{"success":true,"data":{"pressRelease":{"id":"154646","rtpr_id":"nGNE1bXTJ1-20260928","ticker":"EGTX","exchange":"Nasdaq Stockholm","all_tickers":["EGTX"],"title":"Egetis Therapeutics Announces U.S. FDA Approval of EMCITATE® (tiratricol) for Patients with MCT8 Deficiency","author":"Globe Newswire","published_at":"2026-09-28T21:46:00.097Z","article_body":"* First and only FDA-approved treatment of peripheral thyrotoxicosis in adults\nand pediatric patients with monocarboxylate transporter 8 (MCT8) deficiency in\nthe United States\n* Egetis launches Egetis RareLink™, the EMCITATE patient support program, in\npartnership with PANTHERx® Rare\n* FDA granted Egetis a Rare Pediatric Disease Priority Review Voucher (PRV) in\nconnection with the approval \n* Egetis to host a conference call and webcast for analysts and investors on\nTuesday September 29 at 8:00 am CEST (2:00 am EDT)\nSTOCKHOLM, SWEDEN, Sept. 28 2026. (GLOBE NEWSWIRE) – Egetis Therapeutics AB\n(publ) (\"Egetis\" or the \"Company\") (Nasdaq Stockholm: EGTX) today announced\nthat the U.S. Food and Drug Administration (FDA) has approved EMCITATE®\n(tiratricol), a thyroid hormone receptor agonist, for the treatment of\nperipheral thyrotoxicosis in adults and pediatric patients with\nmonocarboxylate transporter 8 (MCT8) deficiency (Allan–Herndon–Dudley\nsyndrome). EMCITATE is not recommended for the treatment of primary\nhypothyroidism. EMCITATE is the first FDA-approved treatment option for\npatients with MCT8 deficiency in the United States.\n\nMCT8 deficiency is a rare, devastating, life-limiting, X-linked disorder\ncaused by pathogenic mutations in the SLC16A2 gene. These mutations impair the\nfunction of MCT8, a critical cell-surface thyroid-hormone transporter\nresponsible for thyroid hormone transport in specific cells, including in the\nbrain. As a result, patients experience disrupted thyroid hormone signaling,\ncharacterized by insufficient thyroid hormone activity in the central nervous\nsystem and excessive exposure to the active thyroid hormone triiodothyronine\n(T3) in peripheral tissues. This leads to a complex disorder characterized by\nsevere neurodevelopmental impairment and persistent peripheral thyrotoxicosis.\nPatients with MCT8 deficiency have a reported median life expectancy of\napproximately 35 years. For further details, please see ‘About MCT8\ndeficiency’ below.\n\nThe FDA approval of EMCITATE was supported by a comprehensive clinical\ndevelopment program evaluating EMCITATE in patients with MCT8 deficiency,\nincluding ReTRIACt, Triac Trial I, Triac Trial II, Erasmus Medical Center\n(EMC) Cohort Study, EMC Survival Study and the U.S. Expanded Access Program.\n \n\n“Today marks a turning point for patients living with MCT8 deficiency and\ntheir caregivers, who have waited long for an approved treatment in the United\nStates. Our immediate focus is ensuring that eligible patients can access\nEMCITATE as quickly as possible,” said Nicklas Westerholm, Chief Executive\nOfficer of Egetis Therapeutics. “We are deeply grateful to the patients,\ncaregivers, investigators, clinicians, and advocacy organizations whose\npartnership and determination made this achievement possible as well as all\nEgetis employees and collaborators for their dedicated and hard work. Today\nalso marks a defining milestone for Egetis as we take steps forward in\ndeveloping medicines that address serious unmet medical needs for patients\nwith rare diseases.”\n\n \"Through my experience caring for patients with MCT8 deficiency, I have seen\nfirsthand the profound impact this complex and life-limiting disorder can have\non patients and their families. Early diagnosis is critical so that patients\ncan be appropriately evaluated, connected with specialists and receive\ncoordinated multidisciplinary care. The FDA approval of EMCITATE provides\nphysicians in the United States with the first approved treatment option for\npatients and represents an important advance for the MCT8 deficiency\ncommunity,” said Andrew J. Bauer, M.D., a pediatric endocrinologist and\nexpert in thyroid hormone disorders and Principal Investigator in ReTRIACt\ntrial and Triac Trial II, evaluating EMCITATE in MCT8 deficiency.