Alpha-Mannosidosis

- Presenting 11 abstracts with range of insights on diagnosis and management of Fabry disease and alpha-mannosidosis and clinical data on pegunigalsidase alfa and velmanase alfa – - Results include 10-year data from study of velmanase alfa – - Organizing and funding two satellite symposia showcasing insights from panel of global clinical experts in lysosomal diseases, focusing on how to optimize treatment strategies – PARMA, Italy, Feb. 3, 2025 /PRNewswire/ -- Chiesi Global Rare Diseases, a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people living with rare diseases, today announced that 11 poster presentations have been accepted at the 21st Annual WORLDSymposium™ Research Meeting to be held February 3-7, 2025, in San Diego, California. The presentations include long-term data up to 10 years of treatment with velmanase alfa in patients with alpha-mannosidosis. Additionally, the presentations cover advances in the understanding of patient tolerability while on treatment and strategies for incorporating both patient and caregiver perspectives in disease management. "We are pleased to have the opportunity to present a wide range of clinical data and insights related to the diagnosis, treatment and appropriate monitoring of people living with Fabry disease and alpha-mannosidosis at the WORLDSymposium," said Giacomo Chiesi, Executive Vice President of Chiesi Global Rare Diseases. "As part of our mission to improve patient outcomes for the lysosomal disease community, we are committed to continual learning, listening and partnering with patients, caregivers and families to improve disease management strategies and evaluating long-term clinical data to better understand the impact of treatment." Poster Presentations Pegunigalsidase alfa and adults with Fabry disease Title: Lower rate of infusion-related reactions in patients with Fabry disease after switching from agalsidase beta to pegunigalsidase alfa Presenter: Nicola Longo, M.D., Chief of the Division of Medical Genetics at the University of Utah Title: Improved tolerability following enzyme replacement therapy switch to pegunigalsidase alfa: A case series from two centers of the expanded access program Presenter: Myrl D. Holida, Physician Assistant at the University of Iowa Health Care Title: Evaluating the relationship between infusion-related reactions and antidrug antibody status: Results from 111 patients with Fabry disease treated with pegunigalsidase alfa Presenter: Robert Hopkin, M.D., Clinical Geneticist at Cincinnati Children's Hospital Medical Center Title: Clinical assessment of disease severity in patients with Fabry disease treated with pegunigalsidase alfa: An integrated analysis Presenter: Derralynn Hughes, M.D., Professor of Experimental Haematology at the University College London Title: Indirect treatment comparisons of pegunigalsidase alfa vs other therapies for left ventricular mass index in Fabry disease Presenter: Eric Wallace, M.D., Co-Director of the University of Alabama at Birmingham Fabry Disease Clinic Title: Awareness of Fabry disease among non-Fabry specialists: Opportunities for education Presenter: Thomas Kenny, Patient Advocacy Lead, Chiesi Limited Title: Maximising engagement through feedback: Insights from shared decision-making toolkit for Fabry disease patients Presenter: Thomas Kenny, Patient Advocacy Lead, Chiesi Limited Title: Speaking the same language: The Fabry lexicon and the implications for how the healthcare community understands the impact of ERT Presenter: Thomas Kenny, Patient Advocacy Lead, Chiesi Limited Velmanase alfa and alpha-mannosidosis Title: Clinical profiles of 134 patients with alpha-mannosidosis from the velmanase alfa clinical program and SPARKLE registry Presenter: Nicole Muschol, M.D., International Center for Lysosomal Disorders (ICLD) at the University Medical Center Hamburg-Eppendorf in Germany Title: Long-term motor function and quality of life outcomes in patients with alpha-mannosidosis: Data from two velmanase alfa extension studies over 10 years Presenter: Nathalie Guffon, M.D., Reference Centre of inherited metabolic disease in Femme Mère Enfant Hospital, Hospices Civils of Lyon, France Title: Real world reflections of the patient odyssey in alpha-mannosidosis: Insights and challenges in diagnosis from caregiver interviews Presenter: Sonia Sgrò, Global Head Patient Advocacy, Lysosomal Storage Disorders (LSDs) at Chiesi Global Rare Diseases Chiesi Global Rare Diseases is also organizing and funding two satellite symposia during the WORLDSymposium: Title: Enzyme Replacement Therapy: Time to Act in Lysosomal Diseases Date and Time: