Comparative analysis of tapered short course deflazacort and acetaminophen versus acetaminophen in treatment of early stage periarthritis of shoulder in adults.

开始日期2022-02-06

申办/合作机构-

CTRI/2021/08/035475 / Not yet recruiting临床4期IIT

Evaluation of the Efficacy and Safety of Deflazacort in Stone Clearance after Extracorporeal Shockwave Lithotripsy when used along with Oral Hydration and Alpha Blockers â?? A Randomized Clinical Trial

开始日期2021-08-10

申办/合作机构-

100 项与地夫可特相关的临床结果

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100 项与地夫可特相关的转化医学

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100 项与地夫可特相关的专利（医药）

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516

项与地夫可特相关的文献（医药）

2024-01-20·Human molecular genetics

Comparison of pharmaceutical properties and biological activities of prednisolone, deflazacort, and vamorolone in DMD disease models

Article

作者: Braughton, Bruce ; Yeh, Shirley ; Narasimhan, Jana ; Liu, Grace ; Jung, Stephen ; Weetall, Marla ; Lennox, William ; Yurkow, Edward ; Voronin, Gregory ; Li, Wencheng ; Rice, Cari ; More, Vijay ; Huarte, Eduardo ; Moon, Young-Choon ; Lipari, Philip ; Gruszka, Barbara ; Mollin, Anna ; Buckendahl, Patricia ; Teplova, Irina ; Ying, Lanqing ; Welch, Ellen ; Ray, Balmiki ; Goodwin, Elizabeth ; McGann, Eric

Abstract:

Duchenne muscular dystrophy (DMD) is a progressive disabling X-linked recessive disorder that causes gradual and irreversible loss of muscle, resulting in early death. The corticosteroids prednisone/prednisolone and deflazacort are used to treat DMD as the standard of care; however, only deflazacort is FDA approved for DMD. The novel atypical corticosteroid vamorolone is being investigated for treatment of DMD. We compared the pharmaceutical properties as well as the efficacy and safety of the three corticosteroids across multiple doses in the B10-mdx DMD mouse model. Pharmacokinetic studies in the mouse and evaluation of p-glycoprotein (P-gP) efflux in a cellular system demonstrated that vamorolone is not a strong P-gp substrate resulting in measurable central nervous system (CNS) exposure in the mouse. In contrast, deflazacort and prednisolone are strong P-gp substrates. All three corticosteroids showed efficacy, but also side effects at efficacious doses. After dosing mdx mice for two weeks, all three corticosteroids induced changes in gene expression in the liver and the muscle, but prednisolone and vamorolone induced more changes in the brain than did deflazacort. Both prednisolone and vamorolone induced depression-like behavior. All three corticosteroids reduced endogenous corticosterone levels, increased glucose levels, and reduced osteocalcin levels. Using micro-computed tomography, femur bone density was decreased, reaching significance with prednisolone. The results of these studies indicate that efficacious doses of vamorolone, are associated with similar side effects as seen with other corticosteroids. Further, because vamorolone is not a strong P-gp substrate, vamorolone distributes into the CNS increasing the potential CNS side-effects.

2024-01-01·Cureus

Medical Management to Treat Chronic Non-healing Ulcers: A Case Series

Article

作者: Kokkatt, Jose K ; Jose, Joseph ; Soni, Bhanushree ; Jose, Saji

INTRODUCTION:

Chronic non-healing leg ulcers are skin defects below the knee that resist healing for more than six weeks. They cause physical, emotional, and economic burdens to patients and society.

OBJECTIVES:

To introduce an innovative medical strategy that targets the chronic inflammation component in non-healing ulcers (NHUs) with rheumatic features and to evaluate its potential effectiveness in achieving complete healing.

METHODS:

We employed an empirical medical therapy regimen, which combined medications like deflazacort, colchicine, dapsone, hydroxychloroquine, and azathioprine. We retrospectively selected 25 patients with chronic pedal ulcers who underwent our therapy.

RESULTS:

The mean duration of ulcers was 7.84 years, and the time to heal was 5.97 months. Among 25 patients, 19 had atypical ulcers, four had venous ulcers, and two had diabetic neuropathy ulcers. Four patients with venous ulcers additionally underwent endovenous laser ablation.

CONCLUSION:

Our medical strategy showed promising results in healing chronic NHUs with rheumatic features without significant steroid-induced adverse effects.

