Last update 25 Dec 2024

Hydrocortisone

Last update 25 Dec 2024

Overview

R&D Status

Clinical Result

Translational Medicine

Overview

Basic Info

SummaryHydrocortisone, a diminutive yet powerful molecule, is renowned for its aptitude to function as a glucocorticoid receptor (GR) agonist, thereby stimulating the GR. This drug boasts a diverse range of clinical applications and is typically employed in the treatment of a multitude of ailments that are closely associated with adrenal insufficiency, including but not limited to congenital adrenal hyperplasia, adrenal hyperplasia, Addison's disease, and isolated ACTH deficiency. Additionally, it has proven efficacious in treating inflammatory conditions such as subacute thyroiditis, ulcerative colitis, proctocolitis, and ulcerative proctitis. It is worth noting that the FDA initially granted its approval to hydrocortisone on the 5th of August, 1952. The development of hydrocortisone is credited to Merck Sharp & Dohme Corp, which pioneered the drug. Owing to its extraordinary anti-inflammatory and immunosuppressive qualities, hydrocortisone has emerged as a definitive remedy for numerous chronic inflammatory disorders.

Drug Type

Small molecule drug

Synonyms

(11β)-11,17,21-trihydroxypregn-4-ene-3,20-dione, 11beta,17alpha,21-Trihydroxy-4-pregnene-3,20-dione, 11beta-hydrocortisone

+ [58]

Target

Mechanism

GR agonists(Glucocorticoid receptor agonists)

Therapeutic Areas

NeoplasmsImmune System DiseasesInfectious Diseases+ [10]

Active Indication

Adrenal Hyperplasia, CongenitalACTH Deficiency, IsolatedAdrenal Insufficiency+ [19]

Inactive Indication

Congenital Adrenal Hyperplasia Due to 21 Hydroxylase Deficiency

Originator Organization

Merck Sharp & Dohme Corp.

Active Organization

ANI Pharmaceuticals, Inc.

Neurocrine Biosciences, Inc.Pharmacia & Upjohn, Inc.

+ [4]

Inactive Organization

Merck & Co., Inc.

Shire Plc

Drug Highest PhaseApproved

First Approval Date

US (05 Aug 1952),

Endocrine System DiseasesHypersensitivityImmune System Diseases+ [1]

RegulationFast Track (US), Orphan Drug (EU), Orphan Drug (AU), Orphan Drug (US)

Structure

Molecular FormulaC21H30O5

InChIKeyJYGXADMDTFJGBT-VWUMJDOOSA-N

CAS Registry50-23-7

467

Clinical Trials associated with Hydrocortisone

NCT06381661 / Not yet recruitingPhase 3IIT

PALETTE- Adaptive Platform Trial for Personnalisation of Sepsis Treatment in Children and Adults: a Multi-national, Treatable Traits-guided, Adaptive, Bayesian Basket Trial"

PALETTE is a perpetual adaptive platform to efficiently study sepsis interventions within 'treatable traits' in all-ages patients enabling prompt evaluation of pandemic treatments. Treatable traits, therapeutic targets identified by phenotypes or endotypes (defined by biological mechanism or by treatment response) through validated biomarkers (measurable characteristic reflecting normal or pathogenic processes, or treatment responses), may include multi-omics, cellular, immune, metabolic, endocrine features, or intelligent algorithms. PALETTE Bayesian adaptive design enables parallel investigations of multiple interventions for sepsis, and quick inclusion of pandemic pathogens. PALETTE's new conceptual model will respond to the challenges of standard approaches, i.e. series of sepsis trials, each investigating one or two interventions, expensive, time consuming, and inappropriate in pandemic context.

Start Date01 Apr 2026

Sponsor / Collaborator

Assistance Publique des Hôpitaux de Paris SA

NCT04231708 / Not yet recruitingPhase 2IIT

Effects of Pharmacological Stress and Repetitive Transcranial Magnetic Stimulation Interventions on Executive Function in Opioid Use Disorder

This preliminary study is designed to evaluate mechanisms by which excitatory dorsolateral prefrontal cortex (dlPFC) repetitive transcranial magnetic stimulation (rTMS) (vs. sham) and pharmacological stress (vs. placebo) alter behavior in non-treatment seeking individuals with opioid use disorder (OUD). Specific Aims are to (1) Evaluate how stress impacts domains of behavior including (1a) executive function and (1b) opioid-seeking behavior; and (2) Determine whether rTMS stimulation attenuates (2a) executive dysfunction, (2b) stress-reactivity, and (2c) opioid-seeking in individuals with OUD not receiving treatment.

