{"success":true,"data":{"pressRelease":{"id":"159566","rtpr_id":"nPn1WQWvta-20261002","ticker":"BMRN","exchange":"NASDAQ","all_tickers":["BMRN"],"title":"BioMarin Announces Five-Year Phase 3 Data for POMBILITI® (cipaglucosidase alfa-atga) + OPFOLDA® (miglustat) at World Muscle Society Annual Congress","author":"PR Newswire","published_at":"2026-10-02T15:00:00.834Z","article_body":"BioMarin Announces Five-Year Phase 3 Data for POMBILITI® (cipaglucosidase alfa-atga) + OPFOLDA® (miglustat) at World Muscle Society Annual Congress\nPR Newswire\n\nSAN RAFAEL, Calif., Oct. 2, 2026\n\nLong-term data reinforce durable efficacy and safety of POMBILITI + OPFOLDA in\nlate-onset Pompe disease\n\nSAN RAFAEL, Calif., Oct. 2, 2026 /PRNewswire/ -- BioMarin Pharmaceutical Inc.\n(Nasdaq: BMRN) today announced new data evaluating the efficacy and safety of\nPOMBILITI(®) (cipaglucosidase alfa-atga) + OPFOLDA(®) (miglustat) in\nlate-onset Pompe disease (LOPD) were presented at the 31st Annual Congress of\nthe World Muscle Society (WMS), in Hiroshima, Japan.\n\nThese results from the PROPEL open-label extension (OLE) study of POMBILITI +\nOPFOLDA, which were shared in a late-breaking presentation, demonstrated\ndurable motor function outcomes and relative stabilization of pulmonary\nfunction over long-term follow-up. The analysis included data from 82\nindividuals who received POMBILITI + OPFOLDA continuously from baseline\nobserved in the initial PROPEL study (PROPEL baseline) to year five. For the\nenzyme replacement therapy (ERT)-experienced cohort, the mean duration of\nprior ERT at baseline was 7.6 years. These data represent long-term benefits\nfor people with LOPD followed in the study, including both ERT-experienced and\nERT-naïve cohorts.\n\n\"These five-year results demonstrate that POMBILITI and OPFOLDA can provide\ndurable, long-term benefits for people living with late-onset Pompe disease,\nincluding those who had been on enzyme replacement therapy for years before\nenrolling in the study,\" said Greg Friberg, M.D., Executive Vice President and\nChief Research & Development Officer at BioMarin. \"Sustaining motor\nfunction and limiting pulmonary decline for up to five years in a progressive\ndisease like LOPD is clinically meaningful, and we remain committed to\ncontinuing to share long-term evidence to inform the care of people with this\nrare genetic condition.\"\n\nTable 1. Efficacy Measures With POMBILITI + OPFOLDA From PROPEL Baseline (BL)\nfor up to Five Years\n                                  ERT-Experienced  ERT-Naïve\n\nto Year 5\nto Year 4.5\n                                  (n=62 at BL)\n(n=20 at BL)\n\n Mean (standard deviation,        0.7% (7.76)      10.5% (11.44)\n SD) change from baseline\n in % predicted six-minute\n walk distance (pp6MWD)\n\n Mean (SD) change from            -2.8% (8.55)     -4.4% (6.89)\n baseline in % predicted\n forced vital capacity\n (ppFVC)\n\nNo new safety signals were identified. 51.2% of participants (n=42)\nexperienced treatment-related adverse events, the majority of which were\nmanageable. Five individuals discontinued treatment for safety reasons,\nincluding two due to serious treatment-related adverse events (chills and\nhypoxia [n=1], anaphylactoid reaction [n=1]).\n\nPhase 1/2 Data Show Positive Dystrophin Expression and Favorable Tolerability\nWith Nivudirsen (BMN 351)\n\nAdditional presentations at WMS included data evaluating nivudirsen (BMN 351),\nan investigational antisense oligonucleotide for ambulatory boys ages 4 to 10\nwith Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. Included\nin these presentations was an update from the ongoing Phase 1/2 351-201 study,\nwhich was previously shared at the 19th International Congress on\nNeuromuscular Diseases in July. Results from 351-201 showed treatment with\nnivudirsen was generally well tolerated and led to consistently positive\noutcomes across multiple measures of therapeutic benefit, including expression\nof near full-length dystrophin.\n\nBelow are key presentations at WMS, with all times listed in Japan Standard\nTime:\n\nPhase 1/2 Results of BMN 351, an Antisense Oligonucleotide for Exon 51\nSkip-Amenable Duchenne Muscular Dystrophy\nPoster Presentation #2.66P\nWednesday, Sept. 30, 5:15 – 6:15 p.m.\n\nComprehensive Panel of Pharmacokinetic, Pharmacodynamic and Biomarker Measures\nDrives Clinical Development of BMN 351 for Duchenne Muscular Dystrophy\nE-Poster Presentation #2.43eP\nWednesday, Sept. 30, 5:15 – 6:15 p.m.\n\nMuscle Composition and Muscle Injury Biomarkers Improve With BMN 351 Treatment\nE-Poster Presentation #2.59eP\nWednesday, Sept. 30, 5:15 – 6:15 p.m.\n\nLong-Term (Five-Year) Efficacy and Safety of Cipaglucosidase Alfa Plus\nMiglustat in Adults With Late-Onset Pompe Disease Treated From PROPEL Baseline\nE-Poster Presentation #3.081LBeP\nFriday, Oct. 2, 2:15 – 3:15 p.m.