The goal of this study is to learn about how the hormone insulin controls blood sugar. The main question it aims to answer is about how much insulin the body actually needs to maintain a normal blood sugar level. People with obesity and high insulin levels will receive eight doses of diazoxide, a drug that suppresses the pancreas's production of insulin, and will have their fasting blood sugar and insulin levels checked daily while taking the drug.

Start Date23 Oct 2024

Sponsor / Collaborator

Columbia University

[+2]

NCT06308107

/ RecruitingPhase 1IIT

Safety and Feasibility of Hyperkalemic Cardioplegia With Diazoxide in Cardiac Surgery (CPG-DZX) Trial

This study aims to confirm the safety and efficacy of diazoxide as an additive to hyperkalemic cardioplegia in patients undergoing cardiac surgery with cardiopulmonary bypass. The investigators hypothesize that diazoxide combined with hyperkalemic cardioplegia provides superior myocardial protection and reduced myocardial stunning compared with standard cardioplegia alone. Thirty patients will receive treatment. Safety will be assessed by comparing mean arterial blood pressure measurements, glucose levels and incidence of adverse events between the two groups. Efficacy will be assessed by comparing right and left ventricular function in pre-operative vs post-operative transesophageal echocardiograms, need for mechanical circulatory support, ease of separation from bypass and Vasoactive Inotrope Score (VIS) between the two groups. The information gained could pave the way for the use of Katp (Potassium-atp) channel openers to prevent stunning, improve patient outcomes, and reduce health care costs related to myocardial stunning that requires inotropic and mechanical support following cardiac surgery.

Start Date08 Jul 2024

Sponsor / Collaborator

The Johns Hopkins University

NCT05729282

/ RecruitingPhase 1IIT

Role of Hyperinsulinemia in Non-Alcoholic Fatty Liver Disease (NAFLD) Pathogenesis: Diazoxide Pilot & Feasibility Study

The goal of this clinical trial is to compare a two-week course of diazoxide (at two different doses) and placebo in people with overweight/obesity and insulin resistance (IR) with, or at high risk for, non-alcoholic fatty liver disease (NAFLD). The main questions it aims to answer are how mitigation of compensatory hyperinsulinemia with diazoxide affects parameters of glucose and lipid metabolism (how people with IR and NAFLD respond to lowering high insulin levels so that the investigators can see what happens to how the liver handles fat and sugar).
Participants will:
* Take 27 doses of diazoxide (at 1 mg per kg of body weight per dose [mpk] or 2 mpk) or of placebo, over 14 days
* Take 32 doses of heavy (deuterated) water (50 mL each) over 14 days
* Have blood drawn and saliva collected after an overnight fast on four mornings over the two-week study period
* Consume their total calculated daily caloric needs as divided into three meals per day
* Wear a continuous glucose monitor for the two-week study period
Researchers will compare fasting blood tests at intervals during the study period in participants randomized (like the flip of a coin) to diazoxide 1 mpk, diazoxide 2 mpk, or placebo, to see how the drug treatment affects plasma glucose, serum insulin, and serum lipid parameters (triglycerides, free fatty acids, and apolipoprotein B). They will also consume heavy (deuterated) water to assess de novo lipogenesis (building of new fatty acids by the liver).

Start Date01 Aug 2023

Sponsor / Collaborator

Columbia University

[+2]

100 Clinical Results associated with Diazoxide

100 Translational Medicine associated with Diazoxide

100 Patents (Medical) associated with Diazoxide

4,568

Literatures (Medical) associated with Diazoxide

01 May 2025·Archives of Disease in Childhood-Fetal and Neonatal Edition

Neonatal hyperinsulinism: a retrospective study of presentation and management in a tertiary neonatal intensive care unit in the UK

Article

Author: Beardsall, Kathy ; Couch, Helen ; Thankamony, Ajay ; Kalogeropoulou, Maria-Sofia

Objective:

Reports of hyperinsulinism typically focus on infants managed by highly specialised services. However, neonates with hyperinsulinism are initially managed by neonatologists and often not referred to specialists. This study aimed to characterise the diversity in presentation and management of these infants.

