BioMarin Announces Two Oral and 19 Poster Presentations at the Society for the Study of Inborn Errors of Metabolism 2015 Annual Meeting

Loading...
Loading...
BioMarin Pharmaceutical Inc.
BMRN
announced today that the company will present data in two oral and 19 poster presentations at the Society for the Study of Inborn Errors of Metabolism 2015 Annual Meeting which will be held on September 1-4 in Lyon, France. In an oral platform session, long-term efficacy and safety data from a Phase 2 extension study of pegvaliase (PEG-PAL, rAvPAL-PEG, BMN 165) in adults with phenylketonuria (PKU) will be presented. PKU is a rare genetic condition in which the body cannot metabolize the essential amino acid phenylalanine. Designated Orphan Drug status in the United States and European Union, pegvaliase is an enzyme substitution therapy that is designed to substitute phenylalanine ammonia lyase for phenylalanine hydroxylase, the enzyme deficient in people with PKU. A separate oral platform presentation will provide an overview of the reveglucosidase alfa (BMN 701) clinical program in late onset Pompe disease. In addition, the overview will summarize the three ongoing trials; the ongoing Phase 3 INSPIRE trial, a switchover study designed to assess safety and efficacy of reveglucosidase alfa in patients previously treated with enzyme replacement therapy; an observational study in patients with late onset Pompe disease; and a transdiaphragmatic stimulation study in late onset Pompe patients treated with reveglucosidase alfa. The overview also includes a review of nonclinical activity as well as interim safety and efficacy results in treatment naïve, late-onset Pompe patients treated with reveglucosidase alfa. Pompe disease is an inherited condition caused by the deficiency in the enzyme acid alpha-glucosidase, which leads to progressive weakening of muscles in the body, including diaphragm muscles essential for breathing. Reveglucosidase alfa is a novel fusion protein of insulin-like growth factor 2 and acid alpha-glucosidase, designed to target delivery to cell structures called lysosomes where the enzyme is most needed. "We are committed to patients with rare inherited metabolic disorders. We continue to evaluate multiple therapies across disease states over the long term to both understand the impact of our medicines on patients, as well as to better understand the biology of the disease," said Hank Fuchs, M.D., Executive Vice President and Chief Medical Officer at BioMarin. "With promising results from our late stage and long term clinical studies, we are one step closer to potentially bringing new treatments to patients in need. Our more than twenty presentations across multiple conditions underscores our growing research to advance the standard of care for children and adults with rare conditions." Additionally, data will be presented from 11 abstracts on mucopolysaccharidosis (MPS), a group of metabolic disorders caused by the deficiency of specific enzymes, including five abstracts evaluating VIMIZIM® (elosulfase alfa) in people with Morquio A Syndrome (MPS IVA), a specific form of the disease. The other studies for MPS also provide understanding into the challenges associated with the disease, such as the management of fertility and pregnancy. Listing of Posters and Presentations at Society for the Study of Inborn Errors of Metabolism 2015 Annual Meeting Oral Presentations   Title Authors An international, phase 3, switchover study of reveglucosidase alfa (BMN 701) in subjects with late onset Pompe disease (INSPIRE study)* Geberhiwot T, Byrne B, Eyskens F, Hughes D, Kissel J, Mengel E, Mozaffar T, Pestronk A, Roberts M, Schoser B, Sivakumar K, Statland J, Young P, Heusner C, Walsh L     Presentation: Wed., Sept. 2, 2015     *Overview of INSPIRE study (trial-in-progress) only and does not include data       Evaluation of long-term safety and efficacy with rAvPAL-PEG (BMN 165) for control of blood phenylalanine levels in adults with phenylketonuria (PKU)* Longo N, Thomas J, Wasserstein M, Burton B, Vockley G, Grange D, Hillman R, Harding C, Dimmock D, Shur N, Adams D, Rizzo W, Whitley