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Real-World LOTUS Study Shows Sustained Efficacy of DAYBUE in Rett Syndrome Patients Over 12 Months

Real World EvidenceRare Disease
  • Acadia Pharmaceuticals published interim results from the LOTUS study showing 71-90% of Rett syndrome patients experienced behavioral improvements with DAYBUE treatment over 12 months.
  • The real-world observational study of 227 patients aged 1-60 years demonstrated improvements in nonverbal communication, alertness, and social interaction consistent with previous clinical trials.
  • Gastrointestinal side effects remained manageable with diarrhea incidence varying from 23-50% in early weeks to 26-38% in later months, with most cases contained in diapers.
  • Quality of life scores improved with a median change of 4.6 points on the QI-Disability questionnaire among patients completing 12-month assessments.

Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome (LAVENDER™)

NCT04181723CompletedPhase 3
ACADIA Pharmaceuticals Inc.
Posted 11/6/2019

Oncotelic Therapeutics Reports Progress Across Six Late-Stage Drug Programs in Oncology and Rare Diseases

Rare DiseaseOncology
  • Oncotelic Therapeutics announced significant clinical and regulatory progress across its late-stage pipeline over the past two years, with multiple drug candidates achieving important milestones.
  • The company's lead program OT-101, a TGF-β inhibitor, has advanced to Phase 3 trials for pancreatic cancer and shows additional applications in ARDS and COVID-19 treatment.
  • The pipeline includes six advanced programs targeting multi-billion-dollar markets, including OXi4503 in Phase 2 for AML/MDS and AL-101 in Phase 2 for Parkinson's disease.
  • The company is leveraging the 505(b)(2) regulatory pathway for its nanomedicine pipeline, offering a faster and more cost-efficient route to market approval compared to traditional NDAs.

Amneal Receives FDA Approval for Generic Sodium Oxybate, Breaking Jazz Pharmaceuticals' Monopoly in Narcolepsy Treatment

Drug ApprovalRare Disease
  • Amneal Pharmaceuticals received FDA approval for its generic sodium oxybate oral solution 500 mg/mL, referencing Jazz Pharmaceuticals' Xyrem for narcolepsy treatment.
  • The approval provides a more affordable alternative for approximately 150,000 narcolepsy patients in the United States who previously had access to only a single manufacturer.
  • Sodium oxybate is considered standard of care therapy for narcolepsy due to its ability to consolidate nighttime sleep and significantly reduce cataplexy episodes.
  • The drug is indicated for treating cataplexy or excessive daytime sleepiness in patients 7 years of age and older with narcolepsy.

Maze Therapeutics' MZE782 Shows 42-Fold Increase in Phenylalanine Excretion in Phase 1 Trial for PKU and CKD

Rare Disease
  • Maze Therapeutics reported positive Phase 1 results for MZE782, an oral SLC6A19 inhibitor, showing up to 42-fold increase in urinary phenylalanine excretion in healthy volunteers.
  • The drug demonstrated dose-dependent eGFR changes similar to SGLT2 inhibitors, suggesting potential kidney protective effects for chronic kidney disease patients.
  • MZE782 showed excellent safety profile with no serious adverse events across all dose levels, supporting advancement to Phase 2 trials in both PKU and CKD in 2026.
  • The compound targets SLC6A19 transporter and represents potential best-in-class therapy for phenylketonuria and first-in-class treatment for chronic kidney disease.

ZETAMET™ BONE GRAFT IN THE REPAIR OF BONE DEFECTS FROM METASTATIC BREAST CANCER IN VERTEBRAL BONES

NCT05280067Active, Not RecruitingPhase 2
Zetagen Therapeutics, Inc
Posted 9/1/2022

Intercept Pharmaceuticals Voluntarily Withdraws OCALIVA from US Market Following FDA Request

Rare Disease
  • Intercept Pharmaceuticals announced the voluntary withdrawal of OCALIVA (obeticholic acid) from the US market for primary biliary cholangitis treatment following an FDA request.
  • The FDA has placed a clinical hold on all US clinical trials involving obeticholic acid, citing ongoing safety concerns about liver injury in patients.
  • OCALIVA received accelerated approval in 2016 but was denied full approval in November 2024 due to insufficient safety and effectiveness data.
  • The drug's conditional authorization was also revoked by European regulators in June 2024, with confirmatory trial recruitment halted due to patient enrollment difficulties.

