Drugs
Pharmaceutical Research and Manufacturers of America (PhRMA) says it appreciates FDA’s attention to container closure systems in light of the significant technological advances that have emerged in material science and drug delivery systems since the agency’s 1999 guidance on the same topic. In a comment letter on an
FDA Policy/General
Former FDA commissioner Scott Gottlieb is calling on Congress to establish a new regulatory pathway for clinical artificial intelligence models developed and used within hospitals, warning that the absence of consistent federal oversight could undermine patient safety and limit broader commercialization of clinical AI technologies. In an October 8 commentary
Drugs
FDA says it is launching a Rare Disease Framework Initiative “to reimagine the development of drugs and biologics for rare diseases.” In a statement, which includes a link to a new white paper on the initiative, the program will “develop an optimized, fit-for-purpose framework that reflects the realities
Drugs
A 4/7-4/18/2025 FDA Bioresearch Monitoring Program inspection at Lovelace Biomedical Research Institute in Albuquerque, NM, found objectionable conditions in the facility’s role in conducting two nonclinical laboratory studies. A 10/6 Warning Letter says the specific violation was: * failure of the quality assurance unit to
Drugs
A 3/26-4/2 FDA inspection of the Silgan Unicep Packaging drug manufacturing facility in Spokane, WA, found significant violations of current good manufacturing practice (CGMP) regulations for finished drugs. A 10/5 Warning Letter says the specific violations were: * failure to thoroughly investigate any unexplained discrepancy or failure
FDA Policy/General
Ultragenyx Pharmaceutical says it has entered into an agreement to sell to an undisclosed buyer a Rare Pediatric Disease Priority Review Voucher (PRV) for $203 million. Ultragenyx won the PRV upon FDA approval of Fayuvi (rebisufligene etisparvovec-hopf), also known as UX111, the first-ever FDA-approved treatment for mucopolysaccharidosis
Biologics
FDA has accepted for review a Pierre Fabre Pharmaceuticals BLA resubmission with a proposed indication as monotherapy for treating adult and pediatric patients 2 years of age and older with Epstein-Barr virus-positive post-transplant lymphoproliferative disease (EBV+ PTLD) who have received at least one prior therapy. The agency
Biologics
FDA has approved Altor BioScience’s Anktiva (nogapendekin alfa inbakicept-pmln) with Bacillus Calmette-Guérin (BCG) for adults with BCG-unresponsive non-muscle invasive bladder cancer (NMIBC). The approval revises the prior indication for adults with BCG-unresponsive NMIBC with carcinoma in situ (CIS) with or without papillary tumors to
FDA Policy/General
The growing use of online prediction markets Kalshi and Polymarket to wager on clinical trial outcomes and FDA drug approvals could compromise trial integrity, encourage insider trading, and undermine public confidence in regulatory decisions, three Boston University researchers warned. Kalshi launched a pilot program in July (see earlier story) allowing
FDA Policy/General
An analysis by two University of Southern California policy researchers finds that between 2013 and 2022, some 38% of FDA advisory committees included either a patient or consumer representative with active ties to an advocacy organization funded by a drug company. Writing in an open-source article for Project Hope,
Independent news and analysis of FDA regulation, policy and enforcement
Former FDA regulatory counsel and now-partner at Spencer Fane Brian Malkin is questioning whether the agency should become more directly involved in the Trump administration's drug-pricing initiatives, raising concerns about the potential direction of FDA policy under commissioner nominee Heidi Overton, whose nomination remains pending in
FDA researchers and academic collaborators identified nearly 80,000 prescription opioid-related cardiovascular adverse event signals in a large-scale analysis of the agency's Adverse Event Reporting System, suggesting that machine learning could improve postmarketing drug safety surveillance. The study, published September 28 in Frontiers in Drug Safety
A 6/1-6/3 FDA inspection at the Eurofins SF Analytical Laboratories drug manufacturing facility in Horsham, PA, found significant violations of current good manufacturing practice (CGMP) regulations for finished drugs. A just-released 10/6 Warning Letter says the three specific violations were: * failure to ensure that laboratory
FDA has accepted for review a Formycon/Zydus Lifesciences BLA for FYB206, a biosimilar to Merck’s blockbuster cancer drug Keytruda (pembrolizumab). A statement says the biosimilar was developed without a comparative Phase 3 efficacy trial, following a development pathway agreed upon with FDA in early 2025. The companies say
The Federal Trade Commission and seven states have asked a federal court to hold Martin Shkreli in civil contempt, alleging that he repeatedly violated a lifetime pharmaceutical industry ban by trading biotechnology stocks and attempting to influence drug companies. Shkreli, a former hedge fund manager and pharmaceutical executive, gained widespread
Congressional Democrats are questioning the Trump administration's proposal to replace combination childhood vaccines with separate injections, a policy that could require new FDA regulatory reviews because single-disease measles, mumps and rubella vaccines are not currently available in the U.S. In a 10/7 letter to CDC
FDA has issued Olympus a 10/7 Warning Letter based on GMP issues raised during inspections of the firm’s facilities in the U.S., Japan, and Europe conducted in late 2025. The items in the Warning Letter primarily concern “aspects of the company’s quality management system and related
A 4/16-4/24 FDA inspection at the Hanlim Pharm Co. drug manufacturing facility in Gyeonggi-do, South Korea, found violations of current good manufacturing practice (CGMP) regulations for finished drugs. A just-released 10/2 Warning Letter says the specific violations were: * failure to establish and follow appropriate
An 11/3-11/7/2025 FDA inspection at the Jiangmen Shuizirun Sanitary Articles Co. drug manufacturing site in Jiangmen, Guangdong, China, found significant violations of current good manufacturing practice (CGMP) regulations for finished drugs. A just-released 9/23 Warning Letter lists the violations as: * failure to establish procedures
Elevar Therapeutics and China's Hengrui Pharmaceuticals have resubmitted a rivoceranib plus camrelizumab as a first-line treatment for patients with unresectable or metastatic hepatocellular carcinoma (HCC). The companies’ resubmission marks the fourth attempt to win U.S. approval, and follows three previous complete response letters (CRLs) issued in
FDA granted approval to Revolution Medicines' Rasonque (daraxonrasib) for metastatic pancreatic cancer despite substantial differences in treatment responses among patients with different KRAS mutations and limited evidence of effectiveness in patients without RAS mutations, according to a newly released approval document. The 321-page review shows that FDA considered