Monthly News Roundup - October 2024

Medically reviewed by Leigh Ann Anderson, PharmD. Last updated on Oct 31, 2024.

FDA Approves AbbVie’s Vyalev to Treat Advanced Parkinson Disease

In October, the US Food and Drug Administration (FDA) approved Vyalev (foscarbidopa and foslevodopa) for the treatment of motor fluctuations in adults with advanced Parkinson disease (PD). Parkinson disease is a long-term, progressive nervous system disorder that affects dopamine-producing neurons in the brain. It leads to problems with movement, such as tremors, stiffness, slowed walking and impaired balance.

Vyalev contains prodrugs of both carbidopa and levodopa and is administered as a 24-hour subcutaneous (under the skin) pump infusion, allowing for individualized dosing needs.
Approval was based on the 12-week, Phase 3 M15-736 study with 130 participants that directly compared Vyalev (plus oral placebo) to oral, immediate-release carbidopa/levodopa (plus a placebo infusion) in patients with advanced PD. "On" time in PD refers to the periods of time when patients are experiencing optimal motor symptom control while "off" time is when symptoms return. The primary endpoint of good "on" time, defined as "on" time without dyskinesia (involuntary muscle movements) plus "on" time with non-troublesome dyskinesia, was collected and averaged over 3 consecutive days and normalized to a typical 16-hour waking period.
The increase in "on" time without troublesome dyskinesia (involuntary muscle movements) at week 12 was 2.72 hours for Vyalev versus 0.97 hours for oral immediate-release carbidopa/levodopa (p=0.0083). Improvements in "on" time were observed as early as the first week and persisted throughout the 12 weeks.
Warnings and precautions include falling asleep during activities of daily living, hallucinations/psychosis, impulse control behaviors, and infusion site reactions and infections.
Common adverse reactions (commonly called side effects) include infusion/catheter site reactions or infections, hallucinations, and dyskinesia (involuntary muscle movements).

FDA Approves First-in-Class Vyloy to Treat Advanced Gastric and GEJ Cancer

This past month the FDA cleared Vyloy (zolbetuximab-clzb) for the first-line treatment of patients with HER2-negative gastric cancer or gastroesophageal junction (GEJ) cancer whose tumors are claudin (CLDN) 18.2 positive. It is used in combination with fluoropyrimidine- and platinum-containing chemotherapy. An FDA-approved test is used to identify patients who may be eligible for Vyloy.

GEJ adenocarcinoma is a cancer that starts at the area where the esophagus joins the stomach. Gastric and gastroesophageal junction (G/GEJ) cancer is the fifth most commonly diagnosed cancer worldwide.
Approval was based on the Phase 3 SPOTLIGHT and GLOW clinical studies. The SPOTLIGHT study evaluated Vyloy plus mFOLFOX6 (oxaliplatin, leucovorin, and fluorouracil) compared to placebo plus mFOLFOX6. The GLOW study evaluated Vyloy plus CAPOX (capecitabine and oxaliplatin) compared to placebo plus CAPOX. Both trials met their primary endpoint, progression-free survival (PFS), as well as a key secondary endpoint, overall survival (OS), in patients treated with Vyloy plus chemotherapy compared to placebo plus chemotherapy.
The recommended first dose of Vyloy is 800 mg/m2 followed by 600 mg/m2 every 3 weeks or 400 mg/m2 every 2 weeks, given as an intravenous (IV) infusion.
The most common adverse reactions (≥15%) for Vyloy in combination with mFOLFOX6 or CAPOX were nausea, vomiting, fatigue, decreased appetite, diarrhea, peripheral sensory neuropathy (weakness, numbness and pain, usually in the hands and feet), abdominal (stomach area) pain, constipation, decreased weight, hypersensitivity (allergic) reactions, and pyrexia (fever).
Vyloy is the first CLDN18.2-targeted therapy approved in the U.S. A Phase 2 trial of zolbetuximab in metastatic pancreatic adenocarcinoma is in progress. Vyloy is manufactured by Astellas Pharma.

