Procysbi (cysteamine bitartrate delayed release) / Chiesi, Amgen - LARVOL DELTA

Clinical features of Cystinosis and practice patterns: A report of the NAPRTCS Cystinosis Registry (IPNA 2025) - "The NAPRTCS Cystinosis Registry has collected clinical information on children and young adults with cystinosis since 2018 and now includes information on 104 patients from 30 centers who were diagnosed from 12/1999 to 8/2023...The type varied over time with an increase in Procysbi use following FDA approval in 2013...Only a few patients (5.1%) were started on indomethacin and a small subset of patients 6.1-13.3% are on indomethacin during longitudinal follow-up...Patients typically present with 6 months of symptoms before a diagnosis is made with failure to thrive present in more than 50%. Children and young adults with cystinosis have complex care needs which are often met through non-local, multispecialty care providers."

Clinical • Endocrine Disorders • Lysosomal Storage Diseases • Metabolic Disorders • Nephrology • Ophthalmology • Pediatrics • Rare Diseases

Adherence to delayed-release cysteamine in nephropathic cystinosis over time: data from the prospective CrYSTobs cohort study. (PubMed, Pediatr Nephrol) - "This study found a fairly good adherence to DR cysteamine over time. Decreasing the number of intakes per day may improve adherence and limit the side effects of the drug, representing an additional argument in favor of its use in patients with cystinosis."

Journal • Rare Diseases • Renal Disease

Clinical Features of Cystinosis and Practice Patterns: A Report of the NAPRTCS Cystinosis Registry (KIDNEY WEEK 2024) - "At enrollment, demographic & clinical data from time of initial presentation with cystinosis, time of enrollment, and every 6 months. Data were collected from 30 centers on 104 subjects diagnosed from 12/1999 to 8/2023 (Table 1).The most commonly reported medications at diagnosis included cystine lowering medications (81.8%), cysteamine eye drops (34.3%), and phosphorus and potassium supplementation (43.4%, 42.4%)...The type varied over time with an increase in Procysbi use following FDA approval in 2013... Children with cystinosis present early in life with majority of patients diagnosed under 2 years of age. Patients typically present with 6 months of symptoms before a diagnosis is made with failure to thrive present in more than 50%. Children and young adults with cystinosis have complex care needs which are often met through non-local, multispecialty care providers."

Clinical • Endocrine Disorders • Nephrology • Ophthalmology

Adherence to delayed-release cysteamine in nephropathic cystinosis over time. A prospective cohort study: CrYSTobs (ESPN 2024) - P=N/A | "Our data describe a good long-term adherence to DR cysteamine, which is an additional argument in favor of its reimbursement."

Adherence • Clinical • Rare Diseases • Renal Disease

ECYSCO, a European cystinosis cohort (ESPN 2024) - "Sixty-seven patients received immediate release formulation (Cystagon®) and 103 received extended release formulation (Procysbi®)... Cystinosis is a good example of a pediatric disease with multiorgan involvement extending into adult care. The high frequency of extra-renal manifestations demonstrates the importance of a multidisciplinary follow up of these patients."

CNS Disorders • Diabetes • Endocrine Disorders • Lysosomal Storage Diseases • Metabolic Disorders • Myositis • Pediatrics • Rare Diseases • Renal Disease

PATIENT REPORTED EXPERIENCES COMPARING IMMEDIATE RELEASE AND DELAYED RELEASE CYSTEAMINE (SSIEM 2024) - "Switching to DR cysteamine was positive for the majority of patients, therapy adherence improved and the burden of treatment decreased. Side effects were reported less frequently."

Clinical • Dental Disorders • Renal Disease

ECYSCO, a European Cystinosis cohort (ERA-EDTA 2024) - "Sixty-seven patients received immediate release formulation (Cystagon®) and 103 received extended release formulation (Procysbi®)...More than half of patients are adults and have reached kidney failure even if age at renal replacement therapy start has increased. The high frequency of extra-renal manifestations demonstrates the importance of a multidisciplinary follow up of these patients."

CNS Disorders • Diabetes • Diabetic Nephropathy • Endocrine Disorders • Lysosomal Storage Diseases • Metabolic Disorders • Musculoskeletal Diseases • Myositis • Orthopedics • Pediatrics • Rare Diseases • Renal Disease • Transplantation

Fibrosing colonopathy associated with cysteamine bitartrate delayed-release capsules in cystinosis patients. (PubMed, Pediatr Nephrol) - "Our case series identified the risk of fibrosing colonopathy in patients taking cysteamine DR and prompted regulatory action by the FDA. As outlined in changes to the U.S. prescribing information for cysteamine DR, healthcare professionals should be aware of the potential risk of fibrosing colonopathy with cysteamine DR, especially as symptoms can be non-specific leading to misdiagnosis or delayed diagnosis. If the diagnosis of fibrosing colonopathy is confirmed, consideration should be given to permanently discontinuing cysteamine DR and switching to cysteamine immediate-release treatment."

