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Monthly News Roundup - July 2025

Medically reviewed by Leigh Ann Anderson, PharmD. Last updated on July 31, 2025.

FDA Approves Ekterly for Oral On-Demand Treatment of Hereditary Angioedema

In July, KalVista Pharmaceuticals announced the approval of Ekterly (sebetralstat) for the on-demand treatment of hereditary angioedema (HAE) attacks in adults and children aged 12 years and older. Treatment guidelines recommend treating HAE attacks as early as possible to prevent further complications.

• Hereditary angioedema (HAE) is a genetic disease characterized by a deficiency or dysfunction in the C1 esterase inhibitor (C1INH) protein and subsequent uncontrolled activation of the kallikrein-kinin system. Symptoms of an HAE attack include painful and debilitating attacks of tissue swelling in the hands, feet, limbs, face, intestinal tract, and airways.
• Sebetralstat is an oral plasma kallikrein inhibitor and works by blocking kallikrein, an enzyme that plays a role in dilating blood vessels and increasing fluid leaking from capillaries. Blocking kallikrein reduces levels of bradykinin, the peptide that causes swelling during HAE attacks.
• Approval was based on data from the KONFIDENT clinical trials. Ekterly met the primary endpoint in the Phase 3 trial with both 300 mg and 600 mg formulations leading to initial symptom relief significantly faster than placebo. The median time to the start of symptom relief was 1.6 hours with Ekterly 300 mg, 1.8 hours with 600 mg and 6.7 hours with placebo.
• Ekterly comes as a tablet taken by mouth and is the first oral on-demand treatment for HAE. Other approved on-demand treatment options require either intravenous (IV) or subcutaneous (under the skin) administration.
• The tablets are administered orally at the earliest sign of an acute attack of HAE and may be repeated after at least 3 hours after the first dose if the symptoms return, worsen or do not improve.
• Avoid use of Ekterly in patients with severe hepatic impairment (Child-Pugh Class C). Common adverse reactions, reported in at least 2% of patients are headaches.

FDA Approves Steroid-Free Anzupgo Cream to Treat Chronic Hand Eczema

The FDA has cleared LEO Pharma’s Anzupgo (delgocitinib) for the topical treatment of moderate to severe chronic hand eczema (CHE) in adults who have not responded well to topical corticosteroids or who should not use them. It is the first U.S. treatment specifically approved for chronic hand eczema.

• Chronic hand eczema (CHE) lasts for more than 3 months or occurs twice or more within a year and is characterized by itchy, painful, blistered, or swollen skin. Some patients can also experience skin that is thickened or leathery with cracks on the hands and wrists. CHE affects approximately one in ten adults worldwide.
• Anzupgo is a steroid-free, topical pan-Janus kinase (JAK) inhibitor and works by blocking the activation of JAK-STAT signaling, which plays a key role in the pathogenesis of CHE.
• Approval was based on DELTA 1 and DELTA 2, two Phase 3, 16-week, vehicle-controlled studies in 960 adults. The primary efficacy endpoint was the proportion of subjects who achieved IGA-CHE treatment success (IGA-CHE TS) at Week 16, defined as a score of 0 (clear) or 1 (almost clear) with at least a 2-point improvement from baseline. Results showed that 20% and 29% who received topical Anzupgo cream achieved IGA-CHE TS, compared to 10% and 7% in the cream vehicle group, respectively (both trials p≤0.0055).
• Anzupgo cream is applied twice daily to the skin of the affected areas only on the hands and wrists. Patients should not use more than 30 grams per 2 weeks or 60 grams per month. Use of Anzupgo in combination with other JAK inhibitors or potent immunosuppressants is not recommended.
• Warnings and precautions associated with Anzupgo include an increased risk of infection, live vaccines, non-melanoma skin cancers and potential risks related to JAK Inhibition.
• Reported adverse reactions (≤1% of subjects) include application site pain, abnormal sensation of tingling or numbness, itching, redness, and bacterial skin infections including finger cellulitis, paronychia (skin infection around nails), other skin infections, and decreased white blood cells.

