BioMarin Pharmaceutical Inc.

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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• 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

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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

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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

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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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• 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

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Entry

Full CANOPY-HCH Phase 3 hypochondroplasia dataset at ESPE 2026 · Data presented at a medical meeting

Drug

VOXZOGO (vosoritide)

For

Hypochondroplasia in children

Stage

Phase 3 · the last stage before a drug can be approved

When

Sep 2026 · the company gave a window, not a day

Source note

BioMarin Q2 2026 earnings release: full Phase 3 dataset to be presented at the European Society for Paediatric Endocrinology Annual Meeting in September 2026.

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• Source: 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
• BMRN headlines on this site

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Entry

VOXZOGO hypochondroplasia sNDA submitted to FDA · Company milestone

Drug

VOXZOGO (vosoritide)

For

Hypochondroplasia in children

Stage

sNDA submission planned

When

Q3 2026 · the company gave a window, not a day

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

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Entry

Phase 3 CANOPY-HCH-3 met primary endpoint: VOXZOGO +2.33 cm/yr in hypochondroplasia · Clinical trial results (readout / topline data)

Drug

VOXZOGO (vosoritide)

For

Hypochondroplasia

Stage

Phase 3 · the last stage before a drug can be approved

When

May 20, 2026

Source note

The Phase 3 CANOPY-HCH-3 study of VOXZOGO in children with hypochondroplasia met its primary endpoint with a statistically significant increase in annualized growth velocity at Week 52 versus placebo (LS mean difference +2.33 cm/yr, p<0.0001), plus significant gains in standing height, height Z-score and arm span. BioMarin planned a US sNDA submission in Q3 2026.

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• Source: 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
• BMRN headlines on this site

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Entry

Phase 3 ENERGY 3: BMN 401 met PPi co-primary but missed RGI-C in ENPP1 deficiency · Clinical trial results (readout / topline data)

Drug

BMN 401

For

ENPP1 deficiency

Stage

Phase 3 · the last stage before a drug can be approved

When

May 18, 2026

Source note

BioMarin reported that the pivotal Phase 3 ENERGY 3 trial of BMN 401 in children aged 1-12 with ENPP1 deficiency met one co-primary endpoint with statistically significant increases in plasma inorganic pyrophosphate through Week 52, but missed the other co-primary endpoint with no improvement in Radiographic Global Impression of Change. BioMarin subsequently discontinued BMN 401 across all indications.

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• Source: 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
• BMRN headlines on this site

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Entry

VOXZOGO Phase 2 trials in Turner, SHOX and ACAN halted after SCFE events · Company milestone

Drug

VOXZOGO (vosoritide)

For

Turner syndrome, SHOX deficiency, ACAN deficiency

Stage

Phase 2 · the stage that tests whether the drug actually works

When

Mar 16, 2026

Source note

BioMarin discontinued dosing and enrollment in its Phase 2 CANOPY trials of VOXZOGO in Turner syndrome, SHOX deficiency and ACAN deficiency after several slipped capital femoral epiphysis events occurred in two investigator-sponsored trials. No SCFE cases had been seen in BioMarin's own trials in these conditions, and the Noonan syndrome and idiopathic short stature arms continued.

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• Source: sec.gov ↗ — 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

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Entry

FDA approval of Roctavian · Regulatory approval

Drug

Roctavian (valoctocogene roxaparvovec)

For

Severe hemophilia A

When

Jun 29, 2023

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• 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