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Monthly News Roundup - November 2024

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

Bimzelx Indications Expanded to Include Hidradenitis Suppurativa, a Debilitating Skin Condition

UCB’s Bimzelx (bimekizumab-bkzx) has been granted a new use for the treatment of adults with moderate to severe hidradenitis suppurativa (HS). Bimzelx, is a humanized interleukin-17A and interleukin-17F antagonist, has been previously approved for the treatment of plaque psoriasis, psoriatic arthritis, non-radiographic axial spondyloarthritis, and ankylosing spondylitis.

• Hidradenitis suppurativa is a chronic (long-lasting) and debilitating inflammatory skin disease that can greatly impact quality of life and may affect 1 out of every 100 people. It is characterized by swollen, painful bumps (nodules), fistulas and abscesses typically located around the armpits, groin, inner thighs, on the buttocks and under the breasts. The exact cause of hidradenitis suppurativa is unknown, but it is thought that the immune system plays a role in the inflammatory symptoms.
• Bimzelx is given as a subcutaneous injection that can be self-injected by the patient under the skin every 2 weeks for the first 16 weeks, then every 4 weeks thereafter.
• Approval was supported by the BE HEARD I and BE HEARD II, Phase 3, multicenter studies in the treatment of 1,014 adults (mean age 37 years) with moderate to severe HS. Results showed that a higher proportion of patients treated with Bimzelx vs. placebo (an inactive agent) achieved a 50% or greater improvement in HS signs and symptoms (total abscess and inflammatory nodule count, with no increase from baseline in abscess or draining tunnel count) at Week 16, as measured by Hidradenitis Suppurativa Clinical Response 50 (HiSCR50), the primary endpoint in both trials. Efficacy results were 48% Bimzelx vs. 29% placebo in trial 1 and 52% Bimzelx vs. 32% placebo in trial 2. Clinical responses were sustained to Week 48.
• Warnings and precautions include suicidal ideation and behavior, infection risk, possible liver injury, and inflammatory bowel disease. Bimzelx should be avoided in patients with active tuberculosis (TB) infection. Avoid live vaccines in patients receiving Bimzelx.
• Common side effects in HS studies include upper respiratory tract infections, oral candidiasis (oral thrush), headache, injection site reactions, tinea (fungal) infections, gastroenteritis, herpes simplex infections, acne, folliculitis (inflamed/infected hair follicles, other candida (yeast) infections, and fatigue.
• Evaluate patients for tuberculosis (TB) infection prior to initiating treatment with Bimzelx. Complete all age-appropriate vaccinations before starting Bimzelx.

FDA Approves Oral Emrosi for the Treatment of Rosacea in Adults

In November, the FDA cleared Emrosi capsule (minocycline hydrochloride), a low-dose, modified-release formulation indicated to treat the inflammatory lesions (papules and pustules) of rosacea in adults. Minocycline is in the tetracycline class of antibiotics and has been approved since 1971.

• Rosacea is a chronic, relapsing, inflammatory skin condition with symptoms such as deep facial redness, acne-like inflammatory lesions (papules and pustules) and spider veins (telangiectasia).
• Emrosi gained FDA approval on Nov. 1, 2024 and is manufactured by Journey Medical.
• Emrosi relieves inflammatory lesions caused by rosacea, such as pimples and bumps. The mechanism of action may be associated with reducing bacterial by-products that contribute to inflammation, promoting skin barrier repair.
• Approval is supported by data from two 16-week, Phase 3 clinical trials which demonstrated a reduction in total inflammatory lesion count and a statistically significant effect over both the current standard treatment, doxycycline, as well as placebo. Participants completed the study with no significant safety issues.
• The recommended dosage of Emrosi for rosacea is 40 mg by mouth once daily.
• Warnings include anaphylaxis (a severe allergic reaction), serious skin reactions (Stevens-Johnson syndrome), erythema multiforme and drug rash with eosinophilia and systemic symptoms (DRESS) syndrome.
• The most common adverse reaction reported by at least 1% of subjects was dyspepsia (indigestion, heartburn).

FDA Approves Danziten Tablets, the First Nilotinib With No Mealtime Restrictions

Azurity Pharmaceuticals has announced the approval of Danziten (nilotinib), a targeted kinase inhibitor used to treat Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML) in adults. Danziten is equivalent in effectiveness to Tasigna (nilotinib), but the re-engineered formulation means patients do not need to take their dose in a fasting state (on an empty stomach).