Start Date01 Jun 2025

Sponsor / Collaborator

Wayne State University (Michigan)

NCT06317662 / Not yet recruitingPhase 2IIT

A Phase 2 Study of Blinatumomab in Combination With Chemotherapy for Infants With Newly Diagnosed Acute Lymphoblastic Leukemia With Randomization of KMT2A-Rearranged Patients to Addition of Venetoclax

This phase II trial tests the addition of venetoclax and/or blinatumomab to usual chemotherapy for treating infants with newly diagnosed acute lymphoblastic leukemia (ALL) with a KMT2A gene rearrangement (KMT2A-rearranged [R]) or without a KMT2A gene rearrangement (KMT2A-germline [G]). Venetoclax is in a class of medications called B-cell lymphoma-2 (Bcl-2) inhibitors. It may stop the growth of cancer cells by blocking Bcl-2, a protein needed for cancer cell survival. Blinatumomab is a monoclonal antibody that may interfere with the ability of cancer cells to grow and spread. Chemotherapy drugs work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Adding venetoclax and/or blinatumomab to standard chemotherapy may be more effective at treating patients with ALL than standard chemotherapy alone, but it may also cause more side effects. This clinical trial evaluates the safety and effectiveness of adding venetoclax and/or blinatumomab to chemotherapy for the treatment of infants with KMT2A-R or KMT2A-G ALL.

Start Date26 Jan 2025

Sponsor / Collaborator

National Cancer Institute

100 Clinical Results associated with Hydrocortisone

100 Translational Medicine associated with Hydrocortisone

100 Patents (Medical) associated with Hydrocortisone

37,465

Literatures (Medical) associated with Hydrocortisone

01 Mar 2025·BIOSENSORS & BIOELECTRONICS

Light source-free smartphone detection of salivary cortisol via colorimetric lateral flow immunoassay using a photoluminescent film

Article

Author: Hong, Donggu ; Choi, Chang Woon ; Kim, Min-Gon

An accurate assay was developed by integrating a novel lateral flow immunoassay (LFIA) design, smartphone-based photoluminescence detection, and computer-aided analysis using machine learning algorithms for the quantitative measurement of cortisol levels in human saliva samples. The unique LFIA strip incorporates a photoluminescent film, which enables photoluminescence detection without an external light source, beneath a nitrocellulose membrane. A smartphone is used to capture images of the LFIA test strips, and specific regions in the captured images are analyzed. The digitized data are then processed using a computer. Machine learning algorithms were employed to interpret the data and quantify cortisol levels in saliva samples obtained from 14 volunteers. The developed assay was shown to be highly accurate, and a low average difference of 18.12% was observed between the predicted cortisol levels and those measured using an established enzyme-linked immunosorbent assay (ELISA) in real saliva samples. The assay has a calculated limit of detection of approximately 139 pg/mL. Furthermore, the strong correlation (r = 0.935) between the results of the developed assay and the ELISA results supports its validity.

01 Mar 2025·PATIENT EDUCATION AND COUNSELING

Embedding an illustrator in the process of co-producing resources to enhance communication and shared decision-making for patients prescribed high-risk medication

Article

Author: James, Delyth H ; Morgan, Amelia Huw

OBJECTIVES:

To examine how embedding an illustrator in the process of co-producing resources to support shared decision-making (SDM) can enhance communication between patients and healthcare professionals (HCPs).

METHODS:

Three case studies were identified involving high-risk medications where the utility of effective visual communication resources is key to SDM due to the risk of information being misinterpreted leading to potential serious adverse effects. An iterative approach was adopted to the phases of co-production, where the Illustrator acted as a conduit between patients and specialist knowledge of the clinical team. The expertise of the Illustrator was harnessed to balance the use of visual and written information, working with the HCPs in co-creating the messaging.

RESULTS:

Case Study 1 relates to the urgent administration of hydrocortisone injection for adrenal crisis in adults and children. Case Study 2 relates to self-management of the need to adjust oral corticosteroid dosing during periods of adrenal insufficiency to prevent adrenal crisis. Case Study 3 focused on women with reproductive potential prescribed or considering sodium valproate for the management of epilepsy. The benefits of involving an Illustrator were evident across all case studies, invoking supportive, empowering and empathic visual communication throughout, leading to removal of some text by the clinical team.

CONCLUSIONS:

Harnessing the expertise of Illustrators as part of a multi-disciplinary team with patients was seen as an enabler for authentic co-production to support an empathic approach to SDM in these three case studies. Further evaluation is needed to assess the impact of the use of visual communication resources of this nature in healthcare, and the impact on patient behaviours and clinical outcomes.