\n\nLong-Term (Four-Year) Efficacy and Safety of Cipaglucosidase Alfa Plus\nMiglustat in Adults With Late-Onset Pompe Disease Treated From PROPEL Baseline\nPoster Presentation #3.56P\nFriday, Oct. 2, 2:15 – 3:15 p.m.\n\nBaseline Disease Burden and Real-World Monitoring Practices: Global Insights\nFrom the followME Pompe Journey Registry\nPoster Presentation #3.57P\nFriday, Oct. 2, 2:15 – 3:15 p.m.\n\nATB200-03/07 Danish Subpopulation: Four-Year Efficacy and Safety of\nCipaglucosidase Alfa Plus Miglustat in Adults With Late-Onset Pompe Disease\nE-Poster Presentation #3.35eP\nFriday, Oct. 2, 2:15 – 3:15 p.m.\n\nAbout the PROPEL Open-Label Extension Study\n\nThe Phase 3 open-label extension (OLE) study (ATB200-07; NCT04138277\n(https://edge.prnewswire.com/c/link/?t=0&l=en&o=4788041-1&h=414769103&u=https%3A%2F%2Fclinicaltrials.gov%2Fstudy%2FNCT04138277&a=NCT04138277)\n) is a multicenter, international study designed to evaluate the long-term\nsafety and efficacy of POMBILITI + OPFOLDA in adults with late-onset Pompe\ndisease (LOPD) who completed the Phase 3 PROPEL study (ATB200-03; NCT03729362\n(https://edge.prnewswire.com/c/link/?t=0&l=en&o=4788041-1&h=56953119&u=https%3A%2F%2Fclinicaltrials.gov%2Fstudy%2FNCT03729362&a=NCT03729362)\n). Of the 118 people treated in the OLE, 82 continued POMBILITI + OPFOLDA\ntreatment from PROPEL (62 ERT-experienced prior to PROPEL; 20 ERT-naïve) and\n37 switched from the comparator arm to POMBILITI + OPFOLDA (29\nERT-experienced; eight ERT-naïve).\n\nThe study evaluates long-term changes from PROPEL baseline in six-minute walk\ndistance (6MWD, the primary endpoint in PROPEL), forced vital capacity (FVC),\ncreatine kinase (CK) and hexose tetrasaccharide (Hex4) levels, and\npatient-reported outcomes, as well as long-term safety.\n\nAbout Study 351-201\n\nThe 351-201 trial (NCT06280209\n(https://edge.prnewswire.com/c/link/?t=0&l=en&o=4788041-1&h=906908441&u=https%3A%2F%2Fclinicaltrials.gov%2Fstudy%2FNCT06280209&a=NCT06280209)\n) is a Phase 1/2 multicenter open-label dose-escalation study evaluating BMN\n351 for the treatment of boys aged 4 to 10 with Duchenne muscular dystrophy\nwith a specific genetic change amenable to exon 51 skipping. The primary\nendpoints examine the safety and tolerability of BMN 351, the secondary\nendpoints assess plasma, urine and muscle pharmacokinetics, and key\nexploratory endpoints include exon-skipping efficiency, dystrophin expression,\nimmunogenicity and functional assessments.\n\nThe study is being conducted in three cohorts, with Cohort 1 being divided\ninto Cohort 1A and Cohort 1B. In Cohort 1A, three boys received increasing\ndoses once every two weeks with a visit to assess safety measures collected\nthe week after dosing prior to escalating doses of BMN 351. In part two, the\nparticipants in Cohort 1A transitioned to once-weekly dosing. The participants\nin Cohort 1B, 2 and 3 will receive low (6 mg/kg), medium (9 mg/kg) and high\n(12 mg/kg) doses of BMN 351 and continue once-weekly dosing at that same dose.\n\nFor more information, please visit clinicaltrials.biomarin.com\n(https://edge.prnewswire.com/c/link/?t=0&l=en&o=4788041-1&h=473382127&u=http%3A%2F%2Fclinicaltrials.biomarin.com%2F&a=clinicaltrials.biomarin.com)\n.\n\nAbout Late-Onset Pompe Disease\n\nLate-onset Pompe disease (LOPD) is an inherited lysosomal disorder caused by\ndeficiency of the enzyme acid alpha-glucosidase (GAA). Reduced or absent\nlevels of GAA lead to accumulation of glycogen in cells, which is believed to\nresult in the clinical manifestations of Pompe disease. LOPD can be severe and\ndebilitating with progressive muscle weakness throughout the body that worsens\nover time, particularly in skeletal muscles and muscles that control\nbreathing.\n\nAbout Duchenne Muscular Dystrophy\n\nDuchenne muscular dystrophy (DMD) is a severe neuromuscular condition that\nleads to progressive muscle weakness and premature mortality. DMD is caused by\ngenetic changes that result in the absence or dysfunction of dystrophin, an\nessential protein that is necessary for preserving muscle function.\n\nAs an X-linked disorder, DMD primarily affects young boys, with onset of\nsymptoms occurring around 2 to 3 years of age. The incidence of DMD is up to 1\nin 3,600 to 6,000 live male births.\n\nIndividuals with DMD experience serious functional impairments, including loss\nof limb strength and ambulation, and respiratory and cardiovascular impacts\nthat require lifelong care.\n\nAbout POMBILITI + OPFOLDA\n\nPOMBILITI + OPFOLDA is a two-component therapy that consists of\ncipaglucosidase alfa-atga, a bis-M6P-enriched rhGAA that facilitates\nhigh-affinity uptake through the M6P receptor while retaining its capacity for\nprocessing into the most active form of the enzyme, and the oral enzyme\nstabilizer, miglustat, that is designed to reduce loss of enzyme activity in\nthe blood.