Monthly News Roundup - April 2024

Medically reviewed by Leigh Ann Anderson, PharmD. Last updated on April 30, 2024.

FDA Approves First-in-Class Anktiva Plus BCG for Bladder Cancer

In April the U.S. Food and Drug Administration (FDA) approved ImmunityBio’s Anktiva (nogapendekin alfa inbakicept-pmln), a cancer immunotherapy for the treatment of patients with a type of bladder cancer that has not responded to treatment with the BCG (Bacillus Calmette-Guérin) vaccine.

Anktiva is approved to treat BCG-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS), with or without papillary tumors. It is used in addition to the BCG vaccine.
Anktiva is classified as an IL-15 (cytokine) agonist. It works by activating the immune system, including immune memory cells, to kill bladder cancer cells.
Approval was based on a multicenter trial with 77 patients who received Anktiva with BCG maintenance therapy for up to 37 months. Overall, 62% of the patients achieved a complete response (CR), with a duration of response (DOR) ranging from 0 to 47 months (results ongoing). The study showed that 58% of patients with CR had a DOR ≥ 12 months and 40% had a DOR ≥ 24 months.
Anktiva is administered intravesically (directly into the bladder through a catheter) with BCG once a week for 6 weeks (induction), then once a week for 3 weeks at months 4, 7, 10, 13 and 19 (maintenance). After instillation is complete, the catheter is removed. The Anktiva and BCG admixture should be retained in the bladder for 2 hours and then voided. Do not repeat the dose if the patient voids before 2 hours. A second induction course may be administered if complete response is not achieved at month 3.
Common (≥15%) side effects include painful urination, blood in the urine, frequent urination, urinary urgency, urinary tract infection (UTI), muscle and joint pain, chills and fever.

Pfizer’s Beqvez is Cleared as a One-Time Gene Therapy for Hemophilia B

The FDA has approved Pfizer’s Beqvez (fidanacogene elaparvovec-dzkt), an adeno-associated virus vector-based gene therapy for the treatment of adults with moderate to severe hemophilia B (congenital factor IX deficiency). This gene therapy allows patients with hemophilia B to produce factor IX themselves rather than needing the regular intravenous (IV) infusions.

Hemophilia B is a rare genetic bleeding disorder caused by a deficiency of a specific protein in the blood called clotting factor IX. People with severe hemophilia often bleed into their muscles or joints, or rarely into other areas like the intracranial space, where bleeding can be fatal.
Beqvez is a one-time IV gene therapy used to treat adults with moderate to severe hemophilia B who are receiving routine factor IX prophylaxis (for bleeding prevention), have life-threatening bleeds, or have repeated serious spontaneous bleeds. Before treatment, a blood test must rule out antibodies to the AAVRh74var virus.
Approval was based on the Phase 3, single arm, open label BENEGENE-2 clinical study in 45 adult male participants (age 18 to 65) with moderately severe to severe hemophilia B. The objective is to evaluate the annualized bleeding rate (ABR) for participants treated with gene therapy versus the factor IX prophylaxis replacement regimen, administered as part of usual care. Clinical trial participants will be followed for up to a total of 15 years.
BENEGENE-2 met its primary endpoint of non-inferiority in the ABR of total bleeds after the Beqvez infusion compared to the factor IX prophylaxis regimen. Bleeds were eliminated in 60% of patients compared to 29% in the prophylaxis arm. A median ABR of zero (range of 0 to 19) was observed compared to the prophylaxis arm in which a median ABR of 1.3 (range of 0 to 53.9) was observed.
The most common adverse reaction (incidence ≥5%) was an increase in transaminases (liver enzymes) seen in 26 out of 60 patients treated at the recommended dose.