• Danziten is indicated for adults with newly diagnosed Philadelphia chromosome positive chronic myeloid leukemia (Ph+ CML) in chronic phase, and adults with chronic phase (CP) and accelerated phase (AP) Ph+ CML resistant to or intolerant to prior therapy that included imatinib (Gleevec).
• Danziten works by targeting BCR-ABL tyrosine kinase, a protein that promotes cancer growth and survival. It triggers cell death (apoptosis) and is particularly effective against cells with the Philadelphia chromosome. It is typically used when other treatments are not effective or not tolerated.
• The blood levels of Tasigna can increase when taken with food, which may significantly prolong QT interval on electrocardiogram (ECG), which signals an abnormal heart rhythm. As such, strict fasting with Tasigna is crucial to avoid cardiotoxicity. The new product Danziten allows for a lower dose and has the potential to improve adherence due to the removal of fasting requirements. However, the Danziten product label still carries a Boxed Warning for QT interval prolongation and sudden death.
• Danziten tablets are taken orally, twice daily. Danziten has different strengths and dosages compared to Tasigna and may not be substitutable with other nilotinib products on a milligram per milligram basis.
• Danziten should not be given to patients with hypokalemia (low potassium), hypomagnesemia (low magnesium), or long QT syndrome (an irregular heart rhythm). Avoid use of Danziten with drugs known to prolong the QT interval and with strong CYP3A4 inhibitors.
• In addition, warnings and precautions include substitution with other nilotinib products and risk of medication errors, bone marrow suppression, cardiac and arterial vascular occlusive events, and pancreatitis and elevated serum lipase, among others.
• Common adverse reactions in adult at least 20% of adult patients include nausea, rash, headache, fatigue, pruritus (itching), vomiting, diarrhea, cough, constipation, arthralgia (joint pain), nasopharyngitis (common cold), pyrexia (fever), and night sweats. Bone marrow suppression, low platelets, and low red and white blood cells have also been reported.

FDA Approves Aucatzyl to Treat Acute Lymphoblastic Leukemia, an Aggressive Blood Cancer

This past month, the FDA cleared Aucatzyl (obecabtagene autoleucel or “obe-cel”), a CD19-directed genetically modified autologous T cell immunotherapy (CAR-T cell therapy) from Autolus Therapeutics. Aucatzyl is approved to treat adults with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (R/R B-ALL).

• Acute lymphoblastic leukemia is an aggressive type of blood cancer that may also involve the lymph nodes, spleen, liver, central nervous system and other organs.
• Aucatzyl is made by genetically modifying a person’s white blood cells to recognize and attack leukemia cells. It is given as a split-dose intravenous (IV) infusion following a chemotherapy regimen that lowers a type of white blood cell in your bone marrow.
• Approval was based on the FELIX clinical trial of obe-cel in adults with R/R B-ALL. The major efficacy outcome was complete remission within 3 months and was achieved in 42% of patients with a median duration of remission (DOR) of 14.1 months. Remission means there are no signs or symptoms of cancer, or the cancer is not growing due to treatment, but does not necessarily mean that the cancer is cured.
• Aucatzyl carries a Boxed Warning for cytokine release syndrome (CRS), immune effector cell-associated neurotoxicity syndrome (ICANS) and secondary hematological malignancies. In studies, it showed low levels of CRS, with 3% Grade 3 events, and no Grade 4 or 5 events. Grade ≥ 3 ICANS was reported in 7% of patients. It is the first CAR T therapy approved by the FDA with no requirement for a Risk Evaluation Mitigation Strategy (REMS) program.
• Warnings and precautions include prolonged cytopenias (low red or white blood cells or platelets), infections, low gamma globulin (antibodies that fight infection), Hemophagocytic Lymphohistiocytosis/Macrophage Activation Syndrome (extreme inflammatory response from the immune system), secondary malignancies (cancers), and serious hypersensitivity (allergic) reactions, among others.
• Common adverse reactions (in at least 20% of patients) include CRS, infections, musculoskeletal pain, viral infections, fever, nausea, bacterial infectious disorders, diarrhea, febrile neutropenia (low white blood cell counts with fever), ICANS, hypotension (low blood pressure), pain, fatigue, headache, encephalopathy (brain changes), and hemorrhage (bleeding).

