Trientine tetrahydrochloride

PITTSBURGH, April 19, 2023 /PRNewswire/ -- PANTHERx® Rare, a leader in rare disease product patient access and support services in the United States, is pleased to announce the launch of CUVRIOR to treat stable Wilson Disease in adults who are de-coppered and able to take penicillamine. "We are pleased to add CUVRIOR to the list of PANTHERx® medications. The PANTHERx team works hard to make a positive difference in the lives of our patients who are dealing with devastating and debilitating diseases and CUVRIOR is yet another way we can make an impact," said Rob Snyder, president of PANTHERx® Rare. Wilson Disease is a rare genetic disorder caused by a mutation in the ATP7B gene, resulting in the accumulation of copper throughout the body. Copper deposits in the body lead to a wide array of symptoms including, but not limited to tremors, anxiety, jaundice due to liver injury, and Kayser-Fleischer rings (brown rings in the corneas of the eyes). Untreated, Wilson Disease can be fatal. Serious complications include problems with the liver, kidneys, neurological (e.g., tremors, clumsy gait, speech difficulties), psychological (e.g., depression, psychosis) and blood problems (anemia, jaundice). CUVRIOR is an innovative oral formulation of the active ingredient trientine created by Orphalan to treat adult patients with stable Wilson Disease who have already been de-coppered. CUVRIOR works by chelating copper in the body to decrease copper absorption and increase urinary copper excretion. In clinical trials, CUVRIOR was found to be well tolerated and non-inferior to penicillamine in reducing non-ceruloplasmin copper (NCC) levels in Wilson Disease patients. About PANTHERx Rare PANTHERx Rare is one of the largest and fastest growing rare pharmacies in the United States. PANTHERx transforms lives by delivering medicine breakthroughs, clinical excellence, and access solutions to people living with rare and devastating conditions. Although the overall incidence of rare diseases is as common as diabetes, less than 7% of the 7,000 known rare and devastating disorders have an FDA-approved therapy. Changes in federal policy and advances in science have led to a surge in FDA orphan drug approvals, providing tremendous hope to the rare disease community. PANTHERx was recently awarded Specialty Pharmacy of the Year by the National Association of Specialty Pharmacy (NASP) and earned the inaugural Accredited Distinction in Rare Diseases and Orphan Drugs from the Accreditation Commission for Health Care (ACHC). PANTHERx is now a five-time winner of the prestigious MMIT Patient Choice Award, including the 2022 honor. PANTHERx is headquartered in Pittsburgh, licensed in all 50 states, and holds accreditations from URAC, NABP, and ACHC. IMPORTANT SAFETY INFORMATION Do not use CUVRIOR if you are allergic to trientine or to any of the ingredients in CUVRIOR. Your Wilson's Disease symptoms could get worse when you start treatment. This could happen because too much copper is removed from the body in a short period of time. Your doctor may need to reduce your dose or stop CUVRIOR treatment. Copper deficiency may develop following treatment with CUVRIOR. Your doctor will do tests to monitor your urine and blood for copper. Iron deficiency may develop while taking CUVRIOR. If this happens, your doctor may tell you to take iron supplements for a limited time. Allergic reactions, such as a rash can occur. If a rash or other allergic reaction occurs, stop taking CUVRIOR and get emergency medical help. The most common side effects are pain in the abdomen, change in bowel habits, rash, hair loss (alopecia), and mood swings. Take CUVRIOR at least 1 hour apart from any other oral medicine. Tell your doctor about all the medicines you're taking, including over-the-counter medicines, vitamins, and herbal supplements. Avoid taking supplements when taking CUVRIOR. Taking CUVRIOR with mineral supplements (e.g., iron, zinc, calcium, magnesium) can reduce the effectiveness of CUVRIOR. If iron supplementation is necessary, take CUVRIOR at least 2 hours before or 2 hours after taking iron; for other mineral supplements (e.g., zinc, calcium, magnesium), take CUVRIOR at least 1 hour before or 2 hours after. Do not start or stop any medicines while taking CUVRIOR without talking to your doctor. These are not all the possible side effects of CUVRIOR. You are encouraged to report negative side effects of prescription drugs. Contact Orphalan at 1-800-961-8320 or FDA at 1-800-FDA-1088 or medwatch. Please see full Prescribing Information. 03/2023 US-ORPH-CUVR-023* - Orphalan reviewed and approved this material exclusively regarding CUVRIOR, Wilson disease and Orphalan related information and claims. SOURCE PANTHERx Rare Pharmacy

An experimental AstraZeneca drug for Wilson disease hit the mark in a Phase III study in which the drug, ALXN1840, was able to achieve three times greater mobilization of copper than the standard of care. In those with Wilson disease, excess copper build-up in organs and tissues caused by the loss of the ATP7B protein required for copper transport can lead to liver disease, as well as neurological and psychiatric symptoms. It is estimated that about 9,000 people in the U.S. have been diagnosed with the disorder. There are only a handful of drugs approved by the FDA to treat this genetic disorder, including Syprine, a medication that’s been around since the 1960s, and the more-recently-approved Cuvrior, developed by France-based Orphalon. Based on data from the Phase III FoCus study, AstraZeneca hopes to join this small group of medications approved by the U.S. Food and Drug Administration. In the Phase III study, which has been billed as the largest global trial in Wilson disease to date, ALXN1840 met its primary endpoint by demonstrating a copper mobilization three times greater than the standard of care, including patients who have previously been treated for at least 10 years. Even after standard of care treatment, some patients continue to see a progression of their disease and a worsening of neurological symptoms, AstraZeneca said. Trial data showed that patients experienced copper mobilization, which is the filtering of copper buildup through the urine, within four weeks and sustained through 48 weeks, the company said. In patients who were symptomatic of Wilson disease at baseline, treatment with ALXN1840 generated greater improvements in neurological scores than the standard of care. This data was a secondary endpoint of the study. However, AstraZeneca noted that in this subgroup, there were no significant differences between treatment groups seen at 48 weeks. AstraZeneca noted that most patients in the trial had low symptom scores at baseline, which it said indicated minimal room for total score improvement. As people with Wilson disease experience a highly varied degree of symptoms, this total score may not reflect the extent of disease severity, AstraZeneca said. ALXN1840 was well tolerated in the FoCus study, and long-term safety and efficacy is being assessed in an up to 60-month extension period. AstraZeneca intends to present full data from the Phase III FoCus study today at the 2022 International Liver Congress in London. ALXN1840, which was developed by AstraZeneca’s rare disease subsidiary Alexion, is an investigational molecule designed to bind with and remove copper from the body. ALXN1840 has been granted Orphan Drug Designation in the United States and orphan designation in the European Union for Wilson disease. Marc Dunoyer, the chief executive officer of Alexion, noted that many Wilson disease patients can continue to experience symptoms even after years of using available therapies. He said that demonstrates a significant need for the re-evaluation of the standard of care used to treat this genetic disorder. “Applying our 30 years of experience in rare disease clinical development, Alexion has conducted rigorous scientific research to bring fresh thinking to Wilson disease around the importance of copper mobilization from the tissues. These data further our efforts to potentially introduce a novel treatment for patients who have gone decades without meaningful innovation,” Dunoyer said in a statement.