Once-Weekly Oral Ojemda First Approval for BRAF-Altered Pediatric Low-Grade Glioma

In April, the FDA granted accelerated approval to One Day Pharmaceutical’s Ojemda (tovorafenib), a type II RAF inhibitor, for the treatment of patients 6 months and older with pediatric low-grade glioma (pLGG) that has come back or not responded to previous treatment and has a certain type of abnormal “BRAF” gene. Continued approval for this use may depend upon results from further clinical trials.

pLGG is the most common brain tumor diagnosed in children, with up to 75% of children having a BRAF alteration. Until now, there had been no medicines approved for patients with pLGG driven by BRAF fusions.
Ojemda is an inhibitor of mutant BRAF V600, wild-type BRAF, and wild-type CRAF kinases. It works by targeting the Mitogen-Activated Protein Kinase (MAPK) signaling pathway, which affects growth and survival of cells.
Accelerated approval was based on response rate and duration of response in the FIREFLY-1 trial of 137 patients across two study groups. Of the 76 patients in group 1, the best overall response rate was 51%, which included 28% partial responses and 11% minor responses. The median time to response following initiation of treatment with Ojemda was 5.3 months. As of the June 2023 data cutoff, the median duration of response was 13.8 months with 66% of patients continuing on treatment.
Ojemda tablets and oral suspension are administered once weekly, with or without food.
Warnings include hemorrhage, skin toxicity / sensitivity to sunlight, liver toxicity, slowed growth in children, fetal harm and increased tumor growth.
The most common adverse reactions (≥30%) were rash, hair color changes, tiredness, viral infection, vomiting, headache, bleeding, fever, dry skin, constipation, nausea, acne, upper respiratory tract infection and laboratory abnormalities.

FDA Clears Selarsdi, the Second Biosimilar to Stelara

In April, Alvotech and Teva Pharmaceuticals announced the approval of Selarsdi (ustekinumab-aekn), the most recent biosimilar to Stelara.

Selarsdi is approved to treat moderate to severe plaque psoriasis and active psoriatic arthritis in adults and pediatric patients 6 years and older. Unlike Stelara, Selarsdi is not approved to treat Crohn's disease or ulcerative colitis.
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.
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.
U.S. sales of the reference product Stelara were close to $7 billion in 2023. Selarsdi is expected to be commercially available in early 2025.
Selarsdi is the second FDA-approved Stelara biosimilar, following the approval of Wezlana (ustekinumab-auub) in 2023.

Oral Antibacterial Pivya Approved to Treat Uncomplicated UTIs

This past month the FDA cleared oral Pivya (pivmecillinam), a penicillin class antibacterial for use in the treatment of uncomplicated urinary tract infections (UTIs) in females 18 years and older. Pivya is indicated to treat susceptible isolates of Escherichia coli, Proteus mirabilis and Staphylococcus saprophyticus.

Uncomplicated UTIs are bacterial infections of the bladder in females with no structural abnormalities of their urinary tract. About 50% of all women experience at least one UTI in their lifetime.
Approval was based on 3 clinical trials comparing different Pivya dosing regimens to placebo, to another oral antibacterial drug and to the antiinflammatory ibuprofen. Efficacy was based on composite response rate (clinical cure and microbiological response) and was assessed 8 to 14 days after patients were enrolled. The composite response was achieved in 62% of those receiving Pivya vs. 10% placebo, in 72% of Pivya vs. 76% on comparator antibacterial, and in 66% of Pivya recipients vs. 22% who received ibuprofen.
The recommended dosage of Pivya is one 185 mg tablet by mouth 3 times a day for 3 to 7 days as clinically indicated, with or without food.
Do not use in patients with serious hypersensitivity to the drug or other beta-lactams, primary or secondary carnitine deficiency or other inborn errors of metabolism, or acute porphyria.
Warnings include allergic reactions, severe skin adverse reactions, carnitine depletion, Clostridioides difficile-associated diarrhea and interference with a newborn screening test for isovaleric acidemia, a rare metabolic disorder. The most common adverse reactions observed in ≥2% of the patients were nausea (4.3%) and diarrhea (2.1%).
Pivya is manufactured by Utility Therapeutics Ltd.

FDA Approves Zevtera, the Latest Cephalosporin Antibiotic

The FDA has approved Basilea Pharmaceutica’s Zevtera (ceftobiprole medocaril sodium for injection) for the treatment of adults with Staphylococcus aureus bloodstream infections (bacteremia) (SAB), including those with right-sided infective endocarditis; adults with acute bacterial skin and skin structure infections (ABSSSI); and adult and pediatric patients (3 months to less than 18 years old) with community-acquired bacterial pneumonia (CABP).