Setting:

Level 3 neonatal intensive care.

Patients:

Neonates with hyperinsulinism, defined as blood glucose <2.8 mmol/mL and insulin level >6 pmol/L.

Design:

7-year retrospective study (January 2015–December 2021).

Results:

99 cases were identified:severe—treated with diazoxide (20%),moderate—clinically concerning hyperinsulinism not treated with diazoxide (30%),mild—biochemical hyperinsulinism (50%). Birth weight z-score was −1.02±2.30 (mean±SD), 42% were preterm, but neither variable correlated with clinical severity. Theseveregroup received a higher concentration of intravenous glucose (27±12%) compared with themoderate(15±7%) andmild(16±10%) groups (p<0.001). At diagnosis, the intravenous glucose intake was similar in thesevere(7.43±5.95 mg/kg/min) andmoderate(5.09±3.86 mg/kg/min) groups, but higher compared with themildgroup (3.05+/2.21 mg/kg/min) (p<0.001). In theseveregroup, term infants started diazoxide earlier (9.9±4.3 days) compared with preterm (37±26 days) (p=0.002). The national congenital hyperinsulinism service was consulted for 23% of infants, and 3% were transferred.

Conclusions:

This study highlights the diversity in clinical presentation, severity and prognosis of neonatal hyperinsulinism, irrespective of birth weight and gestational age. More infants were small rather than large for gestational age, and the majority had transient hyperinsulinism and were not referred to the national centre, or treated with diazoxide. Further research is required to understand the breadth of neonatal hyperinsulinism and optimal management.

01 May 2025·BMJ Case Reports

Congenital hyperinsulinemic hypoglycaemia in a neonate: a rare homozygous ABCC8 gene mutation

Article

Author: Kumar, Mala ; Prabha, Chandra ; Bhriguvanshi, Arpita ; Tripathi, Shalini

Hyperinsulinemic hypoglycaemia (HH) is a heterogeneous disorder causing persistent hypoketotic hypoglycaemia in neonates and infants. Congenital hyperinsulinism (CHI) is a rare cause of HH, resulting from inappropriate insulin secretion by pancreatic β-cells due to genetic defects in key genes, notably ABCC8 and KCNJ11, which encode the SUR1 and Kir6.2 components of the KATPchannels, respectively. We present a case of a neonate with congenital HH with persistent hypoglycaemia since birth, which was managed with high-dose glucose infusions, diazoxide and octreotide. A homozygous pathogenic missense variant, c4253G>A (p.Arg1418His) in Exon 35 of the ABCC8 gene was identified in the neonate, confirming CHI. Despite initial refractoriness to treatment, the infant responded to octreotide therapy, cornstarch and careful management with regular feeds and monitoring, avoiding the need for surgical intervention. This case underscores the critical role of genetic diagnosis and timely management in preventing long-term neurological sequelae.

11 Mar 2025·Cureus Journal of Medical Science

Neonatal Cholestasis Associated With Transient Congenital Hyperinsulinism: A Case Report

Article

Author: Ibi, Kyosuke ; Takahashi, Naoto ; Adachi, Natsuho ; Tanaka, Hiroyuki ; Shitara, Yoshihiko

