Drugs
FDA has granted Tyra Biosciences a rare pediatric disease designation for TYRA-300, an oral FGFR3 selective inhibitor for treating achondroplasia, the most common form of dwarfism. “A specific DNA mutation in FGFR3 causes an estimated 99% of achondroplasia,” the company says. Tyra is planning to submit an IND in
Drugs
A new commentary in the 2/2 online Springer Link by CDER Office of Medical Policy officials Ryan Robinson and Leonard Sacks outlines the advantages and challenges of conducting decentralized clinical trials (DCTs). FDA defines DCTs as “trials where some or all of the trial-related activities occur at locations
Drugs
Seven former FDA commissioners (acting and confirmed) have filed an amicus brief with the Supreme Court urging that a lower court ruling restricting access to the abortion drug mifepristone be reversed. The brief in the closely watched case argues that the Fifth Circuit Court of Appeals should not have substituted
Drugs
FDA has released the forms FDA-483 issued following inspections at four drug manufacturers in three countries. The specific inspection observations for each manufacturer were: * Alvotech Hf, Reykjavik, Iceland — * inadequate environmental/personnel monitoring for a redacted building filling line; * AGC Biologics, Bothell, WA — * written records of investigations into unexplained discrepancies,
Drugs
FDA has cleared a NeuroBo Pharmaceuticals IND for a Phase 1 trial of DA-1726, a novel, dual oxyntomodulin analog agonist that functions as a glucagon-like peptide 1 receptor and glucagon receptor as a treatment for obesity. The company says it plans to start the trial in the first
Drugs
Former FDA deputy commissioner Deborah M. Autor has joined consulting group Healthcare Innovation Catalysts (HIC) as chief executive officer. HIC provides advice to clients on regulatory affairs, clinical issues, compliance, federal partnership, and other strategic services. Autor left FDA in 2013 after serving 11 years to join Mylan as senior
Drugs
As part of its review of an Abeona Therapeutics BLA for pz-cel (prademagene zamikeracel) for recessive dystrophic epidermolysis bullosa, FDA has completed a bioresearch monitoring inspection of Abeona and the BLA mid-cycle review meeting. A company statement says the 1/22-1/24 inspection did not result in
Drugs
FDA has released a Revised Biosimilar User Fee Amendment (BsUFA) Research Roadmap that provides updated information about the biosimilar regulatory science research pilot program’s research priorities. The revised roadmap says the draft that was issued in 1/2023 was updated from public docket comments and a 10/2023 public
Drugs
The House Energy and Commerce Committee’s Subcommittee on Oversight and Investigations will hold a 2/6 public hearing on “Protecting American Health Security: Oversight of Shortcomings in FDA’s Foreign Inspection Program.” In a statement, committee chair Rep. Cathy Morris Rodgers (R-WA) and subcommittee chair Morgan Griffith (R-
Drugs
FDA has issued a warning on South Moon and Rebright or FivFivGo eye drops because of potential risks for eye infections. “These are copycat eye drop products that consumers can easily mistake for Bausch + Lomb’s Lumify brand eye drops, an over-the-counter product approved for redness relief,” the
Drugs
The Biotechnology Innovation Organization (BIO) says it is committed to enhancing clinical trial diversity as part of its BIOEquality Agenda. In a response to an FDA 11/29-11/30/2023workshop on clinical trial diversity, BIO notes that under the FDA Omnibus Reform Act of 2022, the agency was required
Drugs
Some 263 Democrat members of the House and Senate filed an amicus brief with the Supreme Court asking that lower court rulings restricting access to the abortion drug mifepristone be reversed. In a statement from the Senate Judiciary Committee, the Democrats say that at issue in the case are evidence-
Drugs
A 7/17-7/23/2023 FDA inspection at the S&J International Enterprises Public Company Limited drug manufacturing facility in Si Rachi, Thailand, found significant violations of current good manufacturing practice (CGMP) regulations for finished drugs. A 1/26 Warning Letter says specific violations were: * failure of the
Drugs
The DC federal court has ordered FDA to either resolve a Vanda Pharmaceuticals sNDA for its Hetlioz (tasimelteon) to treat jet lag disorder or start a hearing on the sNDA on or before 3/5. The court’s ruling came as it granted Vanda summary judgment in its 9/2022
Drugs
FDA and the European Medicines Agency (EMA) will mark World Cancer Day with a 2/1 virtual public discussion spotlighting innovative cancer treatment advances for patients with chronic myeloid leukemia, multiple myeloma, and melanoma. An agency post says the program will run from 9:30 a.m. to 11 a.
Drugs
FDA has issued Defender Pharmaceuticals a complete response letter (CRL) on its NDA for intranasal scopolamine (DPI-386) for preventing nausea and vomiting induced by motion in adults. The company says it will request a formal meeting with FDA to “fully understand the issues raised in the CRL so we
Drugs
FDA has lifted a 2022-imposed clinical hold against INmune Bio’s XPro (XPro1595; pegipanermin) and its Alzheimer’s disease clinical trial. “The Phase 2 trial is on track to enroll the last patient mid-2024,” the company says, adding that top line data is expected about six months after
Drugs
The Department of Justice (DoJ) is looking at the use of artificial intelligence (AI) embedded in patient records that prompts doctors to recommend specific treatments. Bloomberg Law reports that department investigators want to learn more about generative technology’s role in facilitating anti-kickback and false claims violations. The newsletter
Drugs
FDA has posted a draft guidance entitled “Collection of Race and Ethnicity Data in Clinical Trials and Clinical Studies for FDA-Regulated Medical Products.” The document outlines FDA’s recommendations on using a standardized approach for collecting and reporting race and ethnicity data in submissions, including information collected and reported
Drugs
FDA says that beginning 3/8, Pfizer’s Paxlovid that is labeled under a 12/2021 emergency use authorization will no longer be authorized for adults, and instead patients can be prescribed Paxlovid under a 5/2023 NDA approval for treating mild-to-moderate Covid-19 in adults who are
Drugs
U.S. Senator Tammy Baldwin (D-WI) has written the four biggest drug companies marketing asthma inhalers and urged them to stop unfairly blocking generics from the market. In 1/29 letters (see below) to AstraZeneca, Boehringer Ingelheim, GlaxoSmithKline, and Teva, Baldwin called on the companies to stop improperly listing
Drugs
Merck says data from its Phase 3 KEYNOTE-564 trial evaluating Keytruda (pembrolizumab) as an adjuvant treatment in patients with renal cell carcinoma (RCC) at intermediate-high or high risk of recurrence following nephrectomy, or following nephrectomy and resection of metastatic lesions showed a promising overall survival (OS) benefit. The