C, Goodin K, Decker C, Merilainen M, Li M, Bolt K, Schweighardt B, Shpilberg S, Muthalif M, Zori R     Presentation: Thurs., Sept. 3, 2015       Poster Presentations   Mucopolysaccharidosis (MPS)   Title Authors Urine keratan sulfate (uKS) in Morquio A patients measured via LC-MS/MS method: improved KS detection as compared to dye-based methods and report of age-specific uKS reference ranges Pasquali M, Auray-Blais C, Ellsworth K, Fietz M, Giugliani R, Harmatz P, Izzo E, Lavoie P, la Marca G, Millington D, Trinh M-U, van Vlies N, Varfaj F, Wijburg F, Wood T, Zhang H, Miller N Urine keratan sulfate (uKS) elevation in lysosomal storage disorders (LSDs): comparison of uKS levels in Morquio/mucopolysaccharidosis (MPS) IV versus non-Morquio LSDs Wijburg F, Auray-Blais C, Ellsworth K, Giugliani R, Harmatz P, Izzo E, Lavoie P, Millington D, Wood T, van Vlies N, Zhang H, Miller N. Impact of mucopolysaccharidosis (MPS) on daily living, employment, general health and parenthood of adult patients Lavery C, Wedehase B, Harmatz P, Hendriksz CJ Impact of elosulfase alfa on exercise capacity in patients with Morquio A syndrome in a randomised, double-blind, pilot study Burton B, Berger KI, Lewis GD, Tarnopolsky M, Mitchell JJ, Muschol N, Jones SA, Sutton VR, Pastores GM, Lau H, Sparkes R, Genter F, Shaywitz AJ, Harmatz P Long-term outcomes of treatment with elosulfase alfa for Morquio A Syndrome (mucopolysaccharidosis IVA) Hendriksz C, Jones S, Decker C, Geberhiwot T, Van Tuyl A, Schweighardt B, Slasor P Safety and pharmacodynamic activity of elosulfase alfa in pediatric patients less than 5 years of age with Morquio A Syndrome (Mucopolysaccharidosis IVA) Harmatz P, Jones SA, Bialer M, Parini R, Martin K, Wang H, Shaywitz A Impact of long-term elosulfase alfa treatment on six-minute walk test distance in patients with Morquio A syndrome Harmatz P, Burton BK, Giugliani R, Hughes D, Mitchell JJ, Raiman J, Stewart F, Solano Villarreal ML, Slasor P, Shaywitz AJ Impact of long-term elosulfase alfa treatment on three-minute stair climb test, pulmonary function tests and normalized urine keratan sulfate in patients with Morquio A syndrome Giugliani R, Burton BK, Harmatz P, Hughes D, Mitchell JJ, Raiman J, Solano Villarreal ML, Stewart F1, Slasor P, Shaywitz AJ Management of fertility and pregnancy in individuals with mucopolysaccharidosis (MPS) Stewart F, Harmatz P, Braunlin E, Bentley A, Burton B, Guffon N, Hale S, Johnston T, Kircher S, Kochhar P, Mitchell J, Plöckinger U, Semotok J, Sisic Z Medical issues and other challenges in adult patients with mucopolysaccharidosis (MPS) Mitchell J, Berger K, Quartel A, Braunlin E, Wang R, Pastores GM, White K, Jurecki E Dose- and time-dependent clearance of lysosomal storage in the MPS IIIB mouse model by intracerebroventricular enzyme replacement therapy with BMN 250, a NAGLU-IGFII fusion protein Aoyagi-Scharber M, Vincelette J, Lawrence R, Crippen-Harmon D, Yip BK, Baridon B, Prill H, Minto W, Van Vleet J, Vitelli C, Adintori EG, Strong KM, Christianson T, Tiger PMN, Lo MJ, Holtzinger J, Chen E, Fitzpatrick PA, LeBowitz   Pompe disease   Title Authors Pulmonary function predictors (VC, FVC, MIP, MEP) of ventilator use in late-onset Pompe disease Quartel A, Mozaffar T, Roberts M, Young P, Johnson EM, Berger KI   Phenylketonuria (PKU)   Title Authors Neuropsychiatric comorbidities in adults with phenylketonuria: A retrospective cohort study Bilder DA, Kobori JA, Cohen-Pfeffer JL, Johnson EM, Jurecki ER, Grant ML Evaluation of multiple dosing regimens in phase 2 studies of rAvPAL-PEG for control of blood phenylalanine levels in adults with phenylketonuria Thomas J, Longo N, Zori R, Burton B, Wasserstein M, Grange D, Vockley J, Hillman R, Harding C, Shur N, Adams D, Rice G, Rizzo W, Whitley C, Goodin K, McBride K, Decker C, Merilainen M, Li M, Schweighardt B, Dimmock D. Evaluation of an induction, titration, and maintenance dosing regimen in a phase 2 study of rAvPAL-PEG for control of blood phenylalanine levels in adults with phenylketonuria (PKU) Zori R, Thomas J, Shur N, Rizzo W, Decker C, Merilainen M, Li M, Schweighthardt B, Longo N. Phase 2 studies