Drugs
Beam Therapeutics says it will advance its base-editing therapy BEAM-302 into pivotal development to seek accelerated approval for treating Alpha-1 Antitrypsin Deficiency (AATD) after updated Phase 1/2 data showed the drug can durably raise AAT levels above the protective threshold while sharply reducing the disease-causing
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FDA has released the form FDA-483 with four observations from a 1/30-2/7/2025 inspection at the Micro Labs finished drug products manufacturing facility in Bengaluru, Karnataka, India. The specific observations were: * failure to have in writing and fully follow the responsibilities and procedures applicable to the
Drugs
FDA has approved Corcept Therapeutics’s cortisol-modulating drug Lifyorli (relacorilant) in combination with nab-paclitaxel for patients with platinum-resistant ovarian cancer. The action is backed by data from the Phase 3 ROSELLA trial, where the combo delivered a statistically significant overall survival benefit — a relatively rare outcome in
Drugs
Five Republican members of the Senate HELP Committee want FDA to take “immediate action to prevent misbranded and unapproved versions of the chemical abortion drugs mifepristone and misoprostol from being marketed in the U.S. by online retailers in violation of U.S. law.” In a statement (includes links to
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A 10/21-10/24/2025 FDA inspection at the Yangzhou H&R Plastic Daily Chemical Company drug manufacturing facility in Hangji, Guangling, Yangzhou, Jiangsu, China, found significant violations of current good manufacturing practice (CGMP) regulations for finished drugs. A 3/18 Warning Letter says the violations were: * failure
Drugs
A leading health policy expert is warning that intense price competition in the U.S. generic drug market is undermining manufacturing quality and increasing the risk of shortages, calling for targeted reforms to stabilize the system. In a Brookings commentary that was adapted from recent remarks to the Senate Special
Drugs
FDA has issued a Warning Letter to ImmunityBio, citing misleading promotional claims about its cancer therapy Anktiva (nogapendekin alfa inbakicept-pmln) in both a television advertisement and a podcast appearance by company leadership. In the just-posted 3/13 letter, CDER Office of Prescription Drug Promotion (OPDP) says the company’
Drugs
FDA has granted in part and denied in part an AdventaPharma 10/24/2025 petition asking the agency to take specific actions on ANDAs for nintedanib. The agency’s response says Adventa specifically asked that FDA, before granting full approval: * require that ANDA holders of nintedanib conduct comprehensive risk assessments
Drugs
FDA is requiring updated safety warnings for a widely used class of Parkinson’s disease treatments, citing evidence that they can cause vitamin B6 deficiency and, in some cases, seizures. The agency has notified manufacturers of all drug products containing carbidopa/levodopa to revise prescribing information to include the new
Drugs
A legal update from Wilson Sonsini Goodrich & Rosati (WSGR) addresses FDA’s release earlier this month of a draft guidance (see story) clarifying the scope and eligibility criteria for new clinical investigation exclusivity (3-year exclusivity) under the Federal Food, Drug, and Cosmetic Act. The draft guidance provides detailed
Drugs
FDA accepts for priority review an Ionis Pharmaceuticals NDA for zilganersen, an experimental treatment for Alexander disease. The agency set a user fee review action target date of 9/22. Alexander disease is described as a progressive neurological disorder that primarily affects children and leads to severe motor and cognitive
Drugs
The International Council for Harmonization (ICH) has outlined a roadmap for future global standards on advanced drug manufacturing, highlighting gaps in current regulatory frameworks and calling for new guidelines to support emerging technologies. In a reflection paper recently endorsed by the ICH Assembly, regulators and industry stakeholders identified process modeling,
Drugs
FDA will host a public meeting next month aimed at exploring ways to expand access to nonprescription, or over-the-counter (OTC), medications, as policymakers and stakeholders continue to examine strategies to reduce healthcare costs and improve treatment availability. The meeting, titled “Increasing Access to Nonprescription Drugs,” is scheduled for
Drugs
U.S. Senator Rick Scott (R-FL) is urging FDA and other federal health agencies to review and tighten oversight of clinical trials and drug approvals involving Chinese-linked entities, citing potential national security and patient safety risks. In a letter addressed to HHS secretary Robert F. Kennedy, Jr., NIH
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FDA has approved Bristol Myers Squibb’s Opdivo (nivolumab) for use with doxorubicin, vinblastine, and dacarbazine (AVD) for adult and pediatric patients 12 years and older with previously untreated, Stage III or IV classical Hodgkin lymphoma (cHL). The FDA also granted traditional approval to nivolumab for the following indications in
Drugs
FDA is announcing a 6/12 public hearing to gather input on its Commissioner’s National Priority Voucher (CNPV) Pilot Program, aimed at streamlining review of therapies for critical public health needs. The pilot is designed to explore faster review pathways for certain high-priority medical products. During the hearing,
Drugs
Lilly says its triple agonist retatrutide met all key primary and secondary endpoints in the Phase 3 TRANSCEND T2D-1 trial that evaluated its efficacy and safety in treating adults with type 2 diabetes. A company statement says retatrutide is a first-in-class GIP, GLP-1, and glucagon triple
Drugs
FDA has approved GSK’s Lynavoy (linerixibat), the first U.S.-authorized therapy specifically for cholestatic pruritus in patients with primary biliary cholangitis, according to a company release. The action provides a long-awaited treatment option for a condition that affects up to 89% of people with PBC — an autoimmune
Drugs
Sarepta says it will submit to FDA by the end of April sNDAs to convert the accelerated approvals of its Duchenne muscular dystrophy drugs Amondys 45 (casimersen) and Vyondys 53 (golodirsen) to traditional approvals. A company statement says the decision to submit the sNDAs follows a meeting with FDA on
Drugs
FDA has approved a higher-dose version of Novo Nordisk’s blockbuster weight-loss drug Wegovy (semaglutide) injection, marking the fourth product cleared under the agency’s fast-track National Priority Voucher Program. The new formulation, Wegovy HD (7.2 mg), is indicated for chronic weight management in adults with
Drugs
More than 100 mothers and advocates affected by a group of rare metabolic disorders known as mucopolysaccharidoses (MPS) held a “funeral” in front of the FDA headquarters 3/18 to “mourn the futures” of their children following the agency’s rejection of various rare disease treatments. Newsweek reports that the
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Biopharmaceutical company CSL Limited says it is experiencing a temporary global shortage of its gene therapy Hemgenix, potentially delaying treatment for some patients with hemophilia B. In a letter addressed to the hemophilia B community, the company says the shortage is not related to any concerns about the therapy’s