Drugs
Vanda Pharmaceuticals has filed a lawsuit against FDA seeking an advisory committee meeting to assess the company’s pending NDA for tradipitant and its use in treating symptoms of gastroparesis in adults. Gastroparesis is a rare condition characterized by delayed gastric emptying that results in nausea, vomiting, bloating, fullness after
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A study conducted by CDER and outside researchers of healthcare provider (HCP) considerations of Boxed Warnings when making patient treatment decisions showed that they are only one of several factors that can influence providers’ treatment decisions. A study description says researchers held lengthy interviews with primary care physicians and specialists
Drugs
Denali Therapeutics says it is on track to submit a BLA early next year that will seek accelerated approval for DNL310 (tividenofusp alfa) and its use in treating Mucopolysaccharidosis Type II (MPS II, aka Hunter syndrome). The company says its projection is based on feedback from the agency during a
Drugs
FDA has posted a guidance entitled “Bioresearch Monitoring Technical Conformance Guide — Technical Specifications Document” that provides current agency specifications, recommendations, and general considerations for preparing and submitting clinical study-level information, subject-level data line listings by clinical site, and a summary-level clinical site dataset. The submitted information is
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A 2/28-3/11 FDA Bioresearch Monitoring Program inspection at the Clinical Integrative Research Center of Atlanta operated by Dr. Julio Flamini in Atlanta, GA, found objectionable conditions related to two clinical investigations. An 8/20 Warning Letter says Flamini failed to ensure that the investigations were conducted according
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Pharmaceutical Research and Manufacturers of America (PhRMA) says it is concerned that several of the statements and proposals in an FDA draft guidance on the Platform Technology Designation Program lack clarity or are inconsistent with the law. The PhRMA comment letter recommends that FDA: * not impose extra-statutory limits on
Drugs
Ali Pharmaceutical Manufacturing, a subsidiary of American Laboratories, is asking FDA to require AbbVie to correct what Ali says is the misbranding of an investigational product in a thyroid study. The Ali petition says AbbVie is not using Ali’s Armour Thyroid in a study “to assess the safety and
Drugs
FDA has released the form FDA-483 issued following a 3/13-3/22 inspection at the Otsuka Pharmaceutical Company’s Second Tokushima Factory, a drug product and sterile/non-sterile drug substance manufacturing facility in Tokushima, Japan. The form contains these five inspection observations: * failure to clean, maintain, and
Drugs
CDER and CBER are seeking input to help advance the agency’s model-informed drug development (MIDD) program. FDA has previously described MIDD as an umbrella term covering the “integration and application of statistical, biological, and pharmacological data in support of clinical trial design and quantitative decision making, and includes,
Drugs
CellProthera says it has reached “alignment” with FDA on a planned pivotal Phase 3 trial design for its ProtheraCytes cell therapy after completing a recent IND meeting. “Building on a successful Phase 2 trial in the UK and France, CellProthera is moving into the final phase of clinical development for
Drugs
FDA has cleared a Think Surgical 510(k) for using its TMini Miniature Robotic System with the GMK Sphere and SpheriKA Knee Systems from Medacta International under a collaboration agreement between the two companies. The two knee systems have been added to Think Surgical’s ID-HUB, what it calls
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Merck says it is discontinuing two Keytruda (pembrolizumab) trials — KEYNOTE 867 and KEYNOTE 630. A company statement says the decision to discontinue the trials was based on recommendations from independent data monitoring committees. The 867 trial was evaluating Keytruda in combination with stereotactic body radiotherapy to treat patients with stage
Drugs
CDER director Patrizia Cavazzoni has announced the creation of a CDER Artificial Intelligence (AI) Council to “provide oversight, coordination, and consolidation of AI activities.” In an announcement to Center staff, Cavazzoni says the new council will consolidate and continue the work started by the CDER AI Steering Committee, AI Policy
Drugs
FDA has granted Nuvectis Pharma an orphan drug designation for NXP800 and its use in treating AT-rich interactive domain-containing protein 1a-deficient ovarian, fallopian tube, and primary peritoneal cancers. NXP800 is described as an oral small molecule GCN2 activator currently in a Phase 1b clinical trial for treating
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Topline results from the Phase 3 V-MONO study found that Novartis’ Leqvio (inclisiran) demonstrated clinically meaningful, statistically significant low-density lipoprotein cholesterol (LDL-C) lowering as a monotherapy versus both placebo and Merck’s Zetia (ezetimibe) in patients who were at low or moderate risk of developing atherosclerotic cardiovascular
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FDA has accepted for priority review a Soleno Therapeutics NDA for DCCR (diazoxide choline) for treating Prader-Willi syndrome (PWS) in individuals aged four years and older who have hyperphagia. The agency has set a 12/27 user fee review action target date. The company says the hallmark symptom of
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FDA has accepted for priority review a SpringWorks Therapeutics NDA for mirdametinib, an investigational MEK inhibitor for treating pediatric and adult patients with neurofibromatosis Type 1 (NF1)-associated plexiform neurofibromas. The rare genetic disorder is caused by mutations in the NF1 gene. The agency has set a 2/28/2025
Drugs
Researchers at Yale and Emory Universities say FDA should require postmarket studies for breakthrough therapy-designated indications approved based on surrogate markers, regardless of approval pathway. Writing at JAMA Network Open, the researchers say such postmarket tests would ensure that the drugs’ clinical benefits are confirmed. The study looked at
Drugs
An FDA inspection earlier this month of a Global Calcium manufacturing facility in Hosur, India has resulted in a six-observation Form FDA-483 that cites significant GMP violations. For example, the agency investigators said the firm’s production records do not contain complete information relating to the production and
Drugs
Three stakeholders are recommending changes to an FDA draft guidance on the agency’s platform technology designation program. The National Organization for Rare Disorders (NORD) says the platform technology designation “is a promising regulatory tool of significant potential value to the rare disease community.” It suggests ways to improve the
Drugs
An 11/28-12/6/2023 FDA inspection at the CARsgen Therapeutics Corp. facility in Durham, NC, found significant violations of current good manufacturing practice (CGMP) regulations in the manufacturing of investigational new drugs and biological products that were made available for use in a Phase 2 trial. A just-
Drugs
A 2/6-2/15 FDA inspection at the EyePoint Pharmaceuticals drug manufacturing facility in Watertown, MA, found significant violations of current good manufacturing practice (CGMP) regulations for finished drugs. A just-released 7/12 Warning Letter says specific violations were: * failure to thoroughly investigate any unexplained discrepancy or failure