Drugs
FDA has cleared a Biomea Fusion IND to begin a Phase 1 trial of BMF-219, a selective irreversible menin inhibitor, in adults with relapsed or refractory acute leukemia, including those with an MLL/KM2TA gene rearrangement or an NPM1 mutation. The company says BMF-219 is a synthetic compound
Drugs
FDA has expanded an Eli Lilly emergency use authorization (EUA) for bamlanivimab 700 mg and etesevimab 1,400 mg administered together for post-exposure prophylaxis in certain individuals for preventing Covid-19 infection. Lilly says the combination’s “neutralizing antibodies can now be used together to treat high-risk individuals
Drugs
Researchers in the UK and Italy say there should be more selective use of surrogate endpoints when evaluating new drugs so that they are restricted to use in chronic diseases, especially when collecting data on patient-relevant clinical outcomes requires trials with unattainably long follow-up. Writing in thebmj, the
Drugs
FDA has extended by three months the review of a Calliditas Therapeutics NDA seeking accelerated approval for Nefecon (budesonide) for treating primary IgA nephropathy (IgAN). The major amendment extension was ordered to provide the agency more time to review additional analyses of the company’s NeflgArd trial data which was
Drugs
FDA and the European Medicines Agency (EMA) are starting a pilot program to provide parallel scientific advice to applicants of ANDAs for FDA’s complex generic drug products and marketing authorization applications (MAAs) for EMA’s hybrid products. An FDA statement says the pilot expands the two agencies’ existing parallel
Drugs
Amylyx Pharmaceuticals says it is planning to submit an NDA in the coming months for AMX0035 (sodium phenylbutyrate and taurursodiol) for treating amyotrophic lateral sclerosis (also known as Lou Gehrig’s disease). The decision to file the NDA comes after the company has held recent discussion with FDA reviewers, including
Drugs
FDA has granted accelerated approval for BeiGene’s Brukinsa (zanubrutinib) for treating adult patients with relapsed or refractory marginal zone lymphoma who have received at least one anti-CD20-based regimen. Approval is based on efficacy data from two single-arm clinical trials, with overall response rate (ORR) as the
Drugs
A 9/1 decision by the U.S. Court of Appeals for the Federal Circuit highlights the potential impact of contradictory positions taken before FDA and the U.S. Patent and Trademark Office (USPTO). That’s the view of three Cooley attorneys in an online analysis of Belcher Pharmaceuticals v.
Drugs
Researchers say issues involving innovator and generic drug companies strategically “parking” 180-day generic drug exclusivity to delay generic entry and competitive drug markets now occur infrequently. When parking does occur, the authors write in the Yale Journal of Health Policy, Law, and Ethics, it tends to relate to changes
Drugs
Astellas says the patient in its ASPIROS trial evaluating an investigational drug in patients with X-linked myotubular myopathy who suffered a serious adverse event leading to a pause in the trial has died. A company statement says the cause of death is still pending and the company is gathering
Drugs
FDA says there are things the U.S. Patent and Trademark Office (USPTO) could do to increase drug competition and help address the high cost of medicines. In a 9/10 letter, FDA acting commissioner Janet Woodcock says that while FDA recognizes that patents are critical to fostering innovation, the
Drugs
Jacobus Pharmaceutical Co. has recalled three lots of Ruzurgi (amifampridine) 10 mg tablets due to potential contamination with yeast, mold, and aerobic bacteria. The product is indicated for treating Lambert Eaton Syndrome patients aged six to 17 years. According to a recall notice, oral products heavily contaminated with yeast, mold,
Drugs
FDA has accepted for review an Axsome Therapeutics NDA for AXS-07 (meloxicam-rizatriptan) for the acute treatment of migraine. The submission is supported by data from two Phase 3 randomized, double-blind, controlled trials, MOMENTUM and INTERCEPT, which the company says demonstrated statistically significant elimination of migraine pain compared
Drugs
FDA has lifted a clinical hold against KalVista Pharmaceuticals and its Phase 2 clinical trial of KVD824 for oral prophylactic treatment of hereditary angioedema (HAE). The company says the hold was removed after FDA reviewed its responses to an FDA request for further information and analysis related to certain preclinical
Drugs
FDA has posted a draft International Conference for Harmonization guideline entitled Nonclinical Biodistribution Considerations For Gene Therapy Products ICH S12. The document provides harmonized recommendations for conducting nonclinical biodistribution (BD) studies for developing gene therapy products. It provides recommendations for the overall design of nonclinical BD assessments, and considerations for
Drugs
FDA has accepted for review a BeiGene BLA for its anti-PD-1 antibody tislelizumab as a treatment for patients with unresectable recurrent locally advanced or metastatic esophageal squamous cell carcinoma (ESCC) after prior systemic therapy. The agency has set a user fee review action target date of 7/12/
Drugs
FDA has granted Vor Biopharma a fast track designation for VOR33, Vor’s engineered hematopoietic stem cell therapeutic candidate for treating acute myeloid leukemia (AML). The therapy comprises CRISPR genome-edited hematopoietic stem and progenitor cells that have been engineered to lack CD33, according to the company. “Once infused, VOR33
Drugs
CDER’s Office of Prescription Drug Promotion (OPDP) has proposed a new study to examine whether disclosures in direct-to-consumer (DTC) and healthcare provider-targeted advertising could support allowing clinical benefit information based on a secondary endpoint reported in a product’s approved labeling (secondary claim) to be included
Drugs
FDA has announced a 9/23 symposium entitled “FDA-PDS Symposium X – Data Sharing; The Future of Clinical Trial Data Sharing…The Art of the Possible.” The meeting is intended to provide a forum for open discussion between leaders in oncology treatment and research and development from academia, government, and
Drugs
FDA has granted breakthrough therapy designation to Boehringer Ingelheim and Lilly’s Jardiance (empagliflozin) for treating heart failure with preserved ejection fraction. An American Journal of Managed Care online post says Jardiance is the first drug in its class to gain the designation. The post says results from the EMPEROR
Drugs
FDA has rejected a Humanigen emergency use authorization (EUA) request for the use of its lenzilumab in newly-hospitalized Covid-19 patients. FDA reportedly told the company it was unable to conclude that the known and potential benefits of lenzilumab outweigh the known and potential risks of its use as
Drugs
FDA and the Federal Trade Commission have issued Warning Letters to 10 companies for illegally selling dietary supplements that claim to cure, treat, mitigate, or prevent diabetes. An FDA statement (contains links to the letters) says the letters were issued to: Live Good Inc.; Pharmagenics LLC; Lysulin Inc.: Nuturna International