Drugs
FDA has granted Biogen a breakthrough therapy designation for its experimental antibody litifilimab for treating cutaneous lupus erythematosus (CLE), a chronic autoimmune skin disease for which there are no approved targeted therapies. The designation was based on the totality of data generated to date for litifilimab, including results from the
Drugs
A group of Democratic senators is pressing HHS’ Office of Inspector General to clarify how it will oversee direct-to-consumer prescription drug sales tied to the forthcoming TrumpRx Web site, raising concerns about potential violations of federal fraud and abuse laws. In a 1/29 letter to HHS, Sens.
FDA Policy/General
The current climate at FDA is unprecedented in terms of the depth and breadth of its politicization. That’s the view of University of Southern California economist and associate professor of public policy Genevieve Kanter in an online interview with PharmaVoice. She says that while all presidential administrations use appointed
Drugs
FDA has approved Tenpoint Therapeutics’ Yuvezzi (carbachol and brimonidine tartrate ophthalmic solution) 2.75%/0.1% (previously known as Brimochol PF) as the only dual-agent eye drop to treat presbyopia in adults. A company statement says the approval was based on positive data from two Phase 3 studies enrolling
Drugs
Reduced FDA staffing levels and high turnover are extending regulatory timelines and forcing drug developers to invest more heavily in upfront planning, according to an industry consultant. In a recent interview with Applied Clinical Trials, Charlie Paterson, a partner at PA Consulting, said sponsors are facing longer wait times for
Drugs
A Congressional Research Service (CRS) background paper on “‛Skinny Labels’ for Generic Drugs Under Hatch-Waxman” raises changes that Congress could consider if it wanted to clarify the provisions. The paper gives extensive background on the generic drug approval process under Hatch-Waxman and the role of so-called skinny
Drugs
To meet the legal requirement that it take final action on a petition within 150 days, FDA has denied without commenting on the specifics an 8/29/2025 petition submitted by Foley & Lardner asking for restrictions on approval for any ANDA citing Novo Nordisk’s Ozempic (semaglutide) as the
Drugs
Boehringer Ingelheim says its investigational kidney drug apecotrep reduced proteinuria by 40% compared with placebo in a Phase 2 clinical trial involving patients with primary focal segmental glomerulosclerosis (FSGS), a rare and progressive kidney disease with no approved targeted treatments. The 12-week, placebo-controlled study showed the strongest effect
Drugs
Public Citizen has filed a suit to force the Trump administration to release the texts of the drug pricing agreements it has reached with Pfizer and Eli Lilly. The advocacy group says it was forced to file suit in the DC federal court because both HHS and the Department of
Drugs
FDA has cited Nubratori Inc., a California-based sterile drug outsourcing facility, for multiple manufacturing and contamination-control deficiencies following a December inspection of the company’s Torrance site, according to an agency inspection report. The Form FDA 483 said the company failed to establish adequate written procedures for cleaning
Drugs
HHS’ Office of Inspector General (OIG) has issued new guidance clarifying when pharmaceutical manufacturers can sell prescription drugs directly to patients enrolled in federal health care programs without violating the federal Anti-Kickback Statute. The guidance comes as direct-to-consumer drug sales have begun to expand, including in connection
Drugs
A 7/14-7/18/2025 FDA inspection at the Fulijaya Manufacturing drug manufacturing facility in Kulim, Kedah, Malaysia, found significant violations of current good manufacturing practice (CGMP) regulations for finished drugs. A 1/14 Warning Letter says the specific violations were: * failure to ensure that laboratory records included complete
Drugs
A new Friends of Cancer Research white paper, “Leveraging AI-Enabled Tumor Assessment Tools on Radiological Images to Evaluate Treatment Effect and Support Clinical Trial Endpoints in Solid Tumors,” presents findings from a multi-stakeholder working group it convened to evaluate the current landscape of artificial intelligence (AI)-enabled tumor
Drugs
Roche says its experimental dual GLP-1/GIP receptor agonist CT-388 delivered substantial and statistically significant weight loss in a Phase 2 study of people living with obesity, supporting the drug’s advancement into late-stage development. Topline results from the CT388-103 trial showed that once-weekly subcutaneous
FDA Policy/General
The year 2025 marked a period of significant transformation in FDA and Department of Justice (DoJ) enforcement activities. While traditional priorities around product quality, safety, and clinical research integrity remained central, the enforcement landscape increasingly reflected the current administration’s policy objectives, workforce restructuring, and evolving legal frameworks. A Ropes
Drugs
FDA has lifted a clinical hold on Intellia Therapeutics’ MAGNITUDE-2 Phase 3 trial evaluating nexiguran ziclumeran, or nex-z, for patients with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN), clearing the way for the company to resume patient enrollment and dosing. The gene editing company says FDA removed the
Drugs
Drug companies say that while artificial intelligence (AI) still cannot find new molecules that could lead to breakthrough drugs, it is being used to speed up some of the tedious, time-consuming aspects of drug development and application submission. “AI is helping find participants and sites for clinical trials and
Drugs
A federal judge has sentenced former CytoDyn chief executive Nader Pourhassan to 30 months in prison for misleading investors about the development of an experimental drug for HIV and Covid-19 and selling his personal shares at artificially inflated prices. Between 2018 and 2021, Pourhassan intentionally misled investors about the
Drugs
A newly announced FDA pathway allowing drug approvals based on a “plausible mechanism of action” could accelerate the development of highly individualized treatments for rare genetic diseases, but health policy experts are warning that the approach raises unresolved legal, scientific, and transparency concerns. The pathway for approving certain bespoke, personalized
Drugs
FDA has cited Pine Pharmaceuticals for multiple quality and sterility violations following a 10-day inspection of the company’s drug outsourcing facility in Tonawanda, NY, according to a just-released inspection report issued in mid-November. The inspection identified significant deficiencies in the company’s handling of sterile drug
Drugs
FDA says the majority of novel drugs approved in 2025 moved through accelerated regulatory pathways, underscoring the agency’s continued emphasis on speeding patient access to therapies for serious and unmet medical needs. In its just-released annual report, Advancing Health Through Innovation: New Drug Therapy Approvals 2025, CDER says
Drugs
FDA says it continued to improve the speed and consistency of drug manufacturing oversight in fiscal year 2024, citing strong performance under an integrated inspection and facility evaluation framework that has been in place since 2017. According to an FDA summary released this week, the agency met or exceeded key