\n\nPatient Support Accessing POMBILITI + OPFOLDA\n\nTo reach a BioMarin RareConnections(®) Case Manager, please call, toll-free,\n1-866-906-6100 or email support@biomarin-rareconnections.com\n(mailto:support@biomarin-rareconnections.com) . For more information about\nPOMBILITI + OPFOLDA, please visit www.pombilitiopfolda.com\n(https://edge.prnewswire.com/c/link/?t=0&l=en&o=4788041-1&h=3903822341&u=http%3A%2F%2Fwww.pombilitiopfolda.com%2F&a=www.pombilitiopfolda.com)\n. For additional information regarding these products, please contact BioMarin\nMedical Information at medinfo@bmrn.com (mailto:medinfo@bmrn.com) .\n\nPOMBILITI + OPFOLDA U.S. Indication and Important Safety Information\n\nPOMBILITI in combination with OPFOLDA is indicated for the treatment of adult\npatients with late-onset Pompe disease (lysosomal acid alpha-glucosidase\n[GAA] deficiency) weighing ≥40 kg and who are not improving on their\ncurrent enzyme replacement therapy (ERT).\n\nSAFETY INFORMATION\n\nHYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS: Appropriate medical support\nmeasures, including cardiopulmonary resuscitation equipment, should be readily\navailable. If a severe hypersensitivity reaction occurs, POMBILITI should be\ndiscontinued immediately and appropriate medical treatment should be\ninitiated. INFUSION-ASSOCIATED REACTIONS (IARs): If severe IARs occur,\nimmediately discontinue POMBILITI and initiate appropriate medical treatment.\nRISK OF ACUTE CARDIORESPIRATORY FAILURE IN SUSCEPTIBLE PATIENTS: Patients\nsusceptible to fluid volume overload, or those with acute underlying\nrespiratory illness or compromised cardiac or respiratory function, may be at\nrisk of serious exacerbation of their cardiac or respiratory status during\nPOMBILITI infusion. See the full U.S. Prescribing Information for complete\nBoxed Warning. CONTRAINDICATION: POMBILITI in combination with OPFOLDA is\ncontraindicated in pregnancy. EMBRYO-FETAL TOXICITY: May cause embryo-fetal\nharm. Advise females of reproductive potential of the potential risk to a\nfetus and to use effective contraception during treatment and for at least 60\ndays after the last dose. Adverse Reactions: Most common adverse reactions ≥\n5% are headache, diarrhea, fatigue, nausea, abdominal pain, and pyrexia.\nPlease see U.S. full PRESCRIBING INFORMATION\n(https://edge.prnewswire.com/c/link/?t=0&l=en&o=4788041-1&h=3143792050&u=https%3A%2F%2Famicusrx.com%2Fpi%2Fpombiliti.pdf&a=full+PRESCRIBING+INFORMATION)\n, including BOXED WARNING, for POMBILITI (cipaglucosidase alfa-atga) and full\nPRESCRIBING INFORMATION\n(https://edge.prnewswire.com/c/link/?t=0&l=en&o=4788041-1&h=820450531&u=https%3A%2F%2Famicusrx.com%2Fpi%2Fopfolda.pdf&a=full+PRESCRIBING+INFORMATION)\nfor OPFOLDA (miglustat).\n\nAbout BioMarin\n\nBioMarin is a leading, global rare disease biotechnology company focused on\ndelivering medicines for people living with genetically defined conditions.\nFounded in 1997, the San Rafael, California-based company has a proven track\nrecord of innovation, with nine commercial therapies and a strong clinical and\npreclinical pipeline. Using a distinctive approach to drug discovery and\ndevelopment, BioMarin seeks to unleash the full potential of genetic science\nby pursuing category-defining medicines that have a profound impact on\npatients. To learn more, please visit www.biomarin.com\n(https://edge.prnewswire.com/c/link/?t=0&l=en&o=4788041-1&h=1530424311&u=https%3A%2F%2Fwww.biomarin.com%2F&a=www.biomarin.com)\n.\n\nForward-Looking Statements\n\nThis press release contains forward-looking statements about the business\nprospects of BioMarin Pharmaceutical Inc. (BioMarin), including without\nlimitation, statements about: the data to be presented at the 31st Annual\nCongress of the World Muscle Society (WMS), including the potential benefits\nand safety of POMBILITI + OPFOLDA in late-onset Pompe disease as well as of\nnivudirsen (BMN 351) for ambulatory boys ages 4 to 10 with Duchenne muscular\ndystrophy (DMD) amenable to exon 51 skipping; BioMarin's plans and\nexpectations for the development of POMBILITI + OPFOLDA and nivudirsen; and\nBioMarin's estimate regarding the incidence of DMD. These forward-looking\nstatements are predictions and involve risks and uncertainties such that\nactual results may differ materially from these statements. These risks and\nuncertainties include, among others, results and timing of current and planned\npreclinical studies and clinical trials and the release of data from those\ntrials; any potential adverse events observed in the continuing monitoring of\nthe patients in the clinical trials; the content and timing of decisions by\nthe U.S. Food and Drug Administration, the European Medicines Agency, the\nEuropean Commission and other regulatory authorities; and those factors\ndetailed in BioMarin's filings with the Securities and Exchange Commission,\nincluding, without limitation, the factors contained under the caption \"Risk\nFactors\" in BioMarin's Quarterly Report on Form 10-Q for the quarter ended\nJune 30, 2026, as such factors may be updated by any subsequent reports.