FDA Approves Pfizer’s Hympavzi for Patients with Hemophilia A or B Without Inhibitors

The FDA has approved Hympavzi (marstacimab-hncq), a tissue factor pathway inhibitor (TFPI) antagonist, to help prevent or reduce the frequency of bleeding episodes in adults and children 12 years of age and older with hemophilia A (congenital factor VIII deficiency) without factor VIII inhibitors, or hemophilia B (congenital factor IX deficiency) without factor IX inhibitors.

Hemophilia A and Hemophilia B are the most common types of hemophilia, a group of serious bleeding disorders in which blood doesn't clot properly. People with the condition are particularly vulnerable to bleeding in their joints, muscles, and internal organs, leading to pain, swelling, and joint damage. It can be fatal.
Hympavzi contains marstacimab-hncq, a tissue factor pathway inhibitor (TFPI) antagonist. It works by targeting the Kunitz 2 domain of tissue factor pathway inhibitor (TFPI), a natural anticoagulation protein that functions to prevent the formation of blood clots.
Hympavzi is administered by subcutaneous (under the skin) injection once weekly.
Approval was based on the BASIS study which showed that Hympavzi reduced the annualized bleeding rate (ABR) for treated bleeds by 35% and 92% after a 12-month active treatment period compared to routine prophylaxis (RP) and on-demand (OD) treatment, respectively, in patients with hemophilia A or B without inhibitors.
Warnings and precautions include thromboembolic (blood clotting) events, hypersensitivity (allergic) reactions, and risk to an unborn child.
Adverse reactions include injection site reactions, headache, and pruritus (itching).

FDA Approves Orlynvah to Treat Uncomplicated Urinary Tract Infections in Women

Iterum Therapeutics announced the approval of Orlynvah (sulopenem etzadroxil and probenecid), an oral agent used for the treatment of uncomplicated urinary tract infections (uUTIs) in adult women. It targets Escherichia coli, Klebsiella pneumoniae, or Proteus mirabilis in adult women who have limited or no alternative oral antibacterial treatment options.

Orlynvah is a penem antibacterial and renal tubular transport inhibitor combination. It is the first oral penem approved for use in the U.S. and the second FDA-approved treatment for uUTIs in the past two decades.
Orlynvah is not indicated for the treatment of: complicated urinary tract infections (cUTI) or as step-down treatment after intravenous antibacterial treatment of cUTI; complicated intra-abdominal infections (cIAI) or as step-down treatment after intravenous antibacterial treatment of cIAI.
The recommended dose of Orlynvah is one tablet orally twice daily with food for 5 days. Each tablet contains 500 mg of sulopenem etzadroxil and 500 mg of probenecid.
Approval was based on data from the Phase 3, head-to-head studies SURE 1 and REASSURE in the treatment of adult women with uUTI. SURE 1 showed superiority of Orlynvah compared to ciprofloxacin in fluoroquinolone-resistant infections, while REASSURE showed non-inferiority and statistical superiority of Orlynvah compared to amoxicillin and clavulanate potassium (Augmentin).
Warnings and precautions include potentially serious hypersensitivity (allergic) reactions, Clostridioides difficile-Associated Diarrhea (CDAD), and worsening of gout.
Common adverse reactions include diarrhea, nausea, vaginal yeast infection, headache, and vomiting.

Bimzelx Expanded to Include 320 mg Single-Dose Injection

The FDA has approved a 320 mg pre-filled syringe and pre-filled autoinjector for subcutaneous (under the skin) injection of Bimzelx (bimekizumab-bkzx). These new devices add to the available 160 mg autoinjector and pre-filled syringe options and allow a patient prescribed a 320 mg dose to only use one injection (instead of two).

