— ISTURISA® (osilodrostat) is a cortisol synthesis inhibitor that blocks the enzyme 11beta-hydroxylase to help normalize hypercortisolemia in Cushing's syndrome, a rare endocrine condition that can have significant impact on patients and their families
— The expanded indication is supported by an extensive clinical development program
Today,Recordati RareDiseasesInc.announced that further insights into ISTURISA®as well as its expanded indication will be presented at this year's gathering of theAACE from May15 through May 17,2025,in Orlando, FL.Thisincludes commercial and medical information booths for more information on the expanded indication, a LINC 6 study poster presentation on ISTURISA®, and 2 product theaters, one involving cortisol in Cushing's syndrome and the other, unrelated to ISTURISA®, highlighting the diagnosis and treatment of acromegaly.
Mario Maldonado, MD, Head of Development and Global Endocrinology at Recordati Rare Diseases commented, “The Recordati team is excited to speak at AACE 2025 about the label expansionofISTURISA®toendogenoushypercortisolemiainadultpatients withCushing'ssyndrome for whom surgery is not an option or has not been curative. Aside from that big news, I take great pride in presenting the LINC 6 poster. Given the nature of endogenous Cushing's syndrome, which often requires ongoing management, the LINC 6 study provides crucial data on the long-term safety and efficacy of ISTURISA®. This 2-year real-world interim analysis underscores our commitment to advancing treatment options for those affected by this challenging condition.”
KeydatapresentationsatAACE2025:
— Visit Booth #109 to learn about the expanded indication for ISTURISA® and what it can mean for the treatment of Cushing's syndrome
— LINC 6 Poster (Session #792): Assessing Long-Term Safety and Efficacy of Osilodrostat in Prior- and New-Use Patients With Endogenous Cushing's Syndrome Enrolled in the Non-Interventional, Multinational LINC 6 Study: 2-Year Real-World Interim Analysis
— Presented by MarioMaldonado, MD, Head of Development and Global Endocrinology at Recordati Rare Diseases on May 15, from 1:30 PM to 1:45 PM (EDT)
— Product Theater 1: Is Cortisol the Culprit? An interactive discussion panel to help recognize the signs and symptoms of endogenous hypercortisolemia in patients with Cushing's syndrome
— Friday, May 16, from 12:15 PM to 1:00 PM (EDT) at the Lunch Product Theater, Learning Zone Theater 2, Orlando Ballroom
RECORDATIINDUSTRIACHIMICAEFARMACEUTICAS.p.A.
Registered OfficeViaMatteoCivitali,120148Milano, ItalyTel. +39 02 487871Fax +39 02 40073747www.recordati.com
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— The panel will include AnthonyHeaney,MD,PhD(Professor inResidenceof Medicineat theDavidGeffenSchool ofMedicine,UCLA), LaurenF.Gratian, MD(WilmingtonHealth), and Divya Yogi-Morren, MD (Medical Director of the Pituitary Center, Cleveland Clinic)
— Product Theater 2: Breaking Barriers in Acromegaly: A Personalized Approach to Identifying, Diagnosing,andOptimizingTreatment
— Friday, May 16, from 2:15 PM to 3:00 PM (EDT) at the ET Product Theater, Learning Zone Theater 2, Orlando Ballroom
— The panel will includeWenyu Huang, MD, PhD, (Associate Professor at the Division of Endocrinology, Northwestern University Feinberg School of Medicine) and Michael H. Shanik, MD, FACP, FACE (Managing Partner at Endocrine Associates of Long Island, Smithtown, NY; Associate Professor at Stony Brook University Hospital, Stony Brook, NY)
In April, Recordati announced that the U.S. Food and Drug Administration (FDA) approved the supplemental new drug application (sNDA) for ISTURISA® (osilodrostat) for the treatment of endogenous hypercortisolemiainadultswithCushing'ssyndromeforwhomsurgeryisnotanoptionor has notbeencurative.Thisisanexpansion of thepreviousindication for thetreatmentofpatients with Cushing'sdisease,whichisasub-typeofCushing'ssyndrome.TheISTURISA® indication expansion was supported by theISTURISA®extensiveclinicaldevelopmentprogram, whichincludesover350patients.
ConsumerImportantSafety Information
WHATisISTURISA?
ISTURISA(osilodrostat)isaprescriptionmedicine usedtotreatelevatedlevels ofcortisol in the blood (endogenous hypercortisolemia)inadultswithCushing'ssyndrome:
— who cannot have surgery, or
— who have had surgery which did not cure their Cushing's syndrome It is not known if ISTURISA is safe and effective in children.
IMPORTANTSAFETYINFORMATION
Before startingISTURISA tell yourhealthcareprovideraboutallyourmedicalconditions,includingif you:
— have or had heart problems, such as an irregular heartbeat, including a condition called prolonged QT syndrome (QT internal prolongation). Your healthcare provider will check the electrical signal of your heart (called an electrocardiogram) before you start taking ISTURISA, 1 week after starting ISTURISA, and as needed after that.
— have a history of low levels of potassium or magnesium in your blood.
— have liver problems.
— are or plan to become pregnant. It is not known if ISTURISA will harm your unborn baby. There are risks to the mother and unborn baby associated with active Cushing's syndrome during pregnancy.
