Drugs
FDA has delayed its review decision on an Agios Pharmaceuticals supplemental NDA for mitapivat, its oral therapy for adults with both transfusion-dependent and non-transfusion-dependent forms of alpha- and beta-thalassemia. The agency had been expected issue a decision by 12/7, but it informed Agios that its
Drugs
Dyne Therapeutics is reporting positive topline results from its Phase 1/2 DELIVER trial of z-rostudirsen (DYNE-251), saying the investigational exon-skipping therapy produced large increases in dystrophin and measurable functional benefits in boys and young men with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. It
Drugs
Top House Democrats are pressing FDA for documents and explanations after revelations that a senior FDA official circulated new vaccine development guidelines in an internal email (see earlier story) that the lawmakers say contained misinformation about Covid-19 vaccines. In a 12/5 letter sent FDA commissioner Marty Makary, Energy
Drugs
FDA has reportedly postponed a promised safety review of the abortion drug mifepristone, sources familiar with the matter told Bloomberg. The review, originally intended to assess real-world outcomes and risks, has been delayed at the direction of FDA commissioner Marty Makary until after the 2026 midterm elections. “HHS — through
Drugs
A senior CDRH device compliance official is warning industry that persistent shortcomings in complaint handling, corrective actions, and medical device reporting continue to dominate the Center’s enforcement findings, signaling deeper weaknesses in manufacturers’ risk-management systems as the FDA prepares to implement a sweeping update to device quality regulations
Drugs
FDA has approved Juno Therapeutics’ CAR-T therapy lisocabtagene maraleucel (Breyanzi) for adults with relapsed or refractory marginal zone lymphoma (MZL) who have received at least two prior systemic therapies. The decision gives patients with heavily pretreated MZL — a rare form of non-Hodgkin lymphoma — a new treatment option after
Drugs
Praxis Precision Medicines says its experimental epilepsy drug relutrigine produced positive results in a key study, prompting an independent committee to recommend stopping the trial early for efficacy. The data come from the registrational cohort of the EMBOLD study, which is evaluating relutrigine in patients with SCN2A and SCN8A developmental
Drugs
FDA investigators issued a 24-page Form FDA-483 citing numerous manufacturing violations after a 12-day inspection in May of Apotex’s sterile drug facility in Richmond Hill, Ontario. The just-posted form identifies significant lapses in aseptic operations, cleaning, equipment maintenance, complaint investigations, and data integrity. The 483
Drugs
FDA told an 11/6 Industry Steering Committee meeting as part of the discussions toward the 2027 reauthorization of the Prescription Drug User Fee Act that it intends to present a proposal to charge different user fees depending on whether a company conducts Phase 1 trials in the U.S.
Drugs
The Sedgwick Recall Index for the third quarter of 2025 shows that while the total 97 drug recalls was the same as in the second quarter, the number of recalled units increased 137% to 44.56 million. The report says the average recall size more than doubled between the second
Drugs
CDER Office of Compliance director Jill Furman outlined a year of sweeping operational shifts, heightened enforcement, and emerging use of artificial intelligence during remarks at the Food and Drug Law Institute’s annual enforcement conference in Washington, DC, 12/4. She described the Center’s compliance program navigating two simultaneous
Drugs
The CDER Center for Clinical Trial Innovation says its Bayesian Statistical Analysis demonstration project provides a structured opportunity to use Bayesian approaches for the pre-specified primary analysis, supplementary analysis, or monitoring of simple trials, such as trials with a non-adaptive or straightforward sequential design. A Center online post
Drugs
FDA has lifted a partial clinical hold on one of Vanda Pharmaceuticals’ key tradipitant studies, clearing the company to expand dosing in an ongoing motion-sickness trial. The hold on protocol VP-VLY-686-3403 had previously capped dosing at 90 administrations of tradipitant. The FDA lifted that limitation after
FDA Policy/General
Writing in an online LinkedIn column, BoldApprovals managing director Steven Cohen makes the case that FDA advisory committees matter. “They make a real difference during the approval of a new treatment that has an impact at multiple levels, including public health, public policy, engagement, participation, trust, openness to the media,
Drugs
The CDER Office of Prescription Drug Promotion says a direct-to-consumer video for Daiichi Sankyo’s Turalio (pexidartinib) capsules, for oral use, is false or misleading and thus misbrands the drug. An 11/24 Notice of Violation describes the testimonial of a female patient with tenosynovial giant cell tumor
Drugs
A former senior FDA official says the abrupt resignation of newly appointed CDER director Richard Pazdur — one of the nation’s most influential cancer-drug regulators — signals deepening political interference inside the agency and could trigger further staff departures. In an interview with NewsNation, Peter Pitts, a former FDA associate
Drugs
The FDA Accelerating Rare Disease Cures (ARC) program Year-Three Annual Report says there were 26 rare disease new drug approvals in calendar year 2024. That’s three fewer approvals than ARC reported in its Year-Two report for 2023. Other categories shown on the report’s Year-At-A-
Drugs
In the wake of CDER director Richard Pazdur’s abrupt resignation, reportedly over concerns of growing politicization at the agency, a new report now finds that two Republican lawmakers were barred from attending a 12/3 FDA meeting concerning the potential expanded approval of ImmunityBio’s Anktiva (nogapendekin alfa inbakicept-
Drugs
FDA has granted Eli Lilly traditional approval for Jaypirca (pirtobrutinib) and its use in adults with relapsed or refractory chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) who were previously treated with a covalent BTK inhibitor. The action is based on confirmatory trial data and converts a 2023 accelerated
Drugs
Veristat principal regulatory specialist John Kirk says sponsors looking to participate in the FDA Commissioner’s National Priority Voucher (CNPV) program will do best with the program’s compressed timelines (review in one to two months) if they integrate their biostatistics, regulatory, and medical writing teams from the outset, creating
Drugs
FDA has named physician–epidemiologist Tracy Beth Høeg as CDER acting director, replacing director Richard Pazdur, who submitted his retirement paperwork earlier this week. Høeg, who previously served as senior advisor for clinical sciences in both the Office of the Commissioner and CBER, was selected by FDA commissioner Marty Makary
Drugs
The House has approved the Mikaela Naylon Give Kids a Chance Act, which reauthorizes FDA’s Rare Pediatric Disease Priority Review Voucher program. House Energy and Commerce Committee chairman Brett Guthrie (R-KY) says in a committee statement that the bill “builds on current programs to accelerate research and drug