Monthly News Roundup - March 2025
Medically reviewed by Drugs.com. Last updated on March 30, 2025.
FDA Approves Tremfya for Adults Patients with Active Crohn’s Disease
In March, Johnson & Johnson announced the U.S. Food and Drug Administration (FDA) approval of Tremfya (guselkumab) for the treatment of adults with moderately to severely active Crohn’s disease (CD). It is the first IL-23 inhibitor offering both subcutaneous (SC) and intravenous (IV) induction options, allowing the flexibility of patient SC self-administration from the start of treatment.
Crohn’s disease is a long-term (chronic) inflammatory bowel disease with disruptive stomach symptoms such as abdominal pain and tenderness, frequent bowel movements, rectal bleeding, weight loss and bowel urgency. Crohn's disease typically begins between ages 15 and 40, and there is no cure at this time. Tremfya is an interleukin-23 blocker (IL-23) and CD64 (a receptor on cells that produce IL-23) blocker also approved for the treatment of plaque psoriasis, psoriatic arthritis, and ulcerative colitis. IL-23 is a cytokine (protein) secreted by activated white blood cells and immune cells known to drive immune-mediated diseases including CD. Approval was based on data from the placebo-controlled Phase 3 GRAVITI and GALAXI studies in over 1,300 patients with moderately to severely active CD who failed or were intolerant to conventional therapy (i.e. corticosteroids or immunomodulators) or biologics. Pooled data from the GALAXI clinical program showed Tremfya was superior to Stelara in all pooled endoscopic endpoints. The recommended subcutaneous (SC) induction dosage for CD is 400 mg at Weeks 0, 4 and 8. For the IV induction option, 200 mg IV infusions are administered at Weeks 0, 4 and 8. The recommended maintenance dosage is 100 mg administered by SC injection at Week 16, and every 8 weeks thereafter, or 200 mg administered by SC injection at Week 12, and every 4 weeks thereafter. Warnings and precautions include: serious allergic reactions, infections, tuberculosis, liver toxicity, and to avoid use of live vaccines. Reported adverse reactions in Crohn’s Disease (≥3%) include: respiratory tract infections, abdominal pain, injection site reactions, headache, fatigue, arthralgia, diarrhea, and gastroenteritis. FDA Clears Amvuttra to Reduce Heart-Related Death in ATTR-CM
In March the FDA approved Alnylam’s Amvuttra (vutrisiran), an RNAi therapeutic to treat cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce heart-related death, hospital stays and urgent visits. ATTR-CM often leads to heart failure and premature death.
Transthyretin amyloidosis (ATTR) is a progressive, debilitating and fatal disease caused by misfolded transthyretin (TTR) proteins, which accumulate as amyloid deposits in various parts of the body. This buildup of amyloid deposits can cause a variety of symptoms including shortness of breath, irregular heartbeat, fatigue, leg swelling and fainting. Amvuttra reduces the production of TTR in the liver, where most of it is made. There are two different forms of ATTR - hereditary ATTR (hATTR) which is caused by a TTR gene variant (an inherited gene variant passed down through families) and wild-type ATTR (wtATTR) which occurs without a TTR gene variant (caused by changes associated with aging). FDA approval for ATTR-CM was based on the HELIOS-B Phase 3 study with 654 adults randomized 1:1 to receive 25 mg of Amvuttra (n=326) subcutaneously once every 3 months, or matching placebo (n=328). Results showed statistical significance for Amvuttra compared to placebo on all 10 pre-specified primary and secondary endpoints. In the study, people were shown to live longer and have fewer heart-related hospital visits compared to placebo over 3 years. In the overall population, Amvuttra reduced the risk of all-cause mortality (ACM) and recurrent cardiovascular (CV) events by 28% during the double-blind treatment period of up to 36 months. Amvuttra is administered by a healthcare professional via subcutaneous injection (under the skin) every 3 months (4 doses per year). Warnings and precautions associated with Amvuttra include reduced serum vitamin A levels. Low vitamin A levels can affect vision. Supplementation at the recommended daily allowance (RDA) is recommended. Common adverse reactions (≥5%) include pain in the arms or legs, arthralgia (joint pain), dyspnea (shortness of breath), and decreased vitamin A levels. Amvuttra was previously approved in June 2022 to treat polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults. FDA Approves AstraZeneca’s Imfinzi for Treatment of Muscle Invasive Bladder Cancer
This past month the FDA approved a new use for Imfinzi (durvalumab) with gemcitabine and cisplatin as neoadjuvant treatment (before surgery), followed by single agent durvalumab as adjuvant treatment (after surgery) following radical cystectomy (surgical removal of bladder), for adults with muscle invasive bladder cancer (MIBC). MIBC is a type of bladder cancer that has grown into the muscle wall of the bladder.
