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Race to replace injections for childhood dwarfism

Race to replace injections for childhood dwarfism

New Capabilities

BridgeBio's Oral Therapy Faces Once-Weekly Injection After BioMarin's Daily Shot

February 27th, 2026: FDA Approves YUVIWEL, First Once-Weekly Achondroplasia Treatment

Overview

About 250,000 people worldwide have achondroplasia, the most common genetic cause of dwarfism. Until BioMarin's Voxzogo, the only approved treatment required daily injections.

On February 12, 2026, BridgeBio reported that its oral pill achieved a 2.10 cm/year height growth increase over placebo in a Phase 3 trial, exceeding Voxzogo's 1.57 cm/year gain. It was the first statistically significant improvement in body proportionality. Two weeks later, on February 27, the FDA approved Ascendis Pharma's once-weekly YUVIWEL (navepegritide) for children aged 2 and older.

Voxzogo generated $735 million in 2024 sales; 2025 projections put it at $900 million. The market now has daily injections, a weekly shot, and a pending oral pill. BridgeBio plans regulatory filing in the second half of 2026, while YUVIWEL launches in early Q2 2026.

Play on this story Voices Debate Predict

Key Indicators

2.10 cm/yr
Height gain vs placebo
BridgeBio's infigratinib outperