On September 9, 2026, BioMarin reported Phase 3 CANOPY-HCH-3 results for VOXZOGO (vosoritide) in children with hypochondroplasia. Dauber and colleagues published the same-day paper in NEJM Evidence: "A Phase 3 Trial of Vosoritide in Children with Hypochondroplasia" (DOI 10.1056/EVIDoa2600257). The PubMed and Nemours Pure abstracts match. BioMarin also gave ESPE 2026 late-breaker LBA 1067.
Vosoritide is a C-type natriuretic peptide (CNP) analog. It is FDA-approved for achondroplasia. That is not approval for hypochondroplasia. BioMarin says it submitted an sNDA to expand VOXZOGO to hypochondroplasia. No regulator has approved that use. A 2027 launch, if approved, is a company timeline.
The primary result is a least-squares mean (LSM) annualized growth velocity (AGV) difference of 2.33 cm/year versus placebo at week 52 (95% CI 1.85 to 2.82; two-sided P<0.0001). That is a 52-week signal in 81 randomized children. It is not a U.S. hypochondroplasia label.
This is the 10:07 Research Roundup for 13 September 2026. It is not an Industry News incretin story. Vosoritide is not a GLP-1, GIP, or amylin drug. Yesterday's redasemtide Phase 2 note stays as written. Novapharma News recapped the company sentences. That recap is secondary.
What happened on September 9
BioMarin said CANOPY-HCH-3 met its primary endpoint. Children on vosoritide had statistically significant improvements versus placebo in AGV, standing height, height Z-score, and arm span after 52 weeks. Greg Friberg, M.D., the company's chief R&D officer, said BioMarin submitted the data to FDA "with the goal of securing approval for the first medicine for children with hypochondroplasia." That is a filing statement. It is not an approval.
Andrew Dauber, M.D., lead investigator at Children's National, said the AGV and arm-span changes show how children responded during the study. PeptidePrices reports that quote. It does not adopt "first targeted medicine" marketing as a finding.
The paper is e-pub ahead of print (9 September 2026). The NEJM Evidence HTML returned HTTP 403 here. Abstract numbers come from PubMed PMID 42713641 and Nemours Pure, which match. Standing-height, Z-score, and arm-span LSM differences come from the BioMarin release. This article does not invent extra manuscript tables.
NCT06455059, queried 13 September 2026, still has no posted results module. The September 9 numbers are not on the registry.
Why it matters
Hypochondroplasia is an FGFR3-related skeletal dysplasia with disproportionate short stature. The abstract says it has no available targeted therapies. BioMarin says there are currently no FDA- or EMA-approved medicines for it. The company estimates about 14,000 children in its footprint may be eligible if vosoritide were approved for that use. That 14,000 figure is a company estimate, not a census and not an efficacy result.
VOXZOGO already exists for achondroplasia. FDA's 19 November 2021 note granted accelerated approval to improve growth in children five years and older with achondroplasia and open epiphyses. In that one-year Phase 3, 121 participants were randomized. Participants on Voxzogo grew an average 1.57 centimeters taller than placebo. That 1.57 cm figure is the FDA ACH result. It is not the CANOPY-HCH-3 primary.
The U.S. ACH indication later dropped the age-5 floor. The DailyMed label revised November 2024 lists pediatric patients with achondroplasia and open epiphyses, still under accelerated approval, with safety data from 4.4 months. BioMarin's September 9 text says "all ages" with open epiphyses in the U.S., Japan, and Australia, and 4 months and older in the EU. Those sentences are ACH labels. They do not cover hypochondroplasia.
Do not merge the two sNDAs. The HCH sNDA is the 9 September 2026 expansion package. Separately, BioMarin says an ACH confirmatory sNDA (adult height and outcomes beyond linear growth) has a PDUFA target of 28 February 2027. That date is not an HCH clock.
Vosoritide is a prescription CNP analog, not a catalog "CNP" vial. A COA check can test whether a certificate names a real lab. It cannot turn a listing into VOXZOGO or an ACH label into an HCH label.
Study design
CANOPY-HCH-3 is Study 111-303: a Phase 3, randomized, double-blind, placebo-controlled, multicenter trial. BioMarin is the sponsor. NCT lists the drug as a modified recombinant human CNP.
| Item | Figure | Source |
|---|---|---|
| IDs | CANOPY-HCH-3 / 111-303 / NCT06455059 | NCT; BioMarin |
| Design | Phase 3, quadruple-masked | NCT |
| Population | Ages 3 to <18, genetically confirmed HCH | Abstract; NCT |
| Assignment | Daily SC vosoritide or placebo, 52 weeks, weight-band | Abstract; NCT |
| Randomized N | 81 (41 vs 40) | Abstract |
| Registry N | 80 estimated (20 Jan 2026) | NCT |
| Primary | AGV change at week 52 vs placebo | Abstract; NCT |
| Confirmatory testing | Hierarchical; six key secondaries | Abstract |
| Status | ACTIVE_NOT_RECRUITING; no results posted | NCT |
| Start / paper | 17 June 2024 actual; NEJM Evidence e-pub 9 Sept 2026, BioMarin-funded | NCT; abstract |
NCT says participants roll over from Study 111-902. Inclusion includes a height Z-score of -2.0 SD or below on CDC charts. Exclusion includes other short-stature diagnoses, closed plates, growth hormone / IGF-1 / anabolic steroids, planned limb-lengthening, and prior vosoritide.
