PDR Drug Update - June 2025

Managing Hyperphagia in Prader-Willi Syndrome

Prader-Willi syndrome (PWS) is a complex genetic disorder resulting in deletions or partial deletions on chromosome 15. Characterized by a range of physical, cognitive, and behavioral challenges, PWS is particularly notable for its unique presentation of hyperphagia, which is an intense appetite that, if uncontrolled, leads to excessive eating and obesity. Now, a groundbreaking medication specifically targeting this aspect of PWS may offer renewed hope for improved management of the syndrome.

PWS affects approximately one in 15,000 births and stems from a genetic error that leads to the syndrome occurring randomly. It has a range of manifestations, including hypotonia in infancy, developmental delays, short stature, hypogonadism, and a characteristic facial appearance. Generally, the symptom considered to be the most alarming is hyperphagia. It typically starts in early childhood and involves an insatiable appetite that can lead to food obsession, aggressive food-seeking, and severe obesity if not managed appropriately. Hyperphagia significantly impacts the quality of life and leads to additional serious comorbidities, including diabetes and cardiovascular complications.

Hyperphagia has a neurobiological basis that involves dysfunction in the hypothalamus, where regulation of hunger and satiety occurs in the brain. Individuals with PWS often report overwhelming feelings of food scarcity, leading to persistent food-seeking behaviors. This can pose serious challenges for caregivers and healthcare providers, necessitating stringent environmental controls to limit access to food and acute interventions to manage obesity-related health complications.

The new approval for the first pharmacological intervention designed to treat hyperphagia in individuals with PWS is offering a way to ease some of the challenges that accompany the syndrome. Now approved for targeting hyperphagia in PWS patients aged 4 years and older, Vykat XR (diazoxide choline) works as a selective agonist at certain neuropeptide receptors with the aim of restoring the balance of appetite regulation. By modulating neurotransmission in the hypothalamus, Vykat XR has been shown to help blunt hyperphagic responses that result in compulsive food-seeking behavior in patients with PWS.

Common adverse events have been reported with the use of Vykat XR, including hypertrichosis, edema, hyperglycemia, and rash. The severity of these side effects was generally mild to moderate. Due to the risk of hyperglycemia, monitoring of blood glucose levels is recommended during treatment. Additionally, the risk of fluid overload needs to be monitored, as associated adverse reactions, including pulmonary edema, are possible. Depending on the severity of these events, Vykat XR may require dosage interruption, reduction, or discontinuation to avoid progression to worsened conditions. Physicians managing patients with PWS and prescribing Vykat XR should consider a comprehensive approach that includes dietary management, behavioral modifications, and psychological support alongside pharmacological treatment.

As the clinical understanding of PWS evolves and treatment options increase, the advent of Vykat XR represents a significant step forward. It reflects the importance of targeted therapies in managing complex genetic disorders, helping provide a means to improve the quality of life for affected individuals and their families. Stay informed about the development of other novel therapeutics exploring PDR by ConnectiveRx. You can also stay current by using the official PDR app, mobilePDR, which is available for free from your preferred app store.

FDA New Approvals

Avmapki Fakzynja Co-Pack

The FDA has approved Avmapki Fakzynja Co-Pack (avutometinib capsules; defactinib tablets) for the treatment of adult patients with KRAS-mutated recurrent low-grade serous ovarian cancer who received prior systemic therapy. Learn more about Avmapki Fakzynja Co-Pack

Brekiya

The FDA has approved Brekiya (dihydroergotamine mesylate) injection, the first and currently only dihydroergotamine autoinjector for the acute treatment of migraine with or without aura and the acute treatment of cluster headaches in adults. Learn more about Brekiya

Emrelis

The FDA has approved Emrelis (telisotuzumab vedotin-tllv) for the treatment of adult patients with locally advanced or metastatic, non-squamous non-small cell lung cancer with high c-Met protein overexpression who have received a prior systemic therapy. Learn more about Emrelis

Nuvaxovid

The FDA has approved Nuvaxovid (COVID-19 vaccine, adjuvanted) for individuals aged 12 through 64 years who have at least one underlying condition that puts them at high risk for severe outcomes from COVID-19. Learn more about Nuvaxovid

FDA New Indications

Nucala

The FDA has approved Nucala (mepolizumab) as an add-on maintenance treatment for adult patients with inadequately controlled COPD and an eosinophilic phenotype. Learn more about Nucala

Optison

The FDA has approved a pediatric indication for Optison (perflutren protein-type A microspheres) injectable suspension for intravenous use in patients with suboptimal echocardiograms to opacify the left ventricle and to improve the delineation of the left ventricular endocardial borders. Learn more about Optison

Rinvoq

The FDA has approved Rinvoq (upadacitinib), 15mg, once daily, for the treatment of adults with giant cell arteritis. Learn more about Rinvoq

Susvimo

The FDA has approved Susvimo (ranibizumab) 100mg/mL injection for the treatment of diabetic retinopathy. Susvimo is a refillable implant surgically inserted into the eye during a one-time, outpatient procedure. Learn more about Susvimo

Welireg

The FDA has approved Welireg (belzutifan) for the treatment of adult and pediatric patients aged 12 years and older with locally advanced, unresectable, or metastatic pheochromocytoma or paraganglioma. Learn more about Welireg

Zoryve

The FDA has approved Zoryve (roflumilast) topical foam, 0.3%, for the treatment of plaque psoriasis of the scalp and body in adult and pediatric patients aged 12 years and older. Learn more about Zoryve

Zynyz

The FDA has approved Zynyz (retifanlimab-dlwr) in combination with carboplatin and paclitaxel (platinum-based chemotherapy) for the first-line treatment of adult patients with inoperable locally recurrent or metastatic squamous cell carcinoma of the anal canal (SCAC). In addition, the FDA approved Zynyz as a single agent for the treatment of adult patients with locally recurrent or with metastatic SCAC with disease progression on or intolerance to platinum-based chemotherapy. Learn more about Zynyz

FDA Recalls

EnShiShiXiangNiShangMaoYouXianGongSi Endurance Boost

EnShiShiXiangNiShangMaoYouXianGongSi is voluntarily recalling one lot of Endurance Boost with horny goat weed (20 count) capsules to the consumer level. During the regular sampling, the product was found to be tainted with propoxyphenylsildenafil (a sildenafil analogue) and sildenafil. Learn more about the Endurance Boost recall

Umary Unavy and Umovy

Umary USA is voluntarily recalling all lots of Unavy Acido Hialuronico (30 caplets/850mg) and Umovy Acido Hialuronico (30 caplets/850mg), to the consumer level. FDA laboratory analysis confirmed that these products are tainted with the drug ingredients diclofenac, dexamethasone, and omeprazole. Learn more about the Unavy and Umovy recall

June 2025