Our Pipeline: Status of Selected Key Development Programs as of November 2016; represents ongoing phase of development; does not correspond to initiation or completion of a particular phase.
- Phase 1
- Phase 2
- Phase 3
* Click here for JUXTAPID® (lomitapide) full US prescribing information, including BOXED WARNING for the risk of hepatotoxicity.
** Click here for MYALEPT® (metreleptin) full US prescribing information, including BOXED WARNING for the risk of anti-metreleptin antibodies with neutralizing activity and risk of lymphoma.
*** Plan to submit MAA for metreleptin in EU to treat GL and a subset of PL
Ongoing Research & Development
Novelion is developing a synthetic retinoid product candidate, zuretinol acetate, for the potential treatment of inherited retinal disease (IRD) caused by underlying mutations in RPE65 or LRAT genes, that prevent adequate functioning of the retinoid cycle. The disease area comprises leber congenital amaurosis (LCA) and retinitis pigmentosa (RP).
There are currently no U.S. Food and Drug Administration or European Medicines Agency approved pharmacologic treatments for either LCA or RP, underscoring the significant unmet medical need. Zuretinol has Orphan Drug designation from both FDA and EMA, as well as FDA Fast Track designation. As an oral product, Zuretinol is being studied for its potential to treat the retinas of both eyes simultaneously.