\nInvestors are urged not to place undue reliance on forward-looking statements,\nwhich speak only as of the date hereof. BioMarin is under no obligation, and\nexpressly disclaims any obligation to update or alter any forward-looking\nstatement, whether as a result of new information, future events or otherwise.\n\nBioMarin(®), BioMarin RareConnections(®), POMBILITI(®) and\nOPFOLDA(®) are registered trademarks of BioMarin Pharmaceutical Inc. or its\naffiliates.\n Contacts:\n\n Investors                             Media\n Traci McCarty                         Katherine Powell\n BioMarin Pharmaceutical Inc.          BioMarin Pharmaceutical Inc.\n (415) 455-7558                        (415) 827-2968\n\nView original content to download\nmultimedia:https://www.prnewswire.com/news-releases/biomarin-announces-five-year-phase-3-data-for-pombiliti-cipaglucosidase-alfa-atga--opfolda-miglustat-at-world-muscle-society-annual-congress-302896737.html\n(https://www.prnewswire.com/news-releases/biomarin-announces-five-year-phase-3-data-for-pombiliti-cipaglucosidase-alfa-atga--opfolda-miglustat-at-world-muscle-society-annual-congress-302896737.html)\n\nSOURCE BioMarin Pharmaceutical Inc.\n\n\n\nPhoto: \nhttps://mmx.prnewswire.com/media/MS776208/BioMarin-Pharmaceutical-Inc-Logo.jpg?id=OA2983365\n\nCopyright (c) 2026 PR Newswire Association,LLC. All Rights Reserved.","article_body_html":"","raw_payload":{"data":{"id":"nPn1WQWvta-20261002","title":"BioMarin Announces Five-Year Phase 3 Data for POMBILITI® (cipaglucosidase alfa-atga) + OPFOLDA® (miglustat) at World Muscle Society Annual Congress","author":"PR Newswire","ticker":"BMRN","created":"2026-10-02T15:00:00.834Z","tickers":["BMRN"],"exchange":"NASDAQ","article_body":"BioMarin Announces Five-Year Phase 3 Data for POMBILITI® (cipaglucosidase alfa-atga) + OPFOLDA® (miglustat) at World Muscle Society Annual Congress\nPR Newswire\n\nSAN RAFAEL, Calif., Oct. 2, 2026\n\nLong-term data reinforce durable efficacy and safety of POMBILITI + OPFOLDA in\nlate-onset Pompe disease\n\nSAN RAFAEL, Calif., Oct. 2, 2026 /PRNewswire/ -- BioMarin Pharmaceutical Inc.\n(Nasdaq: BMRN) today announced new data evaluating the efficacy and safety of\nPOMBILITI(®) (cipaglucosidase alfa-atga) + OPFOLDA(®) (miglustat) in\nlate-onset Pompe disease (LOPD) were presented at the 31st Annual Congress of\nthe World Muscle Society (WMS), in Hiroshima, Japan.\n\nThese results from the PROPEL open-label extension (OLE) study of POMBILITI +\nOPFOLDA, which were shared in a late-breaking presentation, demonstrated\ndurable motor function outcomes and relative stabilization of pulmonary\nfunction over long-term follow-up. The analysis included data from 82\nindividuals who received POMBILITI + OPFOLDA continuously from baseline\nobserved in the initial PROPEL study (PROPEL baseline) to year five. For the\nenzyme replacement therapy (ERT)-experienced cohort, the mean duration of\nprior ERT at baseline was 7.6 years. These data represent long-term benefits\nfor people with LOPD followed in the study, including both ERT-experienced and\nERT-naïve cohorts.\n\n\"These five-year results demonstrate that POMBILITI and OPFOLDA can provide\ndurable, long-term benefits for people living with late-onset Pompe disease,\nincluding those who had been on enzyme replacement therapy for years before\nenrolling in the study,\" said Greg Friberg, M.D., Executive Vice President and\nChief Research & Development Officer at BioMarin. \"Sustaining motor\nfunction and limiting pulmonary decline for up to five years in a progressive\ndisease like LOPD is clinically meaningful, and we remain committed to\ncontinuing to share long-term evidence to inform the care of people with this\nrare genetic condition.\"\n\nTable 1. Efficacy Measures With POMBILITI + OPFOLDA From PROPEL Baseline (BL)\nfor up to Five Years\n                                  ERT-Experienced  ERT-Naïve\n\nto Year 5\nto Year 4.5\n                                  (n=62 at BL)\n(n=20 at BL)\n\n Mean (standard deviation,        0.7% (7.76)      10.5% (11.44)\n SD) change from baseline\n in % predicted six-minute\n walk distance (pp6MWD)\n\n Mean (SD) change from            -2.8% (8.55)     -4.4% (6.89)\n baseline in % predicted\n forced vital capacity\n (ppFVC)\n\nNo new safety signals were identified. 