Tuesday, February 4, 2025 from 5:45-6:45 p.m. PT A panel of global clinical experts will discuss current knowledge of lysosomal diseases and appropriate management strategies for patients on enzyme replacement therapy including the potential for switching therapies given the availability of multiple treatment options. Title: Red Light, Green Light: What About the Yellow Light? Exploring the Complexities of Treatment Tolerability in Fabry Disease Date and Time: Wednesday, February 5, 2025 from 12:15-1:15 p.m. PT Presenters will explore the mechanisms and patient-specific factors that influence the tolerability of enzyme replacement therapies in Fabry disease, discuss pegunigalsidase alfa tolerability data, and highlight potential future directions towards enhancing patient outcomes. Indication and Important Safety Information for Elfabrio® (pegunigalsidase alfa-iwxj) Indication Elfabrio® (pegunigalsidase alfa-iwxj) is indicated for the treatment of adults with confirmed Fabry disease. Important Safety Information Prior to Elfabrio administration, consider pretreating with antihistamines, antipyretics, and/or corticosteroids. Inform patients and caregivers of the signs and symptoms of hypersensitivity reactions and infusion-associated reactions (IARs), and instruct them to seek medical care immediately if such symptoms occur. If a severe hypersensitivity reaction (including anaphylaxis) or severe IAR occurs, immediately discontinue Elfabrio administration and initiate appropriate medical treatment. If a mild to moderate hypersensitivity reaction or IAR occurs, consider slowing the infusion rate or temporarily withholding the dose. In clinical trials, 20 (14%) Elfabrio-treated patients experienced hypersensitivity reactions. Four Elfabrio-treated patients (3%) experienced anaphylaxis reactions that occurred within 5 to 40 minutes of the start of the initial infusion. The signs and symptoms of hypersensitivity reactions and anaphylaxis included headache, nausea, vomiting, throat tightness, facial and oral edema, truncal rash, tachycardia, hypotension, rigors, urticaria, intense pruritus, moderate upper airway obstructions, macroglossia, and mild lip edema. In clinical trials, 41 (29%) Elfabrio-treated patients experienced one or more infusion-associated reactions, including hypersensitivity, nausea, chills, pruritus, rash, chest pain, dizziness, vomiting, asthenia, pain, sneezing, dyspnea, nasal congestion, throat irritation, abdominal pain, erythema, diarrhea, burning sensation, neuralgia, headache, paresthesia, tremor, agitation, increased body temperature, flushing, bradycardia, myalgia, hypertension, and hypotension. A case of membranoproliferative glomerulonephritis with immune depositions in the kidney was reported during clinical trials. Monitor serum creatinine and urinary protein-to-creatinine ratio. If glomerulonephritis is suspected, discontinue treatment until a diagnostic evaluation can be conducted. When switching to Elfabrio from a prior enzyme replacement therapy, the risk of hypersensitivity reactions and infusion-associated reactions may be increased in certain patients with pre-existing anti-drug antibodies (ADAs). Consider monitoring IgG and IgE ADAs and clinical or pharmacodynamic response (eg, plasma lyso-Gb3 levels). The most common adverse reactions (≥15%) were infusion-associated reactions, nasopharyngitis, headache, diarrhea, fatigue, nausea, back pain, pain in extremity, and sinusitis. Please see Full Prescribing Information for Elfabrio . Indication and Important Safety Information for Lamzede® (velmanase alfa-tycv) Indication Lamzede® (velmanase alfa-tycv) is indicated for the treatment of non-central nervous system manifestations of alpha-mannosidosis in adult and pediatric patients. Important Safety Information Considerations Due to Hypersensitivity Reactions and/or Infusion-Associated Reactions (IARs) Prior to Lamzede administration, consider pretreating with antihistamines, antipyretics, and/or corticosteroids. Inform patients and caregivers of the signs and symptoms of hypersensitivity reactions and IARs and instruct them to seek medical care immediately if such symptoms occur. If a severe hypersensitivity reaction (including anaphylaxis) or severe IAR occurs, immediately discontinue Lamzede administration and initiate appropriate medical treatment. In the event of a mild to moderate hypersensitivity reaction or a mild to moderate IAR, consider temporarily holding the infusion for 15 to 30 minutes, slowing the infusion rate to 25% to 50% of the recommended rate, and initiating appropriate medical treatment. Hypersensitivity Reactions Including Anaphylaxis