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Here is information on the latest US FDA approvals, the week of August 28 – September 8, 2023.
FDA Approves Ilaris for Treatment of Gout Flares in Adults
The FDA has approved Ilaris (canakinumab) for the treatment of gout flares in adults who either could not tolerate or had an inadequate response to initial treatments, such as NSAIDs, colchicine, or repeated corticosteroid courses. Canakinumab, a humanized anti-interleukin-1β monoclonal antibody, is administered through a single subcutaneous injection of 150 mg. The approval was based on data from two 12-week, double-blind, active-controlled trials. In the β-RELIEVED study (ClinicalTrials.gov Identifier: NCT01029652) Ilaris was approved by the FDA in 2009 for treating children and adults with cryopyrin-associated periodic syndrome (CAPS). It has also been approved for other autoinflammatory diseases, including Still’s disease and recurrent fever syndromes. The latest approval was granted to Novartis.
https://www.novartis.com/us-en/sites/novartis_us/files/ilaris.pdf
FDA Approves Multiple Generics of ADHD Medication
This week the FDA cleared several generic versions of the drug Vyvanse (lisdexamfetamine dimesylate), in an effort to allay shortages of this drug. The now-approved generics range in dose from 10 mg to 70 mg and come in capsule and chewable forms. The approval applies to products from manufacturers Teva, Sun Pharmaceutical, Actavis, Mylan, Hikma and others. Takeda’s patents on the Vyvanse expired August 24, 2023. Vyvanse is also approved for binge-eating disorder and has been in short supply since June due to a manufacturing delay compounded by increased demand. The prescribing information for lisdexamfetamine dimesylate capsules and chewable tablets contains a boxed warning to inform health care providers and patients about the potential risk of abuse and dependence. Takeda’s Vyvanse shortage emerged after another drug, Adderall and its generics began to face similar shortfalls last summer.
https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-multiple-generics-adhd-and-bed-treatment
http://pi.shirecontent.com/PI/PDFs/Vyvanse_USA_ENG.pdf
sNDA for Balversa (erdafitinib) in mUC
A supplemental New Drug Application (sNDA) has been submitted to FDA for the approval of Balversa (erdafitinib) to treat patients with locally advanced or metastatic urothelial carcinoma with FGFR3 or FGFR2 gene alterations, and who have progressed on a prior checkpoint inhibitor or within 12 months of pre- or post-surgical therapy. The manufacturer Janssen submitted data from the phase 3 THOR study which showed that Balversa was superior to standard chemotherapy, with a 36% decrease in risk of death over chemotherapy. Balversa is currently approved for the treatment of adult patients with locally advanced or metastatic urothelial carcinoma (mUC) that has susceptible FGFR3 or FGFR2 genetic alterations and that has progressed on platinum-containing chemotherapy. The current indication is based on accelerated approval granted in April 2019.
https://www.janssenlabels.com/package-insert/product-monograph/prescribing-information/BALVERSA-pi.pdf
INO-3107 for Recurrent Respiratory Papillomatosis
The FDA has granted Breakthrough Therapy designation for INO-3107 as a potential treatment for patients with recurrent respiratory papillomatosis, or RRP. RRP is a debilitating and rare disease caused primarily by HPV-6 and/or HPV-11. RRP is characterized by the development of small, wart-like growths, or papillomas, in the respiratory tract. While papillomas are generally benign, they can cause severe, life-threatening airway obstruction and respiratory complications. RRP can also significantly affect quality of life for patients by affecting the voice box, limiting the ability to speak effectively. Surgery to remove papillomas is the standard of care for RRP; however, the papillomas often grow back because the underlying HPV infection has not been eradicated. INO-3107 is an investigational DNA medicine candidate designed to elicit a targeted T cell response against HPV-6 and HPV-11, the HPV types that cause RRP and other HPV-related disease. The Breakthrough Therapy designation is supported by data from Inovio’s completed Phase 1/2 open-label, multicenter trial that assessed INO-3107’s safety, tolerability, immunogenicity, and efficacy in patients with HPV-6 and/or HPV-11-related RRP. Inovio plans to initiate a pivotal trial of INO-3107 in the first quarter of 2024, subject to FDA clearance.
Crovalimab for PNH
The FDA has accepted a Biologics License Application (BLA) for crovalimab, an investigational, novel anti-C5 recycling monoclonal antibody, for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). The acceptance was based on results from the pivotal Phase III COMMODORE 2 study, which demonstrated that in people with PNH, crovalimab achieved disease control and was well-tolerated. Results from the Phase III COMMODORE 1 study, demonstrating the consistent benefit-risk profile of crovalimab, also supported the application. PNH is a rare and life-threatening blood condition, which affects approximately 20,000 people worldwide. In PNH, red blood cells are destroyed by the complement system – part of the innate immune system. This causes symptoms such as anemia, fatigue and blood clots, and can lead to kidney disease. C5 inhibitors block the complement system cascade and help alleviate the symptoms of PNH. Crovalimab is a novel C5 inhibitor that is recycled within the bloodstream, enabling sustained complement inhibition through low-dose, subcutaneous (SC) administration every four weeks. Crovalimab may be approved by late 2023/early 2024 according to the manufacturer Genentech.
https://www.gene.com/media/press-releases/15000/2023-09-04/fda-accepts-application-for-genentechs-c
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