FDA Approvals Roundup: Tavneos, Verzenio, Dextenza

RoundupsRoundups | 13 October 2021 |  By 

A weekly update on new drug approvals and indications from the US Food and Drug Administration (FDA).
New approvals
Tavneos okayed for ANCA-associated vasculitis
ChemoCentryx’s Tavneos (avacopan) has been approved as an adjunctive treatment for adults with severe active anti-neutrophil cytoplasmic autoantibody-associated vasculitis, also known as ANCA-associated vasculitis, in combination with standard therapy.
ANCA-associated vasculitis is a group of three autoimmune diseases – granulomatosis with polyangiitis (GPA), microscopic polyangiitis (MPA), and eosinophilic granulomatosis with polyangiitis – affecting small blood vessels. They can result in organ damage and failure, particularly with the kidneys, and eventually death, if not treated.
This approval of Tavneos, for GPA and MPA, was based on findings from the Phase 3 randomized, double-blind, active-controlled, double-dummy ADVOCATE trial in which 331 patients from the indicated population were randomized 1:1 to receive Tavneos or oral prednisone together with either cyclophosphamide (followed by azathioprine) or rituximab. Patients in both groups could also receive glucocorticoids if needed.
At week 26, 72% of Tavneos patients and 70% of placebo patients had achieved remission (primary endpoint; noninferior to prednisone). Sustained remission at 52 weeks was 66% and 55%, respectively (secondary endpoint; superior to prednisone).
ChemoCentryx discovered and developed Tavneos, complement 5a receptor (C5aR) antagonist, and owns the commercial rights to the drug in the US. The company’s Kidney Health Alliance with Swiss-based Vifor Pharma provides Vifor with exclusive rights to commercialize the drug outside of US.
New indications
Verzenio wins FDA nod for some early breast cancers
Eli Lilly and Company’s Verzenio (abemaciclib) has been approved for adjuvant treatment of certain patients with early breast cancer, in conjunction with either tamoxifen or an aromatase inhibitor.
Verzenio’s approval is for patients with node-positive early breast cancer that is hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative and deemed at high risk of recurrence. Additionally, the patient’s Ki-67 score, a prognostic marker for tumor proliferation, must be 20% or greater as measured by Agilent’s companion diagnostic Ki-67 IHC MIB-1 pharmDx.
The oral CDK 4/6 inhibitor’s approval is the first for adjuvant treatment of breast cancer, noted FDA in announcing the approval.
Efficacy shown in the randomized, open-label, two-cohort multicenter monarchE trial was the basis of the agency’s approval. Patients in the indicated population received either 2 years of Verzenio plus endocrine therapy chosen by the treating physician, or endocrine therapy alone.
Invasive disease-free survival (IDFS) was the primary efficacy endpoint; IDFS for those receiving Verzenio plus endocrine therapy was 86.1% compared with 79% for those on endocrine therapy alone. At the time the IDFS data analysis was conducted, overall survival data were not yet mature.
Eli Lilly and Company participated in FDA’s real-time oncology review pilot program and made use of the assessment aid for the Verzenio submission.
Keytruda now approved as first-line cervical cancer therapy
Merck’s Keytruda (pembrolizumab) has been approved as first-line therapy for persistent, recurrent or metastatic cervical cancer.
The programmed death-ligand 1 (PD-L1) inhibitor’s new cervical cancer indication is for use with chemotherapy and with or without Genentech’s Avastin (bevacizumab) in patients with PD-L1-expressing tumors, as determined by an FDA-approved test. The PD-L1 ICH22C3 pharmDx manufactured by Dako North America received accelerated FDA approval in June 2018.
Keytruda is also newly approved as a single agent for patients with recurrent or metastatic PD-L1-expressing cervical cancer with progression on, or after completion of, chemotherapy.
FDA looked at data from KEYNOTE-826 in reaching its decisions. The randomized, double-blind, placebo-controlled trial enrolled 617 patients, of whom 548 had PD-L1-expressing tumors; 149 global locations are listed as participating trial sites. Patients received chemotherapy with or without Avastin, with either Keytruda or placebo.
For patients whose tumors expressed PD-L1, the primary outcome measure of median overall survival was 16.3 months in the placebo arm and not reached in the Keytruda arm. The other primary endpoints, progression-free survival, was 10.4 months for those receiving Keytruda, compared with 8.2 months in the placebo arm. Both results were highly statistically significant.
FDA’s Oncology Center of Excellence conducted the Keytruda review under Project Orbis. The application received priority review and FDA, which reached its decision 4 months before the goal date, used the assessment aid during its assessment.
Dextenza’s ophthalmic hydrogel plus steroid okayed for allergic conjunctivitis
Ocular Therapeutix has received a new indication for its ocular intracanalicular insert Dextenza (dexamethasone ophthalmic insert), which is now approved to treat ocular itching associated with allergic conjunctivitis.
The hydrogel-based therapy is placed by a health care provider, delivering steroid to the surface of the eye for up to 30 days. The hydrogel resorbs over that time period, essentially being washed away by tears into the nasolacrimal system.
FDA’s approval of the new indication was based on efficacy seen in three randomized, multicenter, double-masked, parallel group vehicle-controlled trials involving 255 patients with ocular allergies and positive skin test reactions to perennial or seasonal allergens. Patients were challenged with allergens for the trials. Compared with vehicle alone, patients had significantly less eye itching at three time points after allergen challenge at day 8 post-Dextenza placement.
Dextenza had previously been approved to treat ocular inflammation and pain following ophthalmic surgery.


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