\n\n“The FDA approval of EMCITATE is a historic milestone for the MCT8\ndeficiency community and an important step toward ensuring patients have\naccess to a treatment. We are grateful to Egetis and to the researchers,\nclinicians, patients, families, and advocates whose dedication helped bring\nthis treatment to patients. This approval is a testament to what is possible\nwhen a community comes together with a shared commitment to advancing care. We\nlook forward to continuing to advocate for patients and supporting efforts to\nexpand awareness, access and treatment options for the worldwide MCT8\ncommunity.” said the MCT8-AHDS Foundation.\n\nLaunch of Egetis RareLink™: EMCITATE® comprehensive patient support program\n\nEgetis is committed to helping eligible patients gain timely access to\nEMCITATE. The Company expects EMCITATE to be commercially available in the\nUnited States in eight to ten weeks post approval. Through Egetis RareLink,\nits dedicated patient support program, Egetis has established the access\ninfrastructure—including specialty distribution and dedicated support\nresources—to help ensure a seamless experience for patients, caregivers, and\nhealthcare professionals from day one. As part of its U.S. commercialization\nstrategy, Egetis has partnered with PANTHERx® Rare to support medication\naccess, education, care coordination and ongoing treatment services. For more\ninformation about Egetis RareLink, call 1-844-4EGETIS (1-844-434-3847).\n\nPriority review voucher granted\n\nIn connection with the approval of EMCITATE, the FDA granted Egetis a Rare\nPediatric Disease Priority Review Voucher (PRV).  The Company currently\nexpects to explore monetization of the PRV, which could potentially occur in\nthe fourth quarter of 2026, subject to market conditions.\n\nConference call and webcast information\n\nEgetis will host a conference call and webcast for analysts and investors to\ndiscuss the FDA approval of EMCITATE beginning at 8:00 am CEST (2:00 am EDT)\non Tuesday, September 29, 2026.\n\nWebcast link: https://live.events.inderes.com/fda-approval-sep-2026  \n\nTeleconference link:\nhttps://events.inderes.com/live/fda-approval-sep-2026/dial-in   \n\nAfter registration to the teleconference you will be provided phone numbers\nand a conference ID to access the call. You can ask questions verbally via the\nteleconference. A replay of the webcast can be accessed via the webcast link\nabove.\n\nAbout MCT8 deficiency\n\nMonocarboxylate transporter 8 (MCT8) deficiency, also known as\nAllan–Herndon–Dudley syndrome (AHDS), is a rare, devastating,\nlife-limiting, X-linked disorder caused by pathogenic mutations in the SLC16A2\ngene. These mutations impair the function of MCT8, a critical cell-surface\nthyroid hormone transporter responsible for thyroid hormone transport in\nspecific cells, including in the brain. As a result, patients experience\ndisrupted thyroid hormone signaling, characterized by insufficient thyroid\nhormone activity in the central nervous system and excessive exposure to the\nactive thyroid hormone triiodothyronine (T3) in peripheral tissues. This leads\nto a complex disorder characterized by severe neurodevelopmental impairment\nand persistent peripheral thyrotoxicosis.\n\nThe elevated T3 concentrations in peripheral tissues can result in a chronic\nhypermetabolic state affecting multiple organs, including the heart, muscles,\nliver, and kidneys. Clinical manifestations may include failure to thrive,\ncardiovascular strain, muscle wasting, metabolic imbalance, and increased\nsusceptibility to infections. These systemic consequences of persistent\nthyrotoxicosis are believed to contribute significantly to the increased\nmorbidity and premature mortality associated with the disorder. Patients with\nMCT8 deficiency have a reported median life expectancy of approximately 35\nyears.\n\nFor more information about MCT8 deficiency, please visit MCT8deficiency.com or\nLifewithMCT8deficiency.com\n\nINDICATION AND USAGE\n\nEMCITATE is a thyroid hormone receptor agonist indicated for the treatment of\nperipheral thyrotoxicosis in adults and pediatric patients with\nmonocarboxylate transporter 8 (MCT8) deficiency (Allan–Herndon–Dudley\nsyndrome).\n\nLimitation of use: EMCITATE is not recommended for the treatment of primary\nhypothyroidism.