Drugs
FDA has cleared an eGenesis and OrganOx IND for a first-in-human clinical trial to evaluate a novel perfusion system using a genetically engineered porcine liver to treat patients with acute-on-chronic liver failure and no transplant option. An eGenesis statement says its EGEN-5784, a human-compatible,
Drugs
Sanofi says preliminary results from a Phase 2 (NCT05421598) study of amlitelimab in adults with moderate-to-severe asthma show that the “primary endpoint of annualized exacerbation rate at Week 48 was not met at the highest dose level, leading to nominal significance at the medium dose. However, it says
Drugs
Mural Oncology says it is discontinuing all clinical development of its lead drug candidate, nemvaleukin alfa, following disappointing results from two key trials in melanoma and ovarian cancer. The company also said it will begin exploring strategic alternatives to “maximize shareholder value.” The action follows a review of topline data
Drugs
Researchers at China’s Capital Medical University say that compared to the U.S., China appears to take a more stringent regulation toward using surrogate endpoint response rate (RR) data to support cancer drug approvals. In an open-access Cancer Medicine article, the researchers discuss their analysis of all cancer
Drugs
Researchers from FDA and several hospitals say there is increasing interest in using patient-reported outcomes (PROs) as endpoints in heart failure trials and regulatory bodies have shown a willingness to consider heart failure drugs and devices for approval based on improvement in PROs. Writing in the journal Circulation, the
Drugs
FDA has approved Mirum Pharmaceuticals’ tablet formulation of Livmarli (maralixibat) for treating cholestatic pruritus in patients with Alagille syndrome and progressive familial intrahepatic cholestasis. The approval “allows flexibility for patients and physicians, with the liquid dosing used by the youngest patients and a convenient one-tablet per dose option for
Drugs
Ironwood Pharmaceuticals says it will conduct a new Phase 3 clinical trial for its investigational drug apraglutide, following recent discussions with FDA on the drug’s approval path for treating short bowel syndrome with intestinal failure (SBS-IF). FDA is requiring a confirmatory trial after raising concerns with the earlier
Drugs
Pfizer says it is discontinuing development of danuglipron, an experimental oral GLP-1 receptor agonist being evaluated for chronic weight management. Despite promising results from recent dose-optimization studies, including successful achievement of key pharmacokinetic goals and a formulation with potential Phase 3 viability, the company opted to stop further
Drugs
FDA says it seized several hundred units of counterfeit versions of Novo Nordisk’s Ozempic (semaglutide) 1mg injection that had entered the U.S. drug supply chain. The fake drugs were not distributed through the company’s authorized supply channels, according to an agency alert. Novo Nordisk first alerted the
Drugs
Vanda Pharmaceuticals has filed a lawsuit against FDA that seeks a declaratory judgment giving the company protection under the First Amendment to promote Hetlioz (tasimelteon) for the off-label use of treating jet lag. The drug is currently approved for treating non-24-hour sleep-wake disorder, and Vanda had
Drugs
FDA has approved Bristol Myers Squibb’s Opdivo (nivolumab) and Yervoy (ipilimumab) for combination first-line treatment of adult patients with unresectable or metastatic hepatocellular carcinoma (HCC). Approval was based on data from the CHECKMATE-9DW (NCT04039607) trial in 668 adults with unresectable or metastatic HCC. The primary efficacy outcome
Drugs
U.S. senator Rand Paul (R-KY) has introduced legislation to streamline the approval process for biosimilars by “eliminating costly and redundant clinical study requirements.” The Expedited Access to Biosimilars Act is intended to “increase market competition, lower drug prices, and increase access to more life-saving treatments,” Paul says
Drugs
Sen. Richard Blumenthal (D-CT) and Rep. Brian Fitzpatrick (R-PA) have introduced the Bolstering Research and Innovation Now (BRAIN) Act to advance research for brain cancer and improve brain tumor treatment and care. A statement (includes links to the bill text) issued by Blumenthal lists three bipartisan Senate co-
Drugs
FDA says it is phasing out the animal testing requirement for monoclonal antibodies and some other drugs. A 4/10 statement says animal testing would be replaced with “more effective, human-relevant methods. The new approach is designed to improve drug safety and accelerate the evaluation process, while reducing animal
Drugs
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Drugs
Johnson & Johnson is reporting new icotrokinra (JNJ-2113) data from a subgroup analysis of ICONIC-LEAD, a Phase 3 registrational study in moderate-to-severe plaque psoriasis patients. The data show adolescents treated with once-daily icotrokinra achieved higher rates of clear or almost clear skin at Week 16
Drugs
Alzheon says its Phase 3 valiltramiprosate trial in early Alzheimer’s disease (AD) patients did not meet its primary endpoint of slowing cognitive decline in the overall population. While it missed the endpoint, the company says promising results emerged in a prespecified subgroup of patients at the mild cognitive impairment
Drugs
Responding to an FDA draft guidance on considerations for using artificial intelligence (AI) to support regulatory decision-making for drug and biological products, Pharmaceutical Research and Manufacturers of America (PhRMA) says it strongly supports the agency’s efforts with the draft. The association’s 4/7 comment letter lays out
Drugs
Hyman, Phelps & McNamara attorney Kurst Karst is raising concerns about recent staff cuts at FDA that “eradicated” the Office of Generic Drugs’ Division of Policy Development (DPD). The division’s functions are critical to the development and approval of generic drugs, he says in a 4/9 FDA Law
Drugs
A Government Accountability Office (GAO) study of the federal government’s response to drug shortages says HHS should implement a mechanism to coordinate its activities related to shortages since the department says the current coordination function will end in May. The GAO report says that as of 7/31/2024,
Drugs
FDA has approved updated labeling for Idorsia’s Tryvio (aprocitentan) that eliminates the Risk Evaluation and Mitigation Strategy (REMS) requirement. A company statement says Tryvio is a dual endothelin receptor antagonist indicated for treating systemic hypertension in combination with other antihypertensives to lower blood pressure in patients who are not
Drugs
ZyVersa Therapeutics says it is optimistic about a new regulatory development that could significantly shorten the path to approval for treatments targeting focal segmental glomerulosclerosis (FSGS), a rare and debilitating kidney disorder. FSGS affects about 40,000 people in the U.S. and is one of the leading causes of