Journal • Fibrosis • Rare Diseases • Renal Disease

Impact of compliance to oral cysteamine treatment on the costs of Kidney failure in patients with nephropathic cystinosis in the United Kingdom. (PubMed, BMC Nephrol) - "This analysis indicates that lifetime costs of KF in NC can be reduced through improved treatment compliance with oral cysteamine."

Compliance • Journal • Chronic Kidney Disease • Rare Diseases • Renal Disease • Transplantation

Worldwide disparities in access to treatment and investigations for nephropathic cystinosis: a 2023 perspective. (PubMed, Pediatr Nephrol) - "Over the last decade, access to investigations (namely genetics and IL-CL) and to cysteamine have improved in DEing and TrE. However, discrepancies remain with DEed: access to delayed released cysteamine is limited, and reimbursement is still profoundly insufficient, therefore limiting their current use. A higher resolution version of the Graphical abstract is available as Supplementary information."

Journal • Nephrology • Rare Diseases • Renal Disease

Clinical Features of Cystinosis and Practice Patterns: A Report of the NAPRTCS Cystinosis Registry (KIDNEY WEEK 2023) - "Procysbi use progressively increased following FDA approval in 2013... Children with cystinosis present early in life with the majority diagnosed before 2 years of age. Patients typically experience 5 months of symptoms prior to diagnosis, with failure to thrive present in more than 50%. Children and young adults with cystinosis have complex care needs which are often met through multispecialty care providers."

Clinical • Ophthalmology

Enteric-Coated Cysteamine Bitartrate in Cystinosis Patients. (PubMed, Pharmaceutics) - "Treatment with EC-cysteamine improves the quality of life of cystinosis patients because the frequency of intake can be reduced to 2-3 times daily and it has a more favorable pharmacokinetic profile than IR-cysteamine. In particular, cystinosis patients with no access to the only approved delayed-release cysteamine Procysbi could benefit from a cost-effective alternative."

Journal • Dental Disorders • Gastrointestinal Disorder • Metabolic Disorders

Long-term clinical benefits of delayed-release cysteamine bitartrate capsules in patients with nephropathic cystinosis (response to "A comparison of immediate release and delayed release cysteamine in 17 patients with nephropathic cystinosis"). (PubMed, Orphanet J Rare Dis) - "In addition, the article does not include study data to properly support the authors' suggestion of increased dosing effects and benefits. We present a summary of the results from the DR-CYS clinical trial program and evidence of the rigor from which the clinical data for DR-CYS were generated and recommendation for usage as prescribed."

Journal • Rare Diseases • Renal Disease

ECYSCO : A EUROPEAN COHORT DEDICATED TO CYSTINOSIS (ERA-EDTA 2023) - "71 patients received immediate release formulation (Cystagon R ) and 103 received extended release formulation (Procysbi R )... Cystinosis is a good example of a pediatric disease with multiorgan involvement extending into adult care. More than half of patients are adults and have reached ESRD even if age at renal replacement therapy start has increased. The high frequency of extra-renal manifestations demonstrates the importance of a multidisciplinary follow up of these patients."

Chronic Kidney Disease • CNS Disorders • Diabetes • Diabetic Nephropathy • Endocrine Disorders • Lysosomal Storage Diseases • Metabolic Disorders • Myositis • Nephrology • Pediatrics • Rare Diseases • Renal Disease • Transplantation

Cysteamine bitartrate delayed-release capsules control leukocyte cystine levels and promote statural growth and kidney health in an open-label study of treatment-naïve patients <6 years of age with nephropathic cystinosis. (PubMed, JIMD Rep) - "All patients experienced ≥1 adverse event(s) with vomiting (80%) and upper respiratory tract infection (53%) most frequently reported. Based on our study, patients <6 years of age with nephropathic cystinosis without prior treatment can safely and effectively initiate treatment with DR-cysteamine, a delayed-release form of cysteamine bitartrate that can be given every 12 h."

Clinical • Journal • Endocrine Disorders • Infectious Disease • Lysosomal Storage Diseases • Metabolic Disorders • Nephrology • Rare Diseases • Renal Disease • Respiratory Diseases

A comparison of immediate release and delayed release cysteamine in 17 patients with nephropathic cystinosis. (PubMed, Orphanet J Rare Dis) - "A therapy with DR-cysteamine is as effective as IR-cysteamine while less side effects were reported. Our data show that DR-cysteamine should be dosed higher than 70% of the equivalent dose of IR-cysteamine in order to decrease cystine levels over an extended period of time. Moreover, our data suggest increasing the dosing scheme of Procysbi® to three times daily, to prevent a rapid decrease and achieve a steadier decline in cystine levels. Due to the more convenient dosing scheme, DR-cysteamine might ameliorate therapy adherence and improve patients' quality of life."

