Drugs
FDA has accepted for review a BioMarin sBLA seeking an expanded indication for its Voxzogo (vosoritide) for injection to treat children under age 5 with achondroplasia. The agency set a PDUFA target action date of 10/21. The company says the sBLA is supported by results from a Phase 2
Drugs
Covis Pharma says it remains willing to voluntarily withdraw its pre-term birth drug Makena (hydroxyprogesterone caproate injection) and to work with CDER on an orderly wind-down. In a 3/6 letter to FDA commissioner Robert Califf and chief scientist Namandjé Bumpus, responding to the 1/19 report of
Drugs
The FDA Oncology Center of Excellence (OCE) has published a patient-focused glossary of 37 terms to aid patients in understanding complex clinical trial terminology. Writing in The Oncologist, six OCE staff members describe the process OCE took to develop the glossary. “The oncology clinical trial terms and definitions used
Drugs
The United States Pharmacopeia (USP) has launched a Nitrosamines Analytical Hub, the latest tool in its suite of solutions to address the evolving nitrosamines crisis. USP describes the tool as a public, Web-based repository of downloadable analytical procedures to test for nitrosamine impurities and related substances in pharmaceuticals. The
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FDA has extended the PDUFA review date for BioMarin’s BLA for Roctavian (valoctocogene roxaparvovec) gene therapy for adults with severe hemophilia A to 6/30. The agency said it had determined that the company’s submission of the three-year data analysis from the ongoing Phase 3 GENEr8-1
Drugs
An FDA Clinical Pharmacology Corner online post highlights four recent articles demonstrating how the agency says it is using proactive and innovative regulatory research to tackle the nation’s opioid crisis. The post references two articles published in 12/2022 that discuss the agency’s collaborative and innovative research into
Drugs
FDA has announced a 3/30 workshop entitled “Immune Globulin (IG) Hypersensitivity Reactions: Root Causes and Mitigation” that is intended to address “excessive numbers” of hypersensitivity reactions for specific product lots. While hypersensitivity is listed as a possible reaction to IG products, increased reports have led to voluntary market withdrawals
Drugs
Researchers from Johns Hopkins University and elsewhere say drug manufacturers should update labels for drugs that received accelerated approval and don’t contain all the information required by FDA. In an article accepted for publication in Pharmacotherapy, the researchers report that their review of labels of all drugs with indications
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Aclaris Therapeutics says a Phase 2 clinical study investigating the efficacy and safety of zunsemetinib (ATI-450), an investigational oral MK2 inhibitor, and its use in patients with moderate to severe hidradenitis suppurativa (HS), did not meet its primary endpoint of change from baseline in inflammatory nodule/abscess count. The
Drugs
The Association for Accessible Medicines (AAM) says that while some FDA changes to a draft guidance on pre-submission facility correspondence (PFC) related to prioritized generic drug submissions are helpful, it believes that additional changes are needed. The association’s comment letter says the revised draft guidance provides some additional
Drugs
The National Cancer Institute says it has four concerns about an FDA guidance that requires studies of oncologic investigational agents to enroll additional patients on multiple dose levels to assess activity for dose optimization, before the therapy’s clinical effectiveness has been established and before the tolerability of therapy has
Drugs
FDA has announced a 4/12 Nonprescription Drugs Advisory Committee meeting to discuss the adequacy of efficacy data available for over-the-counter (OTC) use of oral phenylephrine as a nasal decongestant. Panel members are also scheduled to discuss whether oral nasal decongestants phenylephrine hydrochloride and phenylephrine bitartrate should be
Drugs
Incyte says it will discontinue the Phase 3 LIMBER-304 myelofibrosis trial after a pre-planned interim analysis performed by an independent data monitoring committee determined that the study is unlikely to meet the primary endpoint in the intent-to-treat patient population. It says safety considerations were not a
Drugs
In response to a World Health Organization (WHO) warning about contaminated medications, including children’s cough/cold syrups found in Gambia with unacceptable amounts of diethylene glycol (DEG) and ethylene glycol (EG), CDER director Patrizia Cavazzoni says the agency is stepping up its actions to prevent contaminated drug products from
Drugs
Novartis has petitioned FDA to require potential generic copies of multiple sclerosis drug Mayzent (siponimod fumaric acid) oral tablets to meet certain requirements before being approved. Mayzent was approved 3/2019 for treating relapsing forms of multiple sclerosis, to include clinically isolated syndrome, relapsing-remitting disease, and active secondary progressive
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FDA has declined a Veru request for an Emergency Use Authorization (EUA) for sabizabulin, a microtubule disruptor for treating hospitalized adult patients with moderate to severe Covid-19 who are at high risk for Acute Respiratory Distress Syndrome. In a statement, the company said the agency’s decision was based
Drugs
Exelixis says its Phase 3 CONTACT-03 study evaluating the company’s Cabometyx (cabozantinib) in combination with atezolizumab (Roche’s Tecentriq) versus cabozantinib alone in certain patients with renal cell carcinoma (RCC) did not meet its primary endpoint of progression-free survival (PFS). The study assessed patients who progressed during
Drugs
Federal Register notice: FDA is canceling for undisclosed reasons a 3/20 joint meeting of the Nonprescription Drugs Advisory Committee and Anesthetic and Analgesic Drug Products Advisory Committee. Panel members were scheduled to discuss a Harm Reduction Therapeutics NDA for naloxone hydrochloride nasal spray, 3 mg/0.1 mL, for
Drugs
FDA has cleared a Bigfoot Biomedical 510(k) for the Android version of the Bigfoot Unity Mobile App, which is a part of the Bigfoot Unity Diabetes Management System. Because 41% of U.S. smartphone users choose Android devices, the clearance is intended to enable expanded access to those with
Drugs
The CDER Drug Safety 2022 Priorities Report highlights the Center’s activities involving Covid-19, the opioid crisis, drug overdoses, and efforts to improve the overall quality of compounded drugs. The report, the eighth in the series, also reviews the FDA Adverse Event Reporting System, Sentinel system, and the Safe
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CDER director Patrizia Cavazzoni says FDA and the Federal Trade Commission (FTC) are working together to help advance competition for biologics, including biosimilars and interchangeable biosimilars. Writing in an online FDA Voices post, Cavazzoni says the agencies’ commitments were detailed in a 2/3/2020 joint statement. That was followed
Drugs
The Sedgwick product safety and recall 2022 report, State of the Nation 2023, says that pharmaceutical recall events increased by 32.5% from 274 in 2021 to 363 in 2022. Comparably, medical device recall events went up 11.4% from 837 in 2021 to 932, the report says. Current good