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FDA approves Omvoh™ (mirikizumab-mrkz)

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On October 26, 2023, Eli Lilly and Company announced that the U.S. Food and Drug Administration approved Omvoh™ (mirikizumab-mrkz) infusion (300 mg/15 mL)/injection (100 mg/mL) for the treatment of moderately to severely active ulcerative colitis (UC) in adults. 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.

Mirikizumab (LY3074828) is a humanized IgG4ҡ monoclonal antibody that blocks the activity of interleukin 23 by targeting the p19 subunit the cytokine. The antibody is engineered with the following mutations: S228P for hinge stabilization, F234A and L235A to abrogate effector function, and K447> del to reduce IgG4 C-terminal heterogeneity.

The recommended induction dosage is 300 mg administered by intravenous infusion over at least 30 minutes at Weeks 0, 4, and 8. The recommended maintenance dosage is 200 mg administered by subcutaneous injection (given as two consecutive injections of 100 mg each) at Week 12, and every 4 weeks thereafter. Lilly received approval for Omvoh in Japan and the European Union earlier in 2023.

FDA approves LOQTORZI™ (toripalimab-tpzi)

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On October 27, 2023, Coherus BioSciences, Inc. and Shanghai Junshi Biosciences Co., Ltd. announced that the U.S. Food and Drug Administration approved LOQTORZI™ (toripalimab-tpzi) in combination with cisplatin and gemcitabine for the first-line treatment of adults with metastatic or recurrent locally advanced nasopharyngeal carcinoma (NPC), and as monotherapy for the treatment of adults with recurrent, unresectable, or metastatic NPC with disease progression on or after platinum-containing chemotherapy.

Toripalimab (marketed as Tuoyi® in China) is an IgG4k anti-PD-1 monoclonal antibody developed by Shanghai Junshi Bioscience Co., Ltd. Coherus partnered with the company to co-develop toripalimab, with Coherus responsible for the development and commercialization of toripalimab in the US and Canada. In 2018, toripalimab became the first anti-PD1 approved in China, and the product is now approved there for multiple types of cancer.

Toripalimab was granted Orphan Drug designations by the FDA for the treatment of NPC, mucosal melanoma, soft tissue sarcoma, esophageal cancer, and SCLC. FDA also granted Breakthrough Therapy designation to toripalimab for the treatment of recurrent or metastatic NPC with disease progression on or after platinum-containing chemotherapy and in combination with gemcitabine and cisplatin as a first-line treatment for patients with recurrent or metastatic NPC.

FDA’s approval was based on results of the JUPITER-02 Phase 3 study and the POLARIS-02 Phase 2 study and is irrespective of a patient’s PD-L1 status. The recommended LOQTORZI dose with cisplatin and gemcitabine is 240 mg every three weeks until disease progression, unacceptable toxicity, or up to 24 months. The recommended LOQTORZI dose as a single agent for previously treated NPC is 3 mg/kg every two weeks until disease progression or unacceptable toxicity.

The Phase 3 JUPITER-02 (NCT03581786) study included patients with recurrent or metastatic NPC and no previous chemotherapy for recurrent or metastatic disease. Patients (n=289)  were randomized (1:1) to receive either toripalimab (240 mg) or placebo in combination with gemcitabine-cisplatin therapy every 3 weeks for up to six cycles, followed by monotherapy with toripalimab or placebo. The primary endpoint was PFS as assessed by a blinded independent review committee according to RECIST v.1.1. The combination of toripalimab and gemcitabine-cisplatin improved the median progression-free survival compared to the chemotherapy arm (11.7 vs 8 months, respectively), the overall response rate (77.4% vs. 66.4% (P = 0.033), respectively) and the median duration of response (10.0 vs. 5.7 months, respectively).

In the POLARIS-02 clinical study, LOQTORZI demonstrated durable antitumor activity in patients with recurrent or metastatic NPC who failed previous chemotherapy, with an objective response rate (ORR) of 20.5%, a disease control rate (DCR) of 40.0%, and a median OS of 17.4 months with an acceptable safety profile.

FDA approves Bimzelx (bimekizumab-bkzx)

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On October 18, 2023, the US Food and Drug Administration approved Bimzelx (bimekizumab-bkzx) for the treatment of moderate to severe plaque psoriasis  in adults who are candidates for systemic therapy or phototherapy. [1] Bimekizumab is a humanized IgG1 kappa antibody that selectively inhibits IL-17A and IL-17F by binding regions that are common to these pro-inflammatory cytokines, which share ~50% sequence identity and are expressed as homodimers and IL-17A/F heterodimers. Bimekizumab is approved at a recommended dose of 320 mg, administered by two subcutaneous injections of 160 mg each every four weeks to week 16 and every eight weeks thereafter.