2023-12-01·Annals of clinical and translational neurology

Comparison of two corticosteroid regimens on brain volumetrics in patients with Duchenne muscular dystrophy

Article

作者: Doorenweerd, Nathalie ; Kan, Hermien E ; Govaarts, Rosanne ; Geuens, Sam ; Ikelaar, Nadine A ; Goemans, Nathalie ; Van Dessel, Jeroen ; Niks, Erik H ; De Waele, Liesbeth ; Meijer, Onno C ; Lemiere, Jurgen

Abstract:

Objective:

Duchenne muscular dystrophy (DMD) is a neuromuscular disorder in which many patients also have neurobehavioral problems. Corticosteroids, the primary pharmacological treatment for DMD, have been shown to affect brain morphology in other conditions, but data in DMD are lacking. This study aimed to investigate the impact of two corticosteroid regimens on brain volumetrics in DMD using magnetic resonance imaging (MRI).

Methods:

In a cross‐sectional, two‐center study, T1‐weighted MRI scans were obtained from three age‐matched groups (9–18 years): DMD patients treated daily with deflazacort (DMDd, n = 20, scan site: Leuven), DMD patients treated intermittently with prednisone (DMDi, n = 20, scan site: Leiden), and healthy controls (n = 40, both scan sites). FSL was used to perform voxel‐based morphometry analyses and to calculate intracranial, total brain, gray matter, white matter, and cerebrospinal fluid volumes. A MANCOVA was employed to compare global volumetrics between groups, with site as covariate.

Results:

Both patient groups displayed regional differences in gray matter volumes compared to the control group. The DMDd group showed a wider extent of brain regions affected and a greater difference overall. This was substantiated by the global volume quantification: the DMDd group, but not the DMDi group, showed significant differences in gray matter, white matter, and cerebrospinal fluid volumes compared to the control group, after correction for intracranial volume.

Interpretation:

Volumetric differences in the brain are considered part of the DMD phenotype. This study suggests an additional impact of corticosteroid treatment showing a contrast between pronounced alterations seen in patients receiving daily corticosteroid treatment and more subtle differences in those treated intermittently.

项与地夫可特相关的新闻（医药）

2024-06-17

·PRNewswire

Pfizer's Setback in Duchenne Muscular Dystrophy Treatment Trial Clears Path for Sarepta, and Many Other Pharma Companies | DelveInsight

