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Orphan 001
- Development stage
- Unknown
- Lead developer
- Alpex Pharma
- Modality
- Small Molecules
- Administration
- Oral
01
Overview
Orphan 001 is an investigational oral sachet formulation developed by Alpex Pharma, a Swiss-based contract manufacturing and development organization. The drug is targeted toward an undisclosed rare disease. According to company pipeline information, Orphan 001 reached the NDA/BLA filing stage with an expected FDA action date in December 2022. As Alpex Pharma primarily operates as a CMO/CRO specializing in delivery technologies (such as effervescent and fast-dissolving systems), specific details regarding the active pharmaceutical ingredient (API) and its molecular mechanism of action remain proprietary and are not publicly specified.
02
Targets
NMT1 (N-myristoyltransferase 1)NMT2 (N-myristoyltransferase 2)
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