FDA Approves Zegfrovy for Non-Small Cell Lung Cancer (NSCLC) With EGFR Mutations

Zegfrovy (sunvozertinib) from Dizal has received accelerated approval for the treatment of adults with locally advanced (has spread to nearby tissue) or metastatic (has spread to distant parts of the body) non-small cell lung cancer (NSCLC), whose disease has progressed on or after platinum-based chemotherapy. Zegfrovy is indicated in NSCLC with EGFR exon 20 insertion (exon20ins) mutations, as detected by an FDA-approved test.

• Sunvozertinib is a targeted, oral, irreversible, epidermal growth factor receptor (EGFR) tyrosine kinase inhibitor (TKI). It blocks EGFR, a protein that promotes cancer growth. In laboratory clinical trials, it effectively stopped cancer cell growth and shrank tumor size in animal models with these specific mutations.
• FDA accelerated approval of Zegfrovy was supported by data from the WU-KONG1 Part B (WU-KONG1B) study. The primary endpoint was confirmed overall response rate (ORR) of 46% (95% CI: 35, 57). An additional efficacy outcome measure was duration of response (DOR) of 11.1 months (95% CI: 8.2, not evaluable).
• Zegfrovy is administered orally by mouth once daily with food until disease progression or unacceptable toxicity.
• Warnings and precautions associated with Zegfrovy include interstitial lung disease (ILD)/pneumonitis, gastrointestinal (stomach area) adverse reactions, dermatologic (skin) adverse reactions, ocular (eye) toxicity, and embryo-fetal toxicity.
• Common (≥20%) adverse reactions include diarrhea, rash, decreased appetite, stomatitis, fatigue, nausea, paronychia, vomiting, constipation, musculoskeletal pain, pruritus, dry skin, urinary tract infection, abdominal pain and decreased weight. Multiple common (≥2%) Grade 3 or 4 laboratory abnormalities were also reported. These are not all of the possible side effects of Zegfrovy.
• This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in additional clinical trials.

FDA Grants Accelerated Approval to Lynozyfic for Treatment of Multiple Myeloma

Regeneron’s Lynozyfic (linvoseltamab-gcpt) has received accelerated approval for the treatment of relapsed or refractory multiple myeloma in patients who have received at least four prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent and an anti‑CD38 monoclonal antibody.

• Lynozyfic is a bispecific B-cell maturation antigen (BCMA)‑directed CD3 T-cell engager. It’s designed to bridge B-cell maturation antigen (BCMA) on multiple myeloma cells with CD3-expressing T cells to facilitate T-cell activation, leading to cancer cell death.
• Multiple myeloma is a blood cancer characterized by the proliferation of cancerous plasma cells (multiple myeloma cells) that crowd out healthy blood cells in the bone marrow, infiltrate other tissues and cause potentially life-threatening organ injury.
• FDA accelerated approval of Lynozyfic was based on data in 80 patients from the Phase 1/2 LINKER-MM1 trial which demonstrated a 70% objective response rate (ORR), with 45% achieving a complete response (CR) or better, and a 0.95 month median time to first response (range: 0.5 to 6 months). The median duration of response (DoR) was not reached (95% Confidence Interval [CI]: 12 months to not estimable).
• Lynozyfic is administered by intravenous (IV) infusion according to a step-up dosing schedule (weekly, then every two-weeks at week 14, and then every four-weeks at week 24 depending on response). The regimen includes hospitalization for safety during the initial step-up dosing period.
• Lynozyfic carries a Boxed Warning for cytokine release syndrome (CRS) and neurologic toxicity including immune effector cell-associated neurotoxicity syndrome (ICANS). Lynozyfic is available only through a restricted program called the Lynozyfic Risk Evaluation and Mitigation Strategy (REMS).
• Warnings and precautions associated with Lynozyfic include serious infections, neutropenia (low white blood cells), hepatotoxicity (liver toxicity), and embryo-fetal toxicity.
• Common adverse reactions (≥20%) include musculoskeletal (muscle/bone) pain, cytokine release syndrome, cough, upper respiratory tract infection, diarrhea, fatigue, pneumonia (lung infection), nausea, headache, and dyspnea (shortness of breath), along with Grade 3 or 4 laboratory abnormalities.
• Continued approval for this use may depend upon verification and description of clinical benefit in a confirmatory clinical trial.