Pfizer's mini-dystrophin gene therapy fordadistrogene movaparvovec failure in the Duchenne muscular dystrophy treatment opens new opportunities for several pharmaceutical companies such as Sarepta Therapeutics (SRP-5051), Santhera Pharmaceuticals/ReveraGen BioPharma (Vamorolone), Taiho Pharmaceutical (TAS-205), FibroGen (Pamrevlumab), and others, to advance their therapies and potentially dominate the DMD market. LAS VEGAS, June 17, 2024 /PRNewswire/ -- Duchenne muscular dystrophy is the most prevalent type of muscular dystrophy found in children. This genetic condition leads to ongoing muscle weakening and degeneration. It is one among nine different forms of muscular dystrophy. According to DelveInsight's estimates, the US accounted for around ~17K total prevalent cases of Duchenne muscular dystrophy in 2023. The highest cases were found in the children of the 5-9 age group, these numbers are expected to grow by the year 2034. As per DelveInsight's analysis, the most commonly associated comorbidities accompanied with DMD included the highest cases of scoliosis accounting for around ~20% of cases followed by ADHD, cardiomyopathy, and obsessive-compulsive disorders accounting for around ~19%, ~18%, and ~15% cases respectively in the US in the year 2023. The standard of care for DMD typically involves a combination of corticosteroids and surgical interventions. Prednisone and deflazacort are commonly prescribed glucocorticoids that have been utilized for over two decades to increase muscular strength in DMD patients. Surgery may be necessary to address contractures, spinal curvature, or scoliosis, which can affect breathing and overall mobility. Additionally, interventions such as pacemakers or other cardiac devices may be utilized to improve heart function. Sarepta Therapeutics is a key player in the DMD market, boasting three approved assets and two in the current pipeline. Despite its dominance, emerging competition is evident. The historical success rate for market entry varies across the 7MM. Notably, the DMD market exhibits geographic disparities in regulatory approvals, with instances of products approved in one jurisdiction and rejected in another, despite identical clinical data, reflecting the distinct standards of regulatory bodies such as EMA and FDA. Learn more about the FDA-approved DMD drugs @ Drugs for Duchenne Muscular Dystrophy Treatment The current US DMD treatment market possesses approved products, EMFLAZA (deflazacort), VYONDYS 53 (golodirsen), EXONDYS 51 (eteplirsen), AMONDYS 45 (casimersen), and VILTEPSO (viltolarsen), ELEVIDYS (delandistrogene moxeparvovec) in patients with DMD. In January 2024, Santhera Pharmaceuticals announced the introduction of its AGAMREE (vamorolone) to treat DMD patients aged four years and above in Germany. Santhera Pharmaceuticals has officially entered the commercial stage of its biopharma journey with the launch of this drug in Germany. ELEVIDYS, the first FDA-approved gene therapy for DMD, received accelerated approval in June 2023. However, ICER recently challenged the potential conversion of Sarepta's gene therapy to full approval, citing concerns about the surrogate endpoint used in the approval process. In the EU4 and the UK, the current market is dominated by steroid therapies along with an approved medication targeting DMD patients with the nonsense mutation, TRANSLARNA (ataluren). In Japan, the only approved treatment is VILTEPSO (viltolarsen). ELEVIDYS is a prescription gene therapy designed for ambulatory children aged 4 to 5 years with DMD and a confirmed dystrophin gene mutation. It received accelerated approval. Patients will take oral corticosteroids before and after receiving ELEVIDYS and will have weekly blood tests to monitor liver enzyme levels for three months post-treatment. In June 2023, the USFDA approved ELEVIDYS as the first gene therapy for treating pediatric patients aged 4-5 years with Duchenne Muscular Dystrophy and a confirmed DMD gene mutation. VYONDYS 53, developed by Sarepta Therapeutics, is prescribed for treating DMD in patients with a specific mutation of the DMD gene that can be addressed by exon 53 skipping. This approval is under the accelerated approval pathway, based on the observed increase in dystrophin production in the skeletal muscle of patients treated with VYONDYS 53. The FDA approved VYONDYS 53 (golodirsen) in December 2019. Additionally, the FDA granted VYONDYS 53 (golodirsen) New Chemical Entity (NCE) exclusivity until December 2024 and Orphan Drug Exclusivity until December 2026. To know more about DMD treatment options, visit @ New Treatment for Duchenne Muscular Dystrophy The DMD market is crowded with so many companies including FibroGen, Santhera Pharmaceutical, Italfarmaco, ReveraGen BioPharma, Cumberland Pharmaceuticals, Sarepta Therapeutics, Antisense Therapeutics, Capricor Therapeutics, and others are running clinical trials to improve the treatment space. The DMD pipeline possesses potential drugs in mid-stage developments to be launched shortly. Some of the promising drugs in the pipeline include Pizuglanstat (TAS-205), SRP-5051, Allogeneic Cardiosphere-derived Cells (CAP-1002), ITF2357 (Givinostat), and others that