51.2% of participants (n=42)\nexperienced treatment-related adverse events, the majority of which were\nmanageable. Five individuals discontinued treatment for safety reasons,\nincluding two due to serious treatment-related adverse events (chills and\nhypoxia [n=1], anaphylactoid reaction [n=1]).\n\nPhase 1/2 Data Show Positive Dystrophin Expression and Favorable Tolerability\nWith Nivudirsen (BMN 351)\n\nAdditional presentations at WMS included data evaluating nivudirsen (BMN 351),\nan investigational antisense oligonucleotide for ambulatory boys ages 4 to 10\nwith Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. Included\nin these presentations was an update from the ongoing Phase 1/2 351-201 study,\nwhich was previously shared at the 19th International Congress on\nNeuromuscular Diseases in July. Results from 351-201 showed treatment with\nnivudirsen was generally well tolerated and led to consistently positive\noutcomes across multiple measures of therapeutic benefit, including expression\nof near full-length dystrophin.\n\nBelow are key presentations at WMS, with all times listed in Japan Standard\nTime:\n\nPhase 1/2 Results of BMN 351, an Antisense Oligonucleotide for Exon 51\nSkip-Amenable Duchenne Muscular Dystrophy\nPoster Presentation #2.66P\nWednesday, Sept. 30, 5:15 – 6:15 p.m.\n\nComprehensive Panel of Pharmacokinetic, Pharmacodynamic and Biomarker Measures\nDrives Clinical Development of BMN 351 for Duchenne Muscular Dystrophy\nE-Poster Presentation #2.43eP\nWednesday, Sept. 30, 5:15 – 6:15 p.m.\n\nMuscle Composition and Muscle Injury Biomarkers Improve With BMN 351 Treatment\nE-Poster Presentation #2.59eP\nWednesday, Sept. 30, 5:15 – 6:15 p.m.\n\nLong-Term (Five-Year) Efficacy and Safety of Cipaglucosidase Alfa Plus\nMiglustat in Adults With Late-Onset Pompe Disease Treated From PROPEL Baseline\nE-Poster Presentation #3.081LBeP\nFriday, Oct. 2, 2:15 – 3:15 p.m.\n\nLong-Term (Four-Year) Efficacy and Safety of Cipaglucosidase Alfa Plus\nMiglustat in Adults With Late-Onset Pompe Disease Treated From PROPEL Baseline\nPoster Presentation #3.56P\nFriday, Oct. 2, 2:15 – 3:15 p.m.\n\nBaseline Disease Burden and Real-World Monitoring Practices: Global Insights\nFrom the followME Pompe Journey Registry\nPoster Presentation #3.57P\nFriday, Oct. 2, 2:15 – 3:15 p.m.\n\nATB200-03/07 Danish Subpopulation: Four-Year Efficacy and Safety of\nCipaglucosidase Alfa Plus Miglustat in Adults With Late-Onset Pompe Disease\nE-Poster Presentation #3.35eP\nFriday, Oct. 2, 2:15 – 3:15 p.m.\n\nAbout the PROPEL Open-Label Extension Study\n\nThe Phase 3 open-label extension (OLE) study (ATB200-07; NCT04138277\n(https://edge.prnewswire.com/c/link/?t=0&l=en&o=4788041-1&h=414769103&u=https%3A%2F%2Fclinicaltrials.gov%2Fstudy%2FNCT04138277&a=NCT04138277)\n) is a multicenter, international study designed to evaluate the long-term\nsafety and efficacy of POMBILITI + OPFOLDA in adults with late-onset Pompe\ndisease (LOPD) who completed the Phase 3 PROPEL study (ATB200-03; NCT03729362\n(https://edge.prnewswire.com/c/link/?t=0&l=en&o=4788041-1&h=56953119&u=https%3A%2F%2Fclinicaltrials.gov%2Fstudy%2FNCT03729362&a=NCT03729362)\n). Of the 118 people treated in the OLE, 82 continued POMBILITI + OPFOLDA\ntreatment from PROPEL (62 ERT-experienced prior to PROPEL; 20 ERT-naïve) and\n37 switched from the comparator arm to POMBILITI + OPFOLDA (29\nERT-experienced; eight ERT-naïve).\n\nThe study evaluates long-term changes from PROPEL baseline in six-minute walk\ndistance (6MWD, the primary endpoint in PROPEL), forced vital capacity (FVC),\ncreatine kinase (CK) and hexose tetrasaccharide (Hex4) levels, and\npatient-reported outcomes, as well as long-term safety.\n\nAbout Study 351-201\n\nThe 351-201 trial (NCT06280209\n(https://edge.prnewswire.com/c/link/?t=0&l=en&o=4788041-1&h=906908441&u=https%3A%2F%2Fclinicaltrials.gov%2Fstudy%2FNCT06280209&a=NCT06280209)\n) is a Phase 1/2 multicenter open-label dose-escalation study evaluating BMN\n351 for the treatment of boys aged 4 to 10 with Duchenne muscular dystrophy\nwith a specific genetic change amenable to exon 51 skipping. The primary\nendpoints examine the safety and tolerability of BMN 351, the secondary\nendpoints assess plasma, urine and muscle pharmacokinetics, and key\nexploratory endpoints include exon-skipping efficiency, dystrophin expression,\nimmunogenicity and functional assessments.\n\nThe study is being conducted in three cohorts, with Cohort 1 being divided\ninto Cohort 1A and Cohort 1B. In Cohort 1A, three boys received increasing\ndoses once every two weeks with a visit to assess safety measures collected\nthe week after dosing prior to escalating doses of BMN 351. In part two, the\nparticipants in Cohort 1A transitioned to once-weekly dosing. The participants\nin Cohort 1B, 2 and 3 will receive low (6 mg/kg), medium (9 mg/kg) and high\n(12 mg/kg) doses of BMN 351 and continue once-weekly dosing at that same dose.