FDA grants Orphan Drug Designation for Tinostamustine in malignant glioma
Tinostamustine is an investigational drug with a potential first-in-class mechanism combining bifunctional alkylating activity and pan histone deacetylase (HDAC) inhibition
Tinostamustine is an investigational drug with a potential first-in-class mechanism combining bifunctional alkylating activity and pan histone deacetylase (HDAC) inhibition
The company is committed to unlocking new frontiers in neuroscience and developing Usnoflast for patients with ALS
MRX-5 is a novel benzoxazole antibiotic developed for the treatment of mycobacteria infections, particularly infections caused by non-tuberculous Mycobacteria
It is the first autotaxin inhibitor to be investigated in cancer patients
CB03 is a candidate drug for the treatment of ALS and other central nerve system (CNS) diseases
ZYIL1 is a novel oral small molecule NLRP3 inhibitor which has demonstrated CSF penetration in non-human primates
The FDA's Orphan Drug Designation is granted to investigational therapies addressing rare medical diseases or conditions that affect fewer than 200,000 people
With this designation, LG Chem will receive benefits such as exclusive sales rights in the US market for seven years, subsidies and tax cuts for clinical testing costs in the US, and preliminary consulting support related to development
TP-3654 is currently being evaluated in a Phase 1/2, multicenter, dose-escalation, open-label trial to assess safety, tolerability, pharmacokinetics, and pharmacodynamics in patients with intermediate or high-risk primary or secondary myelofibrosis.
The treatment of chorea associated with HD is within the scope of this Orphan Drug Designation
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