hold the potential to create a significant positive shift in the DMD market size. Discover which therapies are expected to grab major DMD market share @ Duchenne Muscular Dystrophy Market Report Italfarmaco is developing Givinostat (ITF2357), an oral small molecule that functions as a histone deacetylase inhibitor with potential anti-inflammatory, anti-angiogenic, and antineoplastic properties. ITF2357 increases the production of follistatin protein in muscle cells by inhibiting the HDAC enzyme, leading to increased muscle mass and prevention of muscle degeneration. This action counters the effects of myostatin, helping to alleviate the symptoms of DMD. Givinostat is currently in Phase III trials. The FDA has granted it Orphan Drug, Rare Pediatric Disease, and Fast Track designations for DMD treatment. Additionally, the European Commission has designated Givinostat as an Orphan Medicinal Product for DMD treatment. TAS-205 (pizuglanstat) is a selective inhibitor of hematopoietic prostaglandin D synthase (HPGDS), developed by Taiho Pharmaceutical. It is being advanced as a treatment for Duchenne muscular dystrophy (DMD), effective irrespective of the type of dystrophin gene mutation. By inhibiting HPGDS, which worsens the inflammatory response in the muscles of DMD patients, it helps control the decline in motor function. Currently, it is in Phase III of clinical trials. Discover more about drugs for DMD in development @ Duchenne Muscular Dystrophy Clinical Trials The anticipated launch of these emerging therapies for Duchenne muscular dystrophy are poised to transform the market landscape in the coming years. As these cutting-edge therapies continue to mature and gain regulatory approval, they are expected to reshape the Duchenne muscular dystrophy market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth. DelveInsight estimates that the market size for DMD is expected to grow from USD 2.1 billion in 2023 with a significant CAGR by 2034. The growth of the DMD market is expected to be mainly driven by increased diagnosed incidence, patient awareness, and a robust clinical pipeline during the forecast period (2024–2034). DelveInsight's latest published market report titled as Duchenne Muscular Dystrophy Market Insight, Epidemiology, and Market Forecast – 2034 will help you to discover which market leader is going to capture the largest market share. The report provides comprehensive insights into the DMD country-specific treatment guidelines, patient pool analysis, and epidemiology forecast to help understand the key opportunities and assess the market's underlying potential. The DMD market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into: Total Prevalent Cases of Duchenne Muscular Dystrophy Age-specific Cases of Duchenne Muscular Dystrophy Ambulatory and Non-ambulatory Cases of Duchenne Muscular Dystrophy Mutation-specific Cases of Duchenne Muscular Dystrophy Associated Comorbidities in Duchenne Muscular Dystrophy The report provides an edge while developing business strategies by understanding trends shaping and driving the 7MM DMD market. Highlights include: 11-year Forecast 7MM Analysis Epidemiology-based Market Forecasting Historical and Forecasted Market Analysis upto 2034 Emerging Drug Market Uptake Peak Sales Analysis Key Cross Competition Analysis Industry Expert's Opinion Access and Reimbursement Download this DMD market report to assess the epidemiology forecasts, understand the patient journeys, know KOLs' opinions about the upcoming treatment paradigms, and determine the factors contributing to the shift in the DMD market. Also, stay abreast of the mitigating factors to improve your market position in the DMD therapeutic space. Related Reports Duchenne Muscular Dystrophy Epidemiology Forecast Duchenne Muscular Dystrophy Epidemiology Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted Duchenne muscular dystrophy epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan. Duchenne Muscular Dystrophy Pipeline Duchenne Muscular Dystrophy Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key Duchenne muscular dystrophy companies, including Santhera Pharmaceuticals, Sarepta Therapeutics, Italfarmaco, Wave Life Sciences Ltd, FibroGen, Edgewise Therapeutics, Pfizer, Daiichi Sankyo, Sarepta Therapeutics, Inc., ENCell, Taiho Pharmaceutical, Solid Biosciences, Capricor, Nippon Shinyaku, Hansa Biopharma, Ultragenyx Pharmaceutical, Dyne Therapeutics, Entrada Therapeutics, AAVogen, PepGen, Antisense Therapeutics, BioMarin Pharmaceutical, Avidity Biosciences, Sarepta Therapeutics, Dyne Therapeutics, Solid Biosciences Inc, Regenxbio, Stealth BioTherapeutics, among others. Nonsense Mutation Duchenne Muscular Dystrophy Pipeline Nonsense Mutation Duchenne Muscular Dystrophy Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key nonsense mutation Duchenne muscular dystrophy companies, including Taiho Pharmaceutical, Solid Biosciences, Capricor, Nippon Shinyaku, among others. Becker Muscular Dystrophy Pipeline Becker Muscular Dystrophy Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key becker muscular dystrophy companies, including Epirium Bio, Ultragenyx, Strykagen, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP

临床3期上市批准孤儿药快速通道

2024-06-14

·drugs

FDA Approves Generic Emflaza Oral Suspension for Duchenne Muscular Dystrophy

FRIDAY, June 14, 2024 -- The U.S. Food and Drug Administration has approved the first generic version of Emflaza (deflazacort) oral suspension for Duchenne muscular dystrophy (DMD). Deflazacort oral suspension is a corticosteroid indicated to treat DMD in patients 5 years of age and older but is contraindicated in patients with known hypersensitivity to deflazacort. The most common adverse reactions reported in patients were Cushingoid appearance, weight increase, increase in appetite, upper respiratory tract infection, cough, pollakiuria, hirsutism, central obesity, and nasopharyngitis. "Duchenne muscular dystrophy is a devastating rare disease, and with limited treatment options available there is a critical need for the greater accessibility that a generic therapy can bring," Ketan Mehta, the founder and CEO of Tris Pharma, said in a statement. "This FDA approval is a significant milestone for the patients, caregivers, and physicians who may depend on this medication to treat DMD." Approval of the generic version of Emflaza oral suspension was granted to Cranbury Pharmaceuticals, a subsidiary of Tris Pharma. More Information Whatever your topic of interest, subscribe to our newsletters to get the best of Drugs.com in your inbox.