\n\nFor more information, please visit clinicaltrials.biomarin.com\n(https://edge.prnewswire.com/c/link/?t=0&l=en&o=4788041-1&h=473382127&u=http%3A%2F%2Fclinicaltrials.biomarin.com%2F&a=clinicaltrials.biomarin.com)\n.\n\nAbout Late-Onset Pompe Disease\n\nLate-onset Pompe disease (LOPD) is an inherited lysosomal disorder caused by\ndeficiency of the enzyme acid alpha-glucosidase (GAA). Reduced or absent\nlevels of GAA lead to accumulation of glycogen in cells, which is believed to\nresult in the clinical manifestations of Pompe disease. LOPD can be severe and\ndebilitating with progressive muscle weakness throughout the body that worsens\nover time, particularly in skeletal muscles and muscles that control\nbreathing.\n\nAbout Duchenne Muscular Dystrophy\n\nDuchenne muscular dystrophy (DMD) is a severe neuromuscular condition that\nleads to progressive muscle weakness and premature mortality. DMD is caused by\ngenetic changes that result in the absence or dysfunction of dystrophin, an\nessential protein that is necessary for preserving muscle function.\n\nAs an X-linked disorder, DMD primarily affects young boys, with onset of\nsymptoms occurring around 2 to 3 years of age. The incidence of DMD is up to 1\nin 3,600 to 6,000 live male births.\n\nIndividuals with DMD experience serious functional impairments, including loss\nof limb strength and ambulation, and respiratory and cardiovascular impacts\nthat require lifelong care.\n\nAbout POMBILITI + OPFOLDA\n\nPOMBILITI + OPFOLDA is a two-component therapy that consists of\ncipaglucosidase alfa-atga, a bis-M6P-enriched rhGAA that facilitates\nhigh-affinity uptake through the M6P receptor while retaining its capacity for\nprocessing into the most active form of the enzyme, and the oral enzyme\nstabilizer, miglustat, that is designed to reduce loss of enzyme activity in\nthe blood.\n\nPatient Support Accessing POMBILITI + OPFOLDA\n\nTo reach a BioMarin RareConnections(®) Case Manager, please call, toll-free,\n1-866-906-6100 or email support@biomarin-rareconnections.com\n(mailto:support@biomarin-rareconnections.com) . For more information about\nPOMBILITI + OPFOLDA, please visit www.pombilitiopfolda.com\n(https://edge.prnewswire.com/c/link/?t=0&l=en&o=4788041-1&h=3903822341&u=http%3A%2F%2Fwww.pombilitiopfolda.com%2F&a=www.pombilitiopfolda.com)\n. For additional information regarding these products, please contact BioMarin\nMedical Information at medinfo@bmrn.com (mailto:medinfo@bmrn.com) .\n\nPOMBILITI + OPFOLDA U.S. Indication and Important Safety Information\n\nPOMBILITI in combination with OPFOLDA is indicated for the treatment of adult\npatients with late-onset Pompe disease (lysosomal acid alpha-glucosidase\n[GAA] deficiency) weighing ≥40 kg and who are not improving on their\ncurrent enzyme replacement therapy (ERT).\n\nSAFETY INFORMATION\n\nHYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS: Appropriate medical support\nmeasures, including cardiopulmonary resuscitation equipment, should be readily\navailable. If a severe hypersensitivity reaction occurs, POMBILITI should be\ndiscontinued immediately and appropriate medical treatment should be\ninitiated. INFUSION-ASSOCIATED REACTIONS (IARs): If severe IARs occur,\nimmediately discontinue POMBILITI and initiate appropriate medical treatment.\nRISK OF ACUTE CARDIORESPIRATORY FAILURE IN SUSCEPTIBLE PATIENTS: Patients\nsusceptible to fluid volume overload, or those with acute underlying\nrespiratory illness or compromised cardiac or respiratory function, may be at\nrisk of serious exacerbation of their cardiac or respiratory status during\nPOMBILITI infusion. See the full U.S. Prescribing Information for complete\nBoxed Warning. CONTRAINDICATION: POMBILITI in combination with OPFOLDA is\ncontraindicated in pregnancy. EMBRYO-FETAL TOXICITY: May cause embryo-fetal\nharm. Advise females of reproductive potential of the potential risk to a\nfetus and to use effective contraception during treatment and for at least 60\ndays after the last dose. Adverse Reactions: Most common adverse reactions ≥\n5% are headache, diarrhea, fatigue, nausea, abdominal pain, and pyrexia.\nPlease see U.S. full PRESCRIBING INFORMATION\n(https://edge.prnewswire.com/c/link/?t=0&l=en&o=4788041-1&h=3143792050&u=https%3A%2F%2Famicusrx.com%2Fpi%2Fpombiliti.pdf&a=full+PRESCRIBING+INFORMATION)\n, including BOXED WARNING, for POMBILITI (cipaglucosidase alfa-atga) and full\nPRESCRIBING INFORMATION\n(https://edge.prnewswire.com/c/link/?t=0&l=en&o=4788041-1&h=820450531&u=https%3A%2F%2Famicusrx.com%2Fpi%2Fopfolda.pdf&a=full+PRESCRIBING+INFORMATION)\nfor OPFOLDA (miglustat).