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Here is information on the latest US FDA approvals, the week of August 14 – August 19, 2023.
Elrexfio for R/R Multiple Myeloma
The FDA has granted accelerated approval to Elrexfio (elranatamab-bcmm) for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at least four prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody. Approval was based on the results of the single-arm phase 2 MagnetisMM-3 trial, and continued approval of Elrexfio for this indication is contingent upon verification of clinical benefit in a confirmatory trial(s). Elrexfio is a subcutaneously delivered bispecific antibody (BsAb) immunotherapy that binds to BCMA on myeloma cells and CD3 on T-cells, activating T-cells to kill myeloma cells. The accelerated approval was granted last week to Pfizer. Elrexfio joins Talvey and Tecvayli, two bispecific antibody (BsAb) immunotherapies from Janssen also indicated for relapsed or refractory multiple myeloma.
https://labeling.pfizer.com/ShowLabeling.aspx?id=19669
Liver-Directed Treatment for Metastatic Uveal Melanoma
The FDA has approved Hepzato Kit, a system that delivers melphalan to the liver as a treatment for adults with metastatic uveal melanoma (mUM) with unresectable hepatic metastases. The approval was granted to Delcath Systems. Hepzato Kit is currently the only liver-directed therapy approved by the FDA for the treatment of mUM. The approval was based on results from the phase 3 single arm, multicenter, open label FOCUS Study (NCT02678572). The manufacturer stated that Hepzato Kit may have broad applicability in other tumor types, and “we intend to expand our development efforts beyond uveal melanoma given the high incidence of unresectable hepatic dominant tumors.”
https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/201848s000lbl.pdf
HD Eylea in AMD, DME, and DR
The FDA has approved a higher-dose version of Eylea (aflibercept) Injection for the treatment of patients with wet age-related macular degeneration (wAMD), diabetic macular edema (DME) and diabetic retinopathy (DR). The FDA approval is based on the 48-week results of PULSAR and PHOTON pivotal trials comparing Eylea HD 8 mg at intervals of every 12 or 16 weeks to the standard Eylea injection of 2 mg every 8 weeks. Both trials met their primary endpoint, with Eylea HD demonstrating non-inferior and clinically equivalent vision gains at 48 weeks at longer dosing intervals without additional safety issues compared to lower dose Eylea. Eylea, a VEGF inhibitor, was first approved in November 2011 for Wet AMD. The approval was granted to Regeneron Pharmaceuticals, Inc.
https://www.regeneron.com/downloads/eylea_fpi.pdf
Ingrezza for Chorea Associated With Huntington’s Disease
The FDA has approved INGREZZA® (valbenazine) capsules for the treatment of chorea associated With Huntington’s Disease. The term “chorea” refers to the involuntary, irregular, jerking movements commonly present in individuals with HD. The approval is supported by data from two clinical studies: KINECT®-HD Phase 3 study and the ongoing KINECT®-HD2 open-label extension trial. In the United States, the estimated prevalence of HD is around 5 to 10 cases per 100,000 individuals. INGREZZA is a vesicular monoamine transporter 2 (VMAT2) inhibitor and was approved for the treatment of adults with tardive dyskinesia in 2017. The latest approval was granted to Neurocrine Biosciences. Other agents used for Huntington’s Chorea include Xenazine (tetrabenazine) and Austedo (deutetrabenazine).
https://www.neurocrine.com/assets/2023/08/INGREZZA-Full-Prescribing-Information_PI_Approved.pdf
Sohonos (palovarotene) for FOP
The FDA has approved Sohonos (palovarotene), an oral medication to treat the rare bone disease fibrodysplasia ossificans progressive (FOP) in females ages 8 and older and males ages 10 and older. FOP results from activating mutations in ACVRI/ALK2, a bone morphogenetic protein (BMP) type I receptor and affects an estimated 400 people in the US. As the disease progresses with flare-up episodes causing rapid bone growth, heterotopic ossification severely restricts mobility and function leading to premature death. The FDA approval is based on pivotal efficacy and safety data from the Phase 3 MOVE trial, which showed that Sohonos effectively reduced annualized heterotopic ossification volume compared with no treatment beyond standard of care. Adverse events were consistent with the systemic retinoid class to which Sohonos belongs. Sohonos is selective for the gamma subtype of retinoic-acid receptors, an important regulator of skeletal development and ectopic bone in the retinoid signaling pathway. Sohonos, manufactured by Ipsen, received Orphan Drug and Breakthrough Therapy Designations.
https://www.ipsen.com/press-releases/us-fda-aproves-ipsens-sohonostm-palovarotene-capsules-the-first-and-only-treatment-for-people-with-fibrodysplasia-ossificans-progressiva/
Veopoz for CHAPLE Disease
The FDA has approved the first treatment for CD55-deficient protein-losing enteropathy, also called CHAPLE disease. The treatment, Veopoz (pozelimab-bbfg), is a complement inhibitor, and is indicated for patients 1 year of age and older. CHAPLE—which stands for complement hyperactivation, angiopathic thrombosis, and protein-losing enteropathy— is caused by mutations of the complement regulator CD55 gene and triggers an autoimmune attack of the complement system. Fewer than 100 patients worldwide are thought to have this disease, which is associated with several symptoms including life-threatening thrombotic vascular occlusions. Veopoz received fast track, orphan drug, and rare pediatric disease designations. The approval was granted to Regeneron Pharmaceuticals.
https://classic.clinicaltrials.gov/ct2/show/NCT04209634
https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/761339s000lbl.pdf
Upcoming PDUFA dates
LianBio in partnership with Tarsus has announced the submission of an NDA to the U.S. FDA for TP-03 for the treatment of Demodex blepharitis. The target action date is listed as August 25, 2023. No other are scheduled in the FDA tracking calendar this week.
Did you know, you can access our nascentmc.com/podcast page and search the notes from this podcast! So for example, if you search a term like “atherosclerosis” it will bring up all the diabetes related therapeutic agents approved since the first episode on May 15 of this year. We’re also including the prescribing information with each summary too. Check it out at nascentmc.com/podcast.
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