上市批准

2024-06-12

·BioSpace

Cranbury Pharmaceuticals Receives U.S. FDA Approval for First Generic Version of Emflaza® Oral Suspension (deflazacort) for Duchenne Muscular Dystrophy

– Cranbury, a new subsidiary of Tris Pharma, will market more than 20 generic medicines and advance a diverse pipeline transitioned from Tris – – Deflazacort oral suspension is indicated to treat patients five years of age and older for Duchenne muscular dystrophy, a rare disease with limited treatment options available – – Immediate commercial availability of therapy underscores Cranbury’s commitment to providing patients with access to effective generic medicines across multiple disease states – MONMOUTH JUNCTION, N.J.--(BUSINESS WIRE)-- Cranbury Pharmaceuticals (Cranbury), a wholly-owned subsidiary of Tris Pharma, today announced the U.S. Food and Drug Administration (FDA) approved the Abbreviated New Drug Application (ANDA) for the first generic version of Emflaza® oral suspension (deflazacort) for the treatment of Duchenne muscular dystrophy (DMD). The generic deflazacort oral suspension is now available to patients in the United States. This press release features multimedia. View the full release here: Deflazacort oral suspension is indicated to treat DMD in patients five years of age and older. DMD is a rare genetic disorder characterized by progressive muscle degeneration and weakness. The disease primarily affects boys, with symptom onset in early childhood. DMD occurs in approximately 1 in 5,000 live male births, and it is estimated that about 20,000 children are diagnosed with DMD globally each year.1 “Tris Pharma founded Cranbury as part of the company’s continued commitment to develop high-quality medicines and make them accessible to individuals who need them,” said Janet Penner, president at Cranbury Pharmaceuticals. “The launch of deflazacort oral suspension aligns with this commitment, and we are proud to provide this critical therapy to those with DMD. As a tenured member of the Tris team, I am honored to take on the role of president of our new subsidiary and have the opportunity to provide important generic medicines to patients with a range of disorders and diseases.” Tris founded Cranbury Pharmaceuticals with the vision to create high-quality, patient-friendly generic medicines that leverage Tris’ strengths in product selection, development and commercial launch. Cranbury will market more than 20 generic medicines and advance a diverse pipeline with the potential to address a range of disorders and diseases. The company will operate out of Tris’ headquarters in Monmouth Junction, New Jersey. “Duchenne muscular dystrophy is a devastating rare disease, and with limited treatment options available there is a critical need for the greater accessibility that a generic therapy can bring,” said Ketan Mehta, founder and CEO at Tris Pharma. “This FDA approval is a significant milestone for the patients, caregivers and physicians who may depend on this medication to treat DMD. Our portfolio of differentiated, high-quality generic medications has always been core to our business at Tris, and I look forward to continuing to expand our generic offerings under our new subsidiary, Cranbury Pharmaceuticals.” The spinout of Cranbury Pharmaceuticals follows Tris Pharma's recent launch of Tris Digital Health, a business focused on the development and commercialization of digital diagnostic and therapeutic products for neurological health conditions. The creation of these subsidiaries within Tris facilitates important progress and enables significant continued growth for the company’s digital health and generics portfolios. Tris Pharma will continue to focus on building its commercial portfolio of approved, best-in-class ADHD products and advancing its late-stage pipeline of innovative therapeutics in pain, ADHD, neurological disorders and addiction. INDICATION DEFLAZACORT oral suspension is a corticosteroid indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients 5 years of age and older. IMPORTANT SAFETY INFORMATION Contraindications: DEFLAZACORT is contraindicated in patients with known hypersensitivity to deflazacort or to any of the inactive ingredients in deflazacort oral suspension. Warnings & Precautions Alterations in Endocrine Function: Corticosteroids, such as DEFLAZACORT, oral suspension, can cause serious and life-threatening alterations in endocrine function, especially with chronic use. Monitor patients receiving DEFLAZACORT oral suspension for Cushing’s syndrome, hyperglycemia, and adrenal insufficiency after deflazacort oral suspension withdrawal. In addition, patients with hypopituitarism, primary adrenal insufficiency, or congenital adrenal hyperplasia, altered thyroid function, or pheochromocytoma may be at increased risk for adverse endocrine events. Acute adrenal insufficiency or “withdrawal syndrome” can occur if corticosteroids are withdrawn abruptly and can be fatal. The risk is reduced by gradually tapering the corticosteroid dose when withdrawing treatment. During times of medical stress, corticosteroid dosage may need to be increased. Immunosuppression and Increased Risk of Infection: Increased risk of new, exacerbation, dissemination, or reactivation of latent infections, which can be severe and at times fatal; Signs and symptoms of infection may be masked. Tell patients and/or caregivers to inform their healthcare provider if the patient has had recent or ongoing infections or if they have recently received a vaccine. Warn patients who are on corticosteroids who have not had chickenpox or measles to avoid exposure to chickenpox or measles and to alert their healthcare provider immediately if they are exposed. Alterations in Cardiovascular/Renal Function: Corticosteroids, including DEFLAZACORT oral suspension, can cause elevation of blood pressure, salt, and water retention, and increased excretion of potassium and calcium. DEFLAZACORT oral suspension should be used with caution in patients with congestive heart failure, hypertension, or renal insufficiency. Monitor for elevated blood pressure. Dietary salt restriction and potassium supplementation may be needed. Gastrointestinal Perforation: Increased risk of gastrointestinal perforation during corticosteroid use in patients with certain gastrointestinal disorders such as active or latent peptic ulcers, diverticulitis, recent intestinal anastomoses, and non-specific ulcerative colitis. Signs and symptoms may be masked. Avoid corticosteroids if there is a probability of impending perforation, abscess, or other pyogenic infections; diverticulitis; fresh intestinal anastomoses; or active or latent peptic ulcer. Behavioral and Mood Disturbances: Potentially severe psychiatric adverse reactions may occur with systemic corticosteroids, including DEFLAZACORT oral suspension. Psychiatric adverse reactions usually involve hypomanic or manic symptoms (e.g., euphoria, insomnia, mood swings) during treatment and depressive episodes after discontinuation of treatment. Encourage patients to seek medical attention if symptoms develop or worsen. especially if depressed mood or suicidal ideation is suspected. Effects on Bones: Corticosteroids, including DEFLAZACORT oral suspension, decrease bone formation and increase bone resorption. The risk of osteoporosis increases with prolonged use of DEFLAZACORT, which can predispose patients to vertebral and long bone fractures. Consider a patient’s risk of osteoporosis before initiating corticosteroid therapy. Monitor bone mineral density in patients on long-term treatment with DEFLAZACORT, oral suspension. Ophthalmic Effects: May cause cataracts, infections, and glaucoma; Monitor intraocular pressure if DEFLAZACORT oral suspension is continued for more than 6 weeks. Vaccination: Do not administer live or live attenuated vaccines to patients receiving immunosuppressive doses of corticosteroids. Administer live attenuated or live vaccines at least 4 to 6 weeks prior to starting DEFLAZACORT oral suspension. Serious Skin Rashes: Toxic epidermal necrolysis has been reported with the use of deflazacort with symptoms beginning within 8 weeks of starting treatment. Discontinue at the first sign of rash, unless the rash is clearly not drug related. Effects on Growth and Development: Long-term use of corticosteroids, including DEFLAZACORT oral suspension, can have negative effects on growth and development in children. Thromboembolic Events: Observational studies have shown an increased risk of thromboembolism. Use DEFLAZACORT oral suspension with caution in patients who have or may be predisposed to thromboembolic disorders. Adverse Reactions: The most common adverse reactions (≥10% for DEFLAZACORT Oral suspension and greater than placebo) are Cushingoid appearance, weight increased, increased appetite, upper respiratory tract infection, cough, pollakiuria, hirsutism, central obesity, and nasopharyngitis. Drug Interactions: Give one third of the recommended dosage of DEFLAZACORT oral suspension. Avoid use of moderate or strong CYP3A4 inducers with deflazacort oral suspension, as they may reduce efficacy. To report SUSPECTED ADVERSE REACTIONS, contact Tris Pharma, Inc. at 1-732-940-0358 or FDA at 1-800-FDA-1088 or Please see Full Prescribing Information here. About Cranbury Pharmaceuticals Cranbury Pharmaceuticals is a subsidiary of Tris Pharma focused on the development and commercialization of high-quality, patient-friendly products that leverage the company’s strengths in product selection, development and commercial launch. Driven by patient-focused innovation, Cranbury will market more than 20 generic products and advance a diverse pipeline ready to transform new ideas into approved, effective medicines. Learn more at . About Tris Pharma Tris Pharma is a privately held, innovation-driven biopharmaceutical company that is leveraging its drug development capabilities and proprietary technologies to transform the treatment of ADHD, pain and neurological disorders, including addiction and diseases of the central nervous system. Tris is an established commercial organization with a robust portfolio of best-in-class ADHD products and a promising pipeline of differentiated, near-term drug candidates. More information is available at and on LinkedIn @TrisPharma. References 1 Muscular Dystrophy Association. What is… Duchenne Muscular Dystrophy [Fact sheet]. .