FDA Approves Kirsty (insulin aspart-xjhz), an Interchangeable Biosimilar to NovoLog

In July, the FDA approved Kirsty (insulin aspart-xjhz) from Biocon Biologics, a rapid acting human insulin analog interchangeable biosimilar to NovoLog (insulin aspart) indicated to improve glycemic (blood sugar) control in adults and children with diabetes mellitus.

• Kirsty is the second FDA-approved biosimilar to NovoLog, after Merilog (insulin aspart-szjj) and the first interchangeable biosimilar to NovoLog.
• FDA approval of Kirsty was based on a comprehensive package of analytical, nonclinical and clinical data, which confirmed that Kirsty is highly similar to NovoLog. There were no clinically meaningful differences between Kirsty and NovoLog in terms of safety, efficacy, purity and potency.
• An interchangeable biosimilar is a biologic product that can be substituted automatically for the reference product (in this case, Novolog) by a pharmacist, depending upon state laws. The pharmacist does 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.
• Kirsty is administered as a subcutaneous (under the skin) injection or as an intravenous (IV) injection. It is available as a 100 units/mL (U-100) prefilled pen (3 mL) for subcutaneous use, or as multidose vial (10 mL) for subcutaneous or IV use.
• Warnings and precautions associated with Kirsty include high or low blood glucose with changes in insulin regimen, low blood glucose which may be life-threatening, hypersensitivity (allergic) reactions (including anaphylaxis), hypokalemia (low potassium), medication errors between insulin products, fluid retention and heart failure with concomitant use of thiazolidinediones (TZDs) and hyperglycemia and ketoacidosis due to insulin pump device malfunction.
• Adverse reactions observed with insulin aspart products include hypoglycemia (low blood sugar), allergic reactions, local injection site reactions, lipodystrophy (abnormal fat distribution in certain areas of the body), rash, and itching.

FDA Approves Sephience to Treat Rare Metabolic Disease Phenylketonuria

Sephience (sepiapterin) from PTC Therapeutics is an oral, once daily phenylalanine hydroxylase activator now approved to treat adults and children 1 month and older with hyperphenylalaninemia (HPA) with sepiapterin-responsive phenylketonuria (PKU). It is used in conjunction with a phenylalanine (Phe)-restricted diet.

• Phenylalanine is an essential amino acid found in protein, but if high levels build up in the body it may lead to severe disabilities such as permanent intellectual disability, seizures, delayed development, memory loss, and emotional problems. Phenylketonuria (PKU) is a rare metabolic disease caused by a defect in the gene that helps create the phenylalanine hydroxylase (PAH) enzyme needed to break down phenylalanine.
• Diagnosis of PKU usually takes place during newborn screening programs. There are an estimated 58,000 people living with PKU globally.
• Sephience works in PKU by increasing activity of the PAH enzyme to reduce blood phenylalanine (Phe) levels. Sepiapterin is a precursor compound that’s converted intracellularly to tetrahydrobiopterin (a cofactor of PAH) and has an independent chaperone effect, protecting against PAH misfolding to enhance the enzyme function.
• FDA approval of Sephience was based on the evidence of significant efficacy and safety from the Phase 3 APHENITY trial as well as durability of treatment effect in the APHENITY long-term extension study.
• In the Phase 3 study, the mean (±SE) adjusted change in blood Phe levels from baseline at Weeks 5 and 6 for patients who previously had a ≥30% reduction in Phe levels was found to be -415.8 μmol/L for the Sephience group and -19.9 μmol/L (24.2) for placebo, a significant effect (p <0.0001). The mean percent (%) change in blood Phe from baseline to Weeks 5 and 6 was -62.8% for the Saphience group vs. +1.4% for placebo, a treatment difference of -64.2%.
• Sephience is given by mouth once daily with food. The recommended starting dosage is based on the patient’s age. It comes as an oral powder that is mixed with water, apple juice, strawberry jam, or applesauce.
• Warnings and precautions associated with Sephience include an increased risk of bleeding and hypophenylalaninemia (low levels of phenylalanine in the blood).
• Common adverse reactions (≥2% and greater than placebo) include diarrhea, headache, stomach-area pain, hypophenylalaninemia, feces discoloration (yellow / orange), and mouth / throat pain.

FDA Approves Skytrofa for the Once-Weekly Treatment of Adults with Growth Hormone Deficiency

The FDA has cleared Ascendis Pharma’s Skytrofa (lonapegsomatropin-tcgd), a human growth hormone used for the once-weekly treatment of adults with growth hormone deficiency (GHD), a rare disorder resulting from decreased or total loss of growth hormone production.

