Latest news with #raredisease
Yahoo
a day ago
- Health
- Yahoo
TerSera Therapeutics to Present Real-World Data on Intrathecal Ziconotide (PRIALT®) Prescription Patterns at ASPN 2025
DEERFIELD, Ill., July 18, 2025--(BUSINESS WIRE)--TerSera Therapeutics LLC, a biopharmaceutical company with a focus in oncology, rare disease, and non-opioid pain management, announced today that its collaborative research on intrathecal ziconotide (PRIALT®) prescribing patterns has been accepted for presentation at the 7th Annual Meeting of the American Society of Pain and Neuroscience (ASPN) taking place July 17-20, 2025, in Miami, Florida. The poster will be presented by the primary author, Tolga Suvar, M.D., Assistant Professor, Department of Anesthesiology and Pain Medicine, and Attending Physician at Rush University Pain Center in Chicago, IL. A copy of the poster is available here. The abstract, titled "Real-world Treatment Patterns of Intrathecal Ziconotide", highlights a retrospective, non-interventional study conducted in partnership with Pentec Health, Inc., and other clinical collaborators. The study analyzed real-world data from over 1,000 patients treated with ziconotide between 2017 and 2024, using dispensing and claims records from Pentec, a leading provider of specialty infusion pharmacy and complex in-home clinical services. Evolution of prescribing patterns was analyzed over time and compared with the recommendations for ziconotide in the Polyanalgesic Consensus Conference (PACC) guidelines. Key findings from the study1 include: Shift Toward Monotherapy Use: Use of ziconotide as monotherapy increased from 36.0% in 2017–2018 to 63.1% in 2023–2024, reflecting growing adoption of guideline-aligned prescribing of ziconotide. Lower Starting Doses: The proportion of patients initiated on lower ziconotide doses (<0.5 mcg/day) rose from 3.4% to 20.3% over the study period, indicating a trend toward more conservative initial dosing strategies. Increased Use of Patient-Controlled Analgesia (PCA): The use of basal rate with PCA nearly doubled, from 23.7% to 45.3% over the study period, suggesting increasing use of novel approaches to delivery of intrathecal medications. "These findings underscore the importance of real-world evidence in shaping clinical practice," said Dr. Suvar. "It is encouraging to see increased alignment with PACC guidelines and a growing emphasis on patient-tailored dosing strategies. Precision in dosing—based on the unique characteristics and pathologies of each patient—is essential for optimizing outcomes and minimizing risk." About PRIALT® (Ziconotide Intrathecal Infusion)PRIALT is a non-opioid intrathecal analgesic indicated for the management of severe chronic pain in patients for whom intrathecal therapy is warranted and who are intolerant of or refractory to other treatments. Derived from a marine snail peptide, ziconotide acts as a selective N-type calcium channel blocker, interrupting pain signal transmission in the spinal cord. Ziconotide is administered via continuous intrathecal infusion and is not associated with the risk of addiction or respiratory depression commonly seen with opioid therapies.2 IMPORTANT SAFETY INFORMATION WARNING: NEUROPSYCHIATRIC ADVERSE REACTIONS PRIALT is contraindicated in patients with a preexisting history of psychosis. Severe psychiatric symptoms and neurological impairment may occur during treatment with PRIALT. Monitor all patients frequently for evidence of cognitive impairment, hallucinations, or changes in mood or consciousness. Discontinue PRIALT therapy in the event of serious neurological or psychiatric signs or symptoms. ContraindicationsPRIALT is contraindicated in patients with: A known hypersensitivity to ziconotide or any of its formulation components. Any other concomitant treatment or medical condition that would render intrathecal administration hazardous, such as the presence of infection at the microinfusion injection site, uncontrolled bleeding diathesis, and spinal canal obstruction that impairs circulation of cerebrospinal fluid (CSF). A pre-existing history of psychosis. Warnings and PrecautionsCognitive and Neuropsychiatric Adverse ReactionsSevere psychiatric symptoms and neurological impairment may occur during treatment. Monitor all patients frequently for evidence of cognitive impairment, hallucinations, or changes in mood or consciousness. PRIALT may cause or worsen depression, with the risk of suicide in susceptible patients. In clinical trials, 12% of patients reported hallucinations; other acute psychiatric events included paranoid reactions (3%), hostility (2%), delirium (2%), psychosis (1%), and manic reactions (0.4%). Patients with pretreatment psychiatric disorders may be at an increased risk. Management of psychiatric complications may need to include discontinuation of PRIALT, treatment with psychotherapeutic agents and/or short-term hospitalization. In clinical trials, cognitive adverse reactions included confusion (33%), memory impairment (22%), speech disorder (14%), aphasia (12%), thinking abnormal (8%), and amnesia (1%). Cognitive impairment may appear gradually after several weeks of treatment. Reduce the dose of PRIALT or discontinue the use of PRIALT if signs or symptoms of cognitive impairment develop, but other contributing causes must also be considered. The cognitive effects of PRIALT are generally reversible within 2 weeks after drug discontinuation. The elderly (≥65 years) are at higher risk for confusion. Concomitant use of