— arebreastfeedingorplantobreastfeed.ItisnotknownifISTURISApasses intoyourbreast milk. You should not breastfeed if you take ISTURISA and for 1 week after stopping treatment.
Tell your healthcare provider aboutallthemedicines youtake,includinganyprescriptionandover- the-countermedicines,vitamins,orherbal supplements.
Especiallytellyourhealthcareproviderifyoutakemedicinesusedtotreatcertainheart problems.Ask your healthcare provider if you are not sure whether your medicine is used to treat heart problems.
ISTURISAcancauseserioussideeffectsincluding:
— Low cortisol levels in your blood (hypocortisolism). Tell your healthcare provider right away if you experience more than one of the following symptoms, as these may be symptoms of very low cortisol level, known as adrenal insufficiency: nausea, vomiting, tiredness (fatigue), low blood pressure, problems with body salt (electrolyte) levels in your blood, stomach (abdominal) pain, loss of appetite, dizziness, low blood sugar.
Ifyougetsymptoms ofhypocortisolismwhile takingISTURISA,yourhealthcareprovider may change your dose or ask you to stop taking it.
— Heart problem or a heart rhythm problem, such as an irregular heartbeat which could be a sign of a heart problem called QT prolongation. Call your healthcare provider right away if you have irregular heartbeats.
— Increase in other adrenal hormone levels. Your other adrenal hormones may increase when you take ISTURISA. Your healthcare provider may monitor you for the symptoms associated with these hormonal changes while you are taking ISTURISA:
— Low potassium (hypokalemia).
— High blood pressure (hypertension).
— Swelling (edema)in the legs, ankles or other signs of fluid retention.
— Excessive facial or body hair growth (hirsutism).
— Acne(in women).
Callyourhealthcareprovider ifyouhaveanyofthesesideeffects.
The mostcommonside effects ofISTURISAinclude very low cortisol levels (adrenal insufficiency), tiredness(fatigue),nausea, headache,and swellingofthelegs,anklesorothersignsoffluidretention (edema).
Thesearenotallthepossible sideeffectsofISTURISA.Callyourhealthcareproviderformedical advice about side effects. You are encouraged to report side effects of prescription drugs to the FDA. Call 1-800-FDA-1088 or visit www.fda.gov/medwatch.
PleaseseefullPrescribingInformation
AboutEndogenousHypercortisolemiainCushing'sSyndrome
Hypercortisolemia,which ismarkedbyelevatedlevels ofcortisol,istheunderlyingcauseofendogenous Cushing's syndrome, a rare and serious disease of excess cortisol for any reason (pituitary and nonpituitary). Cushing's disease (a sub-type of Cushing's syndrome) is cortisol elevated on the basis of pituitary overstimulation (ACTH, adrenocorticotropic hormone) of the adrenal glands. Elevated cortisol can lead to a wide range of associated conditions and complications, such as weight gain, high blood glucose, high blood pressure, osteoporosis, thin and fragile skin that bruises easily, muscle weakness, depression, anxiety, and irritability. If endogenous hypercortisolemia in Cushing's syndrome is left untreated, it is associated with severe complications and diseases, including diabetes, osteoporosis, cardiovascularissues, andevenincreased riskofinfectionduetothesuppressionoftheimmune system.
AboutISTURISA®ISTURISA®isacortisolsynthesis inhibitorthatworksbypreventing 11beta-hydroxylase,anenzyme responsible for the final step of cortisol biosynthesis in the adrenal gland, from being created. ISTURISA®is also approved for the treatment of patients with endogenous Cushing's syndrome in multiplecountriesoutside theU.S.includingtheEuropeanUnion(January2020)andChina(September 2024). ISTURISA®received orphan drug designation from the FDA and the European MedicinesAgency for the treatment of endogenous Cushing's syndrome.
RecordatiisaninternationalpharmaceuticalgrouplistedontheItalianStockExchange(XMIL: REC),withrootsdating backtoafamily-runpharmacy inNorthernItaly inthe1920s.Weareuniquelystructuredtoprovidetreatmentsacross specialtyandprimary care,andrarediseases.Ourfullyintegratedoperationsspanclinical development,chemicaland finished product manufacturing, commercialization and licensing. We operate in approximately 150 countries across EMEA, the Americas and APAC with over 4,450 employees. We believe that health is a fundamental right, not a privilege. Today, ourpurpose of”unlocking the full potential of life” aims at empowering individuals to live life to the fullest, whether addressing common health challenges or the rarest.
InvestorRelations
EugeniaLitz+44 7824 394 750investorelations@recordati.it
GianlucaSaletta+39 348 979 4876investorelations@recordati.it
MediaRelations
ICR HealthcareUS:AlexisFeinberg+1 203 939 2225recordatiuspr@westwicke.com
UK,Europe &RestofWorld: Jessica Hodgson+44 7561 424 788recordati@consilium-comms.com
This document contains forward-looking statements relating to future events and future operating, economic and financial results of the Recordati group. By their nature, forward-looking statements involve risk and uncertainty because they depend on the occurrence of future events and circumstances. Actual results may therefore differ materially from those forecast for a variety of reasons, most of which are beyond the Recordati group's control. The information on the pharmaceutical specialties and other products of the Recordati group contained in this document is intended solelyas informationonthe activities ofthe RecordatiGroup,and,as such,itis not intended as a medical scientific indication or recommendation, or as advertising.
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