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Teva's Emrusolmin Receives FDA Fast Track Designation for Multiple System Atrophy Treatment

Orphan Drug
  • Teva Pharmaceuticals received FDA Fast Track designation for emrusolmin (TEV-56286), an investigational small molecule therapy targeting alpha-synuclein protein for Multiple System Atrophy treatment.
  • The therapy is currently in Phase 2 development through a strategic collaboration with German biotech company MODAG GmbH and previously received FDA Orphan Drug designation in 2022.
  • Multiple System Atrophy affects approximately 40,000 people in the US, EU, and Japan with no current treatments available that impact disease progression.
  • Fast Track designation facilitates expedited development and review for drugs addressing serious conditions with urgent unmet medical needs.

A Multi-centered, Double-blind, Randomized, Placebo-controlled Study of TEV-56286 for the Treatment of Multiple System Atrophy

2023-505320-54-00RecruitingPhase 2
Teva Branded Pharmaceutical Products R&D LLC
Posted 10/2/2024

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.

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

NCT04411641CompletedPhase 3
Sanofi
Posted 9/24/2020

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

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

Expanded Access Treatment Protocol With DCA for Patients With PDCD

NCT06931262available
Saol Therapeutics Inc

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

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.

uniQure's AMT-191 Gene Therapy Shows Sustained Enzyme Activity and ERT Discontinuation in Fabry Disease Phase I/IIa Trial

Rare DiseaseOrphan Drug
  • uniQure's AMT-191 gene therapy achieved 27- to 208-fold increases in α-galactosidase A enzyme activity above normal levels in all four patients in the first cohort of its Phase I/IIa trial.
  • All patients in the highest dose cohort successfully discontinued enzyme replacement therapy while maintaining stable plasma lyso-Gb3 biomarker levels through the study cutoff date.
  • The AAV5-based gene therapy demonstrated a manageable safety profile with sustained enzyme activity observed for up to 45 weeks in the first treated patient.
  • AMT-191 represents a potential paradigm shift in the $3.87 billion Fabry disease market by offering a one-time treatment alternative to lifelong enzyme replacement therapy.

Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease

NCT06270316RecruitingPhase 1
UniQure Biopharma B.V.
Posted 6/5/2024

Avadel Presents New LUMRYZ Data at World Sleep 2025, Demonstrating Real-World Efficacy in Narcolepsy Treatment

Drug ApprovalReal World EvidenceOrphan Drug
  • Avadel Pharmaceuticals presented 17 abstracts at World Sleep 2025, including new data from the REFRESH real-world study showing clinically meaningful improvements in narcolepsy symptoms with once-nightly LUMRYZ treatment.
  • The REFRESH study demonstrated that participants experienced improvements in excessive daytime sleepiness scores to within normal range and reduced severity of narcolepsy symptoms from moderate to mild.
  • LUMRYZ, the first and only once-at-bedtime sodium oxybate treatment, showed benefits for both patients switching from twice-nightly oxybates and those new to oxybate therapy.
  • Additional research revealed that patients with narcolepsy have significantly higher rates of sleep, pain, and psychiatric comorbidities compared to matched controls, highlighting the broader disease burden.

Bluejay Therapeutics Initiates Phase 3 AZURE-2 Trial Comparing Brelovitug to Hepcludex for Chronic Hepatitis D

Monoclonal AntibodyOrphan Drug
  • Bluejay Therapeutics has enrolled the first patient in its AZURE-2 phase 3 trial, evaluating brelovitug monotherapy against Hepcludex for chronic hepatitis D treatment.
  • The randomized controlled study will assess a composite primary endpoint of undetectable hepatitis D virus RNA and normalized ALT levels at week 48.
  • Chronic hepatitis D affects approximately 7 million people globally and represents the most severe form of viral hepatitis with limited treatment options.
  • Brelovitug, which received FDA Breakthrough Therapy designation in January 2025, is a monoclonal antibody targeting surface antigens on both hepatitis D and B viruses.

A Trial Evaluating BJT-778 vs Delayed Treatment for the Treatment of Chronic Hepatitis Delta Infection

NCT06907290RecruitingPhase 2
Bluejay Therapeutics, Inc.
Posted 3/25/2025

AL-S Pharma Reports Positive Phase 2 Results for AP-101 in ALS Treatment

Monoclonal AntibodyOrphan Drug
  • AL-S Pharma announced positive topline results from its Phase 2 study of AP-101, a first-in-class monoclonal antibody targeting misfolded SOD1 protein in amyotrophic lateral sclerosis patients.
  • The multicenter, randomized, double-blind, placebo-controlled study met its primary safety and tolerability endpoint in both sporadic ALS (N=52) and mutant SOD1-ALS (N=21) patients over 12 months of treatment.
  • Clinically meaningful changes in exploratory outcome measures related to survival and non-invasive ventilation were observed, along with stabilization of clinical disease-staging and neurofilament biomarkers.
  • This represents the first Phase 2 study to assess a SOD1-targeted therapeutic in both sporadic ALS and genetically determined SOD1-ALS patients, supporting the hypothesis that misfolded SOD1 protein plays a broader role in ALS pathogenesis.

A Study to Evaluate, Safety, Tolerability, Pharmacodynamic (PD) Markers and Pharmacokinetics (PK) of AP-101 in Participants With Amyotrophic Lateral Sclerosis (ALS)

NCT05039099CompletedPhase 2
AL-S Pharma
Posted 9/2/2021

Avadel Pharmaceuticals Acquires Exclusive Global Rights to Valiloxybate for Sleep Disorders

Orphan Drug
  • Avadel Pharmaceuticals has secured exclusive global licensing rights to valiloxybate from XWPharma for $20 million upfront, with potential milestone payments exceeding $205 million.
  • Valiloxybate represents the first salt-free, artificial sweetener-free, once-at-bedtime oxybate therapy designed for treating narcolepsy and idiopathic hypersomnia.
  • The company plans to initiate pharmacokinetic studies in Q4 2025, followed by pivotal trials in H2 2026, leveraging a streamlined bioequivalence pathway.
  • This acquisition strengthens Avadel's position in the expanding sleep medicine market, valued at $5.3 billion in 2024 and projected to reach $8.6 billion by 2033.

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