Drugs
FDA has granted X4 Pharmaceuticals a rare pediatric disease designation for mavorixafor for treating WHIM (Warts, Hypogammaglobulinemia, Infections, and Myelokathexis) syndrome, a rare, inherited, primary immunodeficiency disease caused by genetic mutations in the CXCR4 receptor gene. The company says the drug is currently being investigated in a global pivotal Phase
Drugs
FDA has accepted for filing an Atox Bio NDA for reltecimod to treat suspected organ dysfunction or failure in patients age 12 and older with necrotizing soft tissue infections, in conjunction with surgical debridement, antibiotic therapy, and supportive care. The agency assigned a PDUFA action date of 9/30/21,
Drugs
The American College of Obstetricians and Gynecologists (ACOG) says it strongly supports the recommendation stated in an FDA draft guidance to consider including premenopausal women in breast cancer drug development programs. However, the ACOG comment letter says, it recommends expanding the guidance scope to include survivorship issues, such as fertility
Drugs
Public Citizen is calling on the HHS Inspector General to “investigate the unprecedented and inappropriate close collaboration between FDA and Biogen regarding the company’s application for approval of the Alzheimer’s disease drug aducanumab.” In a 12/9 letter, the advocacy group says that after two identical Phase 3
Drugs
Pfizer says it is pleased with revisions FDA made to a 2010 guidance to eliminate unnecessary testing as shown in a draft guidance on pharmacokinetics in patients with impaired renal function for study design, data analysis, and impact on dosing. A Novartis comment letter says the scope of the draft
Drugs
FDA has awarded a three-year $44.4 million contract to Palantir Technologies to build on the existing partnership with the company to provide an enterprise data management and analytics solution for CDER and the Oncology Center of Excellence. The company says its software will enable information technology leadership, analysts,
Drugs
FDA says its review of the Web site maintained by Natures Boost, Kirkland, WA, determined that the company is marketing two unapproved new drugs in violation of the Federal Food, Drug, and Cosmetic Act. An 11/20 Warning Letter gives some of the promotional statements from the Web site that
Drugs
FDA says its review of the Web site maintained by Valentus, Inc., Sioux Falls, SD, determined that the company takes orders for several products that the Federal Food, Drug, and Cosmetic Act considers to be unapproved new drugs because they are intended for use in the cure, mitigation, treatment, or
Drugs
FDA has published the FDA-483 with six inspection observations issued following a 10/7-10/16 agency inspection at Juno Therapeutics, Bothell, WA. The firm is a CAR-T cell therapy manufacturer. The specific inspection observations were: * failing to thoroughly review any unexplained discrepancy, failure of a batch or
Drugs
FDA has issued two guidances on developing proprietary names for human drug products. One guidance covers prescription drug products and the other covers non-prescription products. The documents say they describe best practices to help minimize proprietary name-related medication errors and otherwise avoid the adoption of proprietary names that
Drugs
The American Civil Liberties Union (ACLU) says the Trump administration has told a federal court that Covid-19 is no longer a serious risk and thus FDA is justified in requiring patients seeking mifepristone to go in-person to a clinical setting solely to sign a form and pick up
Drugs
From 6/2019 to 6/2020, the first full year of the FDA Oncology Center of Excellence’s Project Orbis, the effort received 60 oncology marketing applications for either new molecular entities or supplements adding new indications. Of the 60, 38 were approved. Project Orbis is intended to facilitate oncology
Drugs
FDA has granted Turning Point Therapeutics a breakthrough therapy designation for repotrectinib and its use in treating patients with gene ROS1-positive metastatic non-small cell lung cancer (NSCLC) who have not been treated with a ROS1 tyrosine kinase inhibitor (TKI). The designation was based on positive data from the
Drugs
FDA says it is testing its capability to receive annotated electrocardiogram (aECG) waveform data in electronic format under the Health Level Seven (HL7) Annotated ECG Waveform Data Standard. Those who are interested in voluntarily testing this capability can submit the aECG files directly through the FDA electronic submissions gateway (ESG)
Drugs
FDA is being criticized for its slow response to the need to find alternatives to physical inspections during the worsening Covid-19 pandemic. The issue is even more critical now as a number of drug submission final review actions have been put on the back burner due to the agency’
Drugs
The Justice Department has filed a civil complaint to stop AMARC Enterprises from distributing unapproved and misbranded drugs and adulterated animal drugs. The company sold and distributed “Poly-MVA” and “Poly-MVA for Pets,” which they claim can cure, mitigate, treat, or prevent disease, including cancer. The complaint also alleges
Drugs
FDA is detailing the launch of a new pilot program to provide a pathway for novel drug development tools (DDTs) to be used in regulatory applications for new medical products. The program, Innovative Science and Technology Approaches for New Drugs (ISTAND) Pilot Program, is intended to streamline and potentially speed
Drugs
Expanding on previous direct-to-consumer (DTC) advertising research involving celebrities and other endorsers used in advertisements, CDER’s Office of Prescription Drug Promotion (OPDP) is proposing to examine four types of endorsers (celebrity, physician, patient, non-celebrity influencer) in two separate studies. The studies plan to examine whether the
Drugs
FDA has added content to a question-and-answer appendix in its guidance titled Conduct of Clinical Trials of Medical Products During Covid-19 Public Health Emergency. The updated document’s added question pertains to considerations for disposing unused investigational drug products when a study participant cannot return it to
Drugs
FDA has approved labeling changes stating that Merck’s Januvia (sitagliptin), Janumet (sitagliptin and metformin hydrochloride), and Janumet XR (sitagliptin and metformin hydrochloride extended-release) are not proven to improve glycemic (blood sugar) control in pediatric patients age 10 to 17 with Type 2 diabetes . The drugs are approved to
Drugs
Responding to an FDA draft guidance on developing drugs and biologics for adjuvant treatment of bladder cancer, Novartis says it applauds the agency’s efforts to seek input from stakeholders on actionable policy suggestions that could be implemented in the near-term by CDER Office of New Drugs staff to
Drugs
In a suit filed in a Maryland federal court, Genus Lifesciences says that Lannett Company made untrue statements to FDA in an NDA for its Numbrino, a cocaine-based anesthetic, and the agency has not performed its legal duty to withdraw approval of the NDA. The suit acknowledges that Genus