12
Orphan Designations
4 approved, 4 designated
4
FDA Approvals
Latest: Elevidys (2023)
9
Active Trials
200 recruiting
10
Rare Diseases
across 15 areas
3
News (30d)
Trending
Sarepta Therapeutics, Inc. is a company with 12 orphan drug designations across 10 rare diseases, including 4 FDA-approved therapies. Active clinical trials in 9 indications. 5 active policy bills affect their portfolio. 3 news articles in the past 30 days.
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 |
|---|---|---|
| Becker muscular dystrophy | Amondys 45ElevidysVYONDYS 53peptide-conjugated phosporodiamidate morpholino oligomer (PPMO)Exondys 51 | Des.TrialAppr. |
| Duchenne muscular dystrophy | ElevidysAMONDYS 45VYONDYS 53EXONDYS 51peptide conjugated phosphorodiamidate morpholino oligomer (PPMO)Amondys 45peptide-conjugated phosporodiamidate morpholino oligomer (PPMO)Exondys 51 | Des.TrialAppr. |
| autosomal recessive limb-girdle muscular dystrophy type 2B | recombinant adeno-associated virus serotype rhesus 74 expressing the human gamma-sarcoglycan transgene under the control of a myosin heavy chain creatine kinase muscle-specific promoter (rAAVrh74.MHCK7.hSGCG)self-complementary non-replicating, recombinant adeno-associated virus serotype rhesus 74 expressing the human alpha-sarcoglycan gene under a triple E-box muscle creatine kinase muscle specific promoter (scAAVrh74.tMCK.hSGCA)bidridistrogene xeboparvovecrecombinant adeno-associated virus serotype rhesus 74 expressing the human dysferin gene (AAVrh74.MHCK7.DYSF.DV) | Des.TrialAppr. |
| autosomal recessive limb-girdle muscular dystrophy type 2C | recombinant adeno-associated virus serotype rhesus 74 expressing the human gamma-sarcoglycan transgene under the control of a myosin heavy chain creatine kinase muscle-specific promoter (rAAVrh74.MHCK7.hSGCG) | Des.TrialAppr. |
| autosomal recessive limb-girdle muscular dystrophy type 2D | self-complementary non-replicating, recombinant adeno-associated virus serotype rhesus 74 expressing the human alpha-sarcoglycan gene under a triple E-box muscle creatine kinase muscle specific promoter (scAAVrh74.tMCK.hSGCA) | Des.TrialAppr. |
| autosomal recessive limb-girdle muscular dystrophy type 2E | bidridistrogene xeboparvovec | Des.TrialAppr. |
| autosomal recessive limb-girdle muscular dystrophy type 2I | recombinant adeno-associated virus serotype rhesus 74 expressing the human gamma-sarcoglycan transgene under the control of a myosin heavy chain creatine kinase muscle-specific promoter (rAAVrh74.MHCK7.hSGCG)self-complementary non-replicating, recombinant adeno-associated virus serotype rhesus 74 expressing the human alpha-sarcoglycan gene under a triple E-box muscle creatine kinase muscle specific promoter (scAAVrh74.tMCK.hSGCA)bidridistrogene xeboparvovecrecombinant adeno-associated virus serotype rhesus 74 expressing the human dysferin gene (AAVrh74.MHCK7.DYSF.DV) | Des.TrialAppr. |
| autosomal recessive limb-girdle muscular dystrophy type 2U | recombinant adeno-associated virus serotype rhesus 74 expressing the human dysferin gene (AAVrh74.MHCK7.DYSF.DV) | Des.TrialAppr. |
| muscular dystrophy | - | Des.TrialAppr. |
| symptomatic form of muscular dystrophy of Duchenne and Becker in female carriers | Elevidyspeptide-conjugated phosporodiamidate morpholino oligomer (PPMO) | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
50
overlap in 2+ diseases
0/10
candidate diseases
3
avg importance: 60
5
affecting portfolio
0% of portfolio targets high unmet need diseases
50
overlap in 2+ diseases
0/10
candidate diseases
3
avg importance: 60
5
affecting portfolio