Skytrofa Approved for Adult Growth Hormone Deficiency
On July 28, 2025, the US Food and Drug Administration (FDA) granted Ascendis Pharma approval of Skytrofa® (lonapegsomatropin-tcgd), a long-acting prodrug of human growth hormone, for the treatment of adult growth hormone deficiency (GHD). Initially approved in 2021 for pediatric GHD, Skytrofa uses TransCon™ technology to provide sustained release of unmodified somatropin, which reduces injection frequency and may improve adherence. Approval for adults was based on a phase III trial that demonstrated efficacy and safety compared to daily somatropin and placebo in adults who have GHD. The recommended dose is based on age and concomitant use of estrogen and is administered once weekly via subcutaneous (SC) injection. Key safety considerations include risks of tumor recurrence, intracranial hypertension, hyperglycemia and fluid retention. Ascendis plans to initiate clinical trials for Skytrofa in a variety of other conditions. Here is the updated prescribing information.
Empaveli Gains Approval for Two Rare Kidney Diseases, Including First-Ever IC-MPGN Indication
Apellis Pharmaceuticals’ Empaveli® (pegcetacoplan) SC injection was approved on July 28, 2025, for the treatment of complement 3 glomerulopathy (C3G) and primary immune-complex membranoproliferative glomerulonephritis (IC-MPGN) in adult and pediatric patients aged 12 years and older. This marks two new indications, and the first therapy approved for IC-MPGN. C3G and IC-MPGN are rare, progressive kidney diseases affecting approximately 5,000 people in the US, with nearly half progressing to kidney failure within five to ten years, often requiring transplant or lifelong dialysis. Empaveli is a targeted C3 inhibitor that regulates excessive complement activation, a key driver of these rare kidney diseases. Approval was based on a phase III trial involving both rare diseases, which showed that Empaveli led to a 68% reduction in proteinuria at week 26 and stabilized kidney function. The recommended dose is 1080mg administered SC twice weekly. Initially approved in 2021 for paroxysmal nocturnal hemoglobinuria (PNH), Empaveli requires prescriber certification and patient enrollment in a Risk Evaluation and Mitigation Strategy (REMS) program due to an increased risk of serious bacterial infections, including meningitis. Here is its complete prescribing information.
FDA Lifts Voluntary Hold on Elevidys for Ambulatory DMD Patients
On July 28, 2025, the FDA recommended lifting the voluntary hold on Sarepta Therapeutics’ Elevidys® (delandistrogene moxeparvovec-rokl), an intravenous (IV) gene therapy for the treatment of Duchenne muscular dystrophy (DMD) in ambulatory patients, following a one-week pause due to safety concerns. The FDA’s decision came after concluding that the death of an eight-year-old patient was not related to the therapy. A clinical hold remains in place for non-ambulatory patients after two deaths linked to acute liver failure. The FDA emphasized the importance of patient advocacy in its decision-making. Sarepta will resume shipments to treatment centers immediately.
Leqvio Approved as Monotherapy in Hypercholesterolemia
Novartis’ Leqvio® (inclisiran) was granted an expanded indication on July 31, 2025, for the treatment of hypercholesterolemia in adults with elevated low-density lipoprotein cholesterol (LDL-C) who are at increased risk of cardiovascular disease. The drug can be used as monotherapy along with diet and exercise. Previously approved for treating adults who have heterozygous familial hypercholesterolemia (HeFH), clinical atherosclerotic cardiovascular disease (ASCVD) and hypercholesterolemia in combination with a statin, Leqvio can now be used alone, without statin therapy.Leqvio increases LDL-C uptake and lowers blood levels of LDL-C by blocking proprotein convertase subtilisin/kexin type 9 (PCSK9), an enzyme that affects lipid metabolism. It is administered as a SC injection by a healthcare provider using a prefilled syringe that contains the recommended dose of 284mg. The second dose is given three months after the first, followed by doses every six months. Here is the updated prescribing information.
FDA Mandates Label Changes for Opioids to Highlight Long-Term Risks
The FDA announced labeling changes for all opioid pain medications to underscore the serious risks associated with long-term use on July 31, 2025. These updates, which impact both immediate-release and extended-release formulations, follow findings from two large post-marketing observational studies that revealed significant risks of misuse, addiction, and overdose. The revised labeling removes ambiguous language suggesting indefinite use, strengthens warnings about dose-dependent risks and emphasizes that extended-release opioids should only be used when other treatments are inadequate. Additional updates include guidance on tapering to avoid withdrawal, new warnings about opioid-induced esophageal dysfunction and toxic leukoencephalopathy – a rare brain condition that can happen after overdose – and expanded information on overdose reversal agents. The FDA is also requiring a new randomized controlled trial to further assess long-term opioid efficacy and safety. Manufacturers must submit updated labels within 30 days. The FDA News Release can be found here.
Vizz Approved for Presbyopia
On July 31, 2025, the FDA approved Vizz™ (aceclidine ophthalmic solution – LENZ Therapeutics) 1.44% for the treatment of presbyopia in adults. Presbyopia is a common, age-related, vision condition that impacts the ability to focus on objects close by. It impacts the lives of almost all Americans over 45 years of age with an estimated population of 128 million people. Vizz is an aceclidine-based eye drop approved for this condition, offering the first once-daily solution that provides up to 10 hours of improved near vision demonstrating efficacy within 30 minutes. It works by directly stimulating muscarinic receptors leading to pupil constriction and increased outflow of aqueous humor, reducing intraocular pressure. The most common side effects in clinical trials were mild and included site irritation, dim vision, and headache. It is expected to be available in the US in the fourth quarter of 2025. Cost is not yet known. Here is the prescribing information.
Alhemo Approved for Hemophilia Without Inhibitors
The FDA approved Novo Nordisk’s Alhemo® (concizumab-mtci) for prophylactic treatment to prevent or reduce bleeding episodes in adults and children aged 12 and older with hemophilia A or B without inhibitors. Initially approved in December 2024 for patients with inhibitors, Alhemo is a tissue factor pathway inhibitor (TFPI) antagonist that reduces the activity of TFPI, a protein that keeps blood from clotting and increases the availability of thrombin. Approval was based on a phase III trial showing a 79% reduction in annualized bleeding rate (ABR) in patients with hemophilia B and 86% in patients with hemophilia A without inhibitors compared to no prophylaxis. The recommended dose is a weight-based, once daily SC injection, with potential adjustments based on plasma level. There is a risk of thromboembolic events with treatment. Updated prescribing information is here.
MedWatch Update
FDA Moves to Restrict Potent 7-OH Opioid Products Amid Rising Abuse Concerns On July 29, 2025, the FDA announced a public health action recommending the scheduling of 7-hydroxymitragynine (7-OH) under the Controlled Substances Act (CSA). A highly potent opioid derivative of the kratom plant, 7-OH is sold in concentrated products due to its high abuse potential and strong binding to opioid receptors, with potency exceeding that of morphine. This move does not target natural kratom leaves. The FDA cited growing concerns over the unregulated sale of 7-OH in forms like gummies, drink mixes and ice cream cones, which are often marketed to youth and sold in vape shops, gas stations and online. There are no FDA-approved uses for 7-OH, and it is not permitted in dietary supplements or conventional foods. The DEA will now review the FDA’s recommendation. Here is the full announcement.