central nervous system (CNS) depressants with PRIALT may have additive effects. Meningitis and Other InfectionsMeningitis can occur due to inadvertent contamination of the microinfusion device and other means. In clinical trials, the rate of meningitis was 3% (40 cases) in the PRIALT group using either internal or external microinfusion devices and 1% (1 case) with placebo. In patients with external microinfusion devices and catheters, meningitis occurred in 38 out of 41 patients (93%), 37 of whom received PRIALT and one who received placebo. Patients, caregivers, and healthcare providers must be particularly vigilant for the signs and symptoms of meningitis including, but not limited to, fever, headache, stiff neck, altered mental status (e.g., lethargy, confusion, disorientation), nausea or vomiting, and occasionally seizures. Strict aseptic procedures must be used during the preparation of the PRIALT solution and refilling of the microinfusion device. Reduced Level of ConsciousnessIn clinical trials, 2% of PRIALT-treated patients became unresponsive or stuporous. If reduced levels of consciousness occur, discontinue PRIALT until the event resolves, and other etiologies (e.g., meningitis) must be considered. Elevation of Serum Creatine KinaseIn clinical trials, serum creatine kinase (CK) levels above the upper limit of normal (ULN) were reported in 40% of patients, with 11% of patients having CK levels >3 times ULN. Incidences were higher during the first 2 months of treatment. Serum CK should be monitored periodically. In the setting of new neuromuscular symptoms, evaluate patients, obtain CK measurements, and if symptoms continue and CK levels remain elevated or continue to rise, reduce the dose or discontinue the use of PRIALT. Withdrawal From OpiatesPRIALT is not an opiate and cannot prevent or relieve the symptoms associated with the withdrawal of opiates. To avoid withdrawal syndrome when opiate withdrawal is necessary, do not abruptly reduce or withdraw opioid medications. Driving and Operating MachineryUse of PRIALT has been associated with cognitive impairment and decreased alertness/unresponsiveness. Caution patients against engaging in hazardous activities that require complete mental alertness or motor coordination. Most Common Adverse ReactionsThe most frequently reported adverse reactions (≥25%) in clinical trials (n=1254 PRIALT-treated patients) were dizziness, nausea, confusional state, and nystagmus. Slower titration of PRIALT may result in fewer serious adverse reactions and discontinuations for adverse reactions. IndicationPRIALT® (ziconotide) solution, intrathecal infusion is indicated for the management of severe chronic pain in adult patients for whom intrathecal (IT) therapy is warranted, and who are intolerant of or refractory to other treatment, such as systemic analgesics, adjunctive therapies, or IT morphine. To report suspected adverse reactions, contact the FDA at 1-800-FDA-1088 or You may also contact TerSera Therapeutics at 1-844-334- 4035 or medicalinformation@ Please see full Prescribing Information, including BOXED Warning. About TerSera TherapeuticsTerSera Therapeutics is a biopharmaceutical company with a focus in oncology, rare disease, and non-opioid pain management. Founded in 2016, TerSera is building new cornerstones of care through its portfolio of unique therapeutics, amplifying their ability to deliver meaningful outcomes for patients. For additional information, please visit and follow us on LinkedIn. About Pentec HealthPentec Health is a leader in specialty pharmacy, infusion services, and DME distribution, committed to solving and simplifying complex care challenges to better meet patient and provider needs. Accredited by the Joint Commission and the American Nurses Credentialing Center, with distinctions in Specialty Care Nurse Fellowship, Pentec Health delivers exceptional results through unique solutions, technical expertise, and clinical collaboration. Our proven delivery models reduce administrative burdens and help improve patient outcomes. Pentec Health serves more than 11,000 patients living with complex medical conditions, with care provided nationwide by over 350 clinicians. For additional information, please visit References Suvar, T., Lindley, D., Leatherman, D., Howard, K., Lucia, R., Duran, N., Murray, M., McGlothlen, G., Dagenhart, J., Cannon, J., Sayeed, S., & Lubenow, T. (2025, July). Real-world treatment patterns of intrathecal ziconotide [Poster presentation]. American Society of Pain and Neuroscience (ASPN) 2025 Annual Meeting. PRIALT® (ziconotide). Prescribing information. TerSera Therapeutics LLC. PRIALT is a registered trademark of TerSera Therapeutics LLC. TerSera and the TerSera logo are trademarks of TerSera Therapeutics LLC. ©2025 TerSera Therapeutics LLC. All rights reserved. View source version on Contacts For more information: TerSera Therapeutics: Mark Leonard847-651-9682mleonard@ Pentec Health: Jeremy Sign in to access your portfolio
Yahoo
12-07-2025
- Health
- Yahoo
Health minister says funding of medication for B.C. girl's rare disease denied again
A Langford family that was hoping the province would reverse its decision for drug coverage to treat their daughter's rare disease is angry after coverage was refused for a second time. The minister of health says an independent medical committee that examines these rare drugs has determined the drug, which costs about $1 million a year, will no longer help the girl's condition. Meera Bains reports.