Imfinzi (durvalumab) is a programmed death-ligand 1 (PD-L1) blocking antibody also approved to treat non-small cell lung cancer, small cell lung cancer, biliary tract cancer, hepatocellular (liver) carcinoma, and endometrial cancer. PD-L1 is an immune checkpoint ligand that can be expressed on both tumor cells and tumor-associated immune cells in the tumor microenvironment. By blocking the interaction between PD-L1 and PD-1, durvalumab works to activate the T-cells which then kill the tumor cells. Approval was based on the NIAGARA Phase 3 study that enrolled 1,063 patients eligible for radical cystectomy. Event-free survival (EFS) was the primary outcome, with overall survival (OS) as an additional efficacy measure. At a pre-specified interim analysis, the trial demonstrated a statistically significant improvement in EFS and OS. Median EFS was not reached (NR) (95% CI: NR, NR) in the durvalumab with chemotherapy arm and 46.1 months (95% CI: 32.2, NR) in the chemotherapy arm (hazard ratio 0.68 [95% CI: 0.56, 0.82]; two-sided p-value <0.0001). Median OS was not reached in either arm (hazard ratio 0.75 [95% CI: 0.59, 0.93]; two-sided p-value=0.0106). Dosing for MIBC is based on weight with Imfinzi given as an intravenous (IV) infusion every 3 weeks with chemotherapy (neoadjuvant treatment) and then every 4 weeks as a single agent (adjuvant treatment). Treatment should continue until disease progression that precludes definitive surgery, recurrence, or unacceptable toxicity or a maximum of 8 cycles after surgery. Warnings and precautions include immune-mediated adverse reactions, infusion-related reactions, complications of allogeneic HSCT and embryo-fetal toxicity. Adverse reactions (≥ 20%) were consistent with prior experience with Imfinzi used with platinum-based chemotherapy and may include side effects such as nausea, fatigue/asthenia and alopecia. FDA Approves GSK’s First-in-Class Blujepa to Treat Uncomplicated Urinary Tract Infections
In March, the FDA approved Blujepa (gepotidacin) used for the treatment of uncomplicated urinary tract infections (uUTIs) in females 12 years and older weighing at least 40 kilograms (kg). Blujepa is a first-in-class oral antibiotic with a novel mechanism of action, targeting two essential topoisomerase enzymes. Blujepa treats uUTIs caused by Escherichia coli, Klebsiella pneumoniae, Citrobacter freundii complex, Staphylococcus saprophyticus, and Enterococcus faecalis.
Blujepa contains gepotidacin, a first-in-class triazaacenaphthylene antibiotic that blocks bacterial DNA replication through the inhibition of type II topoisomerase enzymes (bacterial topoisomerase II [DNA gyrase] and topoisomerase IV). An uncomplicated urinary tract infection is a bacterial infection of the lower urinary tract, including the bladder, urethra and associated structures. It occurs in patients without comorbidities such as a fever, catheter in place, diabetes, immune compromised, recent urologic surgery, or pregnancy. Common symptoms include pain or burning during urination (dysuria), frequent urination, a strong urge to urinate, and possibly blood in the urine. FDA approval of Blujepa was based on positive results from the pivotal Phase 3 EAGLE-2 and EAGLE-3 trials which demonstrated non-inferiority to nitrofurantoin, with therapeutic success of 50.6% and 58.5% (Blujepa) compared to 47% and 43.6% (nitrofurantoin), respectively. Blujepa tablets are taken by mouth, twice daily (every 12 hours) after a meal, for 5 days. Warnings and precautions include QTc prolongation (avoid use in patients with a history of QTc prolongation and patients receiving drugs that prolong the QTc interval), acetylcholinesterase inhibition (monitor patients with underlying medical conditions), hypersensitivity reactions including anaphylaxis, and Clostridioides difficile infection. Blujepa should be avoided with strong CYP3A4 inducers, strong CYP3A4 inhibitors and drugs that are extensively metabolized by CYP3A4 and have a narrow therapeutic window. Common adverse reactions (≥1% of patients) include diarrhea, nausea, abdominal (stomach) pain, flatulence (gas), headache, soft feces, dizziness, vomiting, and vaginal yeast (Candida) infections. FDA Approves Celltrion’s Omlyclo, an Interchangeable Biosimilar to Xolair
In March, the FDA cleared Celltrion’s Omlyclo (omalizumab-igec), an anti-IgE antibody, as the first interchangeable biosimilar to Xolair (omalizumab). Omlyclo is used to treat moderate to severe persistent asthma, chronic rhinosinusitis with nasal polyps (CRSwNP), Immunoglobulin E (IgE)-mediated food allergy, and chronic spontaneous urticaria (CSU).