Zevtera is an advanced generation cephalosporin antibiotic for intravenous (IV) administration with rapid activity against a wide range of Gram-positive bacteria, such as Staphylococcus aureus, including methicillin-resistant strains (MRSA), and Gram-negative bacteria. It is given as an IV infusion over 2 hours.
Approval was based on the Phase 3 studies ERADICATE (SAB) and TARGET (ABSSSI) and a phase 3 study in CABP. In ERADICATE, the overall success rate for SAB was 69.8% (132/189) in adults treated with Zevtara and 68.7% (136/198) in patients treated with the comparator antibacterial (daptomycin ± aztreonam).
Warnings include increased mortality with unapproved use in Ventilator-Associated Bacterial Pneumonia (VABP), allergic reactions, seizures and other central nervous system (CNS) side effects and Clostridioides difficile-associated diarrhea (CDAD).
Common adverse reactions among all uses include nausea, vomiting, diarrhea, increased liver enzymes, headache, rash, injection site reaction and fever, among others.

FDA Approves Lumisight Imaging Agent to Detect Breast Cancer Post-Lumpectomy

This past month, the FDA approved Lumicell, Inc.’s Lumisight (pegulicianine), an optical imaging agent for the detection of residual cancerous tissue during breast cancer surgery.

Lumisight is used with the Lumicell Direct Visualization System (DVS) fluorescence imaging device to detect any residual cancerous tissue within the lumpectomy cavity following removal of the primary tumor, potentially sparing some patients from a second surgery due to incomplete tumor removal.
Pegulicianine is an inactive prodrug that works to light up any residual cancerous tissue by producing a fluorescent signal after its peptide chain is cleaved by certain enzymes around the tumor.
Lumisight is administered by intravenous (IV) injection over 3 minutes and is given 2 to 6 hours prior to imaging.
Lumisight carries a Boxed Warning, the FDA’s most stringent safety warning, for serious allergic reactions, including life-threatening anaphylaxis. Warnings and precautions include a risk of misdiagnosis and interference from dyes used for sentinel lymph node mapping.
Common side effects (≥1%) include hypersensitivity and chromaturia (abnormal urine color).

FDA Approves Xolremdi to Treat Patients 12 and Older with WHIM Syndrome

At the end of April, the FDA approved oral Xolremdi (mavorixafor) for use in patients 12 years and older with WHIM (warts, hypogammaglobulinemia, infections and myelokathexis) syndrome to increase the number of circulating mature neutrophils and lymphocytes, types of white blood cells that help fight infection.

WHIM syndrome is a rare, primary immunodeficiency and chronic neutropenic disorder caused by CXC chemokine receptor 4 (CXCR4) pathway dysfunction. People with WHIM syndrome typically have low blood levels of neutrophils and lymphocytes and experience serious or frequent infections.
Xolremdi is a CXCR4 antagonist and works by increasing mobilization and trafficking of white blood cells from the bone marrow into the bloodstream so that the body can better fight infections.
FDA approval was based on the 52-week 4WHIM Phase 3 study which showed an approximate 40% reduction in total infection score and a 60% reduction in the annualized infection rate in Xolremdi-treated patients compared to the placebo group. In addition, improvements in absolute neutrophil counts and absolute lymphocyte counts were above threshold times and statistically significant compared to placebo. Over the 52-weeks, there was no difference in total war change scores between the Xolremdi and placebo treatment arms.
Xolremdi capsules are administered orally, once daily on an empty stomach after an overnight fast, and at least 30 minutes before food.
Warnings and precautions for Xolremdi include fetal harm and QTc interval prolongation (an abnormal heart rhythm). Common side effects include thrombocytopenia (low platelet levels), skin rash, rhinitis (runny nose, common cold symptoms), epistaxis (nose bleed), vomiting, and dizziness.

Posted April 2024

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