Anaphylaxis and severe hypersensitivity signs and symptoms included cyanosis, hypotension, emesis, urticaria, erythema, facial swelling, pyrexia, and tremor. Infusion-Associated Reactions (IARs) The most frequent symptoms of IARs that occurred in >10% of the population were pyrexia, chills, erythema, vomiting, cough, urticaria, rash, and conjunctivitis. Females of Reproductive Potential Advise females of reproductive potential to use effective contraception during treatment and for 14 days after the last dose if Lamzede is discontinued. For females of reproductive potential, verify that the patient is not pregnant prior to initiating treatment with Lamzede. Embryo-Fetal Toxicity Based on findings from animal reproduction studies, Lamzede may cause embryo-fetal harm when administered to a pregnant female. Common Adverse Reactions The most common adverse reactions (incidence >20%) are hypersensitivity reactions including anaphylaxis, nasopharyngitis, pyrexia, headache, and arthralgia. Please see Full Prescribing Information for Lamzede. About Chiesi Global Rare Diseases Chiesi Global Rare Diseases is a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people affected by rare diseases. As a family business, Chiesi Group strives to create a world where it is common to have a therapy for all diseases and acts as a force for good, for society and the planet. The goal of the Global Rare Diseases unit is to ensure equal access so as many people as possible can experience their most fulfilling life. The unit collaborates with the rare disease community around the globe to bring voice to underserved people in the health care system. For more information visit . About Chiesi Group Chiesi is a research-oriented international biopharmaceutical group that develops and markets innovative therapeutic solutions in respiratory health, rare diseases, and specialty care. The company's mission is to improve people's quality of life and act responsibly towards both the community and the environment. By changing its legal status to a Benefit Corporation in Italy, the US, and France, Chiesi's commitment to create shared value for society as a whole is legally binding and central to company-wide decision-making. As a certified B Corp since 2019, we're part of a global community of businesses that meet high standards of social and environmental impact. The company aims to reach Net-Zero greenhouse gases (GHG) emissions by 2035. With over 85 years of experience, Chiesi is headquartered in Parma (Italy), with 31 affiliates worldwide, and counts more than 7,000 employees. The Group's research and development centre in Parma works alongside 6 other important R&D hubs in France, the US, Canada, China, the UK, and Sweden. For further information please visit . Chiesi Group Media Contacts Chiara Travagin Head of Communications, Rare Diseases Tel: +39 348 8818985 Email: [email protected] Jenna Urban CG Life Tel: +1 212 253 8881 Email: [email protected] PP-RA-00518 V1.0 SOURCE Chiesi Global Rare Diseases WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Applications for the independently assessed, expert-led Research Grant Initiative, Find For Rare, are open from today, with experts calling for innovative research to broaden the level of understanding into three lysosomal storage disorders and, ultimately, improve patient care and management. Researchers can apply for grants up to €50,000 – the research grants are designed to support original research projects that aim to advance knowledge in the field of: Fabry disease, alpha-mannosidosis, and cystinosis. All applications will be independently reviewed and assessed by a Steering Committee of leading experts in the lysosomal storage disorders field, who have been identified, contracted and funded by Chiesi, with the successful applicants announced in Q1 2025. PARMA, Italy, Sept. 9, 2024 /PRNewswire/ -- Chiesi Global Rare Diseases, a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people living with rare diseases, has today announced a new Research Grant Initiative, Find For Rare. Researchers are invited to apply for grants up to €50,000 to support their research into lysosomal storage disorders (LSDs). LSDs are inborn errors of metabolism, rare genetic disorders characterized by defective functioning of lysosomes.1 Whilst the disorders can be rare individually, their prevalence is significant at a global level with an estimated 1 in 8000 people diagnosed.1 To address current gaps in understanding, Chiesi Global Rare Diseases is organizing and funding the new call for research into three LSDs – Fabry disease, alpha-mannosidosis, and cystinosis. The