An interchangeable biosimilar can automatically be substituted for the reference product (in this case, Xolair) by a pharmacist, depending upon state laws. The pharmacist will not need to contact the doctor to get an approval. It also means the reference biologic and the new biosimilar can be switched back and forth in a patient without a risk of changes in safety or effectiveness. Interchangeable products should lead to healthcare savings, be more likely to be covered by insurance, and make it easier for patients to access a biologic medicine. Approval for Omlyclo was based on a Phase 3 clinical trial involving 619 adult patients with CSU up to Week 40. The results demonstrated comparable efficacy and safety of Omlyclo to reference product Xolair during both treatment and off-dose periods. Omlyclo is administered by subcutaneous injection every 2 or 4 weeks (every 4 weeks for chronic spontaneous urticaria). Omlyclo carries a Boxed Warning for anaphylaxis and must be given initially in a healthcare setting. Patients must be closely observed for an appropriate period of time after administration. Common adverse reactions vary among indications and include: arthralgia (joint pain), abdominal pain, common cold symptoms (nasopharyngitis), dizziness, headache, fever, injection site reactions, and upper abdominal pain, among others. FDA Approves Shelf-Stable Arbli (losartan potassium) Oral Suspension
In March, the FDA approved Arbli (losartan potassium) from Scienture Holdings, Inc, is an oral liquid formulation of the angiotensin II receptor blocker (ARB) losartan. It is the first ready-to-use liquid, oral losartan in the U.S.
Arbli is approved for the treatment of hypertension (high blood pressure) in patients greater than 6 years old, for the reduction of risk of stroke in patients with hypertension and left ventricular hypertrophy (enlarged heart) and for the treatment of diabetic nephropathy (kidney disease) in certain patients with type 2 diabetes. Losartan is an angiotensin II receptor blocker (ARB) that works by blocking the effect of angiotensin II (a potent vasoconstrictor) to dilate the blood vessels and lower blood pressure. Losartan was first approved in a tablet formulation under the brand name Cozaar in 1995, and is widely available as a generic. Arbli is administered orally once daily. It has a long-term shelf life at room temperature storage. It comes in a 165 mL bottle as a peppermint flavored suspension that does not require refrigeration with a shelf life of 18 months. Arbli comes with a Boxed Warning for fetal toxicity. Drugs that act directly on the renin-angiotensin system can cause injury and death to the developing fetus. Warnings and precautions associated with Arbli include hypotension. Common adverse reactions (incidence ≥2% and greater than placebo) include dizziness, upper respiratory infection, nasal congestion, and back pain. Arbli is expected to be commercially available in the 3rd quarter of 2025. FDA Approves Encelto to Treat Macular Telangiectasia Type 2, a Retinal Eye Disease
In March, Neurotech Pharmaceuticals, Inc. announced the approval of Encelto (revakinagene taroretcel-lwey) ocular implant (for intravitreal use) in adults for the treatment of Macular Telangiectasia type 2 (MacTel), a neurodegenerative disease of the retina. Encelto is the first FDA-approved treatment for MacTel.