Kebilidi is First Gene Therapy Approved for Aromatic L-amino Acid Decarboxylase (AADC) Deficiency

Kebilidi (eladocagene exuparvovec-tneq), from PTC Therapeutics, is a gene replacement therapy for use in the treatment of aromatic L-amino acid decarboxylase (AADC) deficiency in adults and children. Kebilidi was approved under the accelerated approval designation. It is the first US-approved gene therapy for treatment of AADC deficiency and the first administered directly into the brain.

• AADC deficiency is a rare, fatal genetic disorder that affects dopamine neurotransmitter production. Neurotransmitters are chemical messengers that allow cells in the body’s nervous system to communicate with each other. Symptoms of AADC include delays in gross motor function (head control, sitting, standing, and walking), hypotonia (weak muscle tone), and developmental and cognitive skills (thinking, learning, remembering) delays.
• Kebilidi contains the human dopa decarboxylase (DDC) gene and is designed to correct the underlying genetic defect by delivering a functioning DDC gene directly into the putamen of the forebrain, increasing the AADC enzyme and restoring dopamine production.
• Kebilidi is given as 4 infusions into the brain during a single surgical procedure with a qualified medical center and neurosurgeon that specializes in pediatric stereotactic neurosurgery. The procedure is a minimally invasive technique that uses imaging and special equipment to deliver therapies to specific areas in the brain. A child's skull must have reached maturity (via neuroimaging) before Kebilidi can be administered.
• Accelerated approval of Kebilidi was based on an open-label, single-arm clinical study in 13 pediatric patients. Motor milestone assessments were completed at week 48 for 12 of the 13 patients. Efficacy showed gross motor function improvement in 8 of 12 treated patients, which has not been reported in untreated patients with the severe presentation of AADC deficiency.
• Common side effects include involuntary muscle movements like shakes, tics, or tremors (dyskinesia), fever, low blood pressure (hypotension), anemia (low red blood cell count), increased saliva production, insomnia (trouble sleeping), low potassium, phosphate, and magnesium levels and complications during the procedure such as respiratory and cardiac arrest.
• Continued FDA-approval of Kebilidi may depend upon ongoing study results that show a positive clinical benefit at 48 weeks in gross motor function.

FDA Approves First-in-Class Oral Revuforj to Treat Acute Leukemia with a KMT2A Translocation

Revuforj (revumenib), from Syndax Pharmaceuticals, is a first-in-class, oral menin inhibitor now approved for the treatment of relapsed or refractory (R/R) acute leukemia with a lysine methyltransferase 2A gene (KMT2A) translocation in adult and pediatric patients 1 year of age and older. Relapsed or refractory means the disease has come back or has not improved after previous treatment.

• Acute leukemia is a type of blood cancer with immature white blood cells that multiply rapidly in the bone marrow. Over time, these white blood cells crowd out healthy blood cells. Rearrangements of the KMT2A gene (KMT2Ar) occur in about 10% of acute leukemia cases and give rise to an aggressive form of acute leukemia that is associated with a very poor prognosis and high relapse rates.
• KMT2A translocation is a type of gene rearrangement that occurs when part of one chromosome breaks and fuses to a different chromosome. As a menin inhibitor, Revuforj blocks the interaction of both wild-type lysine methyltransferase 2A (KMT2A) and KMT2A fusion proteins with menin to help slow growth and kill cancer cells.
• Approval was based on the results from the Phase 1/2 AUGMENT-101 trial with 104 patients with R/R acute leukemia with a KMT2A translocation. The rate of complete remission (CR) plus CR with partial hematological recovery (CRh) was 21%, the median duration of CR+CRh was 6.4 months, and the median time to CR or CRh was 1.9 months. Twenty-three percent (24/104 pts) of patients were able to undergo hematopoietic stem cell transplantation (HSCT) following treatment with Revuforj.
• Revuforj tablets are given orally twice daily on an empty stomach (fasted) or with a low-fat meal at about the same time each day. Dosage varies by patient weight and use of other medicines (like strong CYP3A4 inhibitors). Treatment is continued until disease progression or unacceptable toxicity, for a minimum of 6 months when possible.
• Labeling carries a Boxed Warning for differentiation syndrome, which can be fatal. Symptoms of differentiation syndrome include cough, fluid build-up, kidney failure, low blood pressure, shortness of breath, and fever among others. Warnings and precautions include QTc interval prolongation (abnormal heart rhythm) and harm to an unborn baby.
• Common adverse reactions (reported in at least 20% of patients) included hemorrhage (bleeding), nausea, muscle pain, infection, febrile neutropenia (low white blood cell counts/fever), bacterial infection, diarrhea, laboratory value changes and abnormal heart rhythm, among others.