This article keeps paper 81 and registry estimated 80 as two figures. It does not invent why they differ. The 41 versus 40 split is the abstract randomized N, not a posted results-module N.
Results
Primary (week 52 AGV). LSM change from baseline was +1.95 cm/year with vosoritide versus -0.39 cm/year with placebo. LSM difference: 2.33 cm/year (95% CI 1.85 to 2.82; two-sided P<0.0001). That pair is in the fetched abstract.
Key secondaries (BioMarin). Standing height +2.35 cm (p<0.0001). Height Z-score +0.39 SDS (p<0.0001). Arm span +1.03 cm (p=0.0082). The abstract says six key secondaries were in the hierarchical test. It does not print those three LSM differences. This article attributes them to the company release.
Quality of life. BioMarin reports numerical improvements and continuing follow-up. Numerical is the company's word. This article does not treat that sentence as a statistically significant secondary.
Safety. Abstract: at least one AE in 87.8% on vosoritide and 72.5% on placebo; no grade 3 or higher AEs, no AE discontinuations, no deaths. BioMarin: most AEs mild, profile consistent with ACH use, no treatment-related serious AEs identified. The percentages are the countable pair. The treatment-related SAE sentence is sponsor language, not a posted results module.
| Endpoint | Vosoritide | Placebo | Contrast |
|---|---|---|---|
| Randomized N | 41 | 40 | 81 total (abstract) |
| LSM AGV change, week 52 | +1.95 cm/year | -0.39 cm/year | LSM difference 2.33 cm/year (95% CI 1.85-2.82; two-sided P<0.0001) |
| Standing height | LSM difference +2.35 cm; p<0.0001 (BioMarin) | ||
| Height Z-score | LSM difference +0.39 SDS; p<0.0001 (BioMarin) | ||
| Arm span | LSM difference +1.03 cm; p=0.0082 (BioMarin) | ||
| Quality of life | Numerical improvements (company) | Not claimed as statistically significant here | |
| Any AE | 87.8% | 72.5% | Abstract |
| Grade 3+ AE / AE discontinuation / death | None reported in the abstract | None reported in the abstract | Abstract |
| Treatment-related SAE | None identified (company) | BioMarin 9 Sept 2026 |
The ACH 1.57 cm FDA figure and the HCH 2.33 cm/year LSM difference are different trials, different diagnoses, and different analysis statements. Do not subtract them into a "HCH beat ACH" ranking.
Trial dose (not an approved hypochondroplasia dose)
The abstract and BioMarin release describe once-daily subcutaneous injections on a weight-band regimen. NCT uses the same rule. None of the fetched sources publish HCH milligrams or mcg per kg.
Hypochondroplasia use is not approved. That does not make a once-daily subcutaneous weight-band schedule an approved hypochondroplasia dose. The rows below are trial and registry protocol fields. They are not a consumer protocol and not medical advice.
| Setting | Reported assignment | Source |
|---|---|---|
| HCH Phase 3 route and schedule | Once-daily subcutaneous injection for 52 weeks | Abstract; NCT06455059 |
| HCH dose rule | Weight-band dosing | Abstract; NCT06455059 |
| HCH milligrams / mcg per kg | Not disclosed in the fetched abstract, BioMarin HTML, or NCT | Fetched 13 Sept 2026 |
| ACH labeled product | Daily subcutaneous injection; dose based on body weight; ACH with open epiphyses only | DailyMed Nov 2024; BioMarin 9 Sept 2026 |
Do not treat the ACH labeled product, a catalog "vosoritide" vial, or a weight-band line as an HCH home schedule. Research-vendor vials, if they appear, are not VOXZOGO. Use the vendor checklist and the COA verification guide before treating any certificate as proof.
Limitations
The paper is e-pub ahead of print. The NEJM Evidence HTML returned HTTP 403. Height, Z-score, and arm-span LSM differences are company-release figures, not fetched abstract cells. A later full text may add intervals and the other key secondaries. Those extras are omitted until fetched.
Fifty-two weeks of AGV is not adult height. BioMarin says follow-up continues. That is not a published final-height result. The abstract safety block has any-AE rates and the no-grade-3 / no-discontinuation / no-death lines. There is no fetched HCH injection-site or blood-pressure table. ACH label warnings stay ACH label text.