\n\nIMPORTANT SAFETY INFORMATION\n\nBOXED WARNING\nNOT FOR TREATMENT OF OBESITY OR FOR WEIGHT LOSS\n\nCONTRAINDICATIONS\nPrimary hyperthyroidism\n\nWARNINGS AND PRECAUTIONS\nThyrotoxicosis: Signs and symptoms of thyrotoxicosis (e.g., increased heart\nrate, elevated blood pressure, diarrhea, hyperhidrosis, irritability,\ninsomnia, nightmares) have occurred with EMCITATE during treatment initiation\nand dose titration. Monitor and adjust the EMCITATE dose as indicated. \nLaboratory Test Interference for T3 Measurement: Tiratricol can cross-react\nwith immunoassays for T3 leading to unreliable T3 results and cause an\noverestimation of T3.\n\nADVERSE REACTIONS\nMost common adverse reactions (≥ 5%): diarrhea, vomiting, rash, and\nhyperhidrosis.\n\nTo report SUSPECTED ADVERSE REACTIONS, contact Egetis Therapeutics US Inc. at\n1-844-4EGETIS (1-844-434-3847) or FDA at 1-800-FDA-1088 or\nwww.fda.gov/medwatch.\n\nPlease see full Prescribing Information here:\nhttps://www.egetis.com/wp-content/uploads/emcitate-full-prescribing-information.pdf\n\n\n\nAbout Egetis Therapeutics\n\nEgetis Therapeutics is a commercial-stage pharmaceutical company focused on\ndeveloping and delivering innovative therapies for patients with rare diseases\nwith significant unmet medical need. Combining scientific and clinical\nexpertise with integrated late-stage development, regulatory and commercial\ncapabilities and manufacturing expertise, Egetis is committed to delivering\ntreatments and dedicated support to underserved patient communities. The\nCompany's lead product, EMCITATE® (tiratricol), is the first and only\napproved treatment for MCT8 deficiency, a rare and life-shortening genetic\ndisorder, with marketing authorizations in the European Union and now in the\nUnited States. Headquartered in Stockholm, Sweden, with operations in the\nU.S. and Europe, Egetis is building a sustainable global rare disease company.\n\nFor more information, visit egetis.com and follow the Company on LinkedIn.\n\n\n\nAbout PANTHERx Rare \n\nPANTHERx Rare makes rare disease care more hyper-personalized and less\noverwhelming by focusing relentlessly on each patient and each therapy.\nPANTHERx experts develop deep personal relationships with patients,\nprescribers, and pharmaceutical partners, serving as trusted advocates to\nensure seamless collaboration and exceptional care. Since its founding in a\ngarage in Pittsburgh, PA in 2011, PANTHERx has grown into the largest\nindependent rare pharmacy in the U.S., leveraging established-company\nresources while maintaining small-company responsiveness, innovation, and\nattention to detail.\n\nPANTHERx is licensed in all 50 states and U.S. territories and was the first\nnational pharmacy to achieve dual accreditations in rare disease from the\nAccreditation Commission for Health Care (ACHC) and Utilization Review\nAccreditation Commission (URAC). PANTHERx is also the nine-time winner of the\nprestigious MMIT Patient Choice Award for patient satisfaction, including the\n2026 honor.\n\nFor more information, please email TheRareSP@pantherxrare.com or visit\nwww.pantherxrare.com\n\nContacts\n\nNicklas Westerholm, CEO\nnicklas.westerholm@egetis.com\n+46 (0) 733 542 062\n\nKarl Hård, Head of Investor Relations, Communications & Business Development\nkarl.hard@egetis.com\n+46 (0) 733 011 944\n\nThis information is information that Egetis Therapeutics is obliged to make\npublic pursuant to the EU Market Abuse Regulation. The information was\nsubmitted for publication, through the agency of the contact persons set out\nabove, at 11:46 pm CEST on September 28, 2026.\n\n\n\nForward-Looking Statements\n\nThis press release contains forward-looking statements within the meaning of\napplicable securities laws, including statements regarding the\ncommercialization and availability of EMCITATE® (tiratricol) in the United\nStates, the timing and outcome of potential monetization of the Rare Pediatric\nDisease Priority Review Voucher, the expected capabilities of the Company’s\npatient access and distribution infrastructure, the anticipated benefits of\nEMCITATE for patients with MCT8 deficiency, and the Company’s broader\nstrategic plans for the development and commercialization of therapies for\nrare diseases. Forward-looking statements can generally be identified by words\nsuch as “expects,” “anticipates,” “intends,” “believes,”\n“estimates,” “plans,” “will,” “may,” “could,”\n“potential,” or similar expressions, although not all forward-looking\nstatements contain these words.