Drugs
X4 Pharmaceuticals has submitted an NDA for a once-daily, oral mavorixafor to treat individuals aged 12 and older with WHIM (warts, hypogammaglobulinemia, infections, and myelokathexis) syndrome, a rare immunodeficiency. The submission is supported by data from the pivotal 4WHIM Phase 3 clinical trial in individuals with WHIM syndrome. The
Drugs
Seagen and Genmab’s cancer drug Tivdak (tisotumab vedotin-tftv) met its primary endpoint of overall survival in their confirmatory Phase 3 innovaTV 301 trial, the companies said 9/5. The study was conducted in recurrent or metastatic cervical cancer patients with disease progression on or after front-line therapy,
FDA Policy/General
The 5th Circuit Court of Appeals has reversed a Texas federal court’s decision dismissing a suit filed by three doctors against FDA and HHS challenging the agencies’ messaging advising against using ivermectin to treat Covid-19. The appeals court sent the case back to the Texas federal court for
Drugs
Roche says recent data from its Phase 3 ALINA study evaluating Alecensa (alectinib) compared with platinum-based chemotherapy met its primary endpoint of disease-free survival (DFS) in patients with anaplastic lymphoma kinase (ALK)-positive non-small cell lung cancer (NSCLC). The data, which was unveiled during a prespecified interim
Drugs
FDA has released a final guidance entitled “Nontuberculous Mycobacterial Pulmonary Disease Caused by Mycobacterium avium Complex (MAC): Developing Drugs for Treatment.” The document addresses clinical trial design issues, choice of trial population, and endpoints for the treating naïve and refractory NTM-PD caused by MAC. The agency notes that the
Drugs
FDA has released a draft guidance entitled “Post-Warning Letter Meetings Under GDUFA” that provides information on implementing the new program that was created during recent generic drug user fee negotiations. Such meetings are described as “a meeting with FDA regarding the facility’s remediation of deficiencies identified in a
Drugs
Sage Therapeutics says it is reorganizing its business operations, including a 40% workforce reduction, to better support the expected launch of Zurzuave (zuranolone) to treat women with postpartum depression in late 2023. A company statement says the reorganization also will refine its pipeline development efforts to advance SAGE-718 and
Drugs
FDA has published a white paper, “CDER’s Quality Management Maturity Program: Practice Areas and Prototype Assessment Protocol Development,” which discusses the assessment approach planned to be used at establishments participating in the Quality Management Maturity (QMM) program. The paper says the program’s goals are to: * foster a strong
Drugs
While progress has been made in many areas of FDA’s four-part drug overdose prevention framework, the crisis has grown into an even more dangerous public health challenge. That’s the assessment of FDA commissioner Robert Califf, in an online FDA Voices 8/31 post to mark International Overdose
Drugs
FDA has published a guidance, Considerations for the Use of Real-World Data and Real-World Evidence to Support Regulatory Decision-Making for Drug and Biological Products, to discuss the applicability of the IND regulations to various clinical study designs that use real-world data (RWD). The document also clarifies
Drugs
FibroGen says its Phase 3 LELANTOS-2 trial of pamrevlumab for treating ambulatory patients with Duchenne muscular dystrophy did not meet the primary endpoint of change in the North Star Ambulatory Assessment total score from baseline to Week 52. Secondary endpoints (four-stair climb velocity, 10-meter walk/run test,
Drugs
FDA has published a guidance, Enhanced Drug Distribution Security at the Package Level Under the Drug Supply Chain Security Act, to assist supply chain stakeholders, particularly trading partners, with requirements for enhanced drug distribution security at the package level under Section 582 of the Federal Food, Drug, and Cosmetic Act.
Drugs
FDA has published two compliance policy guidances establishing a one-year stabilization period to accommodate the additional time that trading partners in the drug supply chain may need to adhere to Drug Supply Chain Security Act (DSCSA) requirements for electronic drug tracing at the package level. The requirements that were
Biologics
FDA has renewed its funding for the University of Maryland Center of Excellence in Regulatory Science and Innovation (M-CERSI) under a new five-year $50 million cooperative agreement. A university statement says M-CERSI is a partnership between two University of Maryland campuses and is the oldest of the
Drugs
FDA has cleared an Aion Biosystems 510 (k) for its iTempShield device and software system for continuous measurement of body temperature. The quarter-sized, skin-wearable device is supported by cloud-based software and proprietary algorithms to measure body temperatures for several clinical applications, such as oncology sepsis monitoring, post-
Drugs
A Reagan-Udall Foundation project, the Real-World Accelerator to Improve the Standard of collection and curation of race and Ethnicity data in healthcare (RAISE) is working to find ways to ensure that race and ethnicity data are included in the real-world data being used to support clinical trials.
Drugs
A 1/18-1/27 and 2/10-2/13 FDA inspection at K.C. Pharmaceuticals in Pomona, CA, found significant violations of current good manufacturing practice (CGMP) regulations for finished drugs. A just-released 8/3 Warning Letter says specific violations were: * failing to establish and follow appropriate written
Drugs
A 2/13-2/17 FDA inspection at Gadal Laboratories in Miami, FL, found significant violations of current good manufacturing practice (CGMP) regulations for finished drugs. An 8/15 Warning Letter says specific violations were: * failing to validate and establish the reliability of a component supplier’s test analyses at
Drugs
Janssen Pharmaceutical has submitted a supplemental NDA that is seeking to convert Balversa’s (erdafitinib) 2019 accelerated approval to full approval. The drug is indicated for treating adult patients with locally advanced or metastatic urothelial carcinoma that has susceptible fibroblast growth factor receptor genetic alterations, and progressed after receiving a
Drugs
FDA has approved a Bristol Myers Squibb BLA supplement for Reblozyl (luspatercept-aamt) for treating anemia in patients with very low- to intermediate-risk myelodysplastic syndromes (MDS) who have not been previously treated with an erythropoiesis stimulating agent (ESA-naïve) and who may require regular red blood cell (RBC) transfusions.
Drugs
Attorneys Dominic DiSabatino and Cortney Inman (SheppardMullin) say an 8/11 Warning Letter issued by the CDER Office of Prescription Drug Promotion (OPDP) to Exeltis USA over its Facebook post for Slynd (drospirenone) oral progestin tablets, demonstrates that OPDP is “closely monitoring social media drug promotion and will respond to
Drugs
Nine bipartisan members of Congress from Georgia, led by Sen. Raphael Warnock (D-GA) and Rep. Earl Carter (R-GA), are asking FDA for information on the agency’s response to the continuing shortage of chemotherapy drugs and what it is doing to ensure the safety of imported replacement drugs.