Drugs
FDA has published a guidance, Alternative Tools: Assessing Drug Manufacturing Facilities Identified in Pending Applications, giving applicants information on how the agency intends to use alternative tools to assess drug manufacturing facilities identified in NDAs, BLAs, ANDAs, or their supplements. The document meets a requirement in the PDUFA 7 and
Drugs
Intercept Pharmaceuticals says it is withdrawing Ocaliva (obeticholic acid) from the U.S. market for treating primary biliary cholangitis (PBC), following an FDA request that cited liver injury concerns. Last December, FDA issued a drug safety communication, saying its review of postmarket clinical trial data identified cases of serious liver
Drugs
While rapid scientific advances are accelerating the development of medical innovations, significant FDA policy and regulatory reforms are needed to fully use the potential of the new technologies. That’s the conclusion in a white paper issued by the University of Southern California Schaeffer Center for Health Policy and Economics.
Drugs
The Securities and Exchange Commission (SEC) says it has accepted an offer from FibroGen to resolve a cease-and-desist order covering false and/or materially misleading statements about the results of certain cardiovascular safety analyses of the firm’s anemia drug roxadustat. A commission order details how the firm’
Drugs
The Clinical Trials Transformation Initiative (CTTI) held a 5/22 “State of Clinical Trials” meeting for influential leaders, high-level agency officials, policymakers, and key CTTI members to hold “solution-driven conversations about advancing the clinical trial enterprise,” to identify concrete, realistic ways to improve efficiency and quality in clinical
Drugs
FDA has issued a draft guidance, Development of Non-Opioid Analgesics for Chronic Pain, to assist sponsors in developing such drugs. The document says it is intended to address two agency priorities — fostering the development of novel analgesic products, and decreasing opioid analgesic exposure and preventing new addiction. The guidance
Drugs
FDA has approved a Johnson & Johnson NDA for Inlexzo (gemcitabine intravesical system), offering a new treatment option for adults with non-muscle invasive bladder cancer (NMIBC) who do not respond to standard Bacillus Calmette-Guérin (BCG) therapy. The therapy is the first intravesical drug-releasing system designed to deliver
Drugs
FDA has granted Greenwich LifeSciences fast track status for GLSI-100, an investigational immunotherapy aimed at preventing breast cancer recurrences. The designation applies to HLA-A*02-genotype patients with HER2-positive breast cancer who have completed standard HER2-targeted therapy. The company says GLSI-100 has already shown promise
Drugs
FDA has accepted and will consider a Corcept Therapeutics NDA for relacorilant to treat patients with platinum-resistant ovarian cancer. The agency set 7/11/2026 as the PDUFA action date for the NDA. A company statement says the NDA is based on data from its pivotal Phase 3 ROSELLA
Drugs
FDA has approved AstraZeneca’s Koselugo (selumetinib) granules and capsules for pediatric patients one year of age and older with neurofibromatosis type 1 (NF1) who have symptomatic inoperable plexiform neurofibromas (PN). The agency previously approved Koselugo for pediatric patients aged two and older with NF1 who have symptomatic, inoperable PN.
Drugs
A 2/3-2/7 FDA inspection at the Hikal Limited drug manufacturing facility in Jigani, Karnataka, India, found significant deviations from current good manufacturing practice (CGMP) regulations for active pharmaceutical ingredients. An 8/20 Warning Letter says the specific deviations were: * failure of the firm’s quality unit to
Drugs
An FDA 8/5-8/23/2024 Bioresearch Monitoring inspection at the clinical site maintained by Dr. Shirish Gadgeel, a lung cancer medical oncologist, in Detroit, MI, found violations in the conduct of three clinical investigations. A 9/4 Warning Letter says the specific violations were: * failure to ensure that
Drugs
Former CBER Office of Therapeutic Products (OTP) deputy director Rachael Anatol, who was removed from her post and placed on administrative leave in June, has been hired by Eli Lilly as associate VP, global regulatory policy and strategy — genetic medicine. “In my new role I will be working to advance
Drugs
Lexicon Pharmaceuticals says it has submitted new clinical data to FDA in an effort to advance its stalled NDA for its diabetes drug Zynquist (sotagliflozin). The company is seeking to address concerns raised in a 12/2024 complete response letter that cited an elevated risk of diabetic ketoacidosis (DKA) with
Drugs
Capricor Therapeutics says it is posting on its Web site the company’s “comprehensive preliminary response” to an FDA complete response letter (CRL) for the BLA for Bytreldo (deramiocel), an investigational cell therapy intended to treat cardiomyopathy associated with Duchenne muscular dystrophy (DMD). While acknowledging in a statement FDA’s
Drugs
NRx Pharmaceuticals has petitioned FDA seeking a ban on all ketamine products formulated with the preservative benzethonium chloride, arguing that the substance poses unnecessary health and environmental risks. In the petition, the company says the preservative was included in early formulations of ketamine hydrochloride (marketed as Ketalar) in the 1970s
Drugs
The 7th Circuit Court of Appeals has affirmed a lower court’s dismissal of a False Claims Act (FCA) retaliation suit against AbbVie (formerly Allergan) that was brought by a former company sales representative. The sales rep, Jeffrey Lewis, said he was denied a promotion and given onerous assignments after
Drugs
FDA is reminding companies and the public that they have until 9/19 to submit comments on a draft guidance entitled E21 Inclusion of Pregnant and Breastfeeding Women in Clinical Trials. The document provides recommendations for the appropriate inclusion and retention of pregnant or breastfeeding women in clinical trials. The
Biologics
The latest Make America Healthy Again (MAHA) Commission report, “Make Our Children Healthy Again: Assessment,” says FDA has a role to play in working with the National Institutes of Health to “build systems for real-world safety monitoring of pediatric drugs and create programs to independently replicate findings from industry-
Drugs
FDA has issued an updated guidance entitled E6(R3) Good Clinical Practice (GCP) that describes GCP principles, emphasizing flexible, modern approaches to clinical trial design and conduct while maintaining participant safety and data integrity. The guidance, aligned with the International Council for Harmonization, notes that well-designed and well-executed
Drugs
Eli Lilly’s Bruton tyrosine kinase (BTK) inhibitor Jaypirca (pirtobrutinib) significantly extended progression-free survival in previously untreated patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL), according to topline results from a Phase 3 study. The company says the BRUIN CLL-313 trial compared pirtobrutinib to chemoimmunotherapy
Drugs
Merck is reporting favorable results from the IDeate-Lung01 Phase 2 trial showing that ifinatamab deruxtecan (I-DXd) demonstrated clinically meaningful response rates in patients with previously treated extensive-stage small cell lung cancer (ES-SCLC). The potential first-in-class B7-H3 directed DXd antibody drug conjugate is being