\nThese statements are based on the Company’s current expectations,\nassumptions, and assessments as of the date of this press release and are\nsubject to risks, uncertainties, and other factors that could cause actual\nresults to differ materially from those expressed or implied. Such factors\ninclude, but are not limited to: risks related to the commercial launch and\nmarket acceptance of EMCITATE in the United States; the ability to establish\nand maintain adequate commercial infrastructure, including distribution,\npatient support, and reimbursement arrangements; the timing and terms of any\nmonetization of the Priority Review Voucher, which is subject to market\nconditions and the availability of interested purchasers; potential\npost-marketing requirements or restrictions imposed by the FDA; the ability to\nmaintain regulatory approvals in the United States and the European Union;\ncompetition from existing or future therapies; the Company’s ability to\nsecure adequate funding for its operations and commercial activities; and\ngeneral economic, market, and business conditions.\nFor a further description of risks and uncertainties that could affect the\nCompany’s business and results, reference is made to the Company’s most\nrecent annual report and other filings with relevant regulatory authorities\navailable at www.egetis.com. The Company undertakes no obligation to publicly\nupdate or revise any forward-looking statements, whether as a result of new\ninformation, future events, or otherwise, except as required by applicable law\nor regulation.","article_body_html":"","raw_payload":{"data":{"id":"nGNE1bXTJ1-20260928","title":"Egetis Therapeutics Announces U.S. FDA Approval of EMCITATE® (tiratricol) for Patients with MCT8 Deficiency","author":"Globe Newswire","ticker":"EGTX","created":"2026-09-28T21:46:00.097Z","tickers":["EGTX"],"exchange":"Nasdaq Stockholm","article_body":"* First and only FDA-approved treatment of peripheral thyrotoxicosis in adults\nand pediatric patients with monocarboxylate transporter 8 (MCT8) deficiency in\nthe United States\n* Egetis launches Egetis RareLink™, the EMCITATE patient support program, in\npartnership with PANTHERx® Rare\n* FDA granted Egetis a Rare Pediatric Disease Priority Review Voucher (PRV) in\nconnection with the approval \n* Egetis to host a conference call and webcast for analysts and investors on\nTuesday September 29 at 8:00 am CEST (2:00 am EDT)\nSTOCKHOLM, SWEDEN, Sept. 28 2026. (GLOBE NEWSWIRE) – Egetis Therapeutics AB\n(publ) (\"Egetis\" or the \"Company\") (Nasdaq Stockholm: EGTX) today announced\nthat the U.S. Food and Drug Administration (FDA) has approved EMCITATE®\n(tiratricol), a thyroid hormone receptor agonist, for the treatment of\nperipheral thyrotoxicosis in adults and pediatric patients with\nmonocarboxylate transporter 8 (MCT8) deficiency (Allan–Herndon–Dudley\nsyndrome). EMCITATE is not recommended for the treatment of primary\nhypothyroidism. EMCITATE is the first FDA-approved treatment option for\npatients with MCT8 deficiency in the United States.\n\nMCT8 deficiency is a rare, devastating, life-limiting, X-linked disorder\ncaused by pathogenic mutations in the SLC16A2 gene. These mutations impair the\nfunction of MCT8, a critical cell-surface thyroid-hormone transporter\nresponsible for thyroid hormone transport in specific cells, including in the\nbrain. As a result, patients experience disrupted thyroid hormone signaling,\ncharacterized by insufficient thyroid hormone activity in the central nervous\nsystem and excessive exposure to the active thyroid hormone triiodothyronine\n(T3) in peripheral tissues. This leads to a complex disorder characterized by\nsevere neurodevelopmental impairment and persistent peripheral thyrotoxicosis.\nPatients with MCT8 deficiency have a reported median life expectancy of\napproximately 35 years. For further details, please see ‘About MCT8\ndeficiency’ below.\n\nThe FDA approval of EMCITATE was supported by a comprehensive clinical\ndevelopment program evaluating EMCITATE in patients with MCT8 deficiency,\nincluding ReTRIACt, Triac Trial I, Triac Trial II, Erasmus Medical Center\n(EMC) Cohort Study, EMC Survival Study and the U.S. Expanded Access Program.