SAN DIEGO, Calif. – Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced that the U.S. Food and Drug Administration (FDA) has approved DAYBUE® STIX (trofinetide) for oral solution, a dye- and preservative-free powder formulation of trofinetide for the treatment of Rett syndrome in adult and pediatric patients two years of age and...
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– Trofinetide met co-primary efficacy endpoints demonstrating statistically significant improvement over placebo in the Rett Syndrome Behaviour Questionnaire (RSBQ) (p=0.0175) and the Clinical Global Impression of Improvement (CGI-I) (p=0.0030) – Trofinetide met key secondary endpoint demonstrating statistically significant improvement over placebo in CSBS-DP-IT–Social (p=0.0064), caregiver scale of ability to communicate...
SAN DIEGO, Calif. — Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced that Health Canada has accepted its New Drug Submission (NDS) for trofinetide for the treatment of Rett syndrome, a rare neurodevelopmental disorder. Health Canada has granted Priority Review for Acadia’s submission. “Rett syndrome is a profoundly debilitating and complex...
SAN DIEGO. Calif. — Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced that the journal Developmental Medicine and Child Neurology published interim results from the caregiver-reported observational online, open-label, ongoing LOTUS study evaluating effectiveness and tolerability outcomes in patients with Rett syndrome who are prescribed DAYBUE® (trofinetide) under routine clinical care in the U.S. Findings included reported...
According to the results of a phase 1/2 study, the Bruton tyrosine kinase (BTK) inhibitor acalabrutinib was safe and efficacious, with durable remissions, in patients with previously untreated chronic lymphocytic leukemia (CLL). The findings were reported in Blood. The single-arm study, ACE-CL-001 (ClinicalTrials.gov Identifier: NCT02029443), evaluated efficacy and safety of acalabrutinib monotherapy...
Acasti Pharma Inc., a late-stage, specialty pharma company advancing three clinical stage drug candidates addressing rare and orphan diseases, recognizes and celebrates Rare Disease Day (February 28), as established by the European Organization for Rare Diseases.
South San Francisco, Calif. – At Denali Therapeutics, where I serve as chief medical officer and head of development, we are eager to develop new treatments for progressive and debilitating neuronopathic mucopolysaccharidoses diseases with our novel technology. We are not alone—several other biopharma companies also have drug candidates that have...
CDER’s Accelerating Rare disease Cures (ARC) Program harnesses CDER’s collective expertise and activities to provide strategic overview and coordination of CDER’s rare disease activities. CDER’s ARC Program is governed by leadership from across CDER’s Office of the Center Director, Office of New Drugs, and the Office of Translational Sciences. The program...
Intas and its subsidiary Accord Healthcare lead commercialisation in Europe and India. In December 2022, serplulimab received orphan drug designation from the EC for the treatment of SCLC, this was recently reviewed by the committee and renewed. ESMO has scored serplulimab 4 out of 5 on their magnitude of clinical benefit scale (MCBS) in...
Stanford, Calif. – A large study by researchers at Stanford Medicine has found that the risk of secondary blood cancers after CAR T-cell therapy — a cell-based cancer treatment that exploded on the scene in 2017 as a treatment for intractable blood cancers — is low, despite a Food and...