Drugs
FDA has granted accelerated approval to Jazz Pharmaceuticals’ Modeyso (dordaviprone), a protease activator, for adult and pediatric patients one year of age and older with diffuse midline glioma harboring an H3 K27M mutation with progressive disease following prior therapy. An agency announcement says Modeyso is the first approval of a
Biologics
FDA has published the FY 2025 Quarter 2 update to the CBER/CDER Data Standards Program Action Plan. The document includes quarterly project updates and a statement that reporting is paused for two projects. The data standards program focuses on four goals: improve data standards for regulatory use, data standards
Drugs
FDA has granted Clarametyx Biosciences both fast track and qualified infectious disease product designations for its lead therapeutic candidate, CMTX-101, and its use in treating chronic bacterial pulmonary infections in people with cystic fibrosis (CF). The designations follow “encouraging interim results” from Clarametyx’s ongoing Phase 1b/2a clinical
Drugs
FDA’s Oncology Center of Excellence will host a two-day (8/27-28) virtual public workshop to advance the development of patient registries in oncology, with a focus on the rare childhood brain tumor known as diffuse midline glioma, also referred to as diffuse intrinsic pontine glioma. The workshop
Drugs
A 1/16-1/27 FDA inspection at the Oasis Medical drug manufacturing facility in Glendora, CA, found significant violations of current good manufacturing practice (CGMP) regulations for finished drugs. A just-released 7/15 Warning Letter says the specific violations were: * failure to establish adequate written procedures for production
Drugs
Vertex Pharmaceuticals says its investigational selective NaV1.8 pain signal inhibitor VX-993 did not meet the primary endpoint in a Phase 2 study evaluating its safety and effectiveness in treating acute pain after bunionectomy surgery. As a result, a company statement says, VX-993 will not be moved into
Drugs
FDA has granted Dyne Therapeutics a breakthrough therapy designation for DYNE-251 for treating patients with Duchenne muscular dystrophy (DMD) that is amenable to exon 51 skipping. The designation is based on data from the ongoing DELIVER clinical trial. “As we’ve previously disclosed, DYNE-251 has demonstrated sustained functional
Drugs
Anixa Biosciences says it is assuming full sponsorship of an IND for its experimental breast cancer vaccine as it heads towards a Phase 2 clinical trial. The IND, which had been held by the Cleveland Clinic during a recently completed Phase 1 study, is now being transferred to Anixa. The
Drugs
FDA has approved Novo Nordisk’s Alhemo (concizumab-mtci) as a once-daily preventive treatment for bleeding episodes in patients 12 years and older with hemophilia A or B without inhibitors. The action expands on the drug’s initial FDA approval in December 2024 for patients with inhibitors. The approval
Drugs
Allogene Therapeutics has announced a major change to its ALPHA3 clinical trial for large B-cell lymphoma (LBCL), choosing to use standard chemotherapy for lymphodepletion following the death of a patient attributed to its investigational ALLO-647, an anti-CD52 monoclonal antibody. Lymphodepletion is described as the process by which
Drugs
FDA has approved LENZ Therapeutics’ Vizz (aceclidine ophthalmic solution) 1.44% for treating presbyopia, a common age-related condition that causes blurry near vision. The eye drops work by selectively constricting the iris sphincter muscle to create a “pinhole effect,” which improves near vision by increasing depth of focus without
Drugs
Eli Lilly is reporting favorable data from the SURPASS-CVOT trial that showed its diabetes drug Mounjaro (tirzepatide) matched the cardiovascular benefits of its older drug Trulicity (dulaglutide). The head-to-head 13,000-patient clinical trial found that Mounjaro was non-inferior to Trulicity in preventing major cardiovascular events
Drugs
FDA has released the form FDA-483 issued following a 6/3-6/14/2024 inspection at the Jubilant HollistierStier General Partnership drug manufacturing facility in Kirkland, Province Quebec, Canada. The 15 inspection observations were: * failure of the statistical quality control criteria to include appropriate acceptance and rejection levels; * failure
Drugs
FDA says the Innovative Science and Technology Approaches for New Drugs (ISTAND) pilot program has transitioned to a permanent drug development tool (DDT) qualification program. An FDA Voices post, written by CDER Office of Translational Sciences director ShaAvhrée Buckman-Garner and Office of New Drugs acting director Mary Thanh Hai,
Drugs
U.S. senators Ted Budd (R-NC) and Martin Heinrich (D-NM) have introduced a bipartisan amendment to FDA’s spending bill to strengthen the agency’s ability to prevent illicit pharmaceuticals and active pharmaceutical ingredients from entering the U.S., particularly from unregistered manufacturing facilities in China. “These illicit
Drugs
In a significant move aimed at curbing long-term misuse of opioid drugs, FDA is announcing new mandatory safety labeling changes for all opioid analgesics. The changes are designed to highlight the serious risks of prolonged use — including addiction, overdose, and death — and are based on new data and a
Drugs
FDA has granted Vivace Therapeutics an orphan drug designation for VT3989 and its use in treating mesothelioma. The company describes the drug as a first-in-class inhibitor targeting the Hippo signaling pathway, a key regulator of cell growth and organ size. Specifically, the drug is designed to block autopalmitoylation
Drugs
FDA says it is aware that some patients and healthcare professionals may look to unapproved GLP-1 drugs (including semaglutide and tirzepatide) as an option for weight loss. “This can be risky for patients, as unapproved versions do not undergo FDA’s review for safety, effectiveness, and quality before they
Drugs
AbbVie has submitted a supplemental NDA seeking approval for a new, fixed-duration, all-oral combination regimen of Venclexta (venetoclax) and acalabrutinib for previously untreated patients with chronic lymphocytic leukemia (CLL). The submission is based on data from the Phase 3 AMPLIFY trial, which showed that the venetoclax-acalabrutinib combination
Drugs
FDA says it terminated its Arthritis Advisory Committee as of 7/30. A Federal Register notice says the committee was established on 4/5/1974 to advise on drugs and other regulated products for human use in arthritic conditions. “The committee has met infrequently in recent years,” the notice says,
FDA Policy/General
FDA commissioner Martin Makary says that while he wants to speed patients’ access to needed drugs, he also wants to reduce the need for pharmaceuticals by addressing the root causes of diseases. In an interview with Yahoo Finance, Makary said he has been focused on ensuring quicker access to drugs,
Drugs
A 9/2-9/6/2024 FDA inspection at the Macsen Drug manufacturing facility in Tehsil Mavli, Udaipur, Rajastahn, India, identified significant deviations from current good manufacturing practice (CGMP) regulations for active pharmaceutical ingredients (APIs). A just-released 3/25 Warning Letter says the specific violations were: * failure of the