\n \n\n“Today marks a turning point for patients living with MCT8 deficiency and\ntheir caregivers, who have waited long for an approved treatment in the United\nStates. Our immediate focus is ensuring that eligible patients can access\nEMCITATE as quickly as possible,” said Nicklas Westerholm, Chief Executive\nOfficer of Egetis Therapeutics. “We are deeply grateful to the patients,\ncaregivers, investigators, clinicians, and advocacy organizations whose\npartnership and determination made this achievement possible as well as all\nEgetis employees and collaborators for their dedicated and hard work. Today\nalso marks a defining milestone for Egetis as we take steps forward in\ndeveloping medicines that address serious unmet medical needs for patients\nwith rare diseases.”\n\n \"Through my experience caring for patients with MCT8 deficiency, I have seen\nfirsthand the profound impact this complex and life-limiting disorder can have\non patients and their families. Early diagnosis is critical so that patients\ncan be appropriately evaluated, connected with specialists and receive\ncoordinated multidisciplinary care. The FDA approval of EMCITATE provides\nphysicians in the United States with the first approved treatment option for\npatients and represents an important advance for the MCT8 deficiency\ncommunity,” said Andrew J. Bauer, M.D., a pediatric endocrinologist and\nexpert in thyroid hormone disorders and Principal Investigator in ReTRIACt\ntrial and Triac Trial II, evaluating EMCITATE in MCT8 deficiency.\n\n“The FDA approval of EMCITATE is a historic milestone for the MCT8\ndeficiency community and an important step toward ensuring patients have\naccess to a treatment. We are grateful to Egetis and to the researchers,\nclinicians, patients, families, and advocates whose dedication helped bring\nthis treatment to patients. This approval is a testament to what is possible\nwhen a community comes together with a shared commitment to advancing care. We\nlook forward to continuing to advocate for patients and supporting efforts to\nexpand awareness, access and treatment options for the worldwide MCT8\ncommunity.” said the MCT8-AHDS Foundation.\n\nLaunch of Egetis RareLink™: EMCITATE® comprehensive patient support program\n\nEgetis is committed to helping eligible patients gain timely access to\nEMCITATE. The Company expects EMCITATE to be commercially available in the\nUnited States in eight to ten weeks post approval. Through Egetis RareLink,\nits dedicated patient support program, Egetis has established the access\ninfrastructure—including specialty distribution and dedicated support\nresources—to help ensure a seamless experience for patients, caregivers, and\nhealthcare professionals from day one. As part of its U.S. commercialization\nstrategy, Egetis has partnered with PANTHERx® Rare to support medication\naccess, education, care coordination and ongoing treatment services. For more\ninformation about Egetis RareLink, call 1-844-4EGETIS (1-844-434-3847).\n\nPriority review voucher granted\n\nIn connection with the approval of EMCITATE, the FDA granted Egetis a Rare\nPediatric Disease Priority Review Voucher (PRV).  The Company currently\nexpects to explore monetization of the PRV, which could potentially occur in\nthe fourth quarter of 2026, subject to market conditions.\n\nConference call and webcast information\n\nEgetis will host a conference call and webcast for analysts and investors to\ndiscuss the FDA approval of EMCITATE beginning at 8:00 am CEST (2:00 am EDT)\non Tuesday, September 29, 2026.\n\nWebcast link: https://live.events.inderes.com/fda-approval-sep-2026  \n\nTeleconference link:\nhttps://events.inderes.com/live/fda-approval-sep-2026/dial-in   \n\nAfter registration to the teleconference you will be provided phone numbers\nand a conference ID to access the call. You can ask questions verbally via the\nteleconference. A replay of the webcast can be accessed via the webcast link\nabove.\n\nAbout MCT8 deficiency\n\nMonocarboxylate transporter 8 (MCT8) deficiency, also known as\nAllan–Herndon–Dudley syndrome (AHDS), is a rare, devastating,\nlife-limiting, X-linked disorder caused by pathogenic mutations in the SLC16A2\ngene. These mutations impair the function of MCT8, a critical cell-surface\nthyroid hormone transporter responsible for thyroid hormone transport in\nspecific cells, including in the brain. As a result, patients experience\ndisrupted thyroid hormone signaling, characterized by insufficient thyroid\nhormone activity in the central nervous system and excessive exposure to the\nactive thyroid hormone triiodothyronine (T3) in peripheral tissues. This leads\nto a complex disorder characterized by severe neurodevelopmental impairment\nand persistent peripheral thyrotoxicosis.