Drugs
A 4/22-4/25 FDA inspection at the Tranzonic Innocore Sales & Marketing drug manufacturing facility in Woodstock, Ontario, Canada, found four repeat Current Good Manufacturing Practice (CGMP) violations in the firm’s production of finished drugs. A 10/16 Warning Letter says the specific violations were: * failure to
Drugs
Acting Office of New Drugs (OND) director Mary Tran Thanh-Hai has been officially named the new OND director, replacing previous head Peter Stein who was forced out of the agency under mass layoffs in April. At the time, Stein was offered a reassignment to a position in patient affairs,
Drugs
FDA has posted a seven-item Form FDA-483 after inspecting Dr. Reddy’s Mirfield, West Yorkshire, UK, active pharmaceutical ingredient (API) manufacturing facility 9/1-5/2025. The key GMP deficiencies noted were: * Failure to establish adequate specifications, sampling plans, and testing procedures to ensure that raw material and
Drugs
Intellia Therapeutics has paused patient dosing and screening for its MAGNITUDE and MAGNITUDE-2 Phase 3 trials of nex-z, a CRISPR-based therapy for transthyretin amyloidosis with cardiomyopathy and polyneuropathy, due to a recent patient injury. The decision follows a 10/24 report on a patient injury in the
Drugs
Senate Special Committee on Aging leaders are demanding answers from HHS on what they describe as serious national security and public health risks tied to America’s dependence on foreign pharmaceutical supply chains. In a 10/24 letter to secretary Robert F. Kennedy Jr., committee chair Rick Scott (R-Fla.
Drugs
BridgeBio Pharma plans to file an NDA next year after reporting positive data from Phase 3 FORTIFY trial evaluating BBP-418, an oral small-molecule therapy for limb-girdle muscular dystrophy type 2I/R9. The progressive genetic muscular dystrophy is caused by FKRP mutations, which impair glycosylation of alpha-dystroglycan
Drugs
FDA has posted a six-item Form FDA-483 after inspecting Hetero Labs’ Vishakapatnam, India, warehouse last month. The findings from the inspection are: * Access to the facility storing active pharmaceutical ingredients, intermediates and key starting materials intended for the U.S. market and associated records were not made readily
Drugs
FDA has approved an expanded indication for Merck’s pulmonary arterial hypertension (PAH) therapy Winrevair (sotatercept-csrk), clearing the drug to reduce the risk of major clinical worsening events, including PAH-related hospitalization, lung transplantation and death. Approval was based on data from the Phase 3 ZENITH trial, which enrolled
Drugs
Inhibrx Biosciences is reporting favorable data for treating chondrosarcoma with its antibody therapy ozekibart (INBRX-109). New data from the Phase 3 ChonDRAgon trial showed the therapy met the primary endpoint in advanced or metastatic, unresectable disease. According to the topline data, treatment cut the risk of progression or death
Drugs
FDA has posted a two-item Form FDA-483 from an August inspection of Sumitomo Pharma’s Suzuka, Japan, manufacturing facility that cited GMP deficiencies. The agency cited the firm over its sampling procedures involving incoming raw materials and excipients. It said Sumitomo had not established a documented rationale or
Drugs
A payroll system error has delayed paychecks for some FDA employees who are designated to continue working during the ongoing government shutdown, according to reporting from Federal News Network. Shutdown-exempt staff at FDA are typically paid on time using carryover funds. However, HHS spokesperson Emily Hilliard told the media
Drugs
FDA has approved Bayer’s Lynkuet (elinzanetant) to treat moderate to severe vasomotor symptoms, or hot flashes, associated with menopause, marking the first U.S. approval for a therapy that targets both neurokinin 1 (NK1) and neurokinin 3 (NK3) receptors. Approval was based on data from Bayer’s Phase 3
Drugs
FDA has issued Sydnexis a complete response letter for SYD-101, a low-dose atropine eye-drop candidate intended to slow disease progression in children with pediatric progressive myopia. The agency concluded that the data submitted do not support the drug’s effectiveness in treating myopia in children, despite acknowledging
Drugs
FDA has granted Partner Therapeutics a breakthrough therapy designation for zenocutuzumab-zbco and its use for treating cholangiocarcinoma defined by NRG1 gene fusions. The designation covers adults with advanced unresectable or metastatic NRG1+ cholangiocarcinoma, the company says, and is based on data from the Phase 2 eNRGy trial. The company
Drugs
FDA has granted Heidelberg Pharma a fast track designation for HDP-101 (pamlectabart tismanitin), the company’s lead amanitin-based antibody drug conjugates candidate. The designation was based on data from the ongoing Phase 1-2a study that is evaluating the drug’s safety and antitumor activity in patients with
Drugs
Ventyx Biosciences is reporting positive Phase 2 data for its oral NLRP3 inhibitor VTX3232 in patients with obesity and cardiovascular risk factors. The 175-participant study examined VTX3232 versus placebo, alone or in combination with semaglutide (Novo Nordisk's Ozempic and Wegovy), and evaluated safety and tolerability as the
Drugs
FDA has approved a GSK BLA for Blenrep (belantamab mafodotin-blmf) in combination with bortezomib (Velcade) and dexamethasone for treating adult patients with relapsed or refractory multiple myeloma who have received at least two prior lines of therapy, including a proteasome inhibitor (PI) and an immunomodulatory (IMID) agent. The BLA
Drugs
FDA has published a guidance, Expanded Access to Investigational Drugs for Treatment Use, providing information in a question-and-answer format for industry, researchers, doctors, institutional review boards, and patients about the implementation of the agency’s 10/13/2009 regulations on expanded access to investigational drugs for treatment use
Drugs
Tango Therapeutics says new data from its ongoing Phase 1-2 study of vopimetostat (TNG462) showed early signs of efficacy across multiple methylthioadenosine phosphorylase (MTAP)-deleted cancers, including pancreatic and lung tumors, paving the way for a pivotal trial next year. Such cancers are described as malignancies characterized by the
Drugs
The investigative newsroom ProPublica says FDA is keeping vital information about issues in generic drug foreign manufacturing from doctors, pharmacists, and patients who could use the information to make more informed decisions. One of the report’s concerns is that for decades, the agency has redacted the names of generic
Drugs
The CDER Office of New Drugs has published a Manual of Policies and Procedures (MAPP) with the policies, responsibilities, and procedures for CDER staff to follow when determining whether there is a basis to refuse to file (RTF) an NDA, sNDA, BLA, or sBLA submitted to the Center. The document
Drugs
To increase transparency and reduce the prevalence of drug regulatory filing deficiencies, FDA has made public the filing checklists CDER uses to determine if a submitted application is complete and reviewable. An agency statement notes that filing deficiencies consume resources and delay promising treatments. “Drug applications should not be derailed