CHIESI Farmaceutici SpA

While FDA is implementing new accelerated approval reforms, Kaiser Permanente says the reforms do not go far enough. The health system, which owns an insurer , said the agency needs to work with Congress this year on new laws that crack down on biopharma companies that don’t complete confirmatory trials. The comments from Kaiser and others are on new FDA draft guidance on accelerated approvals. The intent is to give the FDA a newly strengthened ability to ensure post-approval studies are up and running prior to approval and streamline the agency’s authority to withdraw accelerated approvals. “Although these reforms are a step in the right direction, we believe Congress must go further by statutorily requiring post-approval studies for all products approved through the accelerated approval pathway using surrogate endpoints and considering specific consequences for manufacturers who fail to complete them,” Anthony Barrueta, SVP of government relations at Kaiser, wrote in comments to FDA that were released earlier this month. The agency in December and January released new draft guidance offering more specifics on its expedited withdrawals of new accelerated approvals and how if a confirmatory trial isn’t underway before an accelerated approval, the FDA won’t clear the drug for use. While the FDA currently offers a table of surrogate endpoints that companies have historically relied on for accelerated approvals, Barrueta calls on the FDA to create a publicly available record of successful surrogate and clinical endpoints and their corresponding clinical benefits by indication, “with the evidence base underlying each of those measures.” The left-leaning philanthropy Arnold Ventures raised similar concerns in its comment to FDA, noting “the lack of clear standards for how surrogate endpoints are chosen and in which specific cases they are appropriate.” The comments come as CMS denied coverage for Biogen’s first Alzheimer’s drug Aduhelm in 2022, following an unexpected accelerated approval from the FDA. That coverage denial led to broader questions on if insurers might take a harder look at accelerated approvals. “The accelerated approval program’s current weaknesses deprive physicians, patients, and other stakeholders of vital evidence that can help improve outcomes and avoid paying for ineffective therapies,” Barrueta wrote in his comment. In its comment, Harvard Medical School’s Program On Regulation, Therapeutics, And Law also called for alerts when confirmatory studies are delayed, unpublished or negative, as well as additional benefit-risk reviews to align with the European Medicines Agency’s standards for its conditional approval pathway. Italy-based Chiesi Farmaceutici is the only pharma company to comment on the draft guidance so far, calling on the FDA to describe how the agency may account for the patient and caregiver experience when it comes to confirmatory trials. Chiesi also notes the unintended consequences of provisional accelerated approval qualifications, noting that it recently filed an application for a drug for a rare disease with one available therapy approved under the accelerated pathway 18 years prior. Chiesi’s priority review action date was extended by four months by the FDA, “and during that time the other product approval was converted to a ‘traditional’ approval. As a result, Chiesi’s product was blocked from an accelerated approval just prior to the action date.”

