Miglustat

PRINCETON, N.J., Oct. 04, 2024 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD), today announced that three posters highlighting its development program for Pompe disease will be included at the 29th Annual Congress of the World Muscle Society being held October 8-12, 2024 in Prague, Czechia. Poster Session: Pompe Disease Abstract Title: Cipaglucosidase alfa + miglustat in late-onset Pompe disease: two non-ambulatory patients switching from high-dose, high-frequency alglucosidase alfa (Poster #657P) Presenter: Barry J. Byrne, MD, PhD, University of Florida, Gainesville, FL, U.S.A.Poster Session: Friday, October 11, 3:45 – 4:45 p.m. CETLocation: Forum Hall, The Prague Congress Centre Abstract Title: Post-baseline outcomes of the UK Early Access to Medicines Scheme registry for cipaglucosidase alfa plus miglustat in late-onset Pompe disease (Poster #670P) Presenter: Mark Roberts, MD, Salford Royal NHS Foundation Trust, Greater Manchester Neurosciences Centre, Manchester, U.K.Poster Session: Friday, October 11, 3:45 – 4:45 p.m. CETLocation: Forum Hall, The Prague Congress Centre Abstract Title: Miglustat: a first-in-class enzyme stabilizer for late-onset Pompe disease (Poster #671P) Presenter: Tahseen Mozaffar, MD, Department of Neurology, University of California, Irvine, CA, U.S.A.Poster Session: Friday, October 11, 3:45 – 4:45 p.m. CETLocation: Forum Hall, The Prague Congress Centre For more information on the WMS 2024 Annual Congress, please visit www.wms2024.com About Amicus Therapeutics Amicus Therapeutics (Nasdaq: FOLD) is a global, patient-dedicated biotechnology company focused on discovering, developing and delivering novel high-quality medicines for people living with rare diseases. With extraordinary patient focus, Amicus Therapeutics is committed to advancing and expanding a pipeline of cutting-edge, first- or best-in-class medicines for rare diseases. For more information please visit the company’s website at www.amicusrx.com, and follow on X and LinkedIn. CONTACTS: Investors: Amicus Therapeutics Andrew Faughnan Vice President, Investor Relations afaughnan@amicusrx.com(609) 662-3809 Media: Amicus Therapeutics Diana Moore Head of Global Corporate Communications dmoore@amicusrx.com(609) 662-5079 FOLD–G

IntraBio’s green light likely comes as a blow to Zevra Therapeutics, which snagged a historic approval for Miplyffa in Niemann-Pick disease type C on Friday. While it was just last week that Zevra Therapeutics’ Miplyffa became the first FDA-approved treatment for the lysosomal storage disorder Niemann-Pick disease type C (NPC), IntraBio has already one-upped its rare disease rival.Late Tuesday, the FDA approved IntraBio’s levacetylleucine under the brand name Aqneursa to treat neurological symptoms of NPC in adults and kids weighing at least 15 kg (about 33 pounds).Aqneursa has been approved as a standalone therapy and is available now, according to a release from IntraBio—which hails from the U.K. but recently shifted its headquarters to Austin, Texas, following a $40 million funding round.The green light likely comes as a blow to Zevra, which snagged a historic approval for Miplyffa in NPC on Friday. But, unlike IntraBio’s monotherapy, Miplyffa must be taken with the enzyme inhibitor miglustat. What’s more, Miplyffa isn’t expected to hit the market for another eight to 12 weeks, according to Zevra.In turn, IntraBio’s approval and launch plan have effectively erased Zevra’s head start. NPC, a rare lysosomal storage disorder, prevents the body from moving cholesterol and other lipids in cells. This leads to a buildup of cholesterol and other fats in the liver, spleen or lungs that can cause organ damage. On average, people with NPC only live for about 13 years, the FDA noted in its release.The disease can also impair patients’ speech, cognition, swallowing, movement and fine motor skills.The FDA based its approval of Aqneursa on results from IntraBio’s pivotal trial IB1001-301, which looked at the drug’s effect on neurological symptoms and functioning in adults and kids over the age of 4 with a confirmed NPC diagnosis.The trial met its primary efficacy endpoint and every secondary endpoint across all patient cohorts receiving Aqneursa, IntraBio said. The study found that Aqneursa “significantly” improved neurological signs and symptoms of the disease and led to functional benefits within 12 weeks.The main outcome the FDA looked at in the study was a modified version of the Scale for the Assessment and Rating of Ataxia (SARA)—known as the functional SARA (fSARA)—which measures gait, stability, speech and upper and lower limb coordination across eight individual domains. The fSARA, meanwhile, only assesses gait, sitting, stance and speech disturbance domains with modifications to the scoring responses.Patients on Aqneursa showed greater improvement on fSARA scores versus those on placebo, and the results on the modified assessment were buoyed by consistent outcomes demonstrated in the original test, IntraBio explained.While the most common side effects associated with Aqneursa are abdominal pain, difficult swallowing, upper respiratory tract infection and vomiting, the drug’s prescribing information does carry a warning that Aqneursa may cause embryo-fetal harm if used during pregnancy.Patients should inform their doctors of a known or suspected pregnancy before taking IntraBio’s drug, the FDA noted.Aqneursa is taken orally up to three times a day, with or without food. The dosing varies depending upon the individual patient’s body weight.Aqneursa marks IntraBio’s first commercial product and, with an approval in hand, the company isn’t content to stop at NPC.“Based on our clinical research, we believe that Aqneursa may hold potential for treating other rare and common neurodegenerative and neurodevelopmental disorders, and we will continue to rapidly develop Aqneursa for these additional indications,” Mallory Factor, CEO of IntraBio, said in a statement. In order to help patients access the treatment, IntraBio has launched a support program dubbed Aqneursa Cares. The program will include financial support to “reduce or eliminate” out-of-pocket costs for qualifying patients as well as connect them with third-party resources. The program will also feature educational resources and a team of disease specialists.