\n\nAbout BioMarin\n\nBioMarin is a leading, global rare disease biotechnology company focused on\ndelivering medicines for people living with genetically defined conditions.\nFounded in 1997, the San Rafael, California-based company has a proven track\nrecord of innovation, with nine commercial therapies and a strong clinical and\npreclinical pipeline. Using a distinctive approach to drug discovery and\ndevelopment, BioMarin seeks to unleash the full potential of genetic science\nby pursuing category-defining medicines that have a profound impact on\npatients. To learn more, please visit www.biomarin.com\n(https://edge.prnewswire.com/c/link/?t=0&l=en&o=4788041-1&h=1530424311&u=https%3A%2F%2Fwww.biomarin.com%2F&a=www.biomarin.com)\n.\n\nForward-Looking Statements\n\nThis press release contains forward-looking statements about the business\nprospects of BioMarin Pharmaceutical Inc. (BioMarin), including without\nlimitation, statements about: the data to be presented at the 31st Annual\nCongress of the World Muscle Society (WMS), including the potential benefits\nand safety of POMBILITI + OPFOLDA in late-onset Pompe disease as well as of\nnivudirsen (BMN 351) for ambulatory boys ages 4 to 10 with Duchenne muscular\ndystrophy (DMD) amenable to exon 51 skipping; BioMarin's plans and\nexpectations for the development of POMBILITI + OPFOLDA and nivudirsen; and\nBioMarin's estimate regarding the incidence of DMD. These forward-looking\nstatements are predictions and involve risks and uncertainties such that\nactual results may differ materially from these statements. These risks and\nuncertainties include, among others, results and timing of current and planned\npreclinical studies and clinical trials and the release of data from those\ntrials; any potential adverse events observed in the continuing monitoring of\nthe patients in the clinical trials; the content and timing of decisions by\nthe U.S. Food and Drug Administration, the European Medicines Agency, the\nEuropean Commission and other regulatory authorities; and those factors\ndetailed in BioMarin's filings with the Securities and Exchange Commission,\nincluding, without limitation, the factors contained under the caption \"Risk\nFactors\" in BioMarin's Quarterly Report on Form 10-Q for the quarter ended\nJune 30, 2026, as such factors may be updated by any subsequent reports.\nInvestors are urged not to place undue reliance on forward-looking statements,\nwhich speak only as of the date hereof. BioMarin is under no obligation, and\nexpressly disclaims any obligation to update or alter any forward-looking\nstatement, whether as a result of new information, future events or otherwise.\n\nBioMarin(®), BioMarin RareConnections(®), POMBILITI(®) and\nOPFOLDA(®) are registered trademarks of BioMarin Pharmaceutical Inc. or its\naffiliates.\n Contacts:\n\n Investors                             Media\n Traci McCarty                         Katherine Powell\n BioMarin Pharmaceutical Inc.          BioMarin Pharmaceutical Inc.\n (415) 455-7558                        (415) 827-2968\n\nView original content to download\nmultimedia:https://www.prnewswire.com/news-releases/biomarin-announces-five-year-phase-3-data-for-pombiliti-cipaglucosidase-alfa-atga--opfolda-miglustat-at-world-muscle-society-annual-congress-302896737.html\n(https://www.prnewswire.com/news-releases/biomarin-announces-five-year-phase-3-data-for-pombiliti-cipaglucosidase-alfa-atga--opfolda-miglustat-at-world-muscle-society-annual-congress-302896737.html)\n\nSOURCE BioMarin Pharmaceutical Inc.\n\n\n\nPhoto: \nhttps://mmx.prnewswire.com/media/MS776208/BioMarin-Pharmaceutical-Inc-Logo.jpg?id=OA2983365\n\nCopyright (c) 2026 PR Newswire Association,LLC. All Rights Reserved."},"type":"article","timestamp":"2026-10-02T15:00:00.896006566Z","server_sent_at_ms":1790953200896},"received_at":"2026-10-02T15:00:01.050Z","source_url":"https://www.prnewswire.com/news-releases/biomarin-announces-five-year-phase-3-data-for-pombiliti-cipaglucosidase-alfa-atga--opfolda-miglustat-at-world-muscle-society-annual-congress-302896737.html"},"analysis":{"id":"148166","press_release_id":"159566","analysis_json":{"industry":{"label":"Pharmaceuticals, Biotechnology & Life Sciences","sector":"Health Care"},"redFlags":["five safety discontinuations including two serious treatment-related adverse events (hypoxia, anaphylactoid reaction)","51.2% of participants experienced treatment-related adverse events"],"eventType":"clinical_trial","narrative":"BioMarin presented five-year Phase 3 open-label extension data for POMBILITI + OPFOLDA in late-onset Pompe disease at the World Muscle Society Congress in Hiroshima.