Drugs
FDA is stepping up efforts to close persistent gaps in clinical trial reporting, warning that incomplete or delayed disclosures threaten public trust and scientific progress. An online post from CDER director George Tidmarsh notes that clinical trial transparency is “an ethical obligation under the Declaration of Helsinki” and a cornerstone
Drugs
AbbVie has filed an NDA for tavapadon, an investigational once-daily oral therapy for Parkinson’s disease, based on data from late-stage clinical trials that showed the treatment improved symptoms across different stages of the condition. Tavapadon is described by the company as a selective dopamine D1/D5 receptor
Devices
FDA has granted Essilor a de novo marketing authorization for its Stellest eyeglass lenses, which are designed to both correct vision and slow the progression of pediatric myopia (nearsightedness). The lenses are indicated for children aged 6 to 12 years at the start of treatment, with or without astigmatism. “Today’
Drugs
FDA has approved a Crinetics NDA for Palsonify (paltusotine) for treating adults with acromegaly who have had an inadequate response to surgery and/or for whom surgery is not an option. The rare endocrine disorder causes some bones, organs, and other tissue to grow bigger, according to an FDA release.
Drugs
FDA has issued Novartis Pharmaceuticals an untitled letter citing the company’s allegedly false or misleading direct-to-consumer television advertisement for Fabhalta (iptacopan), saying the ad downplays serious safety risks and exaggerates the drug’s benefits. Fabhalta, approved 12/2023, is a twice-daily oral treatment for paroxysmal nocturnal
Biologics
Capricor Therapeutics says it has reached agreement with FDA on the design and role of its HOPE-3 trial as it works to resubmit its BLA for Deramiocel and its use in treating Duchenne muscular dystrophy (DMD). The update follows a Type A meeting with the agency, convened after it
Drugs
Hansa Biopharma says investigational imlifidase met the primary endpoint in a pivotal Phase 3 study in highly sensitized kidney transplant patients, setting up a potential U.S. regulatory filing later this year. The U.S.-based ConfIdeS trial enrolled adults with a calculated panel reactive antibody level of 99.9%
Drugs
FDA has published its Electronic Submission Gateway NextGen Unified Submission Portal Guide for Industry Users, outlining the functionalities, processes, and requirements for submitting regulatory information through the NextGen Unified Submission Portal (USP). The document says it is intended to facilitate a smooth transition to Electronic Submission Gateway NextGen, providing users
FDA Policy/General
A survey conducted by the University of Pennsylvania’s Annenberg Public Policy Center (APPC) shows that public confidence in FDA and other U.S. health and science agencies has dropped across the board since the 1/20 inauguration of Donald J. Trump as president. An APPC news release says the
Drugs
FDA has approved an Eli Lilly NDA for Inluriyo (imlunestrant, 200 mg tablets), an oral estrogen receptor antagonist for treating adults with estrogen receptor-positive (ER+), human epidermal growth factor receptor 2-negative (HER2–), ESR1-mutated advanced or metastatic breast cancer (MBC) whose disease progressed after at least one line
Biologics
CBER has begun to post, more proactively, inspection-related Form FDA-483s under the Freedom of Information Act’s provision on “frequently requested records.” Under the regulation, FDA can release electronic copies of any records that have been previously released if it determines that the records are likely to be
Devices
FDA has granted Biolinq a de novo authorization for its Shine system, the first fully autonomous, needle-free glucose sensor in the U.S, according to the company. The device features an integrated display and does not require a smartphone to deliver glucose readings. Unlike traditional continuous glucose monitors that
Drugs
In a 9/19 letter to 20 Republican attorneys general, HHS secretary Robert F. Kennedy, Jr., and FDA commissioner Martin Makary say FDA is leading a review of the medication abortion drug mifepristone. Kennedy and Makary wrote in response to a 7/31 letter from the attorneys general raising concerns
Drugs
FDA says it has removed the REMS (Risk Evaluation and Mitigation Strategy) program for Sanofi’s thyroid cancer drug Caprelsa (vandetanib). An agency notice says Caprelsa will remain available with the same prescribing information, but healthcare providers will no longer need special certification or extra monitoring beyond standard clinical care.
Drugs
A 12/3-12/6/2024 FDA Bioresearch Monitoring Program inspection involving two clinical trial protocols at the University of Texas Health Science Center at San Antonio, with Dr. Ralph DeFronzo as clinical investigator, found “objectionable conditions” and a failure to follow the law and regulations governing the conduct of
Drugs
Harmony Biosciences says topline results from its Phase 3 registrational clinical trial (RECONNECT study) of ZYN002 in Fragile X syndrome (FXS) did not meet the primary endpoint of improvement in social avoidance primarily due to a higher-than-expected placebo response rate. “Although the study did not achieve its primary
Biologics
FDA has released a draft guidance outlining methods drugmakers should use to collect long-term safety and efficacy data once cell and gene therapy products are approved. The recommendations reflect growing concern over how to monitor treatments that may have lifelong effects but are tested in relatively small patient populations
Biologics
FDA says that despite repeated requests, Cellebration LLC, headquartered in San Juan, Puerto Rico, has failed to provide records sought by the agency since 10/18/2024. A 9/12 Warning Letter says the Cellebration Web site was reviewed by FDA, which found that the company offers cellular products derived
Drugs
Acadia Pharmaceuticals says it will cease developing intranasal carbetocin after its Phase 3 COMPASS PWS trial of the drug for treating hyperphagia in patients with Prader-Willi syndrome (PWS) did not achieve its primary or secondary endpoints. The 12-week, randomized, placebo-controlled study enrolled 175 children and adults ages
Biologics
FDA has published a draft guidance, Expedited Programs for Regenerative Medicine Therapies for Serious Conditions, with the agency’s recommendations on the expedited development and review of such therapies. The document describes the expedited programs available to sponsors of regenerative medicine therapies for serious conditions, including those products designated as
Biologics
FDA has published a draft guidance, Innovative Designs for Clinical Trials of Cellular and Gene Therapy Products in Small Populations, with recommendations for sponsors who are planning clinical trials of cell and gene therapy (CGT) products intended for use in a disease or condition that affects a small population. The
Drugs
Reps. August Pfluger (R-TX) and Greg Landsman (D-OH) have introduced the Biosimilar Red Tape Elimination Act, which they say would improve patient access to lower-cost prescription medication and increase competition in the biological drug market by automatically deeming biosimilar drugs as interchangeable with their branded counterparts upon