2
Orphan Designations
0 approved
0
FDA Approvals
0
Active Trials
6
Rare Diseases
across 8 areas
0
News (30d)
Quiet
Novartis Institute for BioMedical Research, Inc. is a company with 2 orphan drug designations across 6 rare diseases.
Disease-by-disease pipeline progression from orphan designation through clinical trials to FDA approval.
Source: FDA Orphan Drug Designations, ClinicalTrials.gov, Drugs@FDA
| Disease | Drug(s) | Stage |
|---|---|---|
| FOXG1 disorder | a non-replicating, recombinant adeno-associated virus serotype 9 (AAV9) containing the human Methyl CpG Binding Protein 2B (MECP2B) cDNA under the control of a segment of the murine MECP2 promoter. | Des.TrialAppr. |
| Friedreich ataxia | AAV9 gene vector construct expressing the human Frataxin gene (scAAV9.CB.hFRXN isoform 1) | Des.TrialAppr. |
| Friedreich ataxia 1 | AAV9 gene vector construct expressing the human Frataxin gene (scAAV9.CB.hFRXN isoform 1) | Des.TrialAppr. |
| Friedreich ataxia 2 | AAV9 gene vector construct expressing the human Frataxin gene (scAAV9.CB.hFRXN isoform 1) | Des.TrialAppr. |
| Rett syndrome | a non-replicating, recombinant adeno-associated virus serotype 9 (AAV9) containing the human Methyl CpG Binding Protein 2B (MECP2B) cDNA under the control of a segment of the murine MECP2 promoter. | Des.TrialAppr. |
| atypical Rett syndrome | a non-replicating, recombinant adeno-associated virus serotype 9 (AAV9) containing the human Methyl CpG Binding Protein 2B (MECP2B) cDNA under the control of a segment of the murine MECP2 promoter. | Des.TrialAppr. |
100% of portfolio targets high unmet need diseases
10
overlap in 2+ diseases
0/6
candidate diseases
0
avg importance: 0
0
affecting portfolio
100% of portfolio targets high unmet need diseases
10
overlap in 2+ diseases
0/6
candidate diseases
0
avg importance: 0
0
affecting portfolio