Drugs
Beam Therapeutics says it will submit a complete response in the fourth quarter of this year to the FDA 7/29 clinical hold on the company’s BEAM-201 IND application for the treatment of relapsed/refractory T-cell acute lymphoblastic leukemia/T-cell lymphoblastic lymphoma. The company says it
Drugs
A 2/15-3/7 FDA inspection at the Green Wave Analytical drug contract testing laboratory in San Diego, CA, identified significant violations of current good manufacturing practice (CGMP) regulations for finished drugs. An 8/19 Warning Letter says specific violations were: * failing to establish and document the accuracy, sensitivity,
Drugs
FDA has accepted for priority review a Sanofi BLA for efanesoctocog alfa to treat hemophilia A. The agency set a PDUFA action date of 2/28/23 for its decision on the BLA, the company says. Sanofi says the BLA was supported by data from the pivotal XTEND-1 Phase
Drugs
The state of Florida has filed a lawsuit against FDA for failing to timely respond to a state-sought Freedom of Information Act (FOIA) request seeking information about why its and other states’ requests to begin importing prescription drugs from Canada have stalled at the agency. A rule advanced by
Drugs
A 9/14-28 FDA inspection at Nutritional Laboratories International, doing business as Elite One Source Nutritional Services, Missoula, MT, identified serious violations of the Federal Food, Drug, and Cosmetic Act and applicable regulations. An 8/5 Warning Letter says the inspection found serious violations of the current good manufacturing
Drugs
FDA commissioner Robert Califf says the agency has launched the FDA Overdose Prevention Framework, its vision for undertaking “impactful, creative actions to prevent drug overdoses and reduce deaths.” In an FDA Voices post, Califf says agency activities within four overarching priorities are dynamic to allow it to recalibrate its approach
Drugs
FDA has lifted a partial clinical hold on Curis’ TakeAim Leukemia study that is intended to evaluate emavusertib in a monotherapy phase of the trial. The partial hold remains in place for the combination therapy phase (Phase 1b) and the expansion phase (Phase 2a) of the study until Phase 1a
Drugs
Outlook Therapeutics says it has resubmitted to FDA a BLA for its ONS-5010 (Lytenava) (bevacizumab-vikg) to treat wet age-related macular degeneration. Company officials say they have worked for the past three months to provide the additional information FDA required that was not in the 3/2022 BLA
Drugs
AstraZeneca says new Phase 3 data on Farxiga (dapagliflozin) demonstrated a mortality benefit compared to placebo in patients with heart failure (HF). These results were presented at the European Society of Cardiology Congress 2022 in Barcelona, Spain and simultaneously published in Nature Medicine. “The reduction in risk of cardiovascular (CV)
Drugs
Bristol Myers Squibb and Janssen Pharmaceuticals announced 8/28 that they are planning to begin a Phase 3 trial of their investigational oral factor XIa (FXIa) inhibitor, milvexian, after reporting results from the Phase 2 AXIOMATIC-SSP dose-ranging study that showed a 30% relative risk reduction in recurrent symptomatic
Drugs
FDA has made available two International Council for Harmonization draft guidances on drug analytical procedures. A draft entitled Validation of Analytical Procedures Q2(R2) discusses the elements for consideration during the validation of analytical procedures included as part of regulatory submissions. It provides recommendations on how to derive and evaluate
Drugs
FDA has released a final guidance entitled “E14 and S7B Clinical and Nonclinical Evaluation of QT/QTc Interval Prolongation and Proarrhythmic Potential — Questions and Answers.” The International Council for Harmonization (ICH) document is intended to clarify key issues related to implementing earlier guidances on clinical evaluations of QT/QTc interval
Drugs
FDA has approved an Incyte supplemental NDA for Pemazyre (pemigatinib), a selective fibroblast growth factor receptor (FGFR) inhibitor for treating adults with relapsed or refractory myeloid/lymphoid neoplasms (MLNs) with FGFR1 rearrangement. The rare blood cancers affect about 1 in 100,000 people in the U.S. annually, according to
Drugs
The CDER Office of Pharmaceutical Quality (OPQ) FY 2021 annual report says the data and analyses in the report “provide a picture of a high state of pharmaceutical quality for U.S. consumers and patients with the expectation for continuing improvement.” The report contains key data used to characterize drug
Drugs
Novartis says it is temporarily suspending the dosing of branapalm in the Phase 2b VIBRANT-HD study of the drug in adults with Huntington’s Disease. The decision, which was made by an independent data monitoring committee and supported by the VIBRANT-HD steering committee, was made because early signs
Drugs
A 9/20-9/24/21 FDA inspection at Oregon’s Wild Harvest, Redmond, OR, found serious violations of FDA regulations for current good manufacturing practice (CGMP) in manufacturing, packaging, labeling, or holding operations for dietary supplements. An 8/8 Warning Letter says specific violations were: * failing to establish product
Drugs
A joint reflection paper released by the International Coalition of Medicines Regulatory Authorities, of which FDA is a member, with the International Council for Harmonization, International Pharmaceutical Regulators Program, and Pharmaceutical Inspection Cooperation Scheme, outlines the groups’ multi-stakeholder approach to the harmonization work required to support the development of
Drugs
Last month, the U.S. Court of Appeals for the Second Circuit affirmed a lower court’s decision that a Pfizer-planned copay subsidy program for Tafamidis, indicated for treating a rare heart condition, violated the anti-kickback statute. The program would have offered financial assistance to qualifying patients (including
Drugs
Pharmaceutical Research and Manufacturers of America (PhRMA) is calling on FDA to identify the legal authority for rescinding breakthrough therapy designations (BTDs) as described in a draft guidance. Its comment letter also says the agency should (1) explicitly incorporate procedural details from relevant SOPPs (standard operating policies and procedures) and
Drugs
FDA has approved a Pharmacyclics supplemental NDA for Imbruvica (ibrutinib) for pediatric patients over one year of age with chronic graft versus host disease (cGVHD) after failure of one or more lines of systemic therapy. Approval was based on data from the 47-patient iMAGINE trial that measured overall response
Drugs
Attorneys Joanna Hawana and Benjamin Zegarelli (Mintz) say a recent FDA Warning Letter to Amazon.com “signals that the agency may be adding new tools to its arsenal in the perpetual fight to protect U.S. consumers from adulterated, misbranded, and potentially unsafe products. It also highlights the fact that
Drugs
FDA has denied a citizen petition seeking a “freeze” on all regulatory requests from pharmaceutical and medical device companies headquartered in Russia or with majority Russian ownership due to the country’s invasion of, and war against, Ukraine. The February petition was filed by former FDA Office of Orphan Product