01 Feb 2025·BIOSENSORS & BIOELECTRONICS

Detection of human salivary stress biomarkers using an easy-to-use array sensor based on fluorescent organic molecules

Article

Author: Cavallaro, Alessia ; Pappalardo, Andrea ; Trusso Sfrazzetto, Giuseppe ; Marano, Angela ; Andolina, Manuela ; Tuccitto, Nunzio ; Puglisi, Roberta ; Santonocito, Rossella

During stressful conditions, the human body synthesizes catecholamine neurotransmitters such as dopamine and adrenaline, and cortisol. The monitoring of these three molecules levels is crucial for stress management and holds significant medical applications. Here we developed an analytical device that incorporates a biomimetic (tongue-mimic) associated with an optical physico-chemical transducer, able to detect simultaneously cortisol, dopamine and adrenaline in human saliva without pre-treatments, using an array sensor based on fluorescent chemical receptors (BODIPY, Rhodamine, and Naphthylamides) able to interact by non-covalent interaction with cortisol, adrenaline and dopamine, leading to a change of the emission. Calibration, recovery and selectivity have been performed to validate the device. In particular, the linear responses of the array to concentration range of 1 pM-1 mM of the three analytes were demonstrated by PLS analysis, as well as the high selectivity by PLS-DA analysis performed with artificial saliva samples. Analyses in real human saliva samples, compared to the validated analytical methods, demonstrated that our prototype represents the first point-of-care device able to quantify these three analytes in human saliva with one single analysis in a wider concentration range respect to the other standard methods.

111

News (Medical) associated with Hydrocortisone

20 Dec 2024

·www.globenewswire.com

Eton Pharmaceuticals Closes Acquisition of Increlex® (mecasermin injection)

