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IntraBio Completes Over-Enrollment for Pivotal Niemann-Pick Disease Type C Trial, Data Expected Q2 2023

Rare DiseaseOrphan Drug
  • IntraBio successfully completed recruitment for its Phase III pivotal trial of IB1001 (N-Acetyl-L-Leucine) for Niemann-Pick disease type C, enrolling 130% of target patients across 13 international sites.
  • The company over-enrolled by 125% due to high community interest and unmet medical need, substantially increasing the statistical power of the randomized, placebo-controlled, double-blind crossover study.
  • Data readout is anticipated before the end of Q2 2023, with the trial designed to support accelerated approval pathways with both FDA and EMA for chronic NPC symptom treatment.
  • Upon approval, IntraBio would be eligible for an FDA priority review voucher, which have sold for over $100 million each in the past two years.

A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C

NCT05163288RecruitingPhase 3
IntraBio Inc
Posted 6/30/2022

FDA Approves Zanubrutinib for Waldenström's Macroglobulinemia Treatment

Drug ApprovalRare Disease
  • The FDA has approved zanubrutinib (Brukinsa) for treating adult patients with Waldenström's macroglobulinemia, marking the second therapy specifically approved for this rare lymphoma.
  • The approval was based on the phase 3 ASPEN trial comparing zanubrutinib to ibrutinib, showing a higher very good partial response rate of 28% versus 19% respectively.
  • Zanubrutinib demonstrated improved tolerability compared to the first-generation BTK inhibitor ibrutinib across several clinically important side effects.
  • The drug is administered orally at either 160 mg twice daily or 320 mg once daily, offering patients a convenient single-agent treatment option.

A Study Comparing BGB-3111 and Ibrutinib in Participants With Waldenström's Macroglobulinemia (WM)

NCT03053440CompletedPhase 3
BeiGene
Posted 1/25/2017

Tofersen Shows Promise in Phase 1/2 Trial for Inherited Form of ALS

Rare Disease
  • Biogen's experimental drug tofersen demonstrated safety and successfully lowered levels of disease-causing SOD1 protein in patients with a rare inherited form of ALS in a phase 1/2 clinical trial.
  • The antisense oligonucleotide treatment reduced SOD1 protein concentrations by up to 33% in the high-dose group, with some indications it may slow disease progression, prompting advancement to phase 3 trials.
  • While targeting only approximately 2% of ALS patients with SOD1 mutations, researchers believe this approach could establish a platform for treating other forms of ALS and neurodegenerative conditions through protein regulation.

FDA Approves First Treatment for Primary Hemophagocytic Lymphohistiocytosis, Marking 24-Year Breakthrough

Monoclonal AntibodyDrug ApprovalRare DiseaseOrphan Drug
  • The FDA has approved emapalumab-lzsg (Gamifant) as the first treatment specifically indicated for primary hemophagocytic lymphohistiocytosis (HLH), an ultra-rare and life-threatening immune disorder.
  • In a pivotal phase 2/3 study of 27 patients with refractory disease, 63% demonstrated overall response and 70% proceeded to hematopoietic stem cell transplant.
  • The monoclonal antibody targets interferon gamma, representing the first significant improvement in primary HLH induction therapy in 24 years.
  • Primary HLH typically affects children within the first year of life and has a median survival of less than two months without treatment.

Study to Investigate Safety, Efficacy of an Anti-IFNγ mAb in Children With Primary Haemophagocytic Lymphohistiocytosis

NCT01818492CompletedPhase 2
Swedish Orphan Biovitrum
Posted 7/1/2013

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