- 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

SAN DIEGO--( BUSINESS WIRE )-- Gossamer Bio, Inc. (Nasdaq: GOSS), a clinical-stage biopharmaceutical company focused on the development and commercialization of seralutinib for the treatment of pulmonary arterial hypertension (PAH) and pulmonary hypertension associated with interstitial lung disease (PH-ILD), today announced one oral and three poster presentations related to seralutinib at the Pulmonary Vascular Research Institute (PVRI) 2025 Annual Congress taking place January 29 th through February 1 st in Rio de Janeiro, Brazil. “Gossamer is committed to generating and presenting data that reflect the rapidly evolving pulmonary hypertension treatment landscape to best address the needs of patients. To that end, we are immensely proud to be able to present data from preclinical models that provide evidence of a synergistic treatment effect from the combination of seralutinib and sotatercept, a recently approved therapy for the treatment of PAH,” said Faheem Hasnain, Co-Founder, CEO, and Chairman of Gossamer Bio. “These data underscore seralutinib’s potential to transform the PAH treatment paradigm, if approved, and they highlight the potential promise of these complementary mechanisms of action when utilized in combination. PAH is a progressive disease in which polypharmacy is the standard of care, and these preclinical results further build upon the positive PAH patient data generated in the Phase 2 TORREY Study, in which a majority of patients were on triple background PAH therapy.” “Furthermore, our ongoing global registrational Phase 3 PROSERA Study in PAH allows for the enrollment of a small number of patients on stable background treatment with sotatercept, provided they meet study entry criteria. All other patients who are enrolled in PROSERA may add sotatercept after 24 weeks, where approved and as appropriate. These study design elements will provide a limited, first-in-human combination experience with seralutinib and sotatercept, and importantly, they have been welcomed by patients and investigators, alike,” added Mr. Hasnain. Presentations related to Seralutinib, an Inhaled PDGFR, CSF1R and c-KIT Inhibitor: Preclinical Models Support the Synergistic Potential of Seralutinib and Sotatercept in Treating Pulmonary Arterial Hypertension Oral presentation Session: Risk stratification & treatment in the new era of PH therapeutics Date: Thursday, January 30 th Time: 3:07pm – 3:15pm BRT Location: Windsor Oceanico, Asia, 3 rd floor Presenter: Ravikumar Sitapara, MS, PhD Poster presentation Date: Friday, January 31 st Time: 4:30pm – 6:00pm BRT Location: Windsor Oceanico, Asia, 3 rd floor Sustained Benefit with Seralutinib Treatment: A Post-Hoc Analysis of the TORREY Open-Label Extension Poster presentation Date: Friday, January 31 st Time: 4:30pm – 6:00pm BRT Location: Windsor Oceanico, Asia, 3 rd floor Sustained Effect of Seralutinib on Circulating Biomarkers in the TORREY Phase 2 Open-Label Extension Study Poster presentation Date: Friday, January 31 st Time: 4:30pm – 6:00pm BRT Location: Windsor Oceanico, Asia, 3 rd floor About Gossamer Bio Gossamer Bio is a clinical-stage biopharmaceutical company focused on the development and commercialization of seralutinib for the treatment of pulmonary hypertension. Its goal is to be an industry leader in, and to enhance the lives of patients living with, pulmonary hypertension. Forward-Looking Statements Gossamer cautions you that statements contained in this press release regarding matters that are not historical facts are forward-looking statements. These statements are based on the Company’s current beliefs and expectations. Such forward-looking statements include, but are not limited to, statements regarding: the ability to generate relevant seralutinib clinical data for the evolving PAH treatment landscape and the development and market potential of seralutinib. The inclusion of forward-looking statements should not be regarded as a representation by Gossamer that any of its plans will be achieved. Actual results may differ from those set forth in this press release due to the risks and uncertainties inherent in Gossamer’s business, including, without limitation: potential delays in the commencement, enrollment and completion of clinical trials; disruption to our operations from a pandemic, including clinical trial delays; the Company’s dependence on third parties in connection with product manufacturing, research and preclinical and clinical testing; the results of preclinical studies and early clinical trials are not necessarily predictive of future results; the success of Gossamer’s clinical trials and preclinical studies for seralutinib; regulatory developments in the United States and foreign countries; unexpected adverse side effects or inadequate efficacy of seralutinib that may limit its development, regulatory approval and/or commercialization, or may result in clinical holds, recalls or product liability claims; Gossamer’s ability to obtain and maintain intellectual property protection for seralutinib; Gossamer’s ability to comply with its obligations in collaboration agreements with third parties, including Chiesi, or the agreements under which it licenses intellectual property rights from third parties; unstable market and economic conditions and adverse developments with respect to financial institutions and associated liquidity risk may adversely affect our business and financial condition and the broader economy and biotechnology industry; Gossamer may use its capital resources sooner than it expects; and other risks described in the Company’s prior press releases and the Company’s filings with the Securities and Exchange Commission (SEC), including under the heading “Risk Factors” in the Company’s annual report on Form 10-K and any subsequent filings with the SEC. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof, and Gossamer undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, which is made under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995.