Acquisition bolsters Eton’s commercial pediatric endocrinology portfolioProduct is now available through AnovoRx, a specialty pharmacy dedicated to serving patients with rare and chronic conditions DEER PARK, Ill., Dec. 20, 2024 (GLOBE NEWSWIRE) -- Eton Pharmaceuticals, Inc (“Eton” or “the Company”) (Nasdaq: ETON), an innovative pharmaceutical company focused on developing and commercializing treatments for rare diseases, today announced that it has completed its previously announced asset purchase of Increlex® (mecasermin injection) from Ipsen S.A. (“Ipsen”). Increlex® is a biologic product used to treat pediatric patients 2 years of age and older who suffer from severe primary insulin-like growth factor 1 deficiency (SPIGFD). “We are excited to close this transformational acquisition and add another important treatment to our commercial portfolio. Increlex® is perfectly aligned with our expertise and strong relationships in pediatric endocrinology and we’re well-positioned to leverage our existing sales team to increase awareness of SPIGFD, an underdiagnosed and undertreated condition,” said Sean Brynjelsen, CEO of Eton Pharmaceuticals. “In the U.S., Increlex® is now available through a specialty pharmacy dedicated to rare and chronic conditions and we are proud to be able to continue supplying this crucial product worldwide without disruption.” Increlex® is a biologic product used to treat pediatric patients 2 years of age and older who suffer from severe primary insulin-like growth factor 1 deficiency (SPIGFD) because their bodies do not make enough insulin-like growth factor 1 (IGF-1). The medicine is approved in 41 territories, including the United States (U.S.) and the European Union (EU). It is estimated that approximately 200 patients in the United States and 900-1,000 patients in Europe live with SPIGFD. Increlex® is the only treatment approved by the U.S. Food and Drug Administration (FDA) or European Medicines Agency (EMA) for SPIGFD. Increlex® is now available in the United States exclusively through AnovoRx, a specialty pharmacy dedicated to serving patients with rare and chronic conditions. AnovoRx will administer the Eton Cares Program in partnership with Eton Pharmaceuticals. The program provides prescription fulfillment, insurance benefits investigation, educational support, financial assistance for qualified patients, and other services designed to help patients access treatment. Eton Cares will offer co-pay assistance to allow for $0 co-pays for qualifying patients. Outside the U.S., Ipsen will continue distributing Increlex® during a six-month transition period, after which Eton will take over commercialization. The transaction was financed by Eton’s cash on hand and an expansion of the Company’s existing credit facility with SWK Holdings. Clinicians seeking to prescribe Increlex® can e-prescribe by selecting AnovoRx #5 or fax in a patient referral form to 855-831-2039. Additional product details can be found on the product website, https://www.increlex.com/en-us. For questions regarding prescription fulfillment, please contact AnovoRx at 1-833-343-2500. Important Safety Information Contraindications Hypersensitivity to mecasermin (rhIGF-1), any of the inactive ingredients in INCRELEX®, or who have experienced a severe hypersensitivity to INCRELEX®. Allergic reactions have been reported, including anaphylaxis requiring hospitalization.Intravenous Administration.Closed Epiphyses.Benign and malignant Neoplasia in pediatric patients with active or suspected neoplasia or medical history with an increased risk of benign or malignant neoplasia. Warnings and Precautions Hypoglycemia: INCRELEX® should be administered 20 minutes before or after a meal or snack and should not be administered when the meal or snack is omitted. Glucose monitoring and INCRELEX® dose titration are recommended until a well-tolerated dose is established and as medically indicated.Intracranial Hypertension: Funduscopic examination is recommended at the initiation of and periodically during the course of therapy.Lymphoid Tissue Hypertrophy: Patients should have periodic examinations to rule out potential complications.Slipped Capital Femoral Epiphysis: Carefully evaluate any pediatric patient with the onset of a limp or hip/knee pain during INCRELEX® therapy.Progression of Scoliosis: Patients with a history of scoliosis, treated with INCRELEX®, should be monitored.Cardiomegaly: An echocardiogram is recommended before initiation and at termination of mecasermin treatment in all patientsBenign and malignant neoplasms: There have been postmarketing reports of malignant neoplasia in pediatric patients who received treatment with INCRELEX®. The tumors were observed more frequently in patients who received INCRELEX® at higher than recommended doses or at doses that produced serum IGF-1 levels above the normal reference ranges for age and sex. Monitor all patients receiving INCRELEX® carefully for development of neoplasms. If malignant neoplasia develops, discontinue INCRELEX® treatment.Risk of Serious Adverse Reactions in Infants due to Benzyl Alcohol Preserved Solution: Serious and fatal adverse reactions including “gasping syndrome” can occur in neonates and infants treated with benzyl alcohol-preserved drugs. Use of INCRELEX® in infants is not recommended as well as in children below 3 years old. Adverse Reactions Common adverse reactions include hypoglycemia, local and systemic hypersensitivity, and tonsillar hypertrophy. U.S. Indication INCRELEX® (mecasermin) is indicated for the treatment of growth failure in pediatric patients aged 2 years and older with severe primary IGF-1 deficiency* (IGFD), or with hormone (GH) gene deletion who have developed neutralizing antibodies to GH. Limitations of use: INCRELEX® is not a substitute to GH for approved GH indications. INCRELEX® is not indicated for use in patients with secondary forms of IGFD, such as GH deficiency, malnutrition, hypothyroidism, or chronic treatment with pharmacologic doses of anti-inflammatory steroids. *Severe primary IGF-1 deficiency (IGFD) is defined by height standard deviation score ≤ -3.0 and basal IGF-1 standard deviation score ≤ -3.0 and normal or elevated GH. Full U.S. Prescribing Information for Increlex® is available at: http://increlex.com/pdf/hcp-full-prescribing-information.pdf You are encouraged to report negative effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088. EU Indication In the European Union, INCRELEX is indicated for the long-term treatment of growth failure in children and adolescents from 2 to 18 years with confirmed severe primary insulin-like growth factor 1 deficiency (Primary IGFD). Severe Primary IGFD is defined by: height standard deviation score <–3.0 and basal IGF-1 levels below the 2.5th percentile for age and gender and GH sufficiency. Exclusion of secondary forms of IGF 1 deficiency, such as malnutrition, hypopituitarism, hypothyroidism, or chronic treatment with pharmacologic doses of anti-inflammatory steroids. Severe Primary IGFD includes patients with mutations in the GH receptor (GHR), post-GHR signaling pathway, and IGF 1 gene defects; they are not GH deficient, and therefore, they cannot be expected to respond adequately to exogenous GH treatment. In some cases, when deemed necessary, the physician may decide to assist in the diagnosis by performing an IGF-I generation test. Detailed information on this medicinal product is available on the website of the European Medicines Agency: http://www.ema.europa.eu About Eton Pharmaceuticals Eton is an innovative pharmaceutical company focused on developing and commercializing treatments for rare diseases. The Company currently has six commercial rare disease products: INCRELEX®, ALKINDI SPRINKLE®, PKU GOLIKE®, Carglumic Acid, Betaine Anhydrous, and Nitisinone. The Company has three additional product candidates in late-stage development: ET-400, ET-600, and ZENEO® hydrocortisone autoinjector. For more information, please visit our website at www.etonpharma.com. Forward-Looking Statements Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including statements associated with the expected ability of Eton to undertake certain activities and accomplish certain goals and objectives. These statements include but are not limited to statements regarding Eton’s business strategy, Eton’s plans to develop and commercialize its product candidates, the safety and efficacy of Eton’s product candidates, Eton’s plans and expected timing with respect to regulatory filings and approvals, and the size and growth potential of the markets for Eton’s product candidates. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as “believes,” “anticipates,” “plans,” “expects,” “intends,” “will,” “goal,” “potential” and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Eton’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks associated with the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such drugs. These and other risks concerning Eton’s development programs and financial position are described in additional detail in Eton’s filings with the Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made. Eton undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made. Investor Relations:Lisa M. Wilson, In-Site Communications, Inc.T: 212-452-2793E: lwilson@insitecony.com

