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FDA approved Cuvrior™Treatment of decoppered and penicillamine-resistant adults with Wilson’s disease
Wilson’s disease aka hepatolentiform Nuclear degeneration, a rare inherited disorder of copper transport that primarily affects the liver and brain. 2022year5month2day, < /span>Orphalan SAAnnounces U.S.FDAapprovalits Cuvrior< /span>™(trientine tetrahydrochloride), for decopper treatment and penicillamine-resistant of Wilsondiseaseadult patients. Penicillamine is currently approved in the United States as a first-line treatment for Wilson’s disease, and about one-third of patients develop intolerance. Orphalanrecently completed a globalIIIphase III trialCHELATE , the testmeasured byCuvrior™< span>After treatment with penicillamineserum non-ceruloplasmin-bound copper (NCC) content, span>ProveCuvriorTMefficacynotinferior to penicillamine. (source:Orphalan SAofficial website)< /span>
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New drug for Rett syndrome receives FDA orphan drug designation span>
On May 3, 2022, DepYmed announced that the U.S. FDA has granted orphan drug designation to its lead clinical candidate, a Small molecule PTP1B inhibitor for the treatment of patients with Rett syndrome (Rett). DepYmed is a preclinical pharmaceutical company focused on developing transformative treatments for rare diseases and tumors, based on the protein tyrosine phosphatase (PTP) family. Rett syndrome is a devastating rare genetic neurological disorder that affects almost exclusively women. There are hardly any treatment options currently available. Patients often experience autism-like behaviors, irregular breathing, difficulty swallowing, developmental delays, and seizures. Most cases of Rett syndrome are caused by mutations in the MECP2 gene on the X chromosome. (Source: DepYmed official website)
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ST101 was granted orphan drug designation for the treatment of IIB to Stage IV Melanoma
2022year< span>5month3day,focus on developing peptide therapyTherapeuticsBiotech Company for CancerSapience Therapeuticsannounced,FDA Has granted its lead programST101orphan drugdesignationfor the treatment ofIIB to IV stage melanoma. This followsFDAin 2018year4 MonthGranted Orphan Drug DesignationtreatmentAML, 2020year6monthgranted its treatmentAfter orphan drug designation for glioma, ST101the third time Orphan Drug Designation. ST101is a primary peptide antagonist ofC/EBPβ, currently in progress< span>1/2 Phase 2 evaluation of the clinical study (NCT04478279 ) for difficultPatients with advanced unresectable and metastatic solid tumors receiving standard therapy. (source:Sapience Therapeuticsofficial website)< /span>
Reprinted from: China Rare Disease Alliance