• All News
• Consumer
• Pro
• New Drugs
• Pipeline
• Clinical Trials
• FDA Alerts

Monthly News Roundup - October 2023

Medically reviewed by Leigh Ann Anderson, PharmD. Last updated on Nov 1, 2023.

FDA Clears Loqtorzi as First Treatment for Nasopharyngeal Carcinoma (NPC)

In October, the U.S. Food and Drug Administration (FDA) approved Coherus BioSciences’ Loqtorzi (toripalimab-tpzi) to treat adult patients with metastatic or recurrent locally advanced nasopharyngeal carcinoma (NPC). NPC is an aggressive cancer that starts in the nasopharynx (the throat area behind the nose and near the base of the skull). Due to tumor location, surgery is rarely an option. NPC is rare in the United States, with an yearly incidence of fewer than 1 case per 100,000 people.

• Loqtorzi, a programmed death receptor-1 (PD-1)-blocking antibody, is the first FDA-approved agent for NPC. It works by blocking PD-L1 and PD-L2 ligands to enable the immune system to activate and kill the tumor.
• Loqtorzi is approved to be used for first-line treatment of adults with metastatic (cancer that has spread) or with recurrent (cancer that has returned) locally advanced NPC in combination with cisplatin and gemcitabine, or as a single agent for the treatment of adults with recurrent unresectable (cannot be removed surgically) or metastatic NPC with disease progression on or after a platinum-containing chemotherapy.
• Loqtorzi is given as an intravenous (IV) infusion. When given with cisplatin and gemcitabine, the dose is 240 mg IV every 3 weeks. When used as a single agent, the dose is weight-based at 3 mg/kg IV given every 2 weeks.
• FDA approval was based on results of the Phase 3 JUPITER-02 and Phase 2 POLARIS-02 studies. In JUPITER-02, Loqtorzi combined with chemotherapy significantly improved progression-free survival (PFS), reducing the risk of disease progression or death by 48% compared to chemotherapy alone. PFS is the length of time during and after the treatment of cancer that a patient lives with the disease but it does not get worse. Loqtorzi also reduced the risk of death by 37% compared to chemotherapy alone. In POLARIS-02, 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%. ORR is the percentage of patients who achieve a response to treatment (either a partial or complete response).
• Common (≥ 20%) adverse reactions can include fatigue, hypothyroidism (low thyroid hormones) and musculoskeletal pain when used as a single agent. When combined with chemotherapy, common adverse reactions include nausea, vomiting, decreased appetite, constipation, hypothyroidism, rash, pyrexia (fever), and diarrhea, among others. Warnings include immune-related side effects (which may be severe or fatal), infusion-related reactions, and complications from hematopoietic stem-cell transplantation.
• Loqtorzi is expected to be commercially available in the first quarter of 2024.

Pfizer’s Oral Velsipity is Cleared to Treat Adults with Ulcerative Colitis

This past month the FDA approved Velsipity (etrasimod) for the treatment of moderately-to-severely active ulcerative colitis (UC) in adults. UC affects over 1 million people in the U.S., leading to symptoms such as diarrhea with blood and mucus, stomach area pain, and bowel urgency.

• Velsipity is a non-steroid, oral, once-daily sphingosine-1-phosphate (S1P) receptor modulator. It is thought to work by selectively binding with S1P receptor subtypes 1, 4, and 5 to reduce lymphocyte (white blood cell) migration into the intestines, which may worsen inflammation.
• Approval was based on the ELEVATE UC 52 and ELEVATE UC 12 Phase 3, placebo-controlled studies looking at clinical remission in UC patients who had previously failed or were intolerant to at least one previous UC therapy (conventional, biologic, or Janus kinase/JAK inhibitor therapy). The primary objective was to assess the safety and efficacy of Velsipity 2 mg once daily on clinical remission after both 12 and 52 weeks, based on the 3-domain, modified Mayo score (MMS).
• Statistically significant improvements were obtained in all key primary and secondary endpoints. In ELEVATE UC 52, clinical remission was achieved in 27% of patients receiving Velsipity compared to 7% for the placebo group at week 12, and 32% compared to 7% at week 52. In ELEVATE UC 12, clinical remission was met for 26% of patients receiving Velsipity compared to 15% of patients receiving placebo.
• The recommended dosage is 2 mg orally once daily. Swallow the tablet whole, with or without food.
• The most common side effects include headache, elevated liver tests, and dizziness. Warnings include infections, abnormal heart rhythm and liver injury, among others. Velsipity is primarily metabolized by cytochrome P450 (CYP) enzymes 2C8, 2C9, and 3A4.