Drug ApprovalAcquisition

17 Dec 2024

·ir.etonpharma.com

Eton Pharmaceuticals Announces Final Readout of PKU GOLIKE® Clinical Trial

« Back to news DEER PARK, Ill., Dec. 17, 2024 (GLOBE NEWSWIRE) -- Eton Pharmaceuticals, Inc. (“Eton” or the “Company”) (Nasdaq: ETON), an innovative pharmaceutical company focused on developing and commercializing treatments for rare diseases announced the full readout and compelling results from the clinical trial evaluating PKU GOLIKE as a protein substitute for the treatment of phenylketonuria (PKU) in patients during prolonged fasting periods. The study demonstrated that PKU GOLIKE, administered as the last daily dose and compared to standard amino acid protein substitutes, improved metabolic control by reducing harmful phenylalanine (Phe) levels and increasing beneficial tyrosine (Tyr) levels, both essential for brain function and metabolic health. PKU patients often experience significant fluctuations in blood Phe levels during prolonged fasting periods, particularly at night, when protein breakdown causes Phe concentrations to peak in the early morning. These fluctuations are associated with cognitive difficulties and overall health impacts, making nighttime metabolic control an important focus in PKU management. The study was sponsored by Relief Therapeutics Holding SA, and was a randomized, crossover, controlled clinical study conducted by the Inherited Metabolic Disorders Unit at Birmingham Children’s Hospital, UK, on pediatric patients with classical PKU, the condition’s most severe form. The trial compared PKU GOLIKE to standard amino acid protein substitutes in managing metabolic parameters during overnight fasting, the longest fasting period within 24 hours. At the end of the one-week treatment period, patients receiving PKU GOLIKE as the last daily protein substitute dose showed a statistically significant reduction in blood Phe levels compared to those receiving standard amino acid substitutes (P=0.0002) and a statistically significant increase in blood Tyr levels (P=0.0113). Compared to baseline levels measured prior to the start of treatment, the PKU GOLIKE group achieved an average 17.8% reduction in blood Phe levels (P=0.0484) and an average 33.8% increase in blood Tyr levels (P=0.0008) upon awakening after the overnight fasting period. In comparison, when treated with standard amino acid protein substitutes, the same patients experienced an average 27.6% increase in blood Phe levels (P=0.0063) and no significant improvement in blood Tyr levels. Blood sample analysis at three early morning time points across the two groups revealed no significant differences in peak Phe levels upon reawakening in either group. Highlighting the clinical significance of the findings, Prof. Anita MacDonald, principal investigator and leading dietitian in inherited metabolic disorders at Birmingham Children’s Hospital, stated: “Giving one dose of PKU GOLIKE as the final daily dose of protein substitute resulted in consistently better metabolic control in our cohort of patients with PKU. They all had classical PKU and were a particularly challenging group to control.” These results confirm that PKU GOLIKE’s prolonged-release profile provides clinically and statistically significant improvements in metabolic control during extended fasting periods compared to standard amino acid protein substitutes. Eton expects these findings to support the adoption of PKU GOLIKE among healthcare providers and within the PKU community. The study findings will be presented in a poster titled A Prolonged-Release Formula Has a Positive Impact on Morning Phenylalanine and Tyrosine Fluctuations in Patients with Classical Phenylketonuria at the 2025 American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting, March 18-22, 2025, in Los Angeles, California. Eton promotes PKU GOLIKE with its existing metabolic sales force, which also promotes Eton’s Carglumic Acid, Betaine, and Nitisinone products. PKU patients’ care is typically overseen by metabolic geneticists and their support staff of nurse practitioners and registered dieticians. Medical formulas for PKU are frequently covered by insurance and are regulated by the FDA as medical food products. Patients and healthcare professionals seeking additional information or requesting a product sample can visit pkugolike.com. For more information on this study (NCT05487378), please visit clinicaltrials.gov. About Eton Pharmaceuticals Eton is an innovative pharmaceutical company focused on developing and commercializing treatments for rare diseases. The Company currently has five commercial rare disease products: ALKINDI SPRINKLE®, PKU GOLIKE®, Carglumic Acid, Betaine Anhydrous, and Nitisinone. The Company has three additional product candidates in late-stage development: ET-400, ET-600, and ZENEO® hydrocortisone autoinjector. For more information, please visit our website at etonpharma.com. About Phenylketonuria (PKU) Phenylketonuria (PKU) is caused by a defect of the enzyme needed to break down phenylalanine (Phe), leading to a toxic buildup of Phe from the consumption of foods containing protein or aspartame. Untreated PKU can result in global developmental delay or severe irreversible intellectual disability, as well as growth failure, hypopigmentation, motor deficits, ataxia and seizures. Living with PKU requires a limited diet and very careful management. If left unmanaged, PKU can lead to devastating consequences, such as brain damage. People living with PKU do not have the ability to metabolize Phe, which is found in many foods, and they require supplementation of amino acid-based phenylalanine-free medical formulas as part of an effort to prevent protein deficiency and optimize metabolic control. Medical formulas used in PKU are challenged to provide a range of amino acids slowly and without a medicinal aftertaste. About PKU GOLIKE® PKU GOLIKE® products are foods for special medical purposes (FSMPs) for the dietary management of PKU in both children and adults for use under medical supervision. Developed with Relief’s proprietary, patent-protected Physiomimic Technology™ drug delivery platform, PKU GOLIKE® products are the first prolonged-release amino acid FSMPs, characterized by a special coating that ensures physiological absorption of the amino acids mirroring that of natural proteins. The special coating also masks the unpleasant taste, odor, and aftertaste of the amino acids. PKU GOLIKE PLUS® granules are flavorless and can be mixed with many foods. PKU GOLIKE® products contain all 19 amino acids that people with PKU need to maintain neurological and muscular health and PKU GOLIKE PLUS® granules are fortified with 27 essential vitamins and minerals, including ones normally found in protein-rich foods like iron, calcium and vitamin B12. The PKU GOLIKE® line of products are available in convenient packets (PKU GOLIKE PLUS® 3-16 and 16+) and medical formula bars (PKU GOLIKE BAR®). PKU GOLIKE® products have been commercially available in the U.S. since October 2022. For more information, visit pkugolike.com. (Please note this site is intended for U.S. audiences only). Forward-Looking Statements Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including statements associated with the expected ability of Eton to undertake certain activities and accomplish certain goals and objectives. These statements include but are not limited to statements regarding Eton’s business strategy, Eton’s plans to develop and commercialize its product candidates, the safety and efficacy of Eton’s product candidates, Eton’s plans and expected timing with respect to regulatory filings and approvals, and the size and growth potential of the markets for Eton’s product candidates. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as “believes,” “anticipates,” “plans,” “expects,” “intends,” “will,” “goal,” “potential” and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Eton’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks associated with the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such drugs. These and other risks concerning Eton’s development programs and financial position are described in additional detail in Eton’s filings with the Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made. Eton undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made. Investor Relations: Lisa M. Wilson, In-Site Communications, Inc. T: 212-452-2793 E: lwilson@insitecony.com Source: Eton Pharmaceuticals, Inc. Source: Eton Pharmaceuticals