Roivant Sciences Gains Orphan Drug Designation in Japan for Mosliciguat in Pulmonary Hypertension

Rare DiseaseMonoclonal Antibody
  • Roivant Sciences' subsidiary Pulmovant received orphan drug designation from Japan's Ministry of Health, Labour and Welfare for mosliciguat, an inhaled soluble guanylate cyclase activator.
  • The designation grants regulatory benefits and potential market exclusivity for treating pulmonary hypertension associated with interstitial lung disease, an underserved indication with limited treatment options.
  • Mosliciguat was originally licensed from Bayer after the pharmaceutical giant exited respiratory research, positioning Roivant to advance this promising therapeutic candidate.
  • The company's stock rose 9.4% following the positive regulatory news, reflecting investor optimism about the drug's commercial potential in Japan's orphan disease market.

Arrowhead Pharmaceuticals Files Patent Lawsuit Against Ionis Over Plozasiran FCS Treatment

Orphan DrugRare Disease
  • Arrowhead Pharmaceuticals filed a declaratory judgment complaint against Ionis Pharmaceuticals in Delaware federal court, challenging the validity of Ionis's U.S. Patent No. 9,593,333 and asserting non-infringement by plozasiran.
  • The legal action stems from Ionis's threats of patent infringement litigation against Arrowhead's investigational RNAi therapeutic plozasiran, which is currently under FDA review for treating familial chylomicronemia syndrome.
  • Plozasiran has received multiple FDA designations including Breakthrough Therapy, Orphan Drug, and Fast Track status for FCS treatment, and has demonstrated triglyceride reductions in completed Phase 3 trials.
  • Arrowhead CEO Christopher Anzalone criticized Ionis for prioritizing corporate interests over patient needs, emphasizing that FCS is a severe rare disease that can lead to potentially fatal pancreatitis.

Capsida Suspends Gene Therapy Trial After First Patient Dies Following Treatment for Rare STXBP1 Disorder

Rare DiseaseFDA Approval
  • Capsida Biotherapeutics has suspended its clinical trial of CAP-002 gene therapy after the first participant died shortly after receiving treatment for STXBP1-related developmental and epileptic encephalopathy.
  • The company received FDA approval to begin the trial just four months ago in May, with plans to enroll around 12 children at Weill Cornell Medicine and Children's Hospital of Philadelphia.
  • CAP-002 uses a specially engineered viral vector to deliver therapy to brain neurons and aims to compensate for deficient levels of protein encoded by the STXBP1 gene.
  • The death follows other patient fatalities in gene therapy trials over the past year, raising concerns about safety in this therapeutic area.

A Clinical Trial of CAP-002 Gene Therapy in Pediatric Patients With Syntaxin-Binding Protein 1 (STXBP1) Encephalopathy

NCT06983158SuspendedPhase 1
Capsida Biotherapeutics, Inc.
Posted 7/3/2025

FDA Accepts Sobi's NASP Biologics License Application for Uncontrolled Gout Treatment

Drug ApprovalRare Disease
  • The FDA has accepted Sobi's Biologics License Application for NASP, a novel every 4-week infusion therapy for uncontrolled gout patients, with a PDUFA date set for June 27, 2026.
  • Phase 3 DISSOLVE I and II trials demonstrated that NASP met primary endpoints, achieving serum uric acid reduction below 6mg/dL in 51% and 43% of patients at high and low doses respectively.
  • NASP combines nanoencapsulated sirolimus with pegadricase to address the significant unmet need among approximately 200,000 Americans suffering from uncontrolled gout despite conventional therapies.

FDA Grants Fast Track Designation to NS-229 JAK1 Inhibitor for Rare Autoimmune Disease EGPA

Orphan DrugRare Disease
  • NS Pharma's NS-229, a selective JAK1 inhibitor, receives FDA Fast Track designation for treating eosinophilic granulomatosis with polyangiitis (EGPA), a rare autoimmune disease affecting 5,600-14,500 Americans.
  • The designation follows NS-229's Orphan Drug status granted in April 2025, enabling expedited FDA review and more frequent regulatory collaboration for this unmet medical need.
  • A Phase 2 randomized, placebo-controlled global study is currently evaluating NS-229's efficacy and safety in EGPA patients by targeting the overactive immune response that damages healthy tissues.
  • EGPA causes inflammation in small-to-medium blood vessels, leading to organ damage in lungs, sinuses, nerves, skin, and kidneys, typically following bronchial asthma and allergic rhinitis symptoms.

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