FDA OKs Omvoh, a First-in-Class Agent for Ulcerative Colitis

Omvoh (mirikizumab-mrkz), from Eli Lilly, is the first and only interleukin-23p19 (IL-23p19) antagonist approved for the treatment of moderately to severely active ulcerative colitis (UC) in adults. In studies, rapid improvement of symptoms such as rectal bleeding and stool frequency were observed as early as 3 weeks in patients treated with Omvoh.

• Omvoh is a humanized IgG4 monoclonal antibody that selectively binds to the p19 subunit of human IL-23 cytokine and inhibits its interaction with the IL-23 receptor. IL-23 is known to be involved in intestinal mucosal inflammation in UC.
• Approval was based on the LUCENT Phase 3 studies, a 12-week induction study (UC-1) and a 40-week maintenance study (UC-2) for 52 weeks of continuous treatment. Enrolled patients had failed or could not tolerate past treatments, including biologics or JAK inhibitor treatment. Disease activity was assessed based on the modified Mayo score (mMS).
• Results for the primary endpoints were found to be statistically significant. In UC-1 and UC-2, the primary endpoints were clinical remission at Week 12 and Week 40, respectively. After 12 weeks, 24% in the Omvoh group and 15% in the placebo group achieved remission. After 40 weeks in UC-2 (a total of 52 weeks of treatment), 51% in the Omvoh group and 27% of the placebo group achieved remission. Among those who achieved clinical remission at 12 weeks, approximately two-thirds (66%) of patients maintained clinical remission through one year of continuous treatment compared to placebo (40%).
• Omvoh is given by a healthcare provider as a 300-mg IV infusion (into a vein in the arm) over at least 30 minutes at weeks 0, 4 and 8 for induction. For ongoing maintenance treatment, patients can be taught to self-inject two, 100-mg subcutaneous (under the skin) injections every four weeks, starting at week 12.
• The most common side effects include upper respiratory infections, joint pain, rash, injection site reaction, headache, and herpes viral infection. Warnings include serious allergic reactions, infections and liver problems.

Zymfentra Subcutaneous Form OK’d for Inflammatory Bowel Disease (IBD)

Zymfentra (infliximab-dyyb), from Celltrion, is a tumor necrosis factor (TNF) blocker now approved for the subcutaneous maintenance treatment of moderately to severely active ulcerative colitis (UC) and Crohn’s disease (CD). Zymfentra is used only following induction treatment with an infliximab product administered intravenously.

• Zymfentra works by blocking the action of TNF-alpha, a protein that can be overproduced in response to certain diseases and cause the immune system to attack normal, healthy parts of the body.
• Approval was based on phase III pivotal data (LIBERTY UC and LIBERTY CD) that evaluated the efficacy and safety of Zymfentra as maintenance therapy in patients with moderately to severely active UC and CD. Zymfentra demonstrated superiority in the primary endpoints of clinical remission (UC and CD) and endoscopic response (CD) compared to placebo as maintenance therapy after induction therapy of intravenous formulation of infliximab in patients with UC and CD, over a 54-week study period. No new safety signals were seen.
• Administration of subcutaneous Zymfentra starts at week 10 after completion of an intravenous (IV) induction regimen with an infliximab product. The subcutaneous dose is 120 mg once every two weeks.
• The most common side effects include: COVID-19, joint pain, respiratory infections, (like sinus infections and sore throat), diarrhea, injection site reaction, high blood pressure, headache, urinary tract infections, stomach area pain, dizziness, and liver enzyme changes.
• Zymfentra will be under patent protection until at least 2037.

FDA Approves Bimzelx to Treat Adults with Moderate to Severe Plaque Psoriasis

Bimzelx (bimekizumab-bkzx), from UCB Inc., is a humanized interleukin-17A and interleukin-17F antagonist approved for the treatment of moderate to severe plaque psoriasis in adults who are candidates for systemic therapy or phototherapy. Psoriasis has been shown to affect more than 7.5 million adults in the U.S. and exhibits symptoms like dry skin lesions, rashes and itching, often on the elbows, knees and lower back.

• Bimzelx works by selectively and directly inhibiting IL-17A and IL-17F, two key cytokines linked to plaque psoriasis inflammation. It is the first agent approved in the U.S. that targets both cytokines for plaque psoriasis.
• FDA approval was based on three Phase 3 studies of Bimzelx versus placebo or ustekinumab (BE VIVID), versus placebo (BE READY), and versus adalimumab (BE SURE). Patients treated with Bimzelx achieved superior levels of skin clearance at week 16, compared to those who received placebo or active comparator, as measured by at least a 90% improvement in the Psoriasis Area & Severity Index (PASI 90) and an Investigator’s Global Assessment (IGA) response of clear or almost clear skin (IGA 0/1).
• Ranked secondary endpoints were met and included PASI 75 at week 4 and PASI 100 (complete skin clearance) at week 16. Response with Bimzelx was rapid, with more than 7 out of 10 patients achieving PASI 75 at week 4 following one dose (320 mg).
• The dose of Bimzelx is 320 mg (two subcutaneous injections of 160 mg each) at weeks 0, 4, 8, 12 and 16, then every 8 weeks thereafter. It’s available as an autoinjector and a pre-filled syringe and is expected to be available in the U.S. in mid-November.
• The most common side effects are upper respiratory infections, oral candidiasis (thrush), headache, injection site reactions, tinea (fungal) infections, gastroenteritis (bacterial/viral stomach infection), among others. Warnings include suicidal ideation and behavior, infections, tuberculosis (TB), liver test changes, and Inflammatory Bowel Disease (IBD).