The approval of bimekizumab is supported by data from two Phase 3 studies that compared the effects of bimekizumab to those of either ustekinumab (Stellara®; BE VIVID study; NCT03370133) or adalimumab (Humira®; BE SURE study; NCT03412747) in adults with moderate to severe plaque psoriasis. These pivotal studies met their co-primary endpoints at Week 16, demonstrating superiority of bimekizumab over the active comparator in certain defined measures (e.g., Psoriasis Area and Severity Index). Clinical responses achieved with bimekizumab at Week 16 were maintained up to one year. [2-3] Coprimary endpoints were also met in the Phase 3 BE READY study (NCT03410992), which investigated the efficacy and safety of bimekizumab in patients with moderate to severe plaque psoriasis compared to placebo. [4]

  1. UCB. BIMZELX[®] Approved by the U.S. FDA for the Treatment of Adults with Moderate to Severe Plaque Psoriasis.
  2. Reich K, Papp KA, Blauvelt A, et al. Bimekizumab versus ustekinumab for the treatment of moderate to severe plaque psoriasis (BE VIVID): efficacy and safety from a 52-week, multicentre, double-blind, active comparator and placebo-controlled phase 3 trial. Lancet. 2021;397(10273):487-498.
  3. Warren RB, Blauvelt A, Bagel J, et al. Bimekizumab versus Adalimumab in Plaque Psoriasis. N Engl J Med. 2021;385(2):130-141.
  4. Gordon KB, Foley P, Krueger JG, et al. Bimekizumab efficacy and safety in moderate to severe plaque psoriasis (BE READY): a multicentre, double-blind, placebo-controlled, randomised withdrawal phase 3 trial. Lancet. 2021;397(10273):475-486.

FDA approves Veopoz (pozelimab-bbfg) for CHAPLE disease

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On August 18, 2023, the US Food and Drug Administration (FDA) approved Veopoz (pozelimab-bbfg) injection for the treatment of adult and pediatric patients 1 year of age and older with CD55-deficient protein-losing enteropathy (PLE), also known as CHAPLE disease. Pozelimab (REGN3918) is a human IgG4k antibody targeting complement component 5 (C5) developed by Regeneron. Veopoz received fast track, orphan drug, and rare pediatric disease designations.

Veopoz is the first FDA-approved treatment for CHAPLE disease. An initial dose of 30 m/kg Veopoz is administered intravenously (IV), followed by weekly injections of 10 m/kg given subcutaneously by a health care provider. See the prescribing information for details

The efficacy and safety study of pozelimab in patients with CHAPLE were evaluated in an open-label, single arm Phase 2/3 study (NCT04209634). The study included 10 patients from 3 to 19 years of age with a clinical diagnosis of CD55-deficient PLE disease who received a single loading IV dose on Day 1, then fixed SC doses based on body weight every week over the treatment period. The primary outcome measure of the study was the proportion of patients with active disease at baseline achieving both normalization of serum albumin and clinical outcome improvement. All 10 patients achieved a serum albumin concentration of at least 3.5 g/dL by week 12, which was maintained through at least 72 weeks. All 10 patients also demonstrated a reduction in the number of hospitalizations and number of albumin transfusions over the first 48 weeks of treatment as compared to the 48 weeks prior to treatment.

Interested in data for other antibody therapeutics that have received marketing authorizations? Go to our searchable table of approved antibody therapeutics and those in regulatory review for more information.

Elranatamab-bcmm (Elrexfio) approved for multiple myeloma

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On August 14, 2023, the Food and Drug Administration (FDA) granted accelerated approval to elranatamab-bcmm (Elrexfio, Pfizer, Inc.) for adults with relapsed or refractory multiple myeloma (r/r MM) who have received at least four prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody. The biologics license application (BLA) for elranatamab was granted priority review, breakthrough designation and orphan drug designation by FDA.

Elranatamab (PF-06863135) is a humanized IgG2a T cell-engaging bispecific antibody designed to target BCMA, which is highly expressed on tumor cells, and CD3 found on the T cell surface. Developed by Pfizer, elranatamab is formulated for subcutaneous, rather than intravenous, administration. The recommended elranatamab-bcmm dosages include the following: “step-up dose 1” of 12 mg on Day 1, “step-up dose 2” of 32 mg on Day 4, followed by the first treatment dose of 76 mg on Day 8, and then 76 mg weekly, thereafter, through week 24.

The prescribing information for elranatamab-bcmm includes a Boxed Warning for life threatening or fatal cytokine release syndrome and neurologic toxicity. Elrexfio is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS), called the ELREXFIO REMS.

Elranatamab’s efficacy was evaluated in MagnetisMM-3 (NCT04649359), a Phase 2, open-label, single-arm, multi-center study that included patients with r/r MM who are refractory to at least one proteasome inhibitor, one immunomodulatory drug, and one anti-CD38 antibody. Patients had measurable disease by International Myeloma Working Group (IMWG) criteria at enrollment. The main efficacy outcome measures were objective response rate (ORR) and duration of response (DOR), as assessed by a blinded independent central review based on IMWG criteria. The primary efficacy population consisted of 97 patients naïve to prior BCMA-directed therapy and who had previously received at least 4 prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody. The ORR in the 97 patients receiving the recommended dose was 57.7% (95% CI: 47.3%, 67.7%). With a median follow-up of 11.1 months among responders, the median DOR was not reached (95% CI: 12 months, not reached). The DOR rate at 6 months was 90.4% (95% CI: 78.4%, 95.9%) and at 9 months was 82.3% (95% CI: 67.1%, 90.9%).

FDA’s BLA review was conducted under Project Orbis, which provides a framework for concurrent submission and review of oncology drugs among international partners. The Australian Therapeutic Goods Administration, the Brazilian Health Regulatory Agency, Health Canada, and Swissmedic collaborated with FDA in the review process, and are continuing to review marketing applications for elranatamab.

Interested in data for other antibody therapeutics that have received marketing authorizations? Go to our searchable table of approved antibody therapeutics and those in regulatory review for more information.