\n\nThe elevated T3 concentrations in peripheral tissues can result in a chronic\nhypermetabolic state affecting multiple organs, including the heart, muscles,\nliver, and kidneys. Clinical manifestations may include failure to thrive,\ncardiovascular strain, muscle wasting, metabolic imbalance, and increased\nsusceptibility to infections. These systemic consequences of persistent\nthyrotoxicosis are believed to contribute significantly to the increased\nmorbidity and premature mortality associated with the disorder. Patients with\nMCT8 deficiency have a reported median life expectancy of approximately 35\nyears.\n\nFor more information about MCT8 deficiency, please visit MCT8deficiency.com or\nLifewithMCT8deficiency.com\n\nINDICATION AND USAGE\n\nEMCITATE is a thyroid hormone receptor agonist indicated for the treatment of\nperipheral thyrotoxicosis in adults and pediatric patients with\nmonocarboxylate transporter 8 (MCT8) deficiency (Allan–Herndon–Dudley\nsyndrome).\n\nLimitation of use: EMCITATE is not recommended for the treatment of primary\nhypothyroidism.\n\nIMPORTANT SAFETY INFORMATION\n\nBOXED WARNING\nNOT FOR TREATMENT OF OBESITY OR FOR WEIGHT LOSS\n\nCONTRAINDICATIONS\nPrimary hyperthyroidism\n\nWARNINGS AND PRECAUTIONS\nThyrotoxicosis: Signs and symptoms of thyrotoxicosis (e.g., increased heart\nrate, elevated blood pressure, diarrhea, hyperhidrosis, irritability,\ninsomnia, nightmares) have occurred with EMCITATE during treatment initiation\nand dose titration. Monitor and adjust the EMCITATE dose as indicated. \nLaboratory Test Interference for T3 Measurement: Tiratricol can cross-react\nwith immunoassays for T3 leading to unreliable T3 results and cause an\noverestimation of T3.\n\nADVERSE REACTIONS\nMost common adverse reactions (≥ 5%): diarrhea, vomiting, rash, and\nhyperhidrosis.\n\nTo report SUSPECTED ADVERSE REACTIONS, contact Egetis Therapeutics US Inc. at\n1-844-4EGETIS (1-844-434-3847) or FDA at 1-800-FDA-1088 or\nwww.fda.gov/medwatch.\n\nPlease see full Prescribing Information here:\nhttps://www.egetis.com/wp-content/uploads/emcitate-full-prescribing-information.pdf\n\n\n\nAbout Egetis Therapeutics\n\nEgetis Therapeutics is a commercial-stage pharmaceutical company focused on\ndeveloping and delivering innovative therapies for patients with rare diseases\nwith significant unmet medical need. Combining scientific and clinical\nexpertise with integrated late-stage development, regulatory and commercial\ncapabilities and manufacturing expertise, Egetis is committed to delivering\ntreatments and dedicated support to underserved patient communities. The\nCompany's lead product, EMCITATE® (tiratricol), is the first and only\napproved treatment for MCT8 deficiency, a rare and life-shortening genetic\ndisorder, with marketing authorizations in the European Union and now in the\nUnited States. Headquartered in Stockholm, Sweden, with operations in the\nU.S. and Europe, Egetis is building a sustainable global rare disease company.\n\nFor more information, visit egetis.com and follow the Company on LinkedIn.\n\n\n\nAbout PANTHERx Rare \n\nPANTHERx Rare makes rare disease care more hyper-personalized and less\noverwhelming by focusing relentlessly on each patient and each therapy.\nPANTHERx experts develop deep personal relationships with patients,\nprescribers, and pharmaceutical partners, serving as trusted advocates to\nensure seamless collaboration and exceptional care. Since its founding in a\ngarage in Pittsburgh, PA in 2011, PANTHERx has grown into the largest\nindependent rare pharmacy in the U.S., leveraging established-company\nresources while maintaining small-company responsiveness, innovation, and\nattention to detail.\n\nPANTHERx is licensed in all 50 states and U.S. territories and was the first\nnational pharmacy to achieve dual accreditations in rare disease from the\nAccreditation Commission for Health Care (ACHC) and Utilization Review\nAccreditation Commission (URAC). PANTHERx is also the nine-time winner of the\nprestigious MMIT Patient Choice Award for patient satisfaction, including the\n2026 honor.