initiative's ambition is to provide a platform for innovative research into improving the understanding of the factors affecting the diseases as well as patient-tailored clinical management. All submitted research proposals will be evaluated by the Find For Rare Steering Committee, an independent scientific board of 10 leading experts in the field of LSDs, chaired by three of the experts, including Professor Christoph Wanner. "Find For Rare is calling for all principal investigators and scientists with an interest in lysosomal storage disorders to apply for this fantastic research opportunity," said Professor Christoph Wanner, a Chair of the Find For Rare Steering Committee and Professor of Medicine and Chief of the Division of Nephrology and Hypertension at the University Hospital of Würzburg, Germany. "Whilst we have expanded our knowledge into LSDs in the last decade, we still need to generate greater understanding into the factors affecting the disorders from pathogenesis to progression." "Find For Rare represents an opportunity to discover new thinking and ambition to meet our ultimate goal of improved patient care. The Steering Committee is eagerly awaiting the opportunity to review new ideas from across the globe and hopefully identify excellent candidates to receive the grants." Applications will be evaluated by the independent Steering Committee according to defined assessment criteria: strength of the proposed research project, innovation and novelty, relevance and impact, and potential for success. Depending on the number and quality of applications, based on the review by the Steering Committee, the total number of grants and total amount per project awarded may vary. However, the resources set aside by Chiesi Global Rare Diseases for this initiative amount to a total of €150,000, and researchers can apply for grants up to €50,000 per project. Although Chiesi Global Rare Diseases are organizing and funding the Research Grant Initiative, Chiesi will not in any way influence the application, evaluation, nor grant selection process. Proposals are welcome from all global regions other than the Americas. "Find For Rare is an important platform for the generation of new ideas and identification of future clinical leaders in the rare disease landscape. There is a societal obligation to address issues impacting the estimated 400 million living with a rare disease," said Enrico Piccinini, Head of Europe & International, Chiesi Global Rare Diseases. "Similar to all of our activities, the inspiration behind this initiative is the rare disease community. We pride ourselves on listening closely to their needs and delivering what will truly benefit patients – our real hope is this year's Find For Rare initiative will provide future solutions to today's challenging rare conditions." Further information regarding the initiative and the application process can be found at . The application process is simple and at no cost to the researcher. The application deadline is 31 October 2024, and the grants will be awarded in Q1 2025. About Find For Rare The Find For Rare Research Grant Initiative has been designed to support original research projects that advance knowledge in the fields of Fabry disease, alpha-mannosidosis, and cystinosis. The categories of research eligible for funding are projects aimed at improving understanding of the factors affecting diseases, from pathogenesis to progression, and studies of patient tailored clinical management. Research grants will be provided to organizations operating in the health or scientific research sector. Proposals are welcome from all global regions other than the Americas, with application documents required to be in English. For more information and to apply, visit . The application process is simple and free of charge. Simply complete the application form on the website and submit it with your CV and cover letter to [email protected]. The application deadline is 31 October 2024, and the grants will be awarded in Q1 2025. About lysosomal storage disorders LSDs are inborn errors of metabolism that are characterized by an abnormal build-up of substances in the body's cells as a result of enzyme deficiencies.1 The build-up of these substances can affect different parts of the body, including the skeleton, central nervous system (brain), lungs, heart, and eyes. Whilst there has been progress in clinical knowledge, more research in LSDs can be beneficial.1 About Chiesi Global Rare Diseases Chiesi Global Rare Diseases is a business unit of the Chiesi Group, established to deliver innovative therapies and solutions for people living with rare diseases. As