Here is information on the latest US FDA approvals, the week of October 23 – October 27, 2023
Loqtorzi (toripalimab-tpzi) for Nasopharyngeal Carcinoma
The FDA has approved Loqtorzi (toripalimab-tpzi), a next-generation PD-1 monoclonal antibody, for two key uses in nasopharyngeal carcinoma (NPC): 1) use in combination with cisplatin and gemcitabine as a first-line treatment for metastatic or recurrent locally advanced NPC. Loqtorzi is also approved as a monotherapy for recurrent, unresectable, or metastatic NPC, regardless of PD-L1 status, after platinum-containing chemotherapy. This approval is based on data from the JUPITER-02 Phase 3 study, showing that Loqtorzi significantly improved progression-free survival (48% risk reduction) and overall survival (37% lower risk of death) when combined with chemotherapy. In the POLARIS-02 study, Loqtorzi demonstrated promising antitumor activity, with a 20.5% objective response rate, 40.0% disease control rate, and a median overall survival of 17.4 months in patients who had previously failed chemotherapy. Loqtorzi, manufactured by Coherus and Junshi Biosciences, is expected to be available in the United States in early 2024.
https://loqtorzi.com/pdf/prescribing-information.pdf
Vabysmo for macular edema following RVO
The FDA has approved Vabysmo (faricimab-svoa) as a treatment for macular edema following retinal vein occlusion (RVO), its third approved indication. This bispecific antibody, the first of its kind granted FDA approval for eye-related conditions, targets angiopoietin-2 (Ang-2) and vascular endothelial growth factor-A (VEGF-A), key factors in vision-threatening retinal diseases. It has previously gained approval for wet AMD and diabetic macular edema, conditions impacting approximately 3 million US patients. Two global phase 3 studies, BALATON and COMINO, supported this approval. Faricimab demonstrated early and sustained vision improvement in patients with RVO, meeting the primary endpoint of non-inferior visual acuity gains at 24 weeks compared to aflibercept. Faricimab also rapidly dried retinal fluid and exhibited a generally well-tolerated safety profile, with conjunctival hemorrhage as the most common adverse event (3%). There is an updated Warnings and Precautions section in the label mentioning rare post-marketing cases of retinal vasculitis and/or retinal vascular occlusion, usually in the presence of intraocular inflammation. However, the reported rates of these events align with those of common intravitreal treatments for wet AMD, DME, and RVO. The approval was granted to Genentech.
https://www.gene.com/download/pdf/vabysmo_prescribing.pdf
Omvoh for Moderate-To-Severe Ulcerative Colitis
The FDA has approved Omvoh (mirikizumab-mrkz), to treat adults with moderate-to-severe active ulcerative colitis, a form of chronic inflammatory bowel disease. Omvoh is the only UC treatment that selectively targets the p19 subunit of IL-23, which plays a role in inflammation related to UC. The approval was based on results from the LUCENT program, which included two randomized, double-blind, placebo-controlled Phase 3 clinical trials consisting of one 12-week induction study (UC-1) and one 40-week maintenance study (UC-2) for 52 weeks of continuous treatment. All patients in the LUCENT program had past treatments, including biologic treatments, that did not work, stopped working or that they could not tolerate. This approval of Omvoh follows a setback in April when the FDA initially declined clearance due to manufacturing-related issues. Ulcerative colitis is characterized by immune system-driven inflammation and ulcers in the colon lining, potentially resulting in symptoms like diarrhea, blood in stool, and abdominal pain. The approval was granted to the manufacturer of Omvoh, Eli Lilly. Omvoh will be available in the United States in the coming weeks.
https://pi.lilly.com/us/omvoh-uspi.pdf
Vamorolone for DMD
The FDA has approved Agamree (vamorolone) oral suspension 40 mg/mL, a novel corticosteroid treatment for Duchenne muscular dystrophy (DMD) in patients aged 2 years and older. In clinical trials, vamorolone met the primary endpoint of time to stand (TTSTAND) velocity compared to placebo at the 24-week follow-up of the VISION-DMD phase 2b study (p=0.002) and exhibited a favorable safety profile. DMD affects 11,000 to 13,000 individuals in the United States and leads to muscle function regression, ambulation loss, respiratory failure, and ultimately, mortality. Vamorolone targets both glucocorticoid and mineralocorticoid receptors. In DMD, vamorolone is thought to work by reducing muscle inflammation and atrophy, promoting muscle regeneration and protecting the heart from damage. Common adverse events include mild to moderate cushingoid features, vomiting, and vitamin D deficiency. Vamorolone, manufactured by the Swiss-based company Santhera Pharmaceuticals in partnership with Catalyst Pharmaceuticals, previously received Orphan Drug and Rare Pediatric Disease designations and will gain 7 years of orphan drug exclusivity following this approval. Vamorolone is expected to be available in the first quarter of 2024.
https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/215239s000lbl.pdf
Tibsovo for MDS
The FDA has approved Tibsovo (ivosidenib) as a treatment for adult patients with relapsed/refractory myelodysplastic syndromes (MDS) carrying a susceptible IDH1 mutation, as confirmed by an FDA-approved test. The agency has also approved the Abbott RealTime IDH1 Assay as a companion diagnostic device to aid in patient selection for this treatment. This decision is based on the results of the AG120-C-001 trial, which involved 18 adult patients with relapsed or refractory MDS harboring an IDH1 mutation. A significant proportion of patients achieved transfusion independence. Ivosidenib was generally well-tolerated, with the most common adverse events being manageable. Notably, the label includes a boxed warning for the risk of differentiation syndrome. In addition to MDS, ivosidenib is also approved for certain forms of IDH-mutated acute myeloid leukemia (AML) and metastatic cholangiocarcinoma. This approval was granted to Servier Pharmaceuticals.
https://www.tibsovopro.com/pdf/prescribinginformation.pdf
FluMist for At-Home Administration
The FDA is reviewing AstraZeneca’s application that would allow patients or caregivers to administer its nasal flu vaccine, FluMist Quadrivalent. If approved, this vaccine could become the first self-administered flu vaccine, eliminating the need for healthcare practitioner administration. FluMist was initially approved by the FDA in 2003. AstraZeneca anticipates a decision from the FDA in the first quarter of 2024, with plans to make the vaccine available for self-administration in the United States during the 2024-2025 flu season, provided it receives approval. This supplemental application is supported by data from a usability study, which confirmed that individuals over 18 years of age can successfully self-administer or administer FluMist to eligible patients aged 2-49 years when provided with instructions for use, without the need for additional guidance.
https://www.fda.gov/media/160349/download
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