Drugs
Rep. Abe Hamadeh (R-AZ) is calling on FDA to pause its recent enforcement actions against desiccated thyroid medications in response to allegations made against former CDER director George Tidmarsh (see earlier story). In a statement (contains a link to his letter to FDA commissioner Marty Makary), Hamadeh says he
FDA Policy/General
Despite recent departures among its senior ranks and turmoil caused by the ongoing government shutdown, FDA remains robust, according to commissioner Marty Makary, who spoke at the Milken Institute’s 2025 Future of Health Summit 11/6. Makary emphasized a shift toward a more collaborative culture and outlined ambitious plans
Drugs
Centessa Pharmaceuticals is reporting encouraging clinical data for its oral OX2R agonist ORX750 in patients with narcolepsy Types 1 and 2 (NT1, NT2) and idiopathic hypersomnia (IH). In a third-quarter financial update, the biotech firm says data from the initial dosing cohorts of its ongoing Phase 2a CRYSTAL-1
Drugs
A 3/25-4/8 FDA inspection at the Liebel-Flarsheim drug manufacturing facility in Raleigh, NC, identified significant violations of current good manufacturing practice (CGMP) regulations for finished drugs. A 10/17 Warning Letter says the specific violations were: * failure to thoroughly investigate any unexplained discrepancy or failure of
Drugs
FDA has released the form FDA-483 issued following a 5/26-5/31 inspection at the Alembic Pharmaceuticals active pharmaceutical ingredient manufacturing facility in Tal. Halol, Gujarat, India. The four inspection observations were: * failure to conduct timely laboratory investigations or to thoroughly determine the root cause; * failure of laboratory
Biologics
The 11/5 Medpage Investigative Roundup highlights key findings in a STAT report (subscription required) about chaos at CBER under the leadership of Center director Vinay Prasad. The report indicates that several CBER employees have filed human resources complaints about the director’s management style and practices. It says employees
Drugs
FDA has issued Biohaven a complete response letter, rejecting the company’s NDA for Vyglxia (troriluzole) as a treatment for spinocerebellar ataxia (SCA), a rare and progressive neurodegenerative disease with no approved therapies. The agency letter cited concerns about potential bias and design limitations inherent in the real-world evidence
Drugs
FDA has issued a Warning Letter to Apotex, citing significant GMP violations at the company’s Richmond Hill, Ontario, facility, including failures in sterility assurance, equipment maintenance, and quality investigations. The 10/31 letter follows an inspection conducted from 4/28 to 5/9 at the site, which manufactures sterile
Drugs
A 4/21-5/2 FDA inspection at the Scientific Protein Laboratories drug manufacturing facility in Waunakee, WI, found significant deviations from current good manufacturing practice (CGMP) regulations for active pharmaceutical ingredients (APIs). A 10/10 Warning Letter says the specific deviations were: * failure to ensure that equipment surfaces in
FDA Policy/General
President Donald Trump and the White House have “total confidence” in FDA commissioner Marty Makary as he weathers yet another agency crisis with the abrupt departure of new CDER director George Tidmarsh, who was accused of official misconduct by a drug company. A Politico article quotes from an e-mail
FDA Policy/General
Sydnexis says its SYD-101, a proprietary 0.01% atropine formulation developed with enhanced ocular delivery characteristics and room-temperature stability to slow the progression of myopia in children, met both the primary and key secondary endpoints in the Phase 3 STAR trial. A company statement says treatment effects were
Drugs
FDA has approved a Meitheal Pharmaceuticals NDA for Contepo (fosfomycin) for injection for treating adults with complicated urinary tract infections (cUTI), including acute pyelonephritis, caused by susceptible Escherichia coli and Klebsiella pneumoniae. Fosfomycin is described by the company as a novel epoxide antibacterial and the only intravenous antibiotic of its
FDA Policy/General
Reflecting on the recent FDA turmoil over the resignation of CDER director George Tidmarsh, Axios questions how long industry can “tolerate an underperforming and dysfunctional FDA … before looking to other markets.” Tidmarsh resigned as controversy broke over comments he made on social media that were part of a lawsuit alleging
Drugs
Sarepta Therapeutics says its long-running Phase 3 ESSENCE confirmatory study evaluating its Duchenne muscular dystrophy drugs Amondys 45 (casimersen) and Vyondys 53 (golodirsen) showed mixed data, but the company still plans to meet with FDA to discuss converting the therapies from accelerated to traditional approval. The nine-year, placebo-
Drugs
Compass Pathways says it will accelerate by nine to 12 months the planned launch of its COMP360 psilocybin treatment for treatment-resistant depression. A company statement says moving up the projected launch follows completion of the COMP006 trial enrollment and a positive meeting with FDA on NDA submission strategies, including
Drugs
Otsuka ICU Medical has recalled (Class 1) one lot of its potassium chloride injection, 20 mEq, after discovering that the overwrap label was incorrectly printed as 10 mEq, creating a dosing error risk. The company says the error was due to a manufacturing issue. Because the overwrap conceals the inner
Devices
FDA has given investigational device exemption (IDE) approval for an Aqua Medical pilot clinical trial of its Proximal Intestinal Mucosal Ablation (PIMA) procedure using the company’s proprietary radiofrequency vapor ablation (RFVA) system. A company statement says FDA has already granted the system a breakthrough device designation to treat adults
Drugs
A new report from the Reagan-Udall Foundation for the FDA is calling for broad operational and policy reforms to improve the efficiency, representativeness, and success rates of oncology multi-regional clinical trials. The report, Improving Oncology Multi-Regional Clinical Trials, summarizes findings from a 9/4 roundtable that brought
Drugs
FDA has lifted a clinical hold against a Rein Therapeutics Phase 2 (RENEW) trial evaluating LTI-03 in patients with idiopathic pulmonary fibrosis, allowing the company to resume enrollment in the U.S. The FDA action follows a review of Rein’s complete response submission, which addressed all outstanding regulatory
Drugs
Aurinia Pharmaceuticals’ lawsuit against CDER director George Tidmarsh includes screenshots of troubling text messages and emails from Tidmarsh to Kevin Tang, a biotech investor who chairs Aurinia’s board and heads Tang Capital Partners, the company’s largest investor. Tidmarsh resigned on Sunday, the day the lawsuit was filed in
Drugs
CDER director George Tidmarsh has stepped down after being placed on administrative leave amid an internal ethics investigation and a defamation lawsuit tied to a public post criticizing a company’s drug. Tidmarsh’s abrupt exit — just four months after his appointment to lead the agency’s drug center — comes
Drugs
FDA has approved UCB’s Kygevvi (doxecitine and doxribtimine), which the company describes as the first treatment for thymidine kinase 2 deficiency (TK2d), a life-threatening genetic mitochondrial disease characterized by progressive muscle weakness. The approval covers adults and pediatric patients whose symptoms begin at or before 12 years of