a family business, Chiesi Group strives to create a world where it is common to have a therapy for all diseases and acts as a force for good, for society and the planet. The goal of the Global Rare Diseases unit is to ensure equal access to therapy so as many people as possible can experience their most fulfilling life. The unit collaborates with the rare disease community around the globe to bring a voice to underserved people in the healthcare system. About Chiesi Group Chiesi is a research-oriented, international biopharmaceutical group that develops and markets innovative therapeutic solutions in respiratory health, rare diseases, and specialty care. The company's mission is to improve people's quality of life and act responsibly towards both the community and the environment. By changing its legal status to a Benefit Corporation in Italy, the US, and France, Chiesi's commitment to create shared value for society as a whole is legally binding and central to company-wide decision-making. As a certified B Corp since 2019, we're part of a global community of businesses that meet high standards of social and environmental impact. The company aims to reach Net-Zero greenhouse gases (GHG) emissions by 2035. With over 85 years of experience, Chiesi is headquartered in Parma (Italy), with 31 affiliates worldwide, and counts more than 7,000 employees. The Group's research and development centre in Parma works alongside 6 other important R&D hubs in France, the US, Canada, China, the UK, and Sweden. Media Contact Chiara Travagin Rare Communication Manager, Chiesi Global Rare Diseases Tel: +39 348 8818985 Email: [email protected] Nathan McGlone Associate Director and Media Lead, Aurora Healthcare Communications Tel: +44 7850 775 669 Email: [email protected] References 1 NCBI. Lysosomal Storage Disease. Available at: (LSDs)%20are,to%20great%20morbidity%20and%20mortality. UK-CHI-2400703 | September 2024 Logo -

This year we’re taking the branded drug names that were officially approved by the FDA in 2022 and 2023 - now it's your turn to vote for your winner. LAUNCHED: Wednesday, March 20, 7:00 a.m. ET It’s March, so it’s time for some #FierceMadness. Last year, we channeled the NCAA tournament for the best pharma ad campaigns, but, this year, we’re looking for the best of the best in drug names. Here’s how it works. We’ve chosen 64 of the most distinctive drug names to come out of the FDA’s 2022 and 2023 crop of new approvals. Whittling down to 64 from the 92 drug names available was our staff challenge, but now it’s up to you, readers, to deliberate and vote down to the final winner. Remember, this isn’t about the drugs themselves—neither how well they may work (or not) nor any controversies in how they may have been approved. Our goal is to assess their marketing suitability, considering how well they perform in their respective field and against competitors. Ready to play? Check out the bracket here (PDF) and see the full Madness 64 list below. Then vote is via our poll here. You can vote from now until Friday, March 22, at 6 p.m. ET. Check back here Monday, March 25, for your results, and the chance to vote in the next round.   After that, we’ll be back with a new round each Wednesday and Monday through Monday, April 8, when you can vote for your championship winner. Any remote robo-voting will be noted, and those votes will be discarded. Please play fair. Don’t forget to tell us why you picked what you picked in the voting poll comments section—and what you think each drug name really sounds like. We’ll include the funniest and most out-of-the-box comments in our recaps every round, so make 'em good! New this year: Our sister publication Fierce Biotech is also running its own Madness tournament for the best of the best in biotech company names. Check out and vote in its championship here.  Good luck, and happy Madness! #FierceMadness 2024: The full bracket of 64   Jesduvroq vs. Lamzede Jesduvroq Maker: GSK Indication: Anemia due to chronic kidney disease Sounds like: When you hit your finger with a hammer, but it’s in front of your children so you’re trying not to swear   Lamzede Maker: Chiesi Indication: Non-central nervous system manifestations of alpha-mannosidosis Sounds like: A vegan meal   Pluvicto vs. Lunsumio Pluvicto Maker: Novartis Indication: Prostate-specific membrane antigen-positive metastatic castration-resistant prostate cancer following other therapies Sounds like: A new planet   Lunsumio Maker: Roche Indication: Relapsed or refractory follicular lymphoma, a type of non-Hodgkin lymphoma Sounds like: A Japanese wrestler   Briumvi vs. Camzyos Briumvi Maker: TG