “preferentially damages DNA in cancer cells that over-express certain biomarkers or
Drugs
SciClone Pharmaceuticals has agreed to be acquired for $605 million by a consortium of buyers, including GL Capital Management CP, Bank of China Group Investment, and Ascendent Capital Partners. SciClone is a specialty pharmaceutical company with significant commercial business in China. Its portfolio covers oncology, infectious diseases and cardiovascular disorders.
Drugs
Roche has decided to withdraw the accelerated approval for Tecentriq (atezolizumab) in combination with chemotherapy (Abraxane, albumin-bound paclitaxel; nab-paclitaxel) for treating adults with unresectable locally advanced or metastatic triple-negative breast cancer (mTNBC) whose tumors express PD-L1. The decision, which only affects the mTNBC indication in the
Drugs
Cumberland Pharmaceuticals says it is looking for 48 Duchenne muscular dystrophy (DMD) patients age 7 and older for a Phase 2 FIGHT DMD study funded by FDA to evaluate the safety, efficacy, and duration of effect of its oral ifetroban therapy for cardiomyopathy linked to DMD. A company statement says