• Skytrofa is a prodrug that delivers sustained-released somatropin as the same 191 amino acid sequence in daily somatropin.
• GHD is a condition that occurs when the pituitary gland (a small gland located at the base of the brain) does not produce enough growth hormone (GH). GHD may stem from various causes, including pituitary tumors, genetics, radiation, injury or bleeding.
• Approval was based on Phase 3 study foresiGHt in 259 adults with GHD that compared the efficacy and safety of weekly Skytrofa with weekly placebo and daily somatropin. Results showed a significant reduction in trunk fat (primary endpoint) of -1.7% vs. +0.4%, and an increase in total body lean mass at week 38 in the Skytrofa group vs. placebo, respectively.
• Skytrofa is given by subcutaneous (under the skin) injection from a single-use, prefilled cartridge into the abdomen, buttock, or thigh. Patients or caregivers may administer the medicine using the Skytrofa autoinjector after adequate injection training by a healthcare professional.
• Warnings and precautions associated with Skytrofa include hypersensitivity reactions, increased risk of malignancy progression, decreased insulin sensitivity / diabetes, intracranial hypertension, fluid retention, hypoadrenalism, hypothyroidism, increased risk of slipped capital femoral epiphysis, increased risk of scoliosis progression, and pancreatitis.
• In adults, the most common adverse reaction (≥ 5%) were peripheral edema (fluid retention in areas like the legs, ankles, feet and hands).
• Skytrofa was previously approved in August 2021 for the treatment of children one year and older who weigh at least 11.5 kg (25.4 lb) and have growth failure due to inadequate secretion of endogenous growth hormone (GH).

Pediatric Thrombocytopenia Use and Sprinkle Dosage Form Approved for Doptelet

Sobi has announced that Doptelet (avatrombopag) is now approved for thrombocytopenia in pediatric patients 1 year and older with persistent or chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment.

• This approval also includes a new formulation, Doptelet Sprinkle oral granules for use in children ages 1 to less than 6 years. The sprinkle form is a capsule that contains granules that are mixed with a soft food or liquid for dosing. Doptelet tablets and Doptelet Sprinkle are not substitutable on a mg-to-mg basis. Doptelet oral tablets may also be used in children 6 years and older.
• Doptelet is a second generation, orally administered thrombopoietin (TPO) receptor agonist. It works by mimicking the biologic effects of TPO to stimulate the growth of megakaryocytes (platelet precursor), resulting in increased platelet count.
• Chronic immune thrombocytopenia (ITP) is a rare autoimmune bleeding disorder characterized by low platelet counts and an increased bleeding risk. Bleeding can range from minor, such as bruising, to more severe, like bleeding in the brain. In patients, severe fatigue and restrictions on physical activity may occur due to elevated risk of bleeding.
• Approval was based on the Phase 3 AVA-PED-301 study in children with ITP. The study showed that 27.8% of Doptelet patients achieved the primary endpoint of durable platelet response versus 0% of placebo patients in the absence of rescue medication (p=0.0077, 95% CI 15.8-39.7). A durable response consisted of patients achieving a platelet count >=50,000/uL for 6 of the last 8 weeks in the 12-week core phase of the AVA-PED-301 study.
• In pediatric patients with persistent or chronic immune thrombocytopenia, the most common adverse reactions (≥ 10%) were viral infection, nasopharyngitis (common cold symptoms), cough, pyrexia (fever), and oropharyngeal pain (mouth / throat pain).
• Doptelet is also approved to treat thrombocytopenia in adults with chronic liver disease who are scheduled to undergo a procedure; and for thrombocytopenia in adults with chronic ITP who have had an insufficient response to a previous treatment.

FDA Approves New Dosing Regimen for Lilly’s Kisunla in Alzheimer's Disease

Kisunla (donanemab-azbt) is a once-monthly amyloid beta-directed antibody indicated for the treatment of adults with early symptomatic Alzheimer’s disease (AD), which includes people with mild cognitive impairment (MCI) as well as people in the mild dementia stage of AD, with confirmed amyloid pathology. In July, the FDA cleared an updated recommended dosing regimen for Kisunla to improve safety.