\n\nFor more information, please email TheRareSP@pantherxrare.com or visit\nwww.pantherxrare.com\n\nContacts\n\nNicklas Westerholm, CEO\nnicklas.westerholm@egetis.com\n+46 (0) 733 542 062\n\nKarl Hård, Head of Investor Relations, Communications & Business Development\nkarl.hard@egetis.com\n+46 (0) 733 011 944\n\nThis information is information that Egetis Therapeutics is obliged to make\npublic pursuant to the EU Market Abuse Regulation. The information was\nsubmitted for publication, through the agency of the contact persons set out\nabove, at 11:46 pm CEST on September 28, 2026.\n\n\n\nForward-Looking Statements\n\nThis press release contains forward-looking statements within the meaning of\napplicable securities laws, including statements regarding the\ncommercialization and availability of EMCITATE® (tiratricol) in the United\nStates, the timing and outcome of potential monetization of the Rare Pediatric\nDisease Priority Review Voucher, the expected capabilities of the Company’s\npatient access and distribution infrastructure, the anticipated benefits of\nEMCITATE for patients with MCT8 deficiency, and the Company’s broader\nstrategic plans for the development and commercialization of therapies for\nrare diseases. Forward-looking statements can generally be identified by words\nsuch as “expects,” “anticipates,” “intends,” “believes,”\n“estimates,” “plans,” “will,” “may,” “could,”\n“potential,” or similar expressions, although not all forward-looking\nstatements contain these words.\nThese statements are based on the Company’s current expectations,\nassumptions, and assessments as of the date of this press release and are\nsubject to risks, uncertainties, and other factors that could cause actual\nresults to differ materially from those expressed or implied. Such factors\ninclude, but are not limited to: risks related to the commercial launch and\nmarket acceptance of EMCITATE in the United States; the ability to establish\nand maintain adequate commercial infrastructure, including distribution,\npatient support, and reimbursement arrangements; the timing and terms of any\nmonetization of the Priority Review Voucher, which is subject to market\nconditions and the availability of interested purchasers; potential\npost-marketing requirements or restrictions imposed by the FDA; the ability to\nmaintain regulatory approvals in the United States and the European Union;\ncompetition from existing or future therapies; the Company’s ability to\nsecure adequate funding for its operations and commercial activities; and\ngeneral economic, market, and business conditions.\nFor a further description of risks and uncertainties that could affect the\nCompany’s business and results, reference is made to the Company’s most\nrecent annual report and other filings with relevant regulatory authorities\navailable at www.egetis.com. The Company undertakes no obligation to publicly\nupdate or revise any forward-looking statements, whether as a result of new\ninformation, future events, or otherwise, except as required by applicable law\nor regulation."},"type":"article","timestamp":"2026-09-28T21:46:00.210570739Z","server_sent_at_ms":1790631960210},"received_at":"2026-09-28T21:46:00.399Z","source_url":null},"analysis":{"id":"143405","press_release_id":"154646","analysis_json":{"industry":{"label":"Pharmaceuticals, Biotechnology & Life Sciences","sector":"Health Care"},"redFlags":["Boxed warning: NOT FOR TREATMENT OF OBESITY OR FOR WEIGHT LOSS","PRV monetization timing and value subject to market conditions","Ultrarare disease limits U.S. commercial revenue potential"],"eventType":"fda_approval","narrative":"Egetis Therapeutics received FDA approval of EMCITATE (tiratricol), the first and only approved treatment for peripheral thyrotoxicosis in adults and pediatric patients with MCT8 deficiency in the United States.\n\nThe company expects U.S. commercial availability in eight to ten weeks, supported by the Egetis RareLink patient support program and a partnership with PANTHERx Rare for specialty distribution and access services.