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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.

CAMP4 Therapeutics Secures $100 Million Financing to Advance First-in-Class SYNGAP1 Treatment

Rare DiseaseLeadership Change
  • CAMP4 Therapeutics announced an oversubscribed $100 million private placement led by Coastlands Capital to fund development of its first-in-class SYNGAP1 treatment program.
  • The financing provides $50 million upfront with potential for additional $50 million upon achieving regulatory milestones, including clearance for Phase 1/2 clinical trials expected to begin in 2H 2026.
  • The company appointed Doug Williams as Board Chair and elevated Daniel Tardiff to Chief Scientific Officer to strengthen leadership as it advances its regulatory RNA-targeting therapeutic platform.
  • CAMP4's approach targets regulatory RNAs to upregulate gene expression and restore healthy protein levels in genetic diseases, addressing over 1,200 haploinsufficient disorders.

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.

FDA Issues Complete Response Letter for Saol's SL1009 Treatment for Rare Pediatric Mitochondrial Disease

Orphan DrugRare Disease
  • The FDA issued a Complete Response Letter for Saol Therapeutics' SL1009 (sodium dichloroacetate) New Drug Application, requiring additional deficiencies to be addressed before approval for treating Pyruvate Dehydrogenase Complex Deficiency.
  • PDCD affects fewer than 1,000 individuals in the U.S. with an estimated incidence of 1 in 40,000 live births, and currently has no FDA-approved treatments available.
  • Saol maintains that four years of clinical data from two phase III studies support SL1009's potential as an important treatment option, and the company seeks regulatory flexibility to avoid additional lengthy trials.
  • Patient access to SL1009 continues through ongoing clinical trials and expanded access programs while the company works with the FDA on a path forward.

Relapsing Forms of Multiple Sclerosis (RMS) Study of Bruton's Tyrosine Kinase (BTK) Inhibitor Tolebrutinib (SAR442168) (GEMINI 2)

NCT04410991CompletedPhase 3
Sanofi
Posted 6/11/2020

Nonrelapsing Secondary Progressive Multiple Sclerosis (NRSPMS) Study of Bruton's Tyrosine Kinase (BTK) Inhibitor Tolebrutinib (SAR442168)

NCT04411641CompletedPhase 3
Sanofi
Posted 9/24/2020

Primary Progressive Multiple Sclerosis (PPMS) Study of Bruton's Tyrosine Kinase (BTK) Inhibitor Tolebrutinib (SAR442168) (PERSEUS)

NCT04458051Active, Not RecruitingPhase 3
Sanofi
Posted 8/13/2020

Expanded Access Treatment Protocol With DCA for Patients With PDCD

NCT06931262available
Saol Therapeutics Inc

Relapsing Forms of Multiple Sclerosis (RMS) Study of Bruton's Tyrosine Kinase (BTK) Inhibitor Tolebrutinib (SAR442168) (GEMINI 1)

NCT04410978CompletedPhase 3
Sanofi
Posted 6/30/2020

Trial of Dichloroacetate in Pyruvate Dehydrogenase Complex Deficiency:

NCT02616484Active, Not RecruitingPhase 3
Saol Therapeutics Inc
Posted 7/14/2020

Stanford University Receives Double-Digit Million NIH Grant for Phase II Trial of Tiprelestat in Pulmonary Arterial Hypertension

Rare Disease
  • Stanford University has been awarded a double-digit million dollar NIH grant to conduct a Phase II trial of Tiprelestat for pulmonary arterial hypertension, with patient enrollment expected to begin in mid-2026.
  • Tiprelestat represents a potential disease-modifying therapy that targets underlying inflammation and vascular remodeling in PAH, addressing a critical unmet medical need in a disease with only 57% five-year survival rates.
  • The investigational drug has demonstrated excellent safety profiles across five clinical trials involving over 100 subjects and has received orphan designation in both the U.S. and Europe.
  • The FDA has already issued positive scientific advice on the planned trial design, validating the therapeutic approach for this debilitating rare disease.

Servier Acquires KER-0193 for $450M to Target Fragile X Syndrome, Marking First Neurology Asset

Orphan DrugNeurologyRare Disease
  • Servier acquired KER-0193 from Kaerus Bioscience for up to $450 million, marking the company's first neurology asset acquisition as part of its 2030 strategy to establish a leading neurology franchise.
  • KER-0193 is an orally bioavailable small molecule targeting BK channels for Fragile X syndrome, the most common genetic cause of autism spectrum disorder, with no currently approved treatments available.
  • The drug has received FDA Orphan Drug and Rare Pediatric Drug Designations and successfully completed Phase 1 trials, with Phase 2 studies planned for 2026 in America and Europe.
  • Fragile X syndrome affects approximately 1 in 7,000 males and 1 in 11,000 females globally, representing a significant unmet medical need in neurodevelopmental disorders.

BioMarin Reports Positive Phase 3 Results for PALYNZIQ in Adolescent PKU Patients

Rare DiseaseOrphan Drug
  • BioMarin's Phase 3 PEGASUS trial demonstrated a 49.7% reduction in blood phenylalanine levels in adolescents aged 12-17 with PKU treated with PALYNZIQ compared to diet alone.
  • The study showed 45.2% of PALYNZIQ-treated adolescents achieved ≥50% reductions in phenylalanine levels after 72 weeks, versus only 5.9% in the diet-only group.
  • BioMarin plans to submit regulatory applications for adolescent label expansion in the second half of 2025, with approval anticipated by 2026.
  • The safety profile in adolescents was consistent with that observed in adults, with manageable adverse events and 5.6% serious adverse events leading to discontinuation.

FDA Expands Vonvendi Approval to Include Pediatric von Willebrand Disease Patients

Drug ApprovalRare Disease
  • The FDA has expanded approval of recombinant von Willebrand factor (Vonvendi) to include pediatric patients with von Willebrand disease for acute bleeding treatment and surgical procedures.
  • This marks the first non-plasma-derived VWF therapy available for children in the United States, offering more consistent pharmacokinetics and eliminating plasma-based therapy risks.
  • The approval also extends routine prophylactic use to adults with all types of VWD, previously limited to only severe Type 3 cases.
  • Clinical data showed most non-surgical bleeds in pediatric patients were effectively managed with a single infusion, with a half-life of 14.3 hours in children.

A Study of Recombinant Von Willebrand Factor (rVWF) With or Without ADVATE in Children With Severe Von Willebrand Disease (VWD)

NCT02932618RecruitingPhase 3
Baxalta now part of Shire
Posted 11/6/2017

rVWF IN PROPHYLAXIS

NCT02973087CompletedPhase 3
Takeda
Posted 11/16/2017

A Study of Recombinant Von Willebrand Factor (rVWF) in Pediatric and Adult Participants With Severe Von Willebrand Disease (VWD)

NCT03879135CompletedPhase 3
Baxalta now part of Shire
Posted 4/1/2019

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