Clinical Result

16 Dec 2024

·medcitynews.com

Neurocrine Lands FDA Nod for First New Treatment in Decades for Rare Endocrine Disorder - MedCity News

A rare, inherited endocrine disorder whose standard treatments are steroids that introduce a wide range of side effects now has a new FDA-approved therapy, a non-steroidal Neurocrine Biosciences drug that could give the company another blockbuster seller. The FDA late Friday handed out two approvals for the Neurocrine drug crinecerfont as a treatment for classic congenital adrenal hyperplasia (CAH), a disease that leads to a hormone imbalance that can become fatal. A pill formulation of the San Diego-based biotech’s drug was approved for adults. An oral solution was approved for children age 4 and older. Both formulations will be marketed under the brand name Crenessity. The company claims it’s the first new CAH treatment in 70 years. CAH is an inherited disorder that affects the ability of adrenal glands to produce cortisol, a glucocorticoid hormone that plays a role in tissues and organs throughout the body. Low cortisol levels lead to increased secretion of two other hormones, adrenocorticotropic hormone (ACTH) and androgens. Without treatment, CAH leads to salt wasting (low levels of sodium that affect the brain and kidneys) and dehydration. Extremely low blood levels of cortisol can lead to adrenal crisis, a life-threatening complication. presented by Market Trends to Watch for Health Systems and Their Specialty Pharmacies The future looks bright for the integrated specialty pharmacy model – for health systems, providers, and most importantly, for patients. By Jake Gaffey, MBA, Chief Strategy Officer at Shields Health Solutions Standard CAH treatment includes synthetic versions of glucocorticoids intended to make up for deficient cortisol. But patients need higher doses than what is normally produced by the body to lower levels of ACTH and adrenal androgens. These supraphysiologic doses bring a wide range of complications associated with steroids, including weight gain, diabetes, cardiovascular disease, and osteoporosis. High GC doses can also have psychological and cognitive impacts. Crenessity is an oral small molecule designed to block the corticotropin-releasing factor type 1 receptor. Doing so inhibits secretion of ACTH from the pituitary gland, which in turn reduces production of adrenal androgens. FDA approval of Crenessity is based on results of a global Phase 3 study in adults and children that showed the drug led to significant glucocorticoid reductions compared to placebo after 24 weeks of treatment. The most common adverse reactions reported in the study included fatigue, headache, dizziness, and muscle and joint pain. Neurocrine said these side effects were temporary and mild to moderate in severity. The FDA approval covers the use of Crenessity as an adjunct to glucocorticoid replacement. For adults, the drug is twice daily pill. For children age 4 and older, dosing of the oral solution is according to patient weight. In a note sent to investors Monday, William Blair analyst Myles Minter said the drug’s label is nonrestrictive, permitting treatment of a broad range of CAH patients whether or not clinicians adjust dosing of glucocorticosteroids in patients receiving the two therapies together. Neurocrine said it would disclose Crenessity’s price when the product becomes available later this week. William Blair projects Crenessity will carry a $1,116 per dose price, which works out to about $264,784 per year —falling within the price range of orphan disease drugs. The new Neurocrine drug could achieve about $150 million in 2025 revenue, the firm projects. Peak global sales could reach $1.47 billion depending on regulatory approvals in other markets. Consumer / Employer Health Executives on Digital Transformation in Healthcare Hear executives from Quantum Health, Surescripts, EY, Clinical Architecture and Personify Health share their views on digital transformation in healthcare. By MedCity News “We see significant potential in the CAH market but acknowledge that for this to be a blockbuster $1 billion-plus commercial opportunity, there will need to be both patient and clinician education that supraphysiological glucocorticoid dosing would no longer be the only way to control androgen levels and prevent adrenal crises,” Minter wrote. “With this in mind, we view education activities and partnering with patient advocacy groups like the CARES Foundation as prudent steps ahead of the launch.” Neurocine expanded in endocrine disorders with the 2022 acquisition of Diurnal Group, a Wales-based company whose commercialized products include CAH drug Efmody, a hydrocortisone drug approved for treating CAH in adults and adolescents. At the time of the deal, Crenessity had reached Phase 3 testing. Neurocrine described the Diurnal acquisition as a way to add clinical development and commercial infrastructure in the U.K. The main revenue driver for Neurocrine is Ingrezza, which in 2017 became the first FDA-approved drug for the chronic movement disorder tardive dyskinesia. In the first nine months of 2024, the company reported $1.69 billion in Ingrezza sales, a 27% increase compared to the same period in the prior year. That drug alone accounts for 99% of Neurocrine product revenue, according to the company’s financial reports. FDA approval of Crenessity comes with a priority review voucher that Neurocrine may apply toward speedier regulatory review of a future rare disease drug. But most companies awarded these vouchers sell them to big pharmaceutical companies. Acadia Pharmaceuticals and PTC Therapeutics both received vouchers along with the recent FDA approvals of their respective rare disease drugs. Each voucher sold for $150 million. Illustration: QAI Publishing/Universal Images Group, via Getty Images