contribute to rAvPAL-PEG phase 3 trial design Harding C, Longo N, Thomas J, Burton B, Zori R, Bilder D, Posner J, Lieberman P, Merilainen M, Gu K, Schweighardt B, Weng H, Levy H A randomized, placebo-controlled, double-blind study of sapropterin to treat symptoms of ADHD and executive dysfunction in children and adolescents with phenylketonuria Grant M, Cohen-Pfeffer J, McCandless S, Stahl SM, Bilder D, Jurecki ER, Yu S, Sanchez-Valle A, Dimmock D   CLN2 Disease   Title Authors Neuronal Ceroid Lipofuscinosis-2 (CLN2) disorder, a type of Batten disease caused by TPP1 enzyme deficiency: Current knowledge of the natural history from international experts Schulz A, Cohen-Pfeffer JL, Crystal R, de los Reyes E, Eto Y, Guelbert N, Héron B, Mikhailova S, Miller N, Mink J, Perez-Poyato M, Simonati A, Sims K, Williams RE Real-world experience in the diagnosis of neuronal ceroid lipofuscinosis type 2 (CLN2): Report from an international collaboration of experts Miller N, Mole S, Cohen-Pfeffer JL, Crystal R, de los Reyes E, Eto Y, Fietz M, Heron B, Izzo E, Kohlschutter A, Lourenço CM, Noher de Halac I, Pearce DA, Simonati A, del Socorro Pérez Poyato M, Schulz A Important Safety Information About Vimizim Life-threatening allergic reactions, known as anaphylaxis, can occur during Vimizim® (elosulfase alfa) infusions. Due to the potential for anaphylaxis, appropriate medical support should be readily available when Vimizim is administered and for an appropriate period of time following administration. Hypersensitivity reactions have been observed as early as 30 minutes from the start of infusion but as late as six days after infusion. Frequent symptoms of hypersensitivity reactions included anaphylactic reactions, urticaria, peripheral edema, cough, dyspnea, and flushing. Because of the potential for hypersensitivity reactions, administer antihistamines with or without antipyretics prior to infusion. If severe hypersensitivity reactions occur, immediately stop the infusion of VIMIZIM and initiate appropriate treatment. Patients with acute febrile or respiratory illness at the time of VIMIZIM infusion may be at higher risk of life-threatening complications from hypersensitivity reactions. Sleep apnea is common in MPS IVA patients. Evaluation of airway patency should be considered prior to initiation of treatment with VIMIZIM. Patients using supplemental oxygen or continuous positive airway pressure (CPAP) during sleep should have these treatments readily available during infusion in the event of an acute reaction, or extreme drowsiness/sleep induced by antihistamine use. Spinal or cervical cord compression (SCC) is a known and serious complication of MPS IVA and may occur as part of the natural history of the disease. In clinical trials, SCC was observed both in patients receiving VIMIZIM and patients receiving placebo. Patients with MPS IVA should be monitored for signs and symptoms of SCC (including back pain, paralysis of limbs below the level of compression, urinary and fecal incontinence) and given appropriate clinical care. All patients treated with VIMIZIM 2 mg/kg once per week in the placebo-controlled trial developed anti-drug antibodies. VIMIZIM should be used during pregnancy only if the potential benefit justifies the potential risk to the fetus. It is not known if VIMIZIM is present in human milk. Safety and effectiveness in pediatric patients below 5 years of age have not been established. In clinical trials, the most common adverse reactions (≥10%) occurring during infusion included pyrexia, vomiting, headache, nausea, abdominal pain, chills, and fatigue. The acute reactions requiring intervention were managed by either temporarily interrupting or discontinuing infusion, and administering additional antihistamine, antipyretics, or corticosteroids. Please see full Prescribing Information, including boxed warning, or visit www.VIMIZIM.com.
Loading...
Loading...
Posted In: News
We simplify the market for smarter investing

Trade confidently with insights and alerts from analyst ratings, free reports and breaking news that affects the stocks you care about.

Join Now: Free!

Loading...