NCT still shows estimated enrollment 80 and estimated completion 1 August 2026 (last update 20 January 2026). Quality of life is numerical in the company text, with no QoL p-value in the fetched abstract. The trial is BioMarin-funded; several authors list BioMarin affiliations. Funding does not erase P<0.0001. n=81 is one year of linear growth, not a functional-outcomes monograph.
What this does not mean
- It is not FDA or EMA approval for hypochondroplasia. BioMarin says no regulator has approved vosoritide for that use. The HCH sNDA is a submission.
- ACH approval is not HCH approval. Accelerated approval on 19 November 2021, and the later pediatric open-epiphyses label, cover achondroplasia.
- The ACH sNDA PDUFA date of 28 February 2027 is not the HCH clock. That date is the company's ACH full-approval / confirmatory target.
- 2.33 cm/year is not adult height. It is the week-52 LSM AGV difference versus placebo.
- Numerical quality-of-life improvements are not a significant QoL win in this article. The company used "numerical."
- Weight-band daily subcutaneous dosing is not an approved HCH dose. Hypochondroplasia use is not approved. That does not make the trial schedule an approved dose.
- A research-catalog peptide is not VOXZOGO. Approval of an ACH product, or an HCH sNDA filing, does not validate a gray-market vial.
- This is not medical advice, not a consumer protocol, and not a rewrite of redasemtide or TT-P34.
No PeptidePrices research page for vosoritide existed on 13 September 2026, and none was created. SS-31 / elamipretide is a different peptide with a different FDA story.
What to watch
- FDA review of the HCH sNDA. The September 9 release does not publish an HCH PDUFA date.
- EMA and other regional filings. BioMarin said those submissions are on track. On track is not an approval.
- The ACH PDUFA target of 28 February 2027. Keep it on the ACH confirmatory line.
- Posted NCT06455059 results and the full NEJM Evidence paper. Confirm height, Z-score, arm span, analyzed N, and safety tables.
- Longer follow-up. A 52-week AGV win can still leave adult-height questions. A paper does not turn a catalog listing into VOXZOGO.
FAQs
Is vosoritide (VOXZOGO) approved for hypochondroplasia?
No. It is FDA-approved for achondroplasia with open epiphyses. BioMarin says it submitted an HCH sNDA and that no regulator has approved vosoritide for hypochondroplasia.
What did CANOPY-HCH-3 show?
In 81 randomized children aged 3 to under 18, week-52 LSM AGV change was +1.95 cm/year on vosoritide versus -0.39 cm/year on placebo (LSM difference 2.33 cm/year; 95% CI 1.85 to 2.82; two-sided P<0.0001). BioMarin also reported standing height +2.35 cm, height Z-score +0.39 SDS, and arm span +1.03 cm versus placebo.
How many children were in the trial?
The paper randomized 81 (41 vosoritide, 40 placebo). ClinicalTrials.gov still lists enrollment as 80 estimated. Those figures are not collapsed here.
What dose was used?
Once-daily subcutaneous injections on a weight-band regimen for 52 weeks. The fetched abstract, company release, and NCT record do not publish HCH milligrams. Hypochondroplasia use is not approved. That does not make that schedule an approved dose.
Is this the same filing as the ACH PDUFA date of 28 February 2027?
No. That date is BioMarin's target for the separate ACH confirmatory sNDA. The HCH sNDA is the 9 September 2026 expansion package.
This article is for general informational purposes only and is not medical advice. Talk to a licensed clinician about personal medical decisions. Vosoritide (VOXZOGO) is an FDA-approved CNP analog for achondroplasia with open epiphyses. Hypochondroplasia use remains unapproved. Nothing here is a dosing protocol, an availability claim for a research vendor, or a recommendation to obtain an unapproved product.
Continue your research
- Redasemtide Phase 2 DEB and stroke
- TT-P34 Phase I Parkinson peptide
- How to verify a peptide COA
- COA Authenticity Checker
Sources
- BioMarin. Positive results of Phase 3 VOXZOGO (vosoritide) study in hypochondroplasia published in NEJM Evidence. 9 September 2026
- Dauber A, et al. A Phase 3 trial of vosoritide in children with hypochondroplasia. NEJM Evid. 9 September 2026. DOI 10.1056/EVIDoa2600257
- PubMed PMID 42713641 abstract
- Nemours Pure record of Dauber et al. 2026
- ClinicalTrials.gov NCT06455059. CANOPY-HCH-3 / Study 111-303
- FDA. FDA approves first drug to improve growth in children with the most common form of dwarfism. 19 November 2021
- DailyMed. VOXZOGO (vosoritide) prescribing information. Revised November 2024
- Novapharma News recap of the 2.33 cm/year HCH readout and sNDA (secondary)