Bimzelx (bimekizumab-bkzx) is a humanized interleukin (IL)-17A and interleukin-17F antagonist and inhibits IL-17A and IL-17F, two key cytokines linked to inflammation. It is approved for the treatment of plaque psoriasis, psoriatic arthritis, non-radiographic axial spondyloarthritis, and ankylosing spondylitis.
In the U.S., a 320 mg dose of Bimzelx is recommended for adults with moderate-to-severe plaque psoriasis and adults with active psoriatic arthritis with coexistent moderate-to-severe plaque psoriasis. For these uses, the recommended dose is 320 mg by subcutaneous injection at Weeks 0, 4, 8, 12, and 16, then every 8 weeks thereafter. For patients weighing120 kg or more, consider a dose of 320 mg every 4 weeks after Week 16.
Approval is based on bioequivalence studies of bimekizumab-bkzx 320 mg given as one 2 mL subcutaneous injection, and bimekizumab-bkzx 320 mg given as two 1 mL subcutaneous injections, in healthy study participants.
The most common (≥ 1%) adverse reactions in plaque psoriasis include upper respiratory tract infections, oral candidiasis, headache, injection site reactions, tinea infections, gastroenteritis, herpes simplex infections, acne, folliculitis, other candida infections, and fatigue.
Most common (≥ 2%) adverse reactions in psoriatic arthritis include upper respiratory tract infections, oral candidiasis, headache, diarrhea, and urinary tract infection.
The 320 mg strength will be available in the US in Q1 2025. Bimzelx is manufactured by UCB.

FDA Approves Roche’s Oral Itovebi for PIK3CA-Mutated HR+, HER2- Breast Cancer

In October, the FDA approved Itovebi (inavolisib) targeted therapy to be used with palbociclib (Ibrance) and fulvestrant (Faslodex) for treatment of breast cancer. This regimen is used in adults with breast cancer that is HR-positive, HER2-negative, has an abnormal PIK3CA gene, has spread locally or to other parts of the body, and has come back after treatment (endocrine therapy resistant). A healthcare provider will test the cancer for abnormal PIK3CA genes using an FDA-approved biomarker blood test.

Itovebi is classified as a phosphatidylinositol 3-kinase (PI3K) alpha inhibitor, which kills cancer cells and slows tumor growth. The PIK3CA mutation is found in approximately 40% of HR-positive metastatic breast cancers.
Itovebi (inavolisib) tablets are taken once daily, with or without food, at about the same time each day. Treatment is usually continued until the cancer worsens or there are intolerable side effects.
The Itovebi-based regimen more than doubled progression-free survival, the primary endpoint. In the Phase 3 INAVO120 study with 325 participants, the Itovebi regimen showed a reduced risk of cancer worsening or death by 57% compared with palbociclib and fulvestrant alone (15 months vs. 7.3 months; hazard ratio [HR]=0.43, 95% CI: 0.32-0.59, p<0.0001) in the first-line setting.
Warnings and precautions include hyperglycemia (high blood sugar), severe stomatitis (mouth sores / inflammation), diarrhea, and harm to an unborn baby.
Common adverse reactions include stomatitis (mouth sores / inflammation), diarrhea, fatigue (feeling tired), nausea, rash, decreased appetite, COVID-19 infection, and headache, in addition to multiple laboratory abnormalities.

FDA Clears Imuldosa, the Fifth Biosimilar to Stelara

In October, the FDA approved Imuldosa (ustekinumab-srlf), a biosimilar to Stelara used to treat plaque psoriasis and active psoriatic arthritis in adults and children 6 years and older; and Crohn's disease and ulcerative colitis in adults. Imuldosa is biosimilar to Stelara but does not have an interchangeability designation.

Ustekinumab is a human monoclonal antibody (mAb) that targets the p40 protein in both interleukin (IL)-12 and IL-23 cytokines to help lower inflammation in immune-mediated diseases like plaque psoriasis and psoriatic arthritis.
Biosimilars have been demonstrated to be similar in efficacy and safety to the originator’s reference product (in this case, Stelara), and should offer cost savings to patients and the healthcare system. Interchangeable biosimilars, which can be more affordable or a preferred insurance product, can be switched with the reference product by the pharmacist without prescriber approval. U.S. sales of the reference product Stelara were close to $7 billion in 2023.
Imuldosa, manufactured by Dong-A ST, is administered by subcutaneous or intravenous injection. It is expected to be available in early 2025.
Imuldosa is the 5th FDA-approved Stelara biosimilar, following the approvals of Otulfi (ustekinumab-aauz), Pyzchiva (ustekinumab-ttwe), Selarsdi (ustekinumab-aekn), and Wezlana (ustekinumab-auub).