Therapeutics Indication: Relapsing forms of multiple sclerosis Sounds like: A sixth-generation cheese    Camzyos Maker: Bristol Myers Squibb Indication: Obstructive hypertrophic cardiomyopathy Sounds like: A Spanish holiday resort   Voquezna vs. Mounjaro Voquezna Maker: Phathom Pharmaceuticals Indication: Helicobacter pylori infection Sounds like: A sports drink with a kick   Mounjaro Maker: Eli Lilly Indication: Lowering blood sugar levels in Type 2 diabetes Sounds like: A legendary medieval sword fighter    Vtama vs. Amvuttra Vtama Maker: Dermavant Indication: Plaque psoriasis Sounds like: A mouthful   Amvuttra Maker: Alnylam Indication: Polyneuropathy of hereditary transthyretin-mediated amyloidosis Sounds like: A pope’s gift    Xenpozyme vs. Daxxify Xenpozyme Maker: Sanofi Indication: Acid sphingomyelinase deficiency Sounds like: An alien protein   Daxxify  Maker: Revance Indication: The treatment of cervical dystonia and for the temporary improvement of frown lines  Sounds like: A ray gun to make you sassy    Fruzaqla vs. Sotyktu Fruzaqla Maker: Takeda Pharmaceuticals Indication: Refractory, metastatic colorectal cancer Sounds like: A European swear word   Sotyktu Maker: Bristol Myers Squibb Indication: Moderate to severe plaque psoriasis Sounds like: Gesundheit!   Terlivaz vs. Imjudo Terlivaz Maker: Mallinckrodt Pharmaceuticals Indication: Improve kidney function in adults with hepatorenal syndrome with rapid reduction in kidney function Sounds like: A Hungarian dance   Imjudo Maker: AstraZeneca Indication: Unresectable hepatocellular carcinoma Sounds like: A self-confident martial artist   Elahere vs. Vabysmo Elahere Maker: ImmunoGen Indication: Recurrent ovarian cancer that is resistant to platinum therapy Sounds like: An inebriated attempt at Hawaiian   Vabysmo Maker: Roche Indication: Neovascular (wet) aged-related macular degeneration and diabetic macular edema Sounds like: A new way of saying fantastic   Pyrukynd vs. Rystiggo Pyrukynd Maker: Agios Indication: Hemolytic anemia in pyruvate kinase deficiency Sounds like: A drunk Scrabble game   Rystiggo Maker: UCB Indication: Generalized myasthenia gravis in adults who are anti-acetylcholine receptor- or anti-muscle-specific tyrosine kinase antibody-positive Sounds like: An Italian village     Beyfortus vs. Xdemvy Beyfortus Maker: AstraZeneca Indication: To prevent respiratory syncytial virus lower respiratory tract disease Sounds like: A medieval stronghold   Xdemvy Maker: Tarsus Pharmaceuticals Indication: Demodex blepharitis Sounds like: Elon Musk’s drunk attempt at renaming his business   Tzield vs. Rezlidhia Tzield Maker: Provention Bio Indication: Delay the onset of stage 3 Type 1 diabetes Sounds like: An electronic barrier   Rezlidhia Maker: Rigel Pharmaceuticals  Indication: Relapsed or refractory acute myeloid leukemia with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation Sounds like: A new Disney princess     Krazati vs. Leqembi Krazati Maker: Mirati Therapeutics Indication: KRAS G12C-mutated locally advanced or metastatic non-small cell lung cancer in adults who have received at least one prior systemic therapy Sounds like: A crazy party   Leqembi Maker: Eisai Indication: Alzheimer’s disease Sounds like: An inspirational French aphorism   Cibinqo vs. Brenzavvy Cibinqo Maker: Pfizer Indication: Refractory, moderate to severe atopic dermatitis Sounds like: A game of cheeky bingo   Brenzavvy Maker: TheracosBio Indication: Glycemic control in adults with Type 2 diabetes mellitus as an adjunct to diet and exercise Sounds like: Heard in a copy of "Pirates of the Caribbean"   Jaypirca vs. Skyclarys Jaypirca Maker: Eli Lilly Indication: Relapsed or refractory mantle cell lymphoma in adults who have had at least two lines of systemic therapy, including a BTK inhibitor Sounds like: A strong coffee   Skyclarys Maker: Biogen Indication: Friedreich’s ataxia Sounds like: A high-rise building   Zavzpret vs. Paxlovid Zavzpret Maker: Pfizer Indication: Migraine treatment Sounds like: A Turkish fast-food restaurant   Paxlovid Maker: Pfizer Indication: Mild to moderate COVID-19 in adults at high risk for progression to severe COVID-19 Sounds like: A bizarre Pac-Man sequel   Inpefa vs. Columvi Inpefa Maker: Lexicon Pharmaceuticals Indication: Heart failure Sounds like: An anti-fascist offshoot group   Columvi Maker: Roche Indication: Diffuse large B-cell lymphoma, not otherwise specified, or large B-cell lymphoma arising from follicular lymphoma after two or more lines of systemic therapy Sounds like: An ancient Roman detective   Daybue vs. Zynyz Daybue Maker: Acadia Indication: Rett syndrome Sounds like: How my five-year-old