Drugs
BioMarin Pharmaceutical says it plans to resubmit a BLA for valoctocogene roxaparvovec, a gene therapy for severe hemophilia A, by the end of September that will include previously reported results from the two-year follow-up safety and efficacy data from the Phase 3 GENEr8-1 study. In 8/2020,
Drugs
FDA has extended by three months its review of a TG Therapeutics BLA for ublituximab, indicated for treating patients with relapsing forms of multiple sclerosis (RMS). The revised user fee review action target date is 12/28. The review extension is intended to give the agency additional time to review
Drugs
FDA has announced the authorization of an extension to the shelf-life from 12 months to 18 months for specific lots of the refrigerated Eli Lilly monoclonal antibody, bebtelovimab, which is currently authorized for emergency use for treating Covid-19. The extension applies to all unopened vials of bebtelovimab that
Drugs
An analysis funded by Friends of Cancer Research finds that the breakthrough therapy designation criteria “accurately identify drugs that improve long-term outcomes for patients with cancer and warrant coordinated efforts to ensure timely coverage decisions and access for patients.” The findings were published in Clinical Cancer Research. The researchers
Drugs
Attorney Nancy Taylor (Greenberg Traurig) submitted a 5/24 petition asking FDA to take action to ensure that over-the-counter (OTC) external analgesic drug products in patch, plaster, or poultice (PPP) dosage forms claims are limited only to those that are demonstrated and do not include severe or acute
Drugs
FDA has granted fast track designation for Pfizer’s investigational combination therapy of ervogastat and clesacostat to treat non-alcoholic steatohepatitis. A Pfizer announcement says FDA’s decision was informed by the results of the company’s nonclinical studies and a Phase 2a clinical study of ervogastat/clesacostat that showed
Drugs
FDA has granted Tvardi Therapeutics an orphan drug designation for TTI-101 for treating idiopathic pulmonary fibrosis (IPF). TTI-101 is described as an orally-delivered, small molecule, direct STAT3 inhibitor. “STAT3 has emerged as a central node of fibrosis and has been found to accumulate in the fibrotic lungs
Drugs
FDA has approved Servier Pharmaceuticals’ Tibsovo (ivosidenib) in combination with azacitidine (Bristol-Myers Squibb’s Onureg) for newly-diagnosed acute myeloid leukemia with a susceptible IDH1 mutation as detected by an FDA-approved test in adults age 75 or older who have comorbidities that preclude using intensive induction chemotherapy. An
Drugs
FDA has accepted for priority review a Reata Pharmaceuticals NDA for omaveloxolone for treating patients with Friedreich’s ataxia. The submission is based on data from the MOXIe Part 2 trial and additional supporting data from the MOXIe Part 1 and MOXIe Extension trials, according to the company. The agency
Drugs
U.S. Senators Joe Manchin (D-WV) and Mike Braun (R-IN) 5/26 introduced the Ensuring the FDA Fully Examines Clinical Trial Impact and Vitalness before Endorsement (EFFECTIVE) Act that would give FDA authority to deny a new drug application for an opioid analgesic drug if it isn’t
Drugs
FDA has issued a complete response letter to Verrica Pharmaceuticals for its NDA for VP-102 to treat molluscum contagiosum, described by the Centers for Disease Control as an infection caused by the molluscum contagiosum virus. The result of the infection is usually a benign, mild skin disease characterized by
Drugs
A federal jury has returned guilty verdicts against former Florida pain doctor Steven Chun and former Insys sales rep Daniel Tondre in a healthcare fraud kickback case, according to a Department of Justice statement. Both men were found guilty of conspiring to pay and receive kickbacks and bribes, in the
Drugs
Staff from FDA and the European Medicines Agency say good clinical practice (GCP) inspection findings from 49 common clinical investigator and sponsor/contract research organization inspections were comparable, providing support for continued collaboration between the two regulatory agencies. The two organizations have collaborated in GCP inspections since 9/2009. They
Drugs
Former FDA attorneys William Schultz and Margaret Dotzel (both Zuckerman Spaeder) say the BLOCKING (Bringing Low-Cost Options and Competition While Keeping Incentives for New Generics) Act, if passed, “will not accelerate generic drug entry. Instead, it will upend the critical 180-day exclusivity incentive by making it far less
Drugs
Harvard Medical School researchers say proposed legislation to require dietary supplement manufacturers to provide FDA with some basic information should be revised significantly to make it a true reform and oversight bill. Writing in a New England Journal of Medicine opinion piece, the researchers say the Dietary Supplement Listing Act
Drugs
FDA has granted fast track designation to Elevation Oncology’s seribantumab for the tumor-agnostic treatment of advanced solid tumors that harbor NRG1 gene fusions. The company says it is evaluating seribantumab in the Phase 2 CRESTONE study.
Drugs
FDA has approved a Servier supplemental NDA for Tibsovo (ivosidenib tablets) in combination with azacitidine for treating newly diagnosed IDH1-mutated acute myeloid leukemia (AML) in adults 75 years or older, or those who have comorbidities that preclude use of intensive induction chemotherapy. Approval was based on data from the
Drugs
FDA has accepted for review a Phathom Pharmaceuticals NDA for vonoprazan as a treatment for adults in healing all grades of erosive esophagitis and relief of heartburn, as well as the maintenance of healing and relief of these conditions. Vonoprazan is described as a novel, orally administered investigational potassium-competitive
Drugs
FDA has published a guidance, Importation of Prescription Drugs Final Rule Questions and Answers (Small Entity Compliance Guide), to help small entities better understand the 10/1/20 final rule on the importation of prescription drugs. The agency says the Q&A document restates in plain language the legal
Drugs
FDA has issued Nymox Pharmaceutical a Refusal to File letter for its NDA for fexapotide triflutate for treating benign prostatic hyperplasia (BPH). The letter referenced a “new outstanding issue of longer-term safety data” and it indicated the submission needed to have longer-term safety data, Nymox says. “Longer-term
Drugs
FDA says its review of a Web site and social media pages maintained by Santhigram Kerala Ayurvedic of U.S., Inc., North Brunswick, NJ, found that it is selling products whose uses establish that they are unapproved new drugs. A 5/19 Notice of Violation also says the company describes
Drugs
More than 35 health experts and advocacy groups including U.S. PIRG (Public Interest Research Group) are calling on the Senate HELP Committee to include stronger enforcement of post-market clinical trials for accelerated approval drugs in its coming markup of draft FDA user fee legislation. U.S. PIRG says