Drugs
FDA approved 8/23 changes, including a limitation on authorized use, to the terms of the emergency use authorization for Invivyd’s Pemgarda (pemivibart) injection indicated for pre-exposure prophylaxis of Covid-19. An agency memo lists these changes and the rationale for them: * include Pemgarda pseudotyped virus-like particle
Drugs
FDA has several ongoing initiatives related to real-world data and real-world evidence in what has become a dynamic space. That’s the word from FDA chief medical officer Hilary Marston, speaking on a recent “Q&A with FDA” podcast. “We’ve expanded our use of real-world
Drugs
FDA has granted Ractigen Therapeutics an orphan drug designation for RAG-18, a saRNA product candidate for treating Duchenne Muscular Dystrophy (DMD). The therapy is designed to specifically target and activate UTRN gene expression in muscle cells via the RNAa mechanism, according to the company. “The utrophin protein encoded by
Drugs
FDA has approved Amgen’s Blincyto (blinatumomab) for treating adult and pediatric patients one month or older with CD19-positive Philadelphia chromosome-negative B-cell precursor acute lymphoblastic leukemia (B-ALL) in the consolidation phase, regardless of measurable residual disease (MRD) status. Approval is based on data from the Phase
Drugs
FDA has accepted for priority review a Rhythm Pharmaceuticals supplemental NDA for Imcivree (setmelanotide), a melanocortin-4 receptor agonist for treating obesity due to Bardet-Biedl syndrome or pro-opiomelanocortin (POMC), including proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency in children as young as two
Drugs
FDA has granted BeiGene a fast track designation for BGB-16673 and its use in adult patients with relapsed or refractory (R/R) chronic lymphocytic leukemia or small lymphocytic lymphoma (CLL/SLL) who have been previously treated with at least two prior lines of therapy, including a Bruton’s tyrosine
Drugs
FDA has cleared an Insulet 510(k) for the Omnipod 5 Automated Insulin Delivery System (Omnipod 5) for use by those with Type 2 diabetes (ages 18 years and older). Previously, the insulin pump was FDA-cleared for Type 1 diabetes management. The clearance is based on recent data showing
Drugs
CDER director Patrizia Cavazzoni says agency inspectors and reviewers are seeing deteriorating manufacturing quality in the facilities where biologics and biosimilars are made, and industry needs to address this. Speaking at a Duke Margolis Institute for Health Policy program in Washington, DC 8/20, Cavazzoni noted that BLA and biosimilar
Drugs
FDA has cleared a SurGenTec 510(k) for its proprietary B-MAN Bone Marrow Aspirate Kit. The device is intended to preserve native cell structure and facilitates the extraction of healthier cells by reducing the manipulation that takes place, according to the company. It uses a centrifuge-free method for
Drugs
FDA principal deputy commissioner Namandjé Bumpus has issued an order refusing to approve an NDA submitted by Intarcia Therapeutics for ITCA 650 (exenatide in DUROS device). The order says FDA determined that the approval criteria have not been met because Intarcia failed to demonstrate that ITCA 650 is safe for
Drugs
FDA has granted Wex Pharmaceuticals a fast-track designation for Halneuron (tetrodotoxin for injection), an alternative to narcotics and opioids for treating chemotherapy-induced neuropathic pain (CINP). The drug is currently being evaluated in a Phase 2B clinical trial for CINP, according to the company. Wex says the small molecule
Drugs
FDA has published a new batch of product-specific guidances (PSGs) with recommendations for developing generic drugs and generating evidence to support ANDA approvals. The list of 14 new and 54 revised PSGs contains: * 41 PSGs for products with no approved ANDAs, including 26 complex products; * 33 PSGs (8 new
Drugs
Liquidia Corp. has filed a lawsuit against FDA over the agency’s recent decision to grant United Therapeutics Corp. (UTC) an additional three-year exclusivity period for Tyvaso DPI (treprostinil), which blocks Liquidia’s Yutrepia (treprostinil) from entering the market before 5/23/2025. Treprostinil is indicated for treating pulmonary
Drugs
Pharmaceutical Research and Manufacturers of America (PhRMA) says an FDA draft guidance on considerations in demonstrating interchangeability with a reference product is “a helpful step to provide further transparency into FDA’s evolving approach regarding the need for a switching study or studies to support an interchangeability determination for a
Drugs
FDA has issued Regeneron Pharmaceuticals a complete response letter on its BLA for linvoseltamab in relapsed/refractory (R/R) multiple myeloma (MM) that has progressed after at least three prior therapies. “The sole approvability issue identified is related to findings from a pre-approval inspection at a third-party fill/
Drugs
FDA has published an International Council for Harmonization guidance, E11A Pediatric Extrapolation, to provide recommendations for and promote international harmonization of the use of pediatric extrapolation to support the development and authorization of pediatric medicines. The document says harmonization of the approaches to pediatric extrapolation should reduce the likelihood of
FDA Policy/General
The FDA Bioresearch Monitoring (BIMO) FY 2023 metrics report shows there were 1,073 BIMO inspections across the five FDA Centers. CDER had by far the largest number of inspections at 779, with 141 through CDRH, 126 through CBER, and 26 through CVM. The report also gives common inspection observations
Drugs
The United States Court of Appeals for the DC Circuit has granted a Novartis motion to stay FDA’s approval of MSN Laboratories’ generic version of Novartis’s Entresto (sacubitril/valsartan) until it rules on the matter, which is related to a labeling carve-out dispute. In July, Novartis (see
Drugs
The Federal Trade Commission (FTC) has submitted a comment to FDA expressing its support for a draft guidance removing a prior recommendation that a biosimilar drug applicant had to submit clinical switching studies to demonstrate the biosimilar’s interchangeability with its reference drug. Under the FDA draft, applicants can submit
Drugs
A bipartisan group of four House members is asking FDA commissioner Robert Califf to tell what steps the agency is taking to counter potential issues associated with drug companies conducting trials with medical centers and hospitals associated with China’s People’s Liberation Army (PLA). The letter says such trials
Drugs
A 1/22-2/2 FDA inspection at the Eugia Pharma Specialties Limited drug manufacturing facility in Hyderabad, Telangana, India, identified significant violations of current good manufacturing practice (CGMP) regulations for finished drugs. An 8/15 Warning Letter says specific violations were: * failure to ensure that laboratory records included complete
Drugs
Eli Lilly is reporting “positive topline results” from its SURMOUNT-1 three-year study that assessed tirzepatide (weight-loss drug Zepbound and Type 2 diabetes drug Mounjaro) for long-term weight management and delay in progression to diabetes in adults with pre-diabetes and obesity. “Weekly tirzepatide injections (5 mg,