
Search for companies, drugs, and catalysts
Search for companies, drugs, and catalysts
Pharmaceutical Preparations · market cap $10.6B · as of 2026-06-26
Research and calibrated estimates, not financial advice — verify before acting.
| Asset | Phase | Indication |
|---|---|---|
| Voxzogo | Approved | Achondroplasia |
| Roctavian | Approved | Hemophilia A |
| BMN 331 | Phase 1/2 | Phenylketonuria |
| BMN 110 - Weekly | Phase 3 | Mucopolysaccharidosis IV A |
| Human Growth Hormone | Phase 2 | Idiopathic Short Stature |
| sapropterin dihydrochloride, 6R-BH4, tetrahydrobiopterin | Phase 3 | Phenylketonurias |
| Valoctocogene roxaparvovec | Phase 3 | Hemophilia A With Inhibitor |
| BMN 165 | Phase 3 | Phenylketonuria |
| BMN 190 recombinant human tripeptidyl peptidase-1 (rhTPP1) | Phase 2 | Jansky-Bielschowsky Disease |
| Vosoritide Injection | Phase 2 | Short Stature Homeobox- Containing Gene SHOX Deficiency |
| Kuvan® | Phase 3 | Phenylketonuria |
| rAvPAL-PEG | Phase 2 | Phenylketonuria |
| BMN 110 Weekly | Phase 3 | MPS IV A |
| Phenoptin | Phase 2 | Tetrahydrobiopterin Deficiencies |
| BMN 701 | Phase 2 | Pompe Disease |
| BMN 111 | Phase 3 | Achondroplasia |
| BMN 351 | Phase 2 | Duchenne Muscular Dystrophy |
| 6R-BH4 (sapropterin dihydrochloride) | Phase 2 | Hypertension |
| BMN 307 | Phase 2 | Phenylketonuria (PKU) |
| N-acetylgalactosamine 4-sulfatase | Phase 3 | Mucopolysaccharidosis VI |
Daily closes through 2026-07-01. Realized vol annualized; max drawdown over ~2 years.
The full dossier is a Pro feature
Thesis, bull & bear, the if-approved scenario, financials, smart-money signals and risks — compiled by CeliA. The catalyst path & pipeline above are free.
See ProApproval odds use historical phase base rates; the if-approved scenario is illustrative (see its note). Figures trace to the sources above — verify before acting.
Data from CatalystAlert (ClinicalTrials.gov, SEC EDGAR, openFDA, Financial Modeling Prep). Compiled by CeliA. Not financial advice.