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100 项与地夫可特相关的药物交易

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KEGG	Wiki	ATC	Drug Bank
D03671	地夫可特	H02AB13	DB11921

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适应症	国家/地区	公司	日期
杜氏肌营养不良症	意大利	-	1985-01-01

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适应症	最高研发状态	国家/地区	公司	日期
肢带型肌营养不良2I型	临床3期	美国	PTC Therapeutics, Inc.	2019-10-31
肢带型肌营养不良2I型	临床3期	加拿大	PTC Therapeutics, Inc.	2019-10-31
肢带型肌营养不良2I型	临床3期	丹麦	PTC Therapeutics, Inc.	2019-10-31
肢带型肌营养不良2I型	临床3期	法国	PTC Therapeutics, Inc.	2019-10-31
肢带型肌营养不良2I型	临床3期	德国	PTC Therapeutics, Inc.	2019-10-31
肢带型肌营养不良2I型	临床3期	挪威	PTC Therapeutics, Inc.	2019-10-31
肢带型肌营养不良2I型	临床3期	俄罗斯	PTC Therapeutics, Inc.	2019-10-31
肢带型肌营养不良2I型	临床3期	瑞典	PTC Therapeutics, Inc.	2019-10-31
肝病	临床1期	美国	PTC Therapeutics, Inc.	2014-12-01
肾功能不全	临床1期	美国	PTC Therapeutics, Inc.	2014-12-01