Drug ApprovalPhase 3AcquisitionClinical Result

100 Deals associated with Hydrocortisone

External Link

KEGG	Wiki	ATC	Drug Bank
D00088	Hydrocortisone	D07AA02 A01AC03 S02BA01	DB00741

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Adrenal Hyperplasia, Congenital	EU	Neurocrine Biosciences, Inc.	27 May 2021
Adrenal Hyperplasia, Congenital	IS	Neurocrine Biosciences, Inc.	27 May 2021
Adrenal Hyperplasia, Congenital	LI	Neurocrine Biosciences, Inc.	27 May 2021
Adrenal Hyperplasia, Congenital	NO	Neurocrine Biosciences, Inc.	27 May 2021
ACTH Deficiency, Isolated	JP	Pfizer Japan, Inc.	21 Dec 1972
Adrenal Insufficiency	JP	Pfizer Japan, Inc.	21 Dec 1972
Infectious Diseases	JP	Pfizer Japan, Inc.	21 Dec 1972
Thyroiditis, Subacute	JP	Pfizer Japan, Inc.	21 Dec 1972
Colitis, Ulcerative	US	ANI Pharmaceuticals, Inc.	12 Jan 1966
Proctocolitis	US	ANI Pharmaceuticals, Inc.	12 Jan 1966
Ulcerative proctitis	US	ANI Pharmaceuticals, Inc.	12 Jan 1966
Anaphylaxis	US	Pharmacia & Upjohn, Inc.	15 Dec 1952
Collagen Diseases	US	Pharmacia & Upjohn, Inc.	15 Dec 1952
Edema	US	Pharmacia & Upjohn, Inc.	15 Dec 1952
Eye Diseases	US	Pharmacia & Upjohn, Inc.	15 Dec 1952
Gastrointestinal Diseases	US	Pharmacia & Upjohn, Inc.	15 Dec 1952
Hematologic Diseases	US	Pharmacia & Upjohn, Inc.	15 Dec 1952
Neoplasms	US	Pharmacia & Upjohn, Inc.	15 Dec 1952
Respiratory Diseases	US	Pharmacia & Upjohn, Inc.	15 Dec 1952
Rheumatic Diseases	US	Pharmacia & Upjohn, Inc.	15 Dec 1952

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Addison Disease	Phase 2	DE	Diurnal Ltd.	23 Nov 2021
Addison Disease	Phase 2	GB	Diurnal Ltd.	23 Nov 2021
Congenital Adrenal Hyperplasia Due to 21 Hydroxylase Deficiency	Phase 2	US	Diurnal Ltd.	01 Aug 2007