FDA Grants Accelerated Approval to Ziihera to Treat HER2-Positive Biliary Tract Cancer

This past month the FDA cleared Ziihera (zanidatamab-hrii), a targeted bispecific HER2-directed antibody for the treatment of adults with previously treated, unresectable or metastatic HER2-positive (IHC 3+) biliary tract cancer (BTC), as detected by an FDA-approved test. BTC is a type of bile duct (cholangiocarcinoma) or gallbladder cancer with a poor prognosis and a 5-year survival rate under 5% if the cancer has spread in the body.

• Ziihera works by binding to two extracellular HER2 sites to reduce receptors on the tumor cell surface. This leads to a slowing of tumor growth and tumor cell death via complement-dependent cytotoxicity, antibody-dependent cellular cytotoxicity and antibody-dependent cellular phagocytosis.
• Ziihera is given as an intravenous (into a vein) infusion once every 2 weeks and is used when BTC has been previously treated, cannot be removed by surgery or has spread to other parts of the body (metastatic). Premedications to help prevent infusion-related reactions are recommended.
• Ziihera received FDA-accelerated approval based on the open-label, single arm HERIZON-BTC-01 study with 62 patients (cohort 1) with unresectable or metastatic disease. Patients were required to have received at least one prior gemcitabine-containing systemic chemotherapy regimen in the advanced disease setting and adequate cardiac function (defined as LVEF of at least 50%). Efficacy showed a 52% objective response rate (ORR) and a Kaplan Meier (KM) estimated median duration of response (DOR) of 14.9 months (7.4, NE).
• Ziihera carries a Boxed Warning for harm to an unborn baby. Warnings and precautions include left ventricular dysfunction (weak heart that cannot properly pump blood), infusion-related reactions, and severe diarrhea.
• Common adverse reactions include diarrhea (50%), infusion-related reactions (35%), abdominal (stomach-area) pain (29%), tiredness (24%), rash (19%) and reduced appetite (16%). Administer antidiarrheal treatment as clinically indicated.
• Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial. The Phase 3 HERIZON-BTC-302 confirmatory trial is ongoing to evaluate zanidatamab in combination with standard-of-care therapy versus standard-of-care therapy alone in the first-line setting for patients with HER2-positive BTC.
• Ziihera is manufactured by Jazz Pharmaceuticals.

Attruby Cleared for Patients with ATTR-CM, a Possibly Fatal Heart Muscle Disease

FDA has approved Attruby (acoramidis), an oral transthyretin stabilizer indicated for the treatment of the cardiomyopathy of wild-type or variant transthyretin-mediated amyloidosis in adults to reduce cardiovascular death and cardiovascular-related hospitalization. Attruby is the first approved product with a label specifying near-complete (at least 90%) stabilization of TTR.

• Cardiomyopathy of transthyretin-mediated amyloidosis (ATTR-CM) is a potentially fatal disease of the heart muscle characterized by accumulation of amyloid deposits, leading to an enlarged, stiffened heart (cardiomyopathy) and heart failure. Amyloid is an insoluble protein that can disrupt normal organ function.
• Acoramidis is a selective stabilizer of transthyretin (TTR). Attruby was designed to mimic a naturally-occurring variant of the TTR gene which provides instructions for making a protein called transthyretin (TTR). When TTR is destabilized (usually due to genetics or aging), it can build up to form abnormal amyloid deposits. By stabilizing TTR, Attruby has been shown to preserve the native function of TTR as a transport protein of thyroxine and vitamin A, and to demonstrate benefit on cardiovascular outcomes.
• FDA approval was based on positive results seen in the ATTRibute-CM Phase 3 study with 611 participants (mean age 77 years) with symptomatic ATTR-CM. Participants were randomized 2:1 to receive Attruby or placebo. The trial met the primary endpoint which included all-cause mortality (ACM, 19% Attruby vs 26% placebo) and cumulative frequency of cardiovascular-related hospitalizations (CVH, 27% vs. 43% placebo) over 30 months. Attruby demonstrated significant reductions in death and cardiovascular-related hospitalization and improved quality of life.
• Attruby tablets are administered by mouth twice daily. Common side effects include diarrhea (11.6%) and upper abdominal pain (5.5%).
• It is manufactured by BridgeBio Pharma, Inc.