MacTel causes progressive and irreversible vision loss. Encelto is an allogeneic encapsulated cell-based gene therapy designed to continually deliver therapeutic doses of ciliary neurotrophic factor (CNTF) to the retina to help slow disease progression and preserve vision. CNTF is a protein that supports the growth of nerve cells. Approval was based on two Phase 3 trials which demonstrated that after placement of the implant, Encelto significantly slowed the loss of macular photoreceptors in MacTel patients over 24 months. Encelto is intended for surgical intravitreal implantation by a qualified ophthalmologist. The recommended dose is one Encelto implant per affected eye containing 200,000 to 440,000 allogeneic retinal pigment epithelial cells expressing recombinant human ciliary neurotropic factor (rhCNTF). It is performed as an outpatient surgical procedure. Do not use it in patients with suspected or known eye infections or known hypersensitivity to Endothelial Serum Free Media (Endo-SFM). Warnings and precautions include severe vision loss, infectious endophthalmitis, retinal tears and/or detachment, vitreous hemorrhage, implant extrusion, cataract formation, suture related complications, and delayed dark adaptation. The most common adverse reactions (incidence ≥2%) were conjunctival hemorrhage, delayed dark adaptation, foreign body sensation, eye pain, suture related complications, miosis, and conjunctival hyperemia, among others. Encelto is expected to be available commercially in June of 2025. FDA Approves Vykat XR (diazoxide choline) to Treat Hyperphagia in Prader-Willi Syndrome
The FDA has approved Vykat XR (diazoxide choline) for use in the treatment of hyperphagia (feelings of intense, persistent hunger) in patients with Prader-Willi syndrome (PWS). Vykat XR extended-release tablets are administered orally once daily.
Prader-Willi syndrome (PWS) is a rare genetic disorder characterized by hyperphagia, behavioral problems, cognitive disabilities, low muscle tone, short stature (when not treated with growth hormone), accumulation of excess body fat, developmental delays, and incomplete sexual development. Vykat XR is thought to work in the treatment of hyperphagia in PWS patients through activation of the adenosine triphosphate-sensitive potassium channels in the hypothalamus. Approval was based on data from the Phase 3 Study 2-RWP trial. During the 16-week randomized withdrawal study period, individuals randomized to switch to placebo demonstrated a statistically significant worsening of hyperphagia (feelings of intense, persistent hunger) compared with individuals who remained on Vykat XR. Warnings and precautions include hyperglycemia and risk of fluid overload. Common adverse reactions (incidence ≥10% and at least 2% greater than in placebo) include hypertrichosis (excessive, abnormal hair growth), edema (fluid retention), hyperglycemia (high blood glucose), and rash. Vykat XR is manufactured by Soleno Therapeutics, Inc. FDA Approves Sanofi’s Qfitlia to Treat Hemophilia A or B With or Without Inhibitors
The FDA has cleared Qfitlia (fitusiran), an antithrombin-directed small interfering ribonucleic acid for the prophylactic treatment of people with hemophilia A or B, with or without inhibitors. Qfitlia is indicated for prevention or to reduce the frequency of bleeding episodes in patients aged 12 years and older with hemophilia A or B with or without factor VIII or IX inhibitors.
Hemophilia is a family of rare genetic blood diseases caused by a clotting factor deficiency (factor VIII in hemophilia A and factor IX in hemophilia B) which prevents normal blood clotting, resulting in episodes of bleeding. Qfitlia is a small interfering RNA (siRNA) therapy and the first antithrombin-lowering therapy for use in hemophilia. Qfitlia does not replace the missing clotting factor. Instead, it reduces the amount of a protein called antithrombin, leading to an increase in thrombin, an enzyme critical for blood clotting. FDA approval was based on data from the ATLAS phase 3 studies that demonstrated clinically meaningful bleed protection as measured by annualized bleeding rates (ABR) across hemophilia patients with or without inhibitors. Key results include: Qfitlia is given by subcutaneous injection, starting at once every 2 months. The dose or frequency of administration can be adjusted to maintain antithrombin activity between 15% to 35%. Labeling contains a Boxed Warning for thrombotic events (blood clotting) and gallbladder disease (with some patients requiring gallbladder removal). Qfitlia also has a warning about liver toxicity and the need to monitor liver blood tests at baseline and then monthly for at least 6 months after initiating treatment with Qfitlia or after a dose increase. The most common side effects of Qfitlia are viral infection, common cold symptoms (nasopharyngitis) and bacterial infection.
Posted : 2025-03-31 12:00
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