Drug Profile
Research programme: genetic disorder therapeutics - Enlivex Therapeutics
Alternative Names: BB-FA; BB-OTC; BBrm 01; BBrm02; BBrm1; Intrathecal azithromycin - Enlivex TherapeuticsLatest Information Update: 22 May 2020
Price :
$50
*
At a glance
- Originator Bioblast Pharma
- Class Macrolides; Proteins; Small molecules
- Mechanism of Action Frataxin protein replacements; Gene expression modulators; Ornithine carbamoyltransferase replacements; Survival of motor neuron 2 protein expression stimulants
-
Orphan Drug Status
Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.
- New Molecular Entity No
Highest Development Phases
- No development reported Friedreich's ataxia; Inborn urea cycle disorders; Spinal muscular atrophy
Most Recent Events
- 19 Nov 2018 Bioblas Pharma has been merged into Enlivex Therapeutics to form Enlivex Therapeutics
- 28 Sep 2018 No recent reports of development identified for preclinical development in Friedreich's ataxia in Israel (Parenteral)
- 28 Sep 2018 No recent reports of development identified for preclinical development in Inborn-urea-cycle-disorders in Israel (Parenteral)