10 FDA Headlines You Missed in July 2025

Welcome back to our recap of this month's news and updates from the US Food and Drug Administration (FDA)!

July 2025 continued the FDA’s momentum in advancing therapies across a wide range of disease areas, with key approvals spanning rare diseases, cardiometabolic disorders, dermatology, ophthalmology, and nephrology. The month marked significant firsts, including the approval of the first oral, on-demand treatment for hereditary angioedema and the first FDA-approved therapy for C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN). Expanded indications and label updates for agents like finerenone, inclisiran, and concizumab highlighted evolving standards in heart failure, lipid management, and hemophilia care. At the same time, regulatory setbacks such as a complete response letter for a Duchenne cardiomyopathy therapy underscored ongoing challenges in demonstrating efficacy and manufacturing reliability. Here’s a concise overview of the key FDA decisions and updates from July 2025.

Want to hear from the experts? Look for our Related Content links underneath blurbs for insight into how this decision might influence practice moving forward.

FDA News Month in Review: July 2025

Sebetralstat FDA-Approved as First Oral, On-Demand for Hereditary Angioedema

On July 7, 2025, the FDA approved KalVista Pharmaceuticals’ sebetralstat (Ekterly), the first and only oral, on-demand therapy for the treatment of acute attacks of hereditary angioedema (HAE) in adult and pediatric patients aged 12 years and older. The novel plasma kallikrein inhibitor’s approval was based on data from the phase 3 KONFIDENT and KONFIDENT-S open-label extension trials.

FDA Issues CRL to Deramiocel for Treating Cardiomyopathy Secondary to DMD

On July 11, 2025, the FDA issued a Complete Response Letter (CRL) to Capricor Therapeutics’ Biologics License Application (BLA) for deramiocel, a lead candidate for the treatment of cardiomyopathy associated with Duchenne muscular dystrophy (DMD). The CRL stated that the FDA was incapable of approving the BLA as it did not meet the statutory requirement for substantial evidence of effectiveness and the need for additional clinical data, citing outstanding items in the Chemistry, Manufacturing, and Controls (CMC) section of the application.

Capricor stated their belief that these issues were addressed in prior communications with the FDA, but the materials were not reviewed by the Agency due to the timing of the CRL issuance.

FDA Approves Finerenone (Kerendia) for Heart Failure with Ejection Fraction of 40% or More

On July 14, 2025, the FDA approved Bayer’s finerenone (Kerendia) for adults with heart failure with a left ventricular ejection fraction (LVEF) of 40% or greater. The approval indicates finerenone for reducing the risk of cardiovascular death, hospitalization for heart failure, and urgent heart failure visits in adults with heart failure with mildly reduced ejection fraction (HFmrEF) and preserved ejection fraction (HFpEF) and was supported by data from the FINEARTS-HF trial.

Related Content: Finerenone's FDA Approval Marks a Turning Point in HFpEF Management

FDA Approves Updated VARIPULSE Platform Irrigation Flow Rate for Heart Disease

On July 14, 2025, the FDA approved an update to Johnson & Johnson MedTech’s VARIPULSE Platform’s irrigation flow rate, which is designed to optimize irrigation and support consistent successful operations with low adverse event rates. The approval follows the significant milestone of >10,000 VARIPULSE procedures across all approved countries. The platform has also maintained a neurovascular adverse event rate of <.5%, although the release notes this statistic is calculated from internal customer reported data/complaints rather than a prospective clinical investigation. The rate reported is preliminary and pending publication.

Delgocitinib (Anzupgo) Scores FDA Approval for Chronic Hand Eczema

On July 23, 2025, the FDA approved LEO Pharma’s delgocitinib (Anzupgo) cream for the treatment of moderate-to-severe chronic hand eczema, indicating delgocitinib cream for the topical treatment of chronic hand eczema in adults who have had an inadequate response to, or for whom topical corticosteroids are not advisable based on data from the DELTA 1 and DELTA 2 clinical trials.

FDA Approves Lonapegsomatropin-tcgd (Skytrofa), Formerly TransCon hGH, for Adult Growth Hormone Deficiency

On July 28, 2025, the FDA approved Ascendis Pharma’s lonapegsomatropin-tcgd (Skytrofa), developed as TransCon hGH, for the replacement of endogenous growth hormone in adults with growth hormone deficiency (GHD). The approval was based on results from foresiGHt, a phase 3 randomized, parallel-arm, placebo-controlled, double-blind, and active-controlled clinical trial that compared the efficacy and safety of weekly TransCon hGH with weekly placebo and daily somatropin in adults with GHD.

FDA Approves Pegcetacoplan (Empaveli) for C3 Glomerulopathy, IC-MPGN

On July 28, 2025, the FDA approved Apellis Pharmaceuticals’ pegcetacoplan (Empaveli) for the treatment of C3G and IC-MPGN)in patients aged 12 years and older. The decision was supported by positive 26-week results from the phase 3 VALIANT trial, the largest single trial conducted in patients with C3G and IC-MPGN and the only study to include adolescent and adult patients with native and post-transplant kidneys. The approval indicates the agent for adults and adolescents with C3G or primary IC-MPGN, and post-transplant C3G disease recurrence.

FDA Approves Inclisiran (Leqvio) Label Update as First-Line Monotherapy in Hypercholesterolemia

On July 31, 2025, the FDA approved a label update for Novartis’ inclisiran (Leqvio), enabling its use as monotherapy along with diet and exercise to reduce low-density lipoprotein cholesterol (LDL-C) in adults with hypercholesterolemia. The FDA proactively requested the label update based on the robust LDL-C lowering data for PCSK9-targeting therapies. Specifically, the updated label removes the requirement for inclisiran to be used on top of or in combination with statin therapy and revises "primary hyperlipidemia" to the more specific term of "hypercholesterolemia" throughout the label to more accurately focus on LDL-C reduction.

FDA Approves Aceclidine (VIZZ) for Presbyopia in Adults

On July 31, 2025, the FDA approved LENZ Therapeutics’ aceclidine ophthalmic solution 1.44% (VIZZ) for the treatment of presbyopia in adults. The decision makes aceclidine ophthalmic solution 1.44% the first and only FDA-approved aceclidine-based eye drop for the treatment of presbyopia in adults. According to LENZ Therapeutics, samples are expected in the US as early as October 2025, with commercial product available by mid-Q4 2025.

Expanded Concizumab Approval Offers Subcutaneous Prophylaxis for Hemophilia Without Inhibitors

On July 31, 2025, the FDA approved an expanded indication for Novo Nordisk’s concizumab-mtci (Alhemo) subcutaneous injection as a once-daily prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 years of age and older with hemophilia A or B without inhibitors. The approval follows the original December 2024 approval for adults and children aged at least 12 years with hemophilia A or B with inhibitors and was based on data from the phase 3 explorer8 trial.