PITTSBURGH, April 1, 2024 /PRNewswire/ -- PANTHERx®Rare, a leader in rare disease product patient access and support services in the United States, is pleased to announce that it was selected by Edenbridge Pharmaceuticals for the distribution of Yargesa® (miglustat) capsules, the first oral treatment option for adults with mild to moderate Type 1 Gaucher disease for whom enzyme replacement therapy is not a therapeutic option.
PRINCETON, N.J., Feb. 08, 2024 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD), today announced that WORLDSymposium„¢ has awarded Pombiliti„¢ (cipaglucosidase alfa-atga) + Opfolda„¢ (miglustat)...
Sept 28 (Reuters) - Amicus Therapeutics' (FOLD.O) treatment for a rare muscle disorder called Pompe disease was approved by the U.S. health regulator, the drugmaker said on Thursday, ending its years-long efforts to launch the therapy.
PHILADELPHIA and MARLOW, United Kingdom, Aug. 15, 2023 (GLOBE NEWSWIRE) -- Amicus Therapeutics FOLD, a patient-dedicated global biotechnology company focused on developing and commercializing novel medicines for rare diseases, today announced that the Medicines and Healthcare products Regulatory Agency (MHRA) of the United Kingdom (U.K.) has granted marketing authorizations for Pombiliti® (cipaglucosidase alfa) + Opfolda® (miglustat) for adults living with late-onset Pompe disease (acid ?-glucosidase [GAA] deficiency). The indication for Pombiliti is a long-term enzyme replacement therapy (ERT) used in combination with miglustat for adults with late-onset Pompe disease (LOPD). The indication for Opfolda is an enzyme stabilizer of cipaglucosidase alfa long-term enzyme replacement therapy for adults with LOPD.
Amicus Therapeutics` Opfolda (miglustat) Receives Approval in Europe
Amicus Therapeutics Receives Positive CHMP Opinion for Opfolda® (miglustat) for Late-Onset Pompe Disease
(RTTNews) - Amicus Therapeutics (FOLD) Friday announced that the Committee for Medicinal Products for Human Use of the European Medicines Agency adopted a positive opinion recommending marketing authorization of cipaglucosidase alfa or Pombiliti, a long-term enzyme replacement therapy used in combination with miglustat for adults with late-onset Pompe disease. The Company expects a CHMP opinion of miglustat, the enzyme stabilizer component of AT-GAA, in the second quarter of 2023.
Breckenbridge's Generic Miglustat Receives Approval in the U.S.
Orphazyme’s arimoclomol will likely be used alongside Johnson & Johnson’s Zavesca (miglustat) for most children with Niemann-Pick disease type C (NPC) following a probable FDA approval, experts said.
Edenbridge Pharms`s Generic Miglustat Receives Approval in US