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT01603407 (CTgov)	临床3期	杜氏肌营养不良症	196	Prednisone (Daily Prednisone)	鏇觸範衊餘鏇夢壓鹽艱(鏇觸鏇願獵衊構糧餘積) = 醖醖窪壓願製窪廠鹹廠淵餘鹹簾積簾積夢齋齋 (製積襯繭製憲淵製蓋顧, 蓋構窪觸積夢願齋窪艱 ~ 範夢憲艱鑰窪願鏇鑰鹹) 更多	-	2022-08-12
				Deflazacort (Daily Deflazacort)	鏇觸範衊餘鏇夢壓鹽艱(鏇觸鏇願獵衊構糧餘積) = 餘憲餘襯範積鏇憲壓鏇淵餘鹹簾積簾積夢齋齋 (製積襯繭製憲淵製蓋顧, 憲網鬱積觸艱繭鹽築夢 ~ 壓淵獵襯蓋鬱鏇範製繭) 更多
NCT03783923 (CTgov)	临床3期	肢带型肌营养不良2I型	11	Deflazacort	願鬱糧願積選壓觸構簾(選衊鑰選壓鹹觸觸觸憲) = 繭選網壓鏇範簾窪獵選蓋範鏇齋構選糧製壓鏇 (範齋繭顧製艱鏇獵築襯, 鹹襯窪夢獵鹽憲廠製選 ~ 顧獵鑰壓遞艱廠壓窪齋) 更多	-	2022-06-27
PRO-DMD-01 and Cooperative International Neuromuscular Research Group (CINRG) (MDA2022)	N/A	杜氏肌营养不良症	463	Deflazacort	繭齋繭鬱夢獵艱膚遞顧(鹹鹽遞顧鑰獵鬱膚膚構) = 願願餘製鹽鬱鬱艱構膚積鬱顧鬱艱繭鏇顧簾淵 (憲夢網襯範醖蓋憲築網 ) 更多	积极	2022-03-13
				Prednisone	顧築夢獵選選構繭壓構(襯願遞繭衊築憲襯餘壓) = 衊願壓觸簾膚蓋範糧鹹壓鹹襯襯鑰廠夢願範糧 (襯蓋簾壓願鹹構窪蓋壓 )
MDA2022	N/A	杜氏肌营养不良症	328	Deflazacort	築窪鬱築淵壓願積積選(繭顧齋憲築願襯構製鹹) = 膚網顧簾窪壓膚製觸鏇蓋膚淵窪積鏇廠艱糧願 (鑰蓋選遞淵繭鑰簾觸夢 )	积极	2022-03-13
1847 (AAN2021)	N/A	杜氏肌营养不良症	97	Deflazacort 0.45mg/kg/day	膚齋衊鬱網蓋築餘顧遞(壓鹹艱鑰鏇網窪夢醖構) = 顧遞憲網積醖製願齋網繭襯齋鏇網製鏇醖鑰鬱 (膚齋夢鏇壓簾築鹹簾選, 41.5)	不佳	2021-04-13
				Deflazacort 0.9mg/kg/day	膚齋衊鬱網蓋築餘顧遞(壓鹹艱鑰鏇網窪夢醖構) = 願糧醖製蓋鏇醖鬱獵簾繭襯齋鏇網製鏇醖鑰鬱 (膚齋夢鏇壓簾築鹹簾選, 44)
4950 (AAN2020)	N/A	杜氏肌营养不良症	200	Deflazacort	膚遞選餘餘鑰製顧構襯(願鏇積醖觸築憲壓鏇積) = 網鹹壓範鏇齋糧簾艱艱膚繭糧襯願壓範壓構獵 (糧範壓觸窪網簾廠壓製 ) 更多	积极	2020-04-14
				Prednisone	鹹齋窪觸醖鏇鏇夢衊積(鏇糧選窪鹹膚餘齋積襯) = 壓淵獵膚鑰網繭窪獵糧鏇蓋蓋廠鏇網鹽鬱遞願 (壓獵淵選觸獵範齋鹽觸 ) 更多
P1.6-066 (AAN2019)	临床3期	杜氏肌营养不良症	-	Deflazacort	構繭鑰膚蓋窪選顧夢廠(鹽憲憲蓋築窪鹽鬱鏇鹽) = 顧鬱構選積膚淵構蓋醖鬱醖顧積鬱繭築積願齋 (夢鬱壓製築壓餘艱窪簾, (0.5 ~ 4.1)) 更多	积极	2019-04-09
AAN2018	N/A	杜氏肌营养不良症	-	Deflazacort	製願顧襯餘糧淵鑰顧醖(蓋糧醖鑰廠餘願獵壓簾) = 襯齋夢醖廠襯夢鬱廠膚蓋鬱鑰鬱壓網簾憲遞積 (鏇糧廠醖積願襯觸膚鹽, 13.5)	-	2018-04-10
				Prednisone/Prednisolone	-
CNS2016	临床3期	杜氏肌营养不良症	196	Deflazacort 0.9 mg/kg/day	餘鏇簾齋齋願襯範鹽鬱(餘範範廠築醖簾鹹積夢) = 膚範選醖糧網鹹繭觸襯衊構獵窪鬱醖鹹繭遞壓 (壓繭鬱窪鏇鬱遞鹹夢遞 )	-	2016-10-14
				Deflazacort 1.2 mg/kg/day	淵膚衊製願積窪鬱壓艱(壓醖壓窪觸艱鬱簾願遞) = 鏇膚艱選窪遞遞鑰構夢夢廠醖選鹽獵積壓築鹹 (製簾艱繭鹽鹽衊遞網積 )
P5.037 (AAN2016)	临床3期	杜氏肌营养不良症	196	Deflazacort 0.9 mg/kg/day	獵遞積鑰鑰顧餘範衊遞(鬱遞壓衊膚鬱遞膚顧壓) = 繭壓簾遞築範製願顧淵淵襯艱齋獵鏇壓餘鏇鏇 (鏇蓋廠網壓簾遞繭範衊 )	积极	2016-04-05
				Deflazacort 1.2 mg/kg/day	鏇鬱簾遞製繭製製積鑰(鹹範遞膚製鹽醖廠夢遞) = 襯憲艱艱選製顧願膚願壓憲鹽膚窪鹹窪餘憲膚 (製廠鹽醖選鬱廠鏇鏇淵 )