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03062280 (CTgov)	Phase 3	Adrenal Hyperplasia, Congenital	91	Hydrocortisone	grleueoymn(stgxljhqps) = ulfzeynwec jbvhyycnlq (fgdpyuymct, cbkuqqxgkq - fkgtewqhhy) View more	-	24 Oct 2024
NCT03792256 (CTgov)	Phase 1	Recurrent Adult Acute Lymphoblastic Leukemia \| T-Cell Lymphoma \| acute leukemia ... View more	12	ARAC (Stratum 1 With 50 mg/m^2)	lhgcpqfjvf(plbdposrzs) = ihbsdhdkzt gknsaqltxp (ulcdktmgoe, yvxnwrsvrr - ypzolerihv) View more	-	16 Aug 2024
				ARAC (Stratum PK With 50 mg/m^2)	lhgcpqfjvf(plbdposrzs) = xxbhobplmk gknsaqltxp (ulcdktmgoe, daqcjdrvnu - mtfpqafyxw) View more
NCT03832010 (CTgov)	Phase 4	Eczema \| Dermatitis, Atopic	24	Aquaphor+Triamcinolone ointment+Hydrocortisone Ointment+Crisaborole (Crisaborole)	uucutpwhws(utfplosbyl) = pbsptnluce lvxentlddq (ytjmoitswy, bflndpngww - lgpfuczajc) View more	-	19 Jul 2024
				Aquaphor+Triamcinolone ointment+Hydrocortisone Ointment (Vehicle)	uucutpwhws(utfplosbyl) = fbjdswefpg lvxentlddq (ytjmoitswy, gqdwhznrnk - kvxwvxzsgv) View more
ENDO2024	Not Applicable	Cushing Syndrome	131	Hydrocortisone	ncexwmoroq(ciuixvvghs) = bamomqakqw iyactgcnvc (tmfpwiwlhj ) View more	-	01 Jun 2024
				Prednisone	ncexwmoroq(ciuixvvghs) = uivlyrcuew iyactgcnvc (tmfpwiwlhj ) View more
ENDO2024	Not Applicable	Adrenal Insufficiency	23	Oral Hydrocortisone	iqdbggoakw(mpftewkuhe) = ofhdbjpaou oyitxoudtc (gaiyvecbbg )	Positive	01 Jun 2024
ENDO2024	Not Applicable	Addison Disease	-	Continuous Subcutaneous Hydrocortisone Infusion (CSHI) via Insulin Pump	gpxygvbhfh(daxygheszi) = She had hypoglycemic symptoms at night with weakness which she attributed to adrenal crisis. On several occasions her husband had given her a stress dose of HCT 100 mg intramuscularly with improvement in her symptoms qqpjqwadjm (gcshfkhuqw ) View more	-	01 Jun 2024
ATS2024	Not Applicable	Sepsis \| Shock, Septic	-	Hydrocortisone, Ascorbic Acid, and Thiamine (HAT) Therapy	zdldmbmald(wyokvqzudu) = The use of HAT therapy did not increase incidence of gastrointestinal bleeding compared to placebo/SoC vviaemvehe (mmpvapcmzc ) View more	-	19 May 2024
NCT05299554 (PRNewswire) Manual	Phase 3	Adrenal Hyperplasia, Congenital	-	Chronocort	rbehtkzqfy(umujemrdmx) = MRHC demonstrated improved control of CAH, with an ability to closely replicate cortisol diurnal rhythm when compared to current glucocorticoid treatment pfibftylkj (rkbzadlnds )	Positive	14 May 2024
NCT05222152 (CHAMPAIN, PRNewswire) Manual	Phase 2	Addison Disease	49	Modified-Release Hydrocortisone	yyaccbkcvu(sjccqramcl) = uxymfqpnaf iwkgkrksdw (awpkbxhqcp, 203.50) View more	Positive	14 May 2024
				Plenadren	yyaccbkcvu(sjccqramcl) = ldjnuzwnwn iwkgkrksdw (awpkbxhqcp, 113.87) View more
NCT00625209 (APROCCHSS, Pubmed) Manual	Phase 3	Shock, Septic	1,210	Hydrocortisone plus fludrocortisone	mqxdqfscom(genizfhqxx) = htwumlvgnl dnujtstddy (uxnwdwryiu ) View more	Positive	01 May 2024
				Placebo	mqxdqfscom(genizfhqxx) = kqhcxoiygn dnujtstddy (uxnwdwryiu ) View more

Translational Medicine

Boost your research with our translational medicine data.

view full example data

Deal

Boost your decision using our deal data.

view full example data

Core Patent

Boost your research with our Core Patent data.

view full example data

Clinical Trial

Identify the latest clinical trials across global registries.

view full example data

Approval

Accelerate your research with the latest regulatory approval information.

view full example data

Regulation

Understand key drug designations in just a few clicks with Synapse.

view full example data

Chat with Hiro

Get started for free today!

Accelerate Strategic R&D decision making with Synapse, PatSnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.

Start your data trial now!

Synapse data is also accessible to external entities via APIs or data packages. Empower better decisions with the latest in pharmaceutical intelligence.

Bio

Bio Sequences Search & Analysis

Chemical

Chemical Structures Search & Analysis