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FDA Updates for the Week of Aug. 28, 2023


The FDA has approved Reblozyl as first-line treatment in MDS-related anemia and the first generics of ADHD drug Vyvanse. The agency also issued a CRL for bevacizumab to treat wet AMD. Additionally, two companies have submitted supplemental applications. These include Janssen, which is seeking full approval for Balversa for urothelial carcinoma, and AbbVie, which is seeking approval of Skyrizi for ulcerative colitis.

FDA Approves Reblozyl as First-Line Treatment in MDS-Related Anemia

The FDA has approved Bristol Myers Squibb’s Reblozyl (luspatercept-aamt) to treat anemia in adult patients with very low- to intermediate-risk myelodysplastic syndromes (MDS). It is indicated as first-line therapy for patients who have not received erythropoiesis stimulating agents (ESA).

Reblozy promotes late-stage red blood cell maturation and is also approved to treat anemia in adult patients with beta thalassemia who require regular red blood cell transfusions and in patients who have already received and failed treatment with erythropoiesis stimulating agents. The cost for Reblozyl 25 mg powder is around $4,011 for a supply of one injection, and $12,013.37 for 75 mg powder for one injection, according to Drugs.com. Reblozyl is being developed and commercialized through a global collaboration with Merck as of November 2021.

FDA Approves First Generics of ADHD Drug Vyvanse

The FDA has approved the first generics of Takeda’s Vyvanse (lisdexamfetamine dimesylate) capsules and chewable tablets to treat attention-deficit/hyperactivity disorder (ADHD) in patients six years and older and moderate to severe binge-eating disorder in adults. Approvals have been given to 15 different manufacturers.

Vyvanse was approved by the FDA in 2007 to treat patients with ADHD and in 2015 to treat patients with binge-eating disorder. Vyvanse has experienced some shortages of some products, including the 60 mg capsules and 70 mg capsules in 100 count bottles, because of a manufacturing delay and increased demand, according to the American Society of Health-System Pharmacists. Takeda has Vyvanse 60 mg and 70 mg capsules on intermittent back order and the company estimates this will continue into September 2023.

FDA Issues CRL for Bevacizumab to Treat Wet AMD

The FDA has issued a complete response letter (CRL) for Outlook Therapeutics’ biologics license application for Lytenava (bevacizumab-vikg), an ophthalmic formulation of bevacizumab to treat patients with wet age-related macular degeneration (AMD). Regulators indicated that there were issues related to quality, preapproval manufacturing inspections, and a lack of substantial evidence.

Regulators said the pivotal trials met its safety and efficacy endpoints, which were statistically significant. The company will request a formal meeting to resolve these issues and determine next steps.

Bevacizumab is a recombinant humanized monoclonal antibody that is a vascular endothelial growth factor (VEGF) inhibitor, which blocks blood vessel growth. The product is available as Avastin to treat cancers such as colorectal, lung, glioblastoma, kidney, cervical and ovarian cancer. It is used off-label to treat wet AMD. In addition, several Avastin biosimilars are available, including Mvasi, Zirabev, Alymsys, and Vegzelma, but these are only approved for the cancer indications.

Janssen Seeks Full Approval for Balversa for Urothelial Carcinoma

Janssen has submitted a supplemental new drug application (sNDA) for full approval of Balversa (erdafitinib) to treat patients with metastatic urothelial carcinoma. Balversa is a kinase inhibitor for adult patients who have susceptible fibroblast growth factor receptor (FGFR)3 genetic alterations and who progressed during or following treatment.

The FDA had granted accelerated approval for Balversa for these patients in 2019. Balversa 3 mg has a list price of $19,848.65 for 56 tablets, according to Drugs.com. It is administered through CVS Specialty. Janssen offers a copay program of $5 a prescription, with a maximum benefit of $25,000 annually.

The sNDA is based upon data from Cohort 1 of the phase 3 THOR study, which met its primary endpoint of overall survival (OS). Patients who received Balversa achieved a median overall survival of more than one year, and Balversa reduced the risk of death by 36%.

AbbVie Submits Skyrizi to FDA, EMA for Ulcerative Colitis Indication

AbbVie has submitted applications to the FDA and to European Medicines Agency (EMA) for a new indication for Skyrizi (risankizumab) to treat adult patients with moderately to severely active ulcerative colitis.

Skyrizi blocks interleukin-23, a cytokine involved in inflammatory processes and is thought to be linked to a number of chronic immune-mediated disease. Developed through a collaboration between Boehringer Ingelheim and AbbVie, Skyrizi is already available to treat patients with plaque psoriasis, psoriatic arthritis, and Crohn’s disease. The list price for one dose of Skyrizi is $19,734.61.

The applications for the ulcerative colitis indication were supported by data from two phase 3 clinical trials: an induction study, INSPIRE, and a maintenance study, COMMAND. Significantly more patients treated with Skyrizi in the induction study and at week 52 in the maintenance study achieved the primary endpoint of clinical remission compared with patients receiving placebo.

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