Botox Cosmetic Receives Approval for Vertical Bands Connecting the Jaw and Neck (Platysma Bands)

This past month, the FDA cleared Botox Cosmetic (onabotulinumtoxinA) for muscle injection to temporarily improve the appearance of moderate to severe vertical bands connecting the jaw and neck (called platysma bands) in adults. It works by temporarily reducing the underlying muscle activity. Botox Cosmetic is also approved for use on forehead lines, frown lines, and crow's feet lines.

Platysma bands are vertical bands connecting the jaw and neck that appear as part of the aging process. The bands are thought to be due to muscle contractions that can cause the appearance of neck bands and a less defined jawline. Other treatments for platysma bands are limited but include platysma band surgery (platysmaplasty).
OnabotulinumtoxinA is an acetylcholine release inhibitor and a neuromuscular blocking agent. Approval was based on two Phase 3 studies with 834 participants. In studies, the primary endpoint was met with reduction of the severity of platysma bands assessed by both investigator and the subject at Day 14 compared to placebo. The endpoint in active groups was met by 32% and 31% of subjects in two studies, compared to 2% and 0% with placebo (p<0.0001).
For platysma bands, Botox Cosmetic dosing (units) and number of injections sites are dependent upon the number and area of the platysma bands, as outlined in the product label.
Serious, life-threatening side effects may include spread of toxin and problems with breathing or swallowing. Other side effects may include dry mouth, discomfort or pain at the injection site, tiredness, headache, neck pain, eye problems and allergic reactions, among others. Talk to your health care provider about side effects with Botox Cosmetic.
Botox Cosmetic is manufactured by Allergan Aesthetics, an AbbVie company.

Lumryz Label Expanded for Patients 7 Years of Age and Older with Narcolepsy

In October, Avadel Pharmaceuticals announced the expanded approval of Lumryz (sodium oxybate) for the treatment of cataplexy or excessive daytime sleepiness (EDS) in pediatric patients 7 years of age and older with narcolepsy. Previously, Lumryz was approved for the adult population in May 2023.

Narcolepsy is a nervous system condition that impairs the brain's ability to control the sleep-wake cycle. Symptoms of narcolepsy include EDS and may also include a sudden loss of muscle tone usually triggered by strong emotion (cataplexy), disrupted nighttime sleep, sleep paralysis and hallucinations when falling asleep or waking up.
Sodium oxybate is classified as a central nervous system depressant and is the sodium salt of gamma hydroxybutyrate (GHB). It is thought to work through GABAB actions at noradrenergic and dopaminergic neurons, as well as at thalamocortical neurons. Lumryz is classified as a Schedule III controlled substance.
Approval was supported by the REST-ON study, a placebo-controlled Phase 3 trial in adults with narcolepsy. Lumryz demonstrated statistically significant and clinically meaningful improvements in the three co-primary endpoints: EDS (MWT), clinicians’ overall assessment of patients’ functioning (CGI-I), and cataplexy attacks, for all three evaluated doses when compared to placebo.  
Lumryz comes as a pre-filled, oral suspension powder in packets that contain immediate-release and controlled-release granules. The dose is taken as a single bedtime dose, and at least 2 hours after eating. Middle-of-the-night doses are not needed due to the extended-release formulation.
Lumryz carries a Boxed Warning as a central nervous system depressant, for its potential for abuse and misuse and is available only through a Risk Evaluation and Mitigation Strategy called the Lumryz REMS.
The most common adverse reactions in adults include nausea, dizziness, enuresis (bedwetting), headache, and vomiting. In pediatric patients receiving immediate-release sodium oxybate, the most commonly observed adverse reactions (incidence ≥5%) were nausea, enuresis, vomiting, headache, decreased weight, decreased appetite, dizziness, and sleepwalking.

Opdivo Approved for Expanded Perioperative Use in Resectable Non-Small Cell Lung Cancer (NSCLC)

In October, the FDA approved Opdivo (nivolumab) for use with platinum-doublet chemotherapy as treatment before surgery (neoadjuvant), followed by single-agent Opdivo as treatment after surgery (adjuvant) in patients with non-small cell lung cancer (NSCLC) tumors that can be removed by surgery (resectable) and who do not have known epidermal growth factor receptor (EGFR) or anaplastic lymphoma kinase (ALK) mutations.

