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www.biopharminternational.com Emerging Therapies 2022 eBook BioPharm International ® 27 Quality/Regulations change in best-corrected visual acuity score (the best distance vision a person can achieve—including with correction such as glasses—when reading letters on an eye chart) from baseline. Both studies met their primary endpoint, wherein faricimab-svoa given at intervals of up to four months was found to be non-inferior to aflibercept given every two months. Additionally, average vision gains from baseline at one year in the faricimab-svoa arms were +5.8 and +6.6 letters, respectively, compared to +5.1 and +6.6 letters in the aflibercept arms. YOSEMITE and RHINE had a similar experimental design, except the patient population was 1891 people with diabetic macular edema (940 in YOSEMITE and 951 in RHINE) and each study had three treatment arms, rather than two: faricimab-svoa administered up to every four months after four initial monthly doses using a treat-and-extend approach; faricimab-svoa administered at two-month intervals after six initial monthly doses; and aflibercept administered at fixed two-month intervals after five initial monthly doses. In YOSEMITE, the average vision gains from baseline at one year were +11.6 and +10.7 eye chart letters in the faricimab-svoa treat-and-extend and two-month arms, respectively, and +10.9 letters in the aflibercept arm. In RHINE, the average vision gains from baseline at one year were +10.8 and +11.8 letters in the faricimab-svoa treat-and-extend and two-month arms, respectively, and +10.3 letters in the aflibercept arm. Sanofi's intravenous hemolysis treatment FDA approved Sanofi's Enjamo (sutimilamab-jome) as an intravenous treatment for hemolysis in adults with cold agglutinin disease (CAD) on Feb. 4, 2022. The first and only FDA-approved treatment for people with CAD, sutimilamab-jome is a humanized monoclonal antibody designed to selectively target and inhibit C1s in the classical complement pathway; through this, it inhibits the activation of the complement cascade in the immune system, in turn inhibiting C1-activated hemolysis (and the resulting destruction of healthy red blood cells) in individuals with CAD (4). Sutimilamab-jome's approval was based on the CARDINAL study, a 26-week open label, single arm, pivotal Phase III study examining 24 patients with CAD who had a recent history of blood transfusion. The study met its primary efficacy endpoint, a compos- ite endpoint defined as the proportion of patients who achieved normalization of hemoglobin (Hgb) level ≥12 g/dL and the proportion of patients who demonstrated an increase from baseline in Hgb level ≥2 g/dL at the treatment assessment time point without receiving a blood transfusion or medications prohibited per the protocol in weeks 5 through 26. In the study, 54% of the patients met the composite pri- mary endpoint criteria with 63% of patients achieving hemoglobin ≥ 12 g/dL or an increase of at least 2 g/dL and 71% of patients remaining transfusion-free after week five. All but two patients (92%) did not use other CAD-related treatments. As a whole, the patients also experienced a mean increase in hemoglobin levels of 2.29 g/dL (SE: 0.308) at week 3 and 3.18 g/dL (SE: 0.476) at the 26-week treatment assessment timepoint from the mean baseline level of 8.6 g/dL. Bristol Myers Squibb's oncology drug Bristol Myers Squibb's Opdualag (nivolumab and relat- limab-rmbw) was approved by FDA on March 18, 2022 as a treatment for patients with unresectable or meta- static melanoma. The novel drug is a fixed-dose combi- nation of the LAG-3-blocking antibody relatlimab and the programmed death receptor-1 blocking antibody nivolumab. Prior to its approval, nivolumab and relat- limab-rmbw received Priority Review, Fast Track, and Orphan Drug designations (5). Approval of nivolumab and relatlimab-rmbw was based on the results of RELATIVITY-047, a random- ized, double-blind clinical trial evaluating 714 pa- tients with previously untreated metastatic or un- resectable Stage III or IV melanoma. Patients were randomized to receive 480 mg of nivolumab and 160 mg of relatlimab by intravenous infusion every four weeks or 480 mg of nivolumab by intravenous infu- sion ever y four weeks until disease progression or unacceptable toxicity. The major efficacy outcome measure was progres- sion-free survival (PFS) determined by Blinded Inde- pendent Central Review (BICR). The trial demonstrated a statistically significant improvement in PFS by BICR for nivolumab and relatlimab compared to nivolumab; median PFS was 10.1 months in the nivolumab and relatlimab arm and 4.6 months in the nivolumab arm. An additional analysis of overall survival (OS) was not found to be statistically significant; median OS was not reached in the nivolumab and relatlimab arm and 34.1 months in the nivolumab arm. References 1. FDA, "Novel Drug Approvals for 2022," www.FDA. gov, accessed Aug. 13, 2022. 2. FDA, "Novel Drug Approvals for 2021," www.FDA. gov, accessed Aug. 13, 2022. 3. Genentech, "FDA approves Genentech's Vabysmo, the First Bispecific Antibody for the Eye, to Treat Two Leading Causes of Vision Loss," Press Release, Jan. 28, 2022. 4. Sanofi, "FDA Approves Enjaymo (sutimlimab-jome), First Treatment for Use in Patients with Cold Agglu- tinin Disease," Press Release, Feb. 4, 2022. 5. FDA, "FDA Approves Opdualag for Unresectable or Metastatic Melanoma," Press Release, March 18, 2022. ■