Clinical • Journal • Chronic Kidney Disease • Metabolic Disorders • Nephrology • Rare Diseases • Renal Disease • Transplantation

A novel sustained-release cysteamine bitartrate formulation for the treatment of cystinosis: Pharmacokinetics and safety in healthy male volunteers. (PubMed, Pharmacol Res Perspect) - "This was a randomized, investigator-blinded, three-way cross-over study to compare single doses (600 mg) of PO-001 with Cystagon (immediate-release) and Procysbi (delayed-release)...This study supports advancing this type of sustained-release formulation into a subsequent study to confirm reduced dosing frequency with efficient control of white blood cells (WBCs) cystine levels. Netherlands Trial Registry (NTR) (NL67638.056.18)."

Clinical • Journal • PK/PD data • Gastrointestinal Disorder

PROREAL: Observational Study to Assess the Quality of Life in Nephropathic Cystinosis Patients (clinicaltrials.gov) - P=N/A; N=31; Enrolling by invitation; Sponsor: Chiesi SA/NV; Not yet recruiting ➔ Enrolling by invitation; Trial completion date: Mar 2026 ➔ Jul 2026; Initiation date: Apr 2020 ➔ Jul 2020

Clinical • Enrollment open • HEOR • Trial completion date • Trial initiation date • Rare Diseases • Renal Disease

Nonalcoholic Fatty Liver Disease in Children: Not a Small Matter. (PubMed, Paediatr Drugs) - "Few pharmacologic options such as metformin, probiotics, omega-3 fatty acids, and cysteamine bitartrate have been studied in children, with limited beneficial effects. In this review article, we discuss epidemiology, pathophysiology, and extrahepatic comorbidities of pediatric NAFLD and review existing therapeutic options for children with NAFLD. We also review novel therapeutic strategies studied in adults that could potentially be studied in children in the future."

Clinical • Journal • Review • Biosimilar • Fibrosis • Immunology • Non-alcoholic Fatty Liver Disease • Non-alcoholic Steatohepatitis

"PROCYSBI Approval of @HZNPplc by @HealthCanada 4 Cystinosis https://t.co/10hfWstxWh, more info on Cys https://t.co/OlcWwrvvdb @RareDiseases" (@CheckOrphan)

Biosimilar

Evaluation of Effective Dose from CT Scans for Overweight and Obese Adult Patients Using the VirtualDose Software. (PubMed) - Radiat Prot Dosimetry - "DLP and conversion factors were used to estimate ED, where conversion factors depended on tube voltage, sex, BMI and tissue weight factors. With increasing number of obese individuals, using size-dependence conversion factors will improve accuracy, in estimating patient radiation dose."

Journal • Biosimilar • Obesity

Cumulative radiation exposure and associated cancer risk estimates for scoliosis patients: Impact of repetitive full spine radiography. (PubMed) - Eur J Radiol - "We demonstrate the use of computer simulation and analytic formula to quantitatively obtain the cumulated effective dose and cancer risk at any age of exposure, both of which are valuable information to medical personnel and patients' parents concern about radiation safety in repetitive full spine radiography."

Journal • Biosimilar • Oncology

The effect of food and liquid pH on the integrity of enteric-coated beads from cysteamine bitartrate delayed-release capsules. (PubMed, Drug Des Devel Ther) - "Bead integrity was not maintained when mixed with foods at pH values of ≥5.5 at room temperature. The results from this in vitro dissolution study help in identifying additional foods that may be used for the administration of cysteamine bitartrate DR beads from opened capsules using the sprinkle method. "

Journal • Biosimilar • Renal Disease

MONTE CARLO STUDY OF THE CARDIAC ABSORBED DOSE DURING X-RAY EXAMINATION OF AN ADULT PATIENT. (PubMed) - "Moreover, the provided DCCs were used to present an analytical parameterisation for the calculation of the cardiac absorbed dose for any arbitrary X-ray spectrum and for those patient sizes. Thus, the present work can be considered as an enhancement of the continuous studies performed by medical physicist as part of quality control tests and radiation protection dosimetry."

Journal • Biosimilar

"Horizon Therapeutics plc Announces Availability of PROCYSBI® (Cysteamine Bitartrate) Delayed-Release Oral Granules in Packets in the United States https://t.co/NIwHpbr6CA" (@NewsFromBW)