CTV News
02-07-2025
- Health
- CTV News
B.C. health minister to meet with mother of terminally ill girl after ending drug funding
An emotional mother reacts to the news B.C. will not continue funding an expensive drug treatment for her nine-year-old daughter's rare disease.
CTV News
20-06-2025
- Health
- CTV News
Charleigh Pollock's mother reacts to province defunding daughter's treatment
Vancouver Watch An emotional mother reacts to the news B.C. will not continue funding an expensive drug treatment for her nine-year-old daughter's rare disease.
CBC
15-05-2025
- Health
- CBC
Desperately ill baby healed with personalized gene therapy, doctors say
A baby born with a rare and dangerous genetic disease is growing and thriving after getting an experimental gene editing treatment made just for him, according to the doctors who treated him. Researchers described the case in a new study, saying he's among the first to be successfully treated with a custom therapy that seeks to fix a tiny but critical error in his genetic code that kills half of the infants with the rare disease. Though it may be a while before similar personalized treatments are available for others, doctors hope the technology can someday help the millions left behind even as genetic medicine has advanced because their conditions are so rare. "This is the first step towards the use of gene editing therapies to treat a wide variety of rare genetic disorders for which there are currently no definitive medical treatments," said Dr. Kiran Musunuru, a University of Pennsylvania gene editing expert who co-authored the study published Thursday in the New England Journal of Medicine. The baby, KJ Muldoon of Clifton Heights, Penn., is one of 350 million people worldwide with rare diseases, most of which are genetic. He was diagnosed shortly after birth with severe CPS1 deficiency, estimated by some experts to affect around one in a million babies. Those infants lack an enzyme needed to help remove ammonia from the body, so it can build up in their blood and become toxic. A liver transplant is an option for some. Knowing KJ's odds, parents Kyle and Nicole Muldoon, both 34, worried they could lose him. "We were, like, you know, weighing all the options, asking all the questions for either the liver transplant, which is invasive, or something that's never been done before," Nicole said. "We prayed, we talked to people, we gathered information, and we eventually decided that this was the way we were going to go," her husband added. Within six months, the team at Children's Hospital of Philadelphia and Penn Medicine, along with their partners, created a therapy designed to correct KJ's faulty gene. They used CRISPR, the gene editing tool that won its inventors the Nobel Prize in 2020. Instead of cutting the DNA strand like the first CRISPR approaches, doctors employed a technique that flips the mutated DNA "letter" — also known as a base — to the correct type. Known as "base editing," it reduces the risk of unintended genetic changes. It's "very exciting" that the team created the therapy so quickly, said gene therapy researcher Senthil Bhoopalan at St. Jude Children's Research Hospital in Memphis, who wasn't involved in the study. "This really sets the pace and the benchmark for such approaches." In February, KJ got his first IV infusion with the gene editing therapy, delivered through tiny fatty droplets called lipid nanoparticles that are taken up by liver cells. WATCH | Gene therapy for sickle cell: U.K. approves world's first gene therapy treatment for sickle cell 1 year ago Duration 2:03 While the room was abuzz with excitement that day, "he slept through the entire thing," recalled study author Dr. Rebecca Ahrens-Nicklas, a gene therapy expert at CHOP. After follow-up doses in March and April, KJ has been able to eat more normally and has recovered well from illnesses like colds, which can strain the body and exacerbate symptoms of CPS1. The nine-and-a-half-month old also takes less medication. Considering his poor prognosis earlier, "any time we see even the smallest milestone that he's meeting — like a little wave or rolling over — that's a big moment for us," his mother said. Still, researchers caution that it's only been a few months. They'll need to watch him for years. "We're still very much in the early stages of understanding what this medication may have done for KJ," Ahrens-Nicklas said. "But every day, he's showing us signs that he's growing and thriving." Opens door to advances in other rare diseases Researchers hope what they learn from KJ will help other rare disease patients. Gene therapies, which can be extremely expensive to develop, generally target more common disorders in part for simple financial reasons: more patients mean potentially more sales, which can help pay the development costs and generate more profit. The first CRISPR therapy approved by the U.S. Food and Drug Administration, for example, treats sickle cell disease, a painful blood disorder affecting millions worldwide. Musunuru said his team's work — funded in part by the U.S. National Institutes of Health — showed that creating a custom treatment doesn't have to be prohibitively expensive. The cost was "not far off" from the $800,000 US-plus for an average liver transplant and related care, he said. "As we get better and better at making these therapies and shorten the time frame even more, economies of scale will kick in and I would expect the costs to come down," Musunuru said. Scientists also won't have to redo all the initial work every time they create a customized therapy, Bhoopalan said, so this research "sets the stage" for treating other rare conditions. Carlos Moraes, a neurology professor at the University of Miami who wasn't involved with the study, said research like this opens the door to more advances. "Once someone comes with a breakthrough like this, it will take no time" for other teams to apply the lessons and move forward, he said. "There are barriers, but I predict that they are going to be crossed in the next five to 10 years. Then the whole field will move as a block because we're pretty much ready."