\n\nThe 82-participant analysis showed durable motor function and relative stabilization of pulmonary function, with ERT-naive patients gaining 10.5% in percent-predicted six-minute walk distance and no new safety signals.\n\nBioMarin also shared Phase 1/2 data for nivudirsen (BMN 351) in Duchenne muscular dystrophy, showing near full-length dystrophin expression and favorable tolerability.\n\nThe data are supportive of the commercial franchise but incremental, as POMBILITI + OPFOLDA is already approved and marketed.","sentiment":"bullish","agentHooks":{"shouldPost":false,"suggestedAngle":"Five-year durability data de-risk the Pompe franchise; early BMN 351 dystrophin signal worth monitoring."},"keyFigures":{"drugName":"POMBILITI (cipaglucosidase alfa-atga) + OPFOLDA (miglustat)","phaseOfTrial":"Phase 3 open-label extension","customDimensions":{"bmn351_doses_mg_kg":[6,9,12],"ert_naive_cohort_n":20,"ppfvc_change_ert_naive":"-4.4% (SD 6.89)","pp6mwd_change_ert_naive":"10.5% (SD 11.44) at Year 4.5","safety_discontinuations":5,"ert_experienced_cohort_n":62,"ole_participants_analyzed":82,"treatment_related_ae_rate":"51.2% (n=42)","ppfvc_change_ert_experienced":"-2.8% (SD 8.55)","mean_prior_ert_duration_years":7.6,"pp6mwd_change_ert_experienced":"0.7% (SD 7.76) at Year 5"}},"quotedText":"Sustaining motor function and limiting pulmonary decline for up to five years in a progressive disease like LOPD is clinically meaningful","namedEntities":{"people":[{"name":"Greg Friberg, M.D.","role":"Executive Vice President and Chief Research & Development Officer"},{"name":"Traci McCarty","role":"Investor Relations contact"},{"name":"Katherine Powell","role":"Media contact"}],"products":["POMBILITI (cipaglucosidase alfa-atga)","OPFOLDA (miglustat)","nivudirsen (BMN 351)","BioMarin RareConnections"],"companies":[{"name":"BioMarin Pharmaceutical Inc.","ticker":"BMRN","relationship":"filer"}],"dollarAmounts":[]},"materialImpact":{"score":2,"reasoning":"Long-term OLE data for an already-approved therapy reinforcing durable efficacy/safety; supportive but not a binary regulatory event or new indication. Pipeline read-through from Phase 1/2 BMN 351 is early-stage."},"tickerRelevance":{"others":[],"primary":"BMRN"},"globalImportance":25,"audienceRelevance":30,"eventTypeSecondary":[],"importanceComponents":{"tickerTier":"mid-large-cap biotech","eventGravity":"long-term data for approved asset, conference presentation","issuerAuthored":true,"commercialImpact":"supportive, not new approval or indication"}},"event_type":"clinical_trial","event_type_secondary":null,"sentiment":"bullish","material_impact_score":2,"narrative":"BioMarin presented five-year Phase 3 open-label extension data for POMBILITI + OPFOLDA in late-onset Pompe disease at the World Muscle Society Congress in Hiroshima.\n\nThe 82-participant analysis showed durable motor function and relative stabilization of pulmonary function, with ERT-naive patients gaining 10.5% in percent-predicted six-minute walk distance and no new safety signals.\n\nBioMarin also shared Phase 1/2 data for nivudirsen (BMN 351) in Duchenne muscular dystrophy, showing near full-length dystrophin expression and favorable tolerability.\n\nThe data are supportive of the commercial franchise but incremental, as POMBILITI + OPFOLDA is already approved and marketed.","key_figures":{"drugName":"POMBILITI (cipaglucosidase alfa-atga) + OPFOLDA (miglustat)","phaseOfTrial":"Phase 3 open-label extension","customDimensions":{"bmn351_doses_mg_kg":[6,9,12],"ert_naive_cohort_n":20,"ppfvc_change_ert_naive":"-4.4% (SD 6.89)","pp6mwd_change_ert_naive":"10.5% (SD 11.44) at Year 4.5","safety_discontinuations":5,"ert_experienced_cohort_n":62,"ole_participants_analyzed":82,"treatment_related_ae_rate":"51.2% (n=42)","ppfvc_change_ert_experienced":"-2.8% (SD 8.55)","mean_prior_ert_duration_years":7.6,"pp6mwd_change_ert_experienced":"0.7% (SD 7.76) at Year 5"}},"named_entities":{"people":[{"name":"Greg Friberg, M.D.","role":"Executive Vice President and Chief Research & Development Officer"},{"name":"Traci McCarty","role":"Investor Relations contact"},{"name":"Katherine Powell","role":"Media contact"}],"products":["POMBILITI (cipaglucosidase alfa-atga)","OPFOLDA (miglustat)","nivudirsen (BMN 351)","BioMarin RareConnections"],"companies":[{"name":"BioMarin Pharmaceutical Inc.","ticker":"BMRN","relationship":"filer"}],"dollarAmounts":[]},"model_name":"glm-5.3-flashx","prompt_hash":"sha256:727b4b9429a443af","schema_hash":"sha256:05005c02d9cffac9","created_at":"2026-10-02T15:00:08.422Z","global_importance":25,"audience_relevance":30,"importance_components":{"tickerTier":"mid-large-cap biotech","eventGravity":"long-term data for approved asset, conference presentation","issuerAuthored":true,"commercialImpact":"supportive, not new approval or indication"}},"durationMs":7339,"modelName":"glm-5.3-flashx"}}