FDA Approves Imkeldi, an Imatinib Oral Solution for Leukemia

Shorla Oncology has announced the approval of Imkeldi (imatinib mesylate), an 80 mg/mL oral solution of the approved imatinib tablet (Gleevec). It is used for the treatment of certain forms of leukemia and other types of blood cancers, as well as immune system disorders, skin cancer and certain tumors of the stomach and digestive system. Gleevec, the original brand of imatinib, is now available as a generic option.

• Imkeldi is a strawberry flavored oral solution that may be more convenient for patients who have difficulty swallowing tablets or who require specific dosing tailored to body surface area.
• Imkeldi solution is administered orally, once or twice a day (depending on the dose), with a meal and a large glass of water. Doses of 400 mg or 600 mg should be administered once daily, whereas a dose of 800 mg should be given as 400 mg twice a day. Use an accurate measuring device for doses (not a household teaspoon).
• Imatinab is a kinase inhibitor and inhibits tyrosine kinases associated with the BCR-ABL, PDGF (platelet-derived growth factor), SCF (stem cell factor) and the KIT protein. These enzymes are proteins that are involved in the development of blood cancers like chronic myelogenous leukemia (CML), acute lymphoblastic leukemia (ALL), certain skin cancers and some types of tumors of the stomach or intestine. Blocking these enzymes may help to slow cancer growth.
• Your doctor can have a blood test performed to detect the presence of the BCR-ABL gene, or other genes or mutations that may be fueling the cancer growth.
• Warnings and precautions include edema and severe fluid retention, low blood cells counts like red or white cells or platelets, congestive heart failure and left ventricular dysfunction, liver toxicity, kidney toxicity; hemorrhage (bleeding); gastrointestinal (digestive tract) perforations, heart has trouble pumping blood and oxygen, serious skin reactions (erythema multiforme and Stevens-Johnson syndrome), low thyroid levels, harm to an unborn baby, slowed growth in children, and tumor lysis syndrome.
• Common adverse reactions (in at least 30% of patients) include edema (fluid retention), nausea, vomiting, muscle cramps, muscle and bone pain, diarrhea, rash, fatigue (tiredness), and abdominal (stomach area) pain.

FDA Approves Rapiblyk for Supraventricular Tachycardia (SVT), an Abnormal Heart Rhythm

The FDA has approved Rapiblyk (landiolol), a short-term treatment for supraventricular tachycardia (SVT) including atrial fibrillation and atrial flutter to reduce the ventricular rate. Rapiblyk is a short-acting beta-adrenergic blocker that helps decrease heart rate (negative chronotropic effect) with less lowering of blood pressure. It is given as an intravenous (IV) infusion in a monitored setting.

• Supraventricular tachycardia (SVT) is a condition that causes your heart to beat much faster than it should. SVTs (including atrial fibrillation and atrial flutter) can occur both in patients with and without heart disease. They require immediate medical attention because they can impair heart function.
• Approval for Rapiblyk was based on data from 5 randomized, double-blind, placebo-controlled studies with 317 adults with SVT. A “heart rate decrease” was defined as a greater than 20% decrease in heart rate or a heart rate less than 100 beats per minute; or at least intermittent cessation of the arrhythmia. A heart rate decrease was seen in 40% to 90% of patients treated with Rapiblyk within about 10 minutes, compared to 0% to 11% of patients receiving placebo (an inactive injection).
• Warnings and precautions include a risk of hypotension (low blood pressure), bradycardia (slowed heart rate), and cardiac failure (heart cannot pump adequate blood), worsening of reactive airway (lung) disease, diabetes mellitus, and myocardial ischemia (when the heart muscle does not receive adequate blood flow and oxygen).
• The most common adverse reaction in 9.9% of patients was hypotension (low blood pressure).
• Rapiblyk is from AOP Orphan Pharmaceuticals GmbH (AOP Health).

Posted November 2024

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