Drugs
FDA has cited major quality control and data integrity concerns at an Indian manufacturing facility operated by Intas Pharmaceuticals, including repeated stability failures tied to a drug product distributed in the U.S. and allegations that electronic batch records were altered without proper documentation. The findings were detailed in a
Drugs
Dyne Therapeutics has submitted a BLA that seeks accelerated approval for its experimental Duchenne muscular dystrophy (DMD) therapy z-rostudirsen for patients amenable to exon 51 skipping. The submission is based on data from the registrational expansion cohort of the Phase 1/2 DELIVER study, in which the therapy demonstrated
FDA Policy/General
FDA Matters founder Steven Grossman says it’s hard to imagine a new FDA commissioner taking office before December 2026, and the early months of 2027 would be even more likely. Writing in his online newsletter, Grossman says, “It’s highly unlikely that anyone will be nominated soon, and no
Devices
Senator Tom Cotton (R-AR) is pressing FDA and government cybersecurity officials to review Chinese-made medical devices cleared before updated cybersecurity requirements took effect, citing concerns that vulnerable devices could expose Americans to espionage, fraud and potentially dangerous remote manipulation. In a 5/26 letter to acting FDA commissioner
FDA Policy/General
The long-term impact of the Supreme Court’s Loper Bright decision in the health landscape remains unclear, according to an article in the Yale Journal on Regulation. Immediately after the decision was announced, many commentators warned of a coming “seismic” impact. Written by staff and a student at the
Drugs
Biogen and Denali Therapeutics say they will limit the development of their experimental Parkinson’s disease drug BIIB122 after the therapy failed to meet primary and secondary endpoints in a Phase 2b clinical trial. The companies reported that the LUMA study showed BIIB122, also known as DNL151, did not slow
Drugs
FDA has approved Daiichi Sankyo’s Datroway (datopotamab deruxtecan-dlnk) for adults with unresectable or metastatic triple-negative breast cancer (TNBC) who are not candidates for PD-1/PD-L1 inhibitor therapy, expanding treatment options for patients with one of the most aggressive forms of breast cancer. The drug was
Drugs
FDA has granted Gilead Sciences an accelerated approval for Hepcludex (bulevirtide-gmod) for treating chronic hepatitis delta virus (HDV) infection, marking the first FDA-approved therapy for the rare but potentially life-threatening liver disease. The approval covers treatment in adults with chronic hepatitis delta virus infection who do not
Devices
FDA has issued an early alert about a potentially high-risk software problem involving certain heart pump controllers manufactured by Abiomed that could temporarily stop circulatory support during treatment. The agency says Abiomed has initiated an urgent medical device correction affecting all Automated Impella Controllers used with left ventricular Impella
FDA Policy/General
FDA’s recent draft guidance on Form-483 responses is signaling tougher expectations for how drug manufacturers respond to inspection observations, according to a regulatory affairs analysis published on clinicalleader.com by Rivka Zaibel of ADRES Bio. The draft guidance, which outlines FDA expectations for responses following GMP inspections, emphasizes
Drugs
The Biotechnology Innovation Organization (BIO) says an FDA draft guidance on general considerations for using new approach methodologies (NAMs) in drug development “marks an important step toward establishing a flexible science-based framework for integrating NAMs into drug development.” The BIO comment letter highlights areas where the association believes additional
Devices
React Health is recalling (Class 1) certain VOCSN V+Pro ventilators due to a manufacturing issue that could lead to undetected oxygen leaks and potentially cause serious injury or death, according to an FDA notice. The recall affects VOCSN V+Pro Package devices with catalog number PRT-01198-000 and
Devices
FDA says the Bolton Medical 8/23 recall (correction) notice, with updated use instructions for all RelayPro Thoracic Stent Graft Systems, is a Class 1 recall, the most serious type of medical device recall. An agency notice says the company also recommended that customers use alternative stent-graft options before
Drugs
FDA has published the International Council for Harmonization (ICH) M11 Template: Clinical Electronic Structured Harmonized Protocol, which is intended for interventional clinical trial protocols and is said to be suitable for all phases and therapeutic areas of clinical research. The document is marked “For Reference Only.” It says that existing
Drugs
In response to an FDA request for comments on increasing access to non-prescription drugs, Pharmaceutical Research and Manufacturers of America (PhRMA) says it commends the agency’s commitment to expanding the appropriate availability of non-prescription drugs. “While PhRMA is aligned with the goal of increasing access to non-
Biologics
FDA is announcing a 6/18 advisory committee meeting to review Moderna’s mRNA-based seasonal influenza vaccine. The Vaccines and Related Biological Products Advisory Committee is scheduled to review Moderna’s MFLUSIVA (influenza vaccine, mRNA) for preventing influenza disease caused by influenza virus subtypes A and B represented in
Biologics
HHS secretary Robert F. Kennedy Jr. is facing renewed scrutiny from Senate Democrats over the role of longtime anti-vaccine activist David Geier at the Department, as lawmakers say the agency has failed to provide details about his employment despite earlier promises to do so. According to a 2/21
Drugs
Eli Lilly says its investigational obesity drug retatrutide produced weight-loss results approaching those historically associated with bariatric surgery in a pivotal Phase 3 clinical trial, strengthening the company’s position in the increasingly competitive obesity treatment market. The drug met all primary and key secondary endpoints in the TRIUMPH-
Drugs
A 3/17-3/20/2025 FDA Bioresearch Monitoring Program inspection of a clinical trial conducted by Dr. Sourav Mishra at the All India Institute of Medical Sciences in Bhubaneswar, Odisha, India, found that the clinical investigation failed to meet statutory and regulatory requirements for the conduct of clinical investigations
Drugs
A 2/14-3/13/2025 FDA Bioresearch Monitoring Program inspection at a clinical site operated by cardiologist Naseem Jaffrani in Alexandria, LA, found he did not follow statutory requirements and regulations governing the conduct of clinical investigations and the protection of human subjects. A 5/4 Warning Letter says
FDA Policy/General
Former FDA associate commissioner Peter Pitts, now president of the Center for Medicine in the Public Interest, says the recent resignation of FDA commissioner Marty Makary can be a positive development for American public health if whoever is appointed as his successor can learn from Makary’s mistakes and change
Biologics
FDA has to create a protocol to review Intellia Therapeutics’ nex-z, a permanent, single-dose genomic edit in 60,000 base pairs of a living patient’s hepatocytes that targets transthyretin amyloidosis (ATTR). An online Clinical Trial Vanguard post says the therapy uses liquid nanoparticles to deliver CRISPR-Cas9