\n\nThe FDA also granted a Rare Pediatric Disease Priority Review Voucher, which Egetis may look to monetize as early as Q4 2026, providing a potential non-dilutive cash inflection alongside the U.S. launch.","sentiment":"bullish","agentHooks":{"shouldPost":true,"suggestedAngle":"First-in-class FDA approval for Egetis with U.S. launch in 8-10 weeks and a monetizable priority review voucher on top."},"keyFigures":{"drugName":"EMCITATE (tiratricol)","phaseOfTrial":"ReTRIACt, Triac Trial I, Triac Trial II, EMC Cohort Study, EMC Survival Study","customDimensions":{"prv_granted":true,"median_life_expectancy_mct8":"approximately 35 years","commercial_availability_weeks":"eight to ten weeks post approval"}},"quotedText":"Today marks a turning point for patients living with MCT8 deficiency and their caregivers, who have waited long for an approved treatment in the United States.","namedEntities":{"people":[{"name":"Nicklas Westerholm","role":"CEO"},{"name":"Andrew J. Bauer, M.D.","role":"pediatric endocrinologist, Principal Investigator in ReTRIACt and Triac Trial II"},{"name":"Karl Hård","role":"Head of Investor Relations, Communications & Business Development"}],"products":["EMCITATE (tiratricol)","Egetis RareLink"],"companies":[{"name":"Egetis Therapeutics AB","ticker":"EGTX","relationship":"filer"},{"name":"PANTHERx Rare","relationship":"specialty pharmacy partner"},{"name":"MCT8-AHDS Foundation","relationship":"patient advocacy organization"},{"name":"Erasmus Medical Center","relationship":"clinical research collaborator"}],"dollarAmounts":[]},"materialImpact":{"score":5,"reasoning":"FDA approval of the company's lead asset EMCITATE (tiratricol), the first-ever approved treatment for MCT8 deficiency in the U.S. This is a binary regulatory event that transitions Egetis into a U.S. commercial-stage company, plus a Rare Pediatric Disease PRV that could be monetized in Q4 2026."},"tickerRelevance":{"others":[],"primary":"EGTX"},"globalImportance":55,"audienceRelevance":30,"eventTypeSecondary":["product_launch","partnership"],"importanceComponents":{"tickerTier":"small-cap European-listed (Nasdaq Stockholm)","eventGravity":"FDA approval of lead asset","sectorWeight":"biotech binary event","householdBrandBoost":0,"retailFavoriteBoost":0}},"event_type":"fda_approval","event_type_secondary":["product_launch","partnership"],"sentiment":"bullish","material_impact_score":5,"narrative":"Egetis Therapeutics received FDA approval of EMCITATE (tiratricol), the first and only approved treatment for peripheral thyrotoxicosis in adults and pediatric patients with MCT8 deficiency in the United States.\n\nThe company expects U.S. commercial availability in eight to ten weeks, supported by the Egetis RareLink patient support program and a partnership with PANTHERx Rare for specialty distribution and access services.\n\nThe FDA also granted a Rare Pediatric Disease Priority Review Voucher, which Egetis may look to monetize as early as Q4 2026, providing a potential non-dilutive cash inflection alongside the U.S. launch.","key_figures":{"drugName":"EMCITATE (tiratricol)","phaseOfTrial":"ReTRIACt, Triac Trial I, Triac Trial II, EMC Cohort Study, EMC Survival Study","customDimensions":{"prv_granted":true,"median_life_expectancy_mct8":"approximately 35 years","commercial_availability_weeks":"eight to ten weeks post approval"}},"named_entities":{"people":[{"name":"Nicklas Westerholm","role":"CEO"},{"name":"Andrew J. Bauer, M.D.","role":"pediatric endocrinologist, Principal Investigator in ReTRIACt and Triac Trial II"},{"name":"Karl Hård","role":"Head of Investor Relations, Communications & Business Development"}],"products":["EMCITATE (tiratricol)","Egetis RareLink"],"companies":[{"name":"Egetis Therapeutics AB","ticker":"EGTX","relationship":"filer"},{"name":"PANTHERx Rare","relationship":"specialty pharmacy partner"},{"name":"MCT8-AHDS Foundation","relationship":"patient advocacy organization"},{"name":"Erasmus Medical Center","relationship":"clinical research collaborator"}],"dollarAmounts":[]},"model_name":"glm-5.3-flashx","prompt_hash":"sha256:727b4b9429a443af","schema_hash":"sha256:05005c02d9cffac9","created_at":"2026-09-28T21:46:05.879Z","global_importance":55,"audience_relevance":30,"importance_components":{"tickerTier":"small-cap European-listed (Nasdaq Stockholm)","eventGravity":"FDA approval of lead asset","sectorWeight":"biotech binary event","householdBrandBoost":0,"retailFavoriteBoost":0}},"durationMs":5421,"modelName":"glm-5.3-flashx"}}