VOXZOGO hypochondroplasia sNDA submitted to FDA
VOXZOGO hypochondroplasia sNDA submitted to FDA, in Q3 2026 (Jul–Sep). A company milestone for BioMarin (VOXZOGO (vosoritide) for Hypochondroplasia in children): a step in the program rather than a decision or a data readout.
Plain-English summary generated from this entry’s own fields (drug, condition, stage, date). Glossary
- Company
- BioMarin Pharmaceutical Inc. BMRN
- Drug
- VOXZOGO (vosoritide)
- For
- Hypochondroplasia in children
- Stage
- sNDA submission planned
- When
- Q3 2026 · the company gave a window, not a day; the calendar never invents one
- Source note
- the known Q3 2026 HCH sNDA milestone has occurred — Aug 6, 2026 Q2 PR says the sNDA was 'recently submitted'; FDA acceptance/PDUFA date not yet announced. Full CANOPY-HCH dataset to be a late-breaking oral at ESPE in September (already tracked).
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- Source: prnewswire.com ↗ — where this entry was taken from
- SEC filings (8-K) ↗ — press releases are attached to 8-K filings
- ClinicalTrials.gov ↗ — trials of this drug
- BMRN headlines on this site
BMN 351 DMD program update by year-end 2026, expected in Q4 2026 (Oct–Dec) (no exact date yet). A company milestone for BioMarin (BMN 351 for Duchenne muscular dystrophy (exon 51 skipping)): a step in the program rather than a decision or a data readout.
More details
- Entry
- BMN 351 DMD program update by year-end 2026 · Company milestone
- Drug
- BMN 351
- For
- Duchenne muscular dystrophy (exon 51 skipping)
- Stage
- Early clinical
- When
- Q4 2026 · the company gave a window, not a day
- Source note
- Q2 2026 earnings PR (Aug 6, 2026) frames the known H2 2026 BMN 351 update as 'by year-end', i.e., Q4 2026 under the date-mapping rules.
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- Checked by a person when it was added
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- Source: prnewswire.com ↗ — where this entry was taken from
- SEC filings (8-K) ↗ — press releases are attached to 8-K filings
- ClinicalTrials.gov ↗ — trials of this drug
- BMRN headlines on this site
Plain-English summary generated from this entry’s own fields (drug, condition, stage, date). Glossary · Open this catalyst’s page
The FDA is due to decide by Feb 28, 2027 whether to approve BioMarin’s VOXZOGO (vosoritide) for Achondroplasia in children (conversion of accelerated approval to full approval). Approval clears it for sale in the US; a rejection usually arrives as a letter listing what has to be fixed first.
More details
- Entry
- PDUFA date for VOXZOGO full-approval sNDA in achondroplasia · FDA decision due (PDUFA date)
- Drug
- VOXZOGO (vosoritide)
- For
- Achondroplasia in children (conversion of accelerated approval to full approval)
- Stage
- sNDA under FDA review
- When
- Feb 28, 2027
- Source note
- FDA accepted BioMarin's sNDA for full approval of VOXZOGO in children with achondroplasia with a PDUFA target action date of February 28, 2027, supported by long-term data including adult height, body proportionality, and arm span.
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- Source: investors.biomarin.com ↗ — where this entry was taken from
- SEC filings (8-K) ↗ — press releases are attached to 8-K filings
- ClinicalTrials.gov ↗ — trials of this drug
- Drugs@FDA ↗ — search the drug name for approval letters
- BMRN headlines on this site
Plain-English summary generated from this entry’s own fields (drug, condition, stage, date). Glossary · Open this catalyst’s page
BioMarin expects to report results from its Phase 2/3 study of BMN 333 for Achondroplasia during 2027 (no exact date yet). Phase 2/3 is a combined mid- to late-stage study.
More details
- Entry
- BMN 333 long-acting CNP Phase 2/3 achondroplasia data update · Clinical trial results (readout / topline data)
- Drug
- BMN 333
- For
- Achondroplasia
- Stage
- Phase 2/3 · a combined mid- to late-stage study
- When
- 2027 · the company gave a window, not a day
- Source note
- Q2 2026 earnings PR (Aug 6, 2026); no half/quarter given.
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- Source: prnewswire.com ↗ — where this entry was taken from
- SEC filings (8-K) ↗ — press releases are attached to 8-K filings
- ClinicalTrials.gov ↗ — trials of this drug
- BMRN headlines on this site
Plain-English summary generated from this entry’s own fields (drug, condition, stage, date). Glossary · Open this catalyst’s page
BioMarin expects the FDA to decide on VOXZOGO (vosoritide) for Hypochondroplasia during 2027 (no exact date yet). An approval lets the company start selling it in that market.
More details
- Entry
- Potential FDA approval/launch of VOXZOGO in hypochondroplasia · Regulatory approval
- Drug
- VOXZOGO (vosoritide)
- For
- Hypochondroplasia
- Stage
- sNDA under FDA review
- When
- 2027 · the company gave a window, not a day
- Source note
- Q2 2026 earnings PR (Aug 6, 2026); a PDUFA date should follow FDA acceptance of the recently submitted sNDA.
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- Checked by a person when it was added
- Verify it
- Source: prnewswire.com ↗ — where this entry was taken from
- SEC filings (8-K) ↗ — press releases are attached to 8-K filings
- ClinicalTrials.gov ↗ — trials of this drug
- Drugs@FDA ↗ — search the drug name for approval letters
- BMRN headlines on this site
Plain-English summary generated from this entry’s own fields (drug, condition, stage, date). Glossary · Open this catalyst’s page