For some patients with early-stage NSCLC, surgery alone may be used as an option for treatment. However, 30% to 55% of patients can develop cancer recurrence, contributing to a need for perioperative treatment options administered before surgery (neoadjuvant) and after surgery (adjuvant) to improve long-term outcomes.
Approval of Opdivo for perioperative use was based on results from the Phase 3 CheckMate-77T trial which looked at 461 adult patients with resectable NSCLC given either Opdivo plus platinum-doublet chemotherapy before surgery, followed by Opdivo alone after surgery; or placebo plus platinum-doublet chemotherapy followed by surgery and adjuvant placebo after surgery.
The Opdivo arm demonstrated an improved event free survival (EFS), a primary endpoint, compared to the chemotherapy and placebo treatment arm. In cancer studies, EFS is the length of time after cancer treatment ends that the patient remains free of certain complications that the treatment was intended to prevent or delay. In this study, the risk of cancer recurrence, cancer progression or death was reduced by 42% (EFS Hazard Ratio [HR] 0.58; 95% Confidence Interval [CI]: 0.43 to 0.78; P =0.00025) in patients treated in the Opdivo arm, compared to the chemotherapy/placebo arm, with a median follow-up of 25.4 months.
In addition, 18-month EFS was demonstrated in 70% of patients in the Opdivo arm, compared to 50% of patients in the chemotherapy and placebo arm.
The most common adverse reactions (≥20%) in 228 patients receiving Opdivo in combination with chemotherapy were anemia (39.5%), constipation (32%), nausea (28.9%), fatigue (28.1%), hair loss (25.9%) and cough (21.9%).
Opdivo is a programmed death receptor-1 (PD-1) blocking antibody from Bristol Myers Squibb approved for the treatment of wide-ranging types of cancer, including: melanoma (advanced skin cancer); pleural mesothelioma (cancer that affects the lining of the lungs and chest wall); kidney cancer (renal cell carcinoma); classical Hodgkin lymphoma (a blood cancer); squamous cell cancer of the esophagus or head and neck; bladder cancer (urothelial carcinoma); liver cancer; colorectal (colon) cancer; or cancer of the stomach and esophagus.

Abrysvo Vaccine Use Expanded in Adults Aged 18 to 59 at Increased Risk Due to RSV

Pfizer’s Abrysvo (Respiratory Syncytial Virus Vaccine) was FDA-approved in October for active immunization for the prevention of lower respiratory tract disease (LRTD) caused by Respiratory Syncytial Virus (RSV) in individuals 18 through 59 years of age who are at increased risk for LRTD caused by RSV.

RSV is a common but highly contagious virus that can be spread by touching contaminated surfaces, or from respiratory droplets spread by an infected person. The symptoms begin like a cold (runny or stuffy nose, sore throat, fever, cough) but can become serious and lead to wheezing, shortness of breath and more severe illness or death.
Over 9% of US adults ages 18 to 49 have health conditions that increase the risk for more serious RSV-associated LRTD, which rises to 24.3% among those 50 to 64 years of age.
Abrysvo vaccine injection is given as a single, intramuscular dose. Approval was based on the Phase 3 study MONeT (RSV I M munizati ON Study for Adul T s at Higher Risk of Severe Illness), which investigated the safety, tolerability, and immunogenicity of Abrysvo in adults at risk of RSV-associated disease due to certain chronic medical conditions.
The most commonly reported solicited local and systemic adverse reactions in studies in individuals 18 through 59 years of age (≥10%) were pain at the injection site (35.3%), muscle pain (24.4%), joint pain (12.4%), and nausea (11.8%)
Abrysvo is the only RSV vaccine indicated for adults aged 18 to 49 at increased risk for the disease at this time. Formerly, Abrysvo was also approved for immunization against RSV in people 60 years of age and older (May 2023) and for pregnant individuals (32 through 36 weeks of gestation) to protect infants from birth up to 6 months of age (August 2023).
In September 2023, Advisory Committee on Immunization Practices (ACIP) recommended maternal immunization to help protect newborns from RSV seasonally where the vaccine should be administered from September through January in most of the continental US. In June 2024, the ACIP voted to update its recommendation of RSV vaccines for use in adults aged ≥75 years and adults aged 60-74 years who are at increased risk for severe RSV disease.

Posted October 2024

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