insists debut is spelled    Zynyz Maker: Incyte Indication: Metastatic or recurrent locally advanced Merkel cell carcinoma Sounds like: Drug naming companies may be running out of ideas   Rezzayo vs. Qalsody Rezzayo Maker: Melinta Therapeutics Indication: Candidemia and invasive candidiasis Sounds like: A phrase used when you’ve had a great nap in the day   Qalsody Maker: Biogen Indication: Amyotrophic lateral sclerosis in adults who have a SOD1 gene mutation Sounds like: From the makers of “Better Call Saul” comes “better Qalsody”   Elfabrio vs. Veozah Elfabrio Maker: Chiesi Indication: Fabry disease Sounds like: The lost Spanish Mario brother   Veozah Maker: Astellas Indication: Moderate to severe hot flashes caused by menopause Sounds like: A Broadway play     Epkinly vs. Zurzuvae Epkinly Makers: AbbVie/Genmab Indication: Relapsed or refractory diffuse large B-cell lymphoma and high-grade B-cell lymphoma after two or more lines of systemic therapy Sounds like: Someone who watches too many streaming episodes   Zurzuvae Makers: Sage/Biogen Indication: Postpartum depression Sounds like: A mouthful   Izervay vs. Talvey Izervay Maker: Iveric Bio Indication: Geographic atrophy secondary to age-related macular degeneration Sounds like: A new app for scanning the land   Talvey Maker: Johnson & Johnson Indication: Relapsed or refractory multiple myeloma who have received at least four prior therapies Sounds like: A little too close to talc…   Elrexfio vs. Veopoz Elrexfio Maker: Pfizer Indication: Relapsed or refractory multiple myeloma who have received at least four prior lines of therapy Sounds like: A new dinosaur found in Mexico   Veopoz Maker: Regeneron Pharmaceuticals Indication: Patients 1 year old and older with CD55-deficient protein-losing enteropathy Sounds like: Double-plus good!   Zilbrysq vs. Ojjaara Zilbrysq Maker: UCB Indication: Generalized myasthenia gravis in adults who are anti-acetylcholine receptor antibody positive Sounds like: Absolute zero   Ojjaara Maker: GSK Indication: Intermediate or high-risk myelofibrosis in adults with anemia Sounds like: “What’s my name?”     Exxua vs. Pombiliti Exxua Maker: Fabre-Kramer Pharmaceuticals Indication: Major depressive disorder Sounds like: Someone who’s always leaving   Pombiliti Maker: Amicus Indication: Late-onset Pompe disease Sounds like: The ability to treat Pompe disease (this feels a little on the nose)    Rivfloza vs. Velsipity Rivfloza Maker: Novo Nordisk Indication: Urinary oxalate levels in patients 9 years and older with primary hyperoxaluria type 1 and relatively preserved kidney function Sounds like: An exuberant DJ   Velsipity Maker: Pfizer Indication: Moderately to severely active ulcerative colitis in adults Sounds like: A deodorant brand you wouldn’t want to buy   Bimzelx vs. Omvoh Bimzelx Maker: UCB Indication: Moderate to severe plaque psoriasis in adults who are candidates for systemic therapy or phototherapy Sounds like: Throwing away unwanted antacids   Omvoh Maker: Eli Lilly Indication: Ulcerative colitis Sounds like: A new age prayer   Loqtorzi vs. Defencath Loqtorzi Maker: Coherus Biosciences Indication: Recurrent or metastatic nasopharyngeal carcinoma when used together with or following other therapies Sounds like: How you close the door in a magical kingdom   Defencath Maker: CorMedix Indication: Reduce the incidence of catheter-related bloodstream infections in adults with kidney failure receiving chronic hemodialysis Sounds like: A Dungeons and Dragons character   Augtyro vs. Ryzneuta Augtyro Maker: Bristol Myers Squibb Indication: ROS1-positive non-small cell lung cancer Sounds like: A virtual reality game where you’re the bad guy   Ryzneuta Maker: Acrotech Biopharma Indication: Neutropenia Sounds like: Getting rid of someone’s charisma   Truqap vs. Ogsiveo Truqap Maker: AstraZeneca Indication: Breast cancer that meets certain disease criteria Sounds like: A limit on truth   Ogsiveo Maker: SpringWorks Indication: Progressing desmoid tumors who require systemic treatment Sounds like: Oh, give us a go!     Quviviq vs. Ztalmy Quviviq Maker: Idorsia Indication: Insomnia Sounds like: A producer asking a biotech founder turned politician to get ready for his TV appearance   Ztalmy Maker: Marinus Indication: Seizures in cyclin-dependent kinase-like 5 deficiency disorder Sounds like: A nefarious Yevgeny Zamyatin character   Opdualag vs. Wainua Opdualag Maker: Bristol Myers Squibb Indication: Unresectable or metastatic melanoma Sounds like: A Russian prison   Wainua Maker: AstraZeneca Indication: Polyneuropathy of hereditary transthyretin-mediated amyloidosis Sounds like: A Premier League footballer