• Donanemab-azbt is a humanized IgG1 antibody that targets a modified form of beta amyloid plaque called N3pG. Kisunla is thought to work by helping the body remove the excessive buildup of amyloid plaques in the brain. Amyloid is a protein produced naturally in the body that can clump together to create plaques in the brain that may lead to memory and thinking issues associated with Alzheimer's disease.
• ARIA-E, a temporary swelling in the brain, is a side effect of amyloid plaque-targeting therapies, including Kisunla. ARIA-E is usually asymptomatic, although serious and fatal events can occur. ARIA is detected via magnetic resonance imaging (MRI) brain scans. Enhanced clinical detection for ARIA is recommended during the first 24 weeks of treatment.
• Kisunla is administered as an intravenous (IV) infusion over approximately 30 minutes every four weeks. The new dosing recommendation differs from the original dosing by shifting a single vial from the first dose to the third dose, delivering the same amount of Kisunla by week 24. The new recommended dosing regimen for Kisunla is 350 mg (dose 1), 700 mg (dose 2), 1,050 mg (dose 3), and 1,400 mg (dose 4 and beyond). Kisunla is supplied as a 350 mg/20 mL (17.5 mg/mL) single-dose vial.
• In the TRAILBLAZER-ALZ 6 study, the modified dosing significantly lowered the incidence of ARIA-E by 41% at 24 weeks and by 35% at 52 weeks versus the original dosing schedule, while still achieving similar levels of amyloid plaque removal and P-tau217 reduction. No new adverse reactions were identified in this study, although higher rates of hypersensitivity reactions and infusion-related reactions were observed.
• The Kisunla label carries a Boxed Warning for amyloid-related imaging abnormalities (ARIA), such as edema (ARIA-E), and ARIA with hemosiderin deposition (ARIA-H). ARIA-E is usually asymptomatic, although serious and fatal events can occur. Use caution with antithrombotic drugs or in patients who are ApoE ε4 homozygotes. Warnings and precautions also include injection-site reactions.
• The most common adverse reactions (at least 10% and higher incidence compared to placebo) are ARIA-E, ARIA-H microhemorrhage, ARIA-H superficial siderosis, and headache.
• Kinsula is from Eli Lilly and Co and was first approved by the FDA in July 2024 for adults with early symptomatic Alzheimer's disease.

FDA Approves Apellis’ Empaveli for People 12 and Older with Rare, Serious Kidney Diseases

Empaveli (pegcetacoplan) is now approved to treat adult and pediatric patients aged 12 years and older with C3 glomerulopathy (C3G) or primary immune-complex membranoproliferative glomerulonephritis (IC-MPGN), to reduce proteinuria.

• C3G and IC-MPGN are rare and debilitating kidney diseases that can lead to kidney failure. Approximately one-half of patients with C3G and primary IC-MPGN develop kidney failure within 5 to 10 years of diagnosis, requiring a kidney transplant or lifelong dialysis.
• Excessive C3 deposits are a key marker of disease activity, which can lead to kidney inflammation, damage, and failure. Emapveli is a targeted C3 inhibitor and works by binding to complement protein C3 and its activation fragment C3b to regulate excessive activation of the complement cascade.
• Approval was based on the Phase 3 VALIANT study, where Empaveli demonstrated a 68% reduction in proteinuria, stabilization of kidney function, and substantial clearance of C3 deposits as measured by C3 staining, compared to placebo. The positive results were consistent across adolescent and adult patients with C3G and primary IC-MPGN, and in C3G patients with post-transplant disease recurrence.
• Empaveli is administered subcutaneously (under the skin) twice weekly by subcutaneous infusion via a commercially available infusion pump or with the Empaveli Injector.
• The Empaveli product label carries a Boxed Warning for the increased risk of meningococcal and other serious infections caused by encapsulated bacteria. Because of this risk, Empaveli is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS).
• Common adverse reactions (incidence ≥10%) in patients with C3G or primary IC-MPGN include infusion site reactions, pyrexia, nasopharyngitis, influenza, cough, and nausea. Empaveli may also interfere with certain laboratory coagulation tests using silica reagents.
• Emapveli, from Apellis Pharmaceuticals, was previously approved for the treatment of paroxysmal nocturnal hemoglobinuria in adults in May 2021.

Posted July 2025

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