Penbraya Vaccine Provides Broad Serogroup Coverage for Meningococcal Disease

Pfizer’s Penbraya vaccine is now approved for active immunization to prevent invasive meningococcal disease caused by Neisseria meningitidis serogroups A, B, C, W, and Y. Penbraya is approved by the FDA for use in individuals 10 through 25 years of age.

• Meningococcal disease is an uncommon but serious bacterial infection that can infect the spinal cord and brain, causing meningitis and blood infections. It can be fatal within 24 hours or lead to permanent and disabling medical problems.
• Penbraya combines the components from two meningococcal vaccines into one injection: Trumenba (meningococcal group B vaccine) and meningococcal groups A, C, W-135, and Y conjugate vaccine. Penbraya helps to protect against the most common serogroups that cause the majority of invasive meningococcal disease (IMD) globally.
• Approval was based on data from Phase 2 and Phase 3 trials that showed that Penbraya has robust immunogenicity non-inferior to Trumenba + groups A, C, W-135, and Y conjugate vaccine for all serogroups and was well-tolerated with a favorable safety profile.
• Penbraya, a suspension for intramuscular injection, is administered as a two-dose series given 6 months apart. It reduces the total number of doses needed for individuals to be fully vaccinated against the five most common serogroups.
• The most commonly reported side effects were pain at the injection site, fatigue, headache, muscle pain, injection site redness, injection site swelling, joint pain, and chills.
• On October 25th, the CDC’s Advisory Committee on Immunization Practices (ACIP) voted to recommend Penbraya for use in people 16 through 23 years of age when both MenACWY and MenB are indicated at the same visit. These provisional recommendations will be reviewed and finalized by the director of the CDC and the Department of Health and Human Services (HHS).

Agamree Oral Suspension Approved to Treat Duchenne Muscular Dystrophy

Agamree (vamorolone) 40 mg/mL oral suspension is a novel corticosteroid approved for the treatment of Duchenne Muscular Dystrophy (DMD) in patients 2 years of age and older.

• DMD is a rare, incurable, muscle wasting disease typically associated with people assigned male at birth. Patients weaken, lose the ability to walk, have serious issues related to the heart and lungs and may only live into their 20’s or 30’s.
• Agamree is a novel corticosteroid drug that acts through the glucocorticoid receptor to exert antiinflammatory and immunosuppressive effects. The mechanism of action is based on differential effects on glucocorticoid and mineralocorticoid receptors which may lead to a better-tolerated side effect profile, but the exact mechanism is not fully known.
• In the pivotal Phase 2b VISION-DMD study, Agamree was administered at doses ranging from 2 to 6 mg/kg/day, extending for a period of up to 48 months. Agamree met the primary endpoint Time to Stand (TTSTAND) velocity versus placebo at 24 weeks of treatment. TTSTAND velocity is a measure of muscle function that records the time required for the patient to fully stand from a position on the floor.
• Compared with current standard-of-care corticosteroids, Agamree treatment exhibited comparable efficacy, with data suggesting a reduction in adverse events, notably related to bone health, growth trajectory, and behavior.
• The recommended dosage is 6 mg/kg taken orally once daily preferably with a meal, up to a maximum daily dosage of 300 mg for patients weighing more than 50 kg. Lower doses suggested for patients with liver impairment.
• The most commonly reported adverse events include cushingoid features (for example: round face, weight gain, fatty hump, bruising easily, fatigue), mental health disorders, vomiting, increased weight, and vitamin D deficiency. Warnings include changes in endocrine function, immunosuppression, increased risk of infection, and changes in heart and kidney function.
• Agamree is marketed by Catalyst Pharmaceuticals, Inc. and is expected to launch in the first quarter of 2024.

Posted October 2023

More news resources

• FDA Medwatch Drug Alerts
• Daily MedNews
• News for Health Professionals
• New Drug Approvals
• New Drug Applications
• Drug Shortages
• Clinical Trial Results
• Generic Drug Approvals

Subscribe to our newsletter

Whatever your topic of interest, subscribe to our newsletters to get the best of Drugs.com in your inbox.