Congenital hyperinsulinism (CHI) and neonatal cholestasis (NC) are occasionally observed in neonatal settings; however, the causes have not been identified despite thorough investigation. Surgical evaluation is essential for patients with cholestasis to rule out biliary atresia because timely surgical intervention is critical. Few case reports have described the co-occurrence of CHI and NC. Herein, we report a case of a boy born as one of dichorionic-diamniotic twins via planned cesarean delivery. Apparent ischemic events were not present, with Apgar scores of 8 and 9 at one and five minutes, respectively; however, the umbilical artery blood gas pH was 7.17. He experienced a hypoglycemic attack on day two and was diagnosed with CHI. The patient was put on diazoxide therapy, following which his blood glucose levels were stable without intravenous glucose infusion. However, cholestasis was observed from day 14. Workups did not indicate any specific clinical condition, and biliary atresia was ruled out on day 44 by cholangiography. He exhibited prolonged fasting hypoglycemia because of lipid malabsorption until he recovered from cholestasis. He was free of diazoxide at 16 months of age and subsequently acquired normal physical and mental development. This case highlights the potential co-occurrence of CHI and NC. The etiologies remain unidentified; however, both may have resulted from perinatal distress. Hypoglycemic episodes prior to the diagnosis of CHI may also trigger cholestasis. Transient CHI can be accompanied by benign NC, and such patients can experience persistent hypoglycemia and require nutritional management until the cholestasis is relieved.

News (Medical) associated with Diazoxide

15 May 2025

·www.globenewswire.com

Soleno Therapeutics Announces Presentations Featuring VYKAT(TM) XR in Prader-Willi Syndrome at PES 2025

REDWOOD CITY, Calif., May 15, 2025 (GLOBE NEWSWIRE) -- Soleno Therapeutics, Inc. (Soleno) (NASDAQ: SLNO), a biopharmaceutical company developing novel therapeutics for the treatment of rare diseases, today announced poster presentations from its VYKATTM XR (diazoxide choline) extended-release tablets, previously referred to as DCCR, clinical development program for the treatment of hyperphagia associated PWS at the Pediatric Endocrine Society (PES) Meeting 2025, which is being held May 15-18, in National Harbor, Maryland. Details of the presentations are as follows: PES 2025 Title: Long-term administration of diazoxide choline extended-release (DCCR) tablets in people with Prader-Willi syndrome: changes in lean body mass and lean mass Index Format: PosterSession: Poster Session 3 Date/Time: May 17, 12:45 - 2:30 PM Presenter: Jack Yanovski, MD PhD Title: Resuming diazoxide choline extended-release (DCCR) after 16-week randomized withdrawal is associated with significant improvements in hyperphagia and behavioral symptoms in PWS (Study C614)Format: PosterSession: Poster Session 3 Date/Time: May 17, 12:45 - 2:30 PM Presenter: Jack Yanovski, MD PhD About PWSThe Prader-Willi Syndrome Association USA estimates that PWS occurs in one in every 15,000 live births. The hallmark symptom of this disorder is hyperphagia, a chronic and life-threatening condition characterized by feelings of intense, persistent hunger, food pre-occupation, and an extreme drive to seek and consume food, which can severely diminish the quality of life for individuals with PWS and their families. Additional characteristics of PWS include behavioral problems, cognitive disabilities, low muscle tone, short stature (when not treated with growth hormone), the accumulation of excess body fat, developmental delays, and incomplete sexual development. Hyperphagia can lead to significant mortality (e.g., stomach rupture, choking, accidental death due to food seeking behavior) and longer term, co-morbidities such as diabetes, obesity, and cardiovascular disease. About VYKAT XRVYKAT XR was approved by the U.S. Food and Drug Administration (FDA) on March 26, 2025, and is now commercially available to U.S. patients. VYKAT XR is indicated for the treatment of hyperphagia in adults and pediatric patients 4 years of age and older with Prader-Willi syndrome (PWS). IMPORTANT SAFETY INFORMATION ContraindicationsUse of VYKAT XR is contraindicated in patients who have a known hypersensitivity to diazoxide, other components of VYKAT XR, or to thiazides. Warnings and Precautions HyperglycemiaHyperglycemia, including diabetic ketoacidosis, has been reported. Before initiating VYKAT XR, test fasting plasma glucose (FPG) and HbA1c; optimize blood glucose in patients who have hyperglycemia. During treatment, regularly monitor fasting glucose (FPG or fasting blood glucose) and HbA1c. Monitor fasting glucose more frequently during the first few weeks of treatment in patients with risk factors for hyperglycemia. Risk of Fluid OverloadEdema, including severe reactions associated with fluid overload, has been reported. Monitor for signs or symptoms of edema or fluid overload. VYKAT XR has not been studied in patients with compromised cardiac reserve and should be used with caution in these patients. Adverse ReactionsThe most common adverse reactions (incidence ≥10% and at least 2% greater than placebo) included hypertrichosis, edema, hyperglycemia, and rash. Please see the full Prescribing Information, including Medication Guide. About Soleno Therapeutics, Inc.Soleno is focused on the development and commercialization of novel therapeutics for the treatment of rare diseases. The company’s first commercial product, VYKAT XR (diazoxide choline) extended-release tablets, formerly known as DCCR, is a once-daily oral treatment for hyperphagia in adults and children 4 years of age and older with Prader-Willi syndrome. For more information, please visit www.soleno.life. Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of Section 21E of the Securities Exchange Act of 1934, as amended. All statements other than statements of historical facts contained in this press release are forward-looking statements. In some cases, you can identify forward-looking statements by terms such as "may," "will," "should," "expect," "plan," "anticipate," "could," "intend," "target," "project," "contemplates," "believes," "estimates," "predicts," "potential" or "continue" or the negative of these terms or other similar expressions. These forward-looking statements speak only as of the date of this press release and are subject to a number of risks, uncertainties and assumptions, including those described in the company's prior press releases and in the periodic reports it files with the SEC. The events and circumstances reflected in the company's forward-looking statements may not be achieved or occur and actual results could differ materially from those projected in the forward-looking statements. Except as required by applicable law, the company does not plan to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise. Corporate Contact:Brian RitchieLifeSci Advisors, LLC212-915-2578 Media Contact:Soleno Therapeutics media@soleno.life 650-213-8444, #2

Drug Approval

27 Mar 2025

·www.pharmaceutical-technology.com

Soleno wins FDA approval for Prader-Willi hyperphagia treatment

Hyperphagia is one of the most serious and defining characteristics of Prader-Willi syndrome. Credit: Catherine Falls Commercial via Getty Images. Soleno Therapeutics has won US Food and Drug Administration (FDA) approval for diazoxide choline – which will be branded as Vykat XR – to treat extreme hunger in patients with Prader-Willi syndrome. This approval follows a three-month delay from the FDA. In November 2024, the agency classified responses from Soleno as a “major amendment” to its new drug application (NDA), requiring additional time to review. However, the agency did not raise concerns about the drug’s safety, efficacy, or manufacturing. Prader-Willi syndrome is a rare genetic disorder caused by abnormalities in chromosome 15. Symptoms include low muscle tone, developmental delays, and behavioural challenges. Extreme hunger – known as hyperphagia, is one of the most serious and defining characteristics of the disorder and can lead to severe obesity and associated health complications if unmanaged. Vykat XR is now the only FDA-approved treatment specifically addressing hyperphagia in Prader-Willi syndrome. The FDA approval of Vykat XR was based on data from the Phase III Study C602-RWP clinical trial (NCT03714373). Efficacy was demonstrated during a 16-week randomised withdrawal study period. Patients who switched to a placebo demonstrated a statistically significant worsening of hyperphagia compared to those who remained on Vykat XR. The drug was also studied in the Phase III DESTINY-PWS trial (NCT03440814), which enrolled 127 individuals with genetically confirmed Prader-Willi syndrome. The trial did not demonstrate a statistically significant difference in hyperphagia reduction overall, but Soleno reported “nominally significant” reductions in fat mass, and general improvements in condition as assessed by investigators. Vykat XR is set to launch in the US in April 2025, according to Soleno’s 26 March announcement. Vykat XR – an extended-release tablet containing a crystalline salt formulation of diazoxide – works by activating the adenosine triphosphate (ATP)-sensitive potassium (KATP) channel, which plays a role in hunger regulation. The approval was welcomed by the Prader-Willi Syndrome Association: “Today marks a historic day for the PWS community. The FDA’s approval of Vykat XR represents a monumental step forward in addressing the longstanding unmet needs of individuals living with PWS and their families,” said Stacy Ward, CEO of the Association. Soleno also has its eyes set on Europe, having announced previously that it plans to file for approval of Vykat XR in the EU in the first half of 2025. According to GlobalData’s Pharma Intelligence Center, Vykat XR is set to generate $876m in sales by 2030. GlobalData is the parent company of Pharmaceutical Technology.

Phase 3Drug ApprovalClinical Result

27 Mar 2025

·www.biospace.com

Soleno Wins First FDA Approval for Hyperphagia in Prader-Willi Syndrome

iStock, MariaGisina Soleno’s Vykat XR is the first drug approved for the rare disease that directly targets its hallmark symptom. The FDA signed off on Soleno Therapeutics’ treatment for a major symptom of the rare disease Prader-Willi syndrome. The drug, diazoxide choline extended-release tablet, will now carry the brand name Vykat XR. Vykat XR is specifically indicated to address hyperphagia, or the abnormally strong sensation of hunger, which often leads to excessive eating. Its approval covers both adult and pediatric patients with Prader-Willi syndrome (PWS) aged 4 years and up. Soleno is working to make the drug available in the U.S. next month, as per its Wednesday release. Vykat XR is the first drug that targets hyperphagia in PWS. In a note to investors, Stifel analysts called the approval “a significant win” for Soleno, pointing to Vykat XR’s “very favorable/clean” label, the lack of a boxed warning or exclusions according to the severity of hyperphagia. “We think the drug’s well-positioned to launch strongly into what we see as a blockbuster opportunity.” PWS is a rare and genetic neurodevelopmental disorder that affects one in every 15,000 live births. Hyperphagia is the “defining symptom” of PWS, according to Soleno, pointing to the “extreme drive to consume food” that not only compromises quality of life for patients and their families but can also become life-threatening without treatment. Vykat XR’s active ingredient is diazoxide, a molecule that has been used “ for decades ” to manage rare diseases in infants, though never for PWS, according to a June 2024 news release from Soleno. To assess the drug’s potential in PWS, the biotech ran a Phase III clinical program of diazoxide consisting of two trials: C601, a randomized, double-blinded and placebo-controlled study, and C602, an open-label extension study. Soleno presented long-term data from these studies in June 2022, touting significant improvements in metabolic parameters, showing that the drug could tamp down on hyperphagia. At the one-year follow-up, patients had significantly better lean body mass, fasting insulin and leptin levels, insulin sensitivity and adiponectin, a cardioprotective marker. Vykat XR’s approval continues biopharma’s rally in the rare disease space. Last week, for instance, Novartis’ factor B inhibitor Fabhalta won the FDA’s go-ahead for C3 glomerulopathy to lower proteinuria, becoming the first-ever drug for the indication. And last month, Mirum notched a nod for Ctelix for use in cerebrotendinous xanthomatosis—also an industry first. Similarly, the FDA in February signed off on SpringWorks’ rare disease drug Gomekli, which can now be used for adults and children with neurofibromatosis type 1 with symptomatic plexiform neurofibromas.

Drug ApprovalPhase 3Clinical Result

100 Deals associated with Diazoxide

External Link

KEGG	Wiki	ATC	Drug Bank
D00294	Diazoxide	V03AH01 C02DA01	DB01119

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Hypertension	United States	Schering Corp.	22 Jan 1973
Hypoglycemia	United States	Schering Corp.	22 Jan 1973

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Multiple Sclerosis, Relapsing-Remitting	Phase 2	Germany	Neurotec Pharma SL	01 Jun 2011
Multiple Sclerosis, Relapsing-Remitting	Phase 2	Spain	Neurotec Pharma SL	01 Jun 2011
Multiple Sclerosis	Phase 2	Spain	Neurotec Pharma SL	-
Amyotrophic Lateral Sclerosis	Preclinical	Spain	Neurotec Pharma SL	-

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT01028846 (CTgov)	Phase 4	Diabetes Mellitus, Type 2 \| Hyperglycemia	10	Diazoxide (Diazoxide)	jwcfzalrcw(evnmyfsnba) = gnpoupofpo mtxflsciht (czgygezqfi, 0.13) View more	-	14 Mar 2025
				Placebo (Placebo)	jwcfzalrcw(evnmyfsnba) = lebovybiro mtxflsciht (czgygezqfi, 0.17) View more
SAT157 (ENDO2023)	Not Applicable	Chronic Kidney Diseases	4	Diazoxide 50 mg TID	gmsxnkdfza(nojjzrmbrk) = aiqsjpttlq wxvjhtrjcg (xuabtpocpk ) View more	Positive	05 Oct 2023
				Diazoxide 70 mg BID	gmsxnkdfza(nojjzrmbrk) = vxtawzivih wxvjhtrjcg (xuabtpocpk ) View more
PSUN340 (ENDO2022)	Not Applicable	Congenital Hyperinsulinism ABCC8 \| KCNJ11 \| GCK ... View more	2	diazoxide (Case 1)	pyvlhrjosh(fujihvjfpp) = zdvfwhvthz fdhbhwxsae (ycesrtzpid ) View more	-	01 Nov 2022
				diazoxide (Case 2)	pyvlhrjosh(fujihvjfpp) = lshlvibncd fdhbhwxsae (ycesrtzpid ) View more
ESPE2022	Not Applicable	Congenital Hyperinsulinism HADH mutation	-	Diazoxide	hjrsnfckcu(lowaigaxhm) = kamkkzyeyt gfrzsumsfj (hxrzmhufft, 0.9 - 7.1) View more	-	15 Sep 2022
				Diazoxide	hmoqznpyfm(hsjojnytbg) = ucqiauokqm zaewwlllrb (bvcaykgoxv )
ESPE2022	Not Applicable	Hemochromatosis	99	Diazoxide 2mg/kg/day	wqylzmadxf(wbegtbvxfd) = Adverse events can still be observed with DZX low doses, but at a lower incidence compared to the STD mnpaijbxqy (nqwegsmmjs ) View more	Positive	15 Sep 2022
				Diazoxide 3mg/kg/day
ENDO2021	Not Applicable	Glut1 Deficiency Syndrome SLC2A1	1	Diazoxide	vkhiiiqpxc(yiaackpyoi) = xmhzozuuaq sagumbibsx (renvtlovaa ) View more	Positive	03 May 2021
				Diazoxide (Ketogenic Diet)	vkhiiiqpxc(yiaackpyoi) = prnqsycbrz sagumbibsx (renvtlovaa ) View more
ENDO2021	Not Applicable	Neonatal hypoglycemia	46	Diazoxide	ulieqrkyul(yqujcyinjj) = nndsaofyxc xxvgfdvyis (bysnfcazis ) View more	Positive	03 May 2021
MON-911 (ENDO2020)	Not Applicable	Insulinoma 5-HIAA \| chromogranin A \| IGF-1	-	MON-911 (Insulinoma)	vaoslmjcmn(hhggtzvorp) = txlaimoygu idysvszcpx (gennsqeekx, 298 - 2350) View more	-	08 May 2020
MON-073 (ENDO2020)	Not Applicable	Hyperinsulinism ABCC8 Dominant Mutation	-	Diazoxide (ABCC8 Dominant Mutation)	xgyvlyzphc(bapddpacbo) = ldujjrdeeu ejzhrvqqsw (ytdpsdhtnl ) View more	-	08 May 2020
ESPE2019	Not Applicable	Congenital Hyperinsulinism ABCC8 \| KCNJ11	-	Diazoxide	tagtceviuw(izamqsmnvw) = due to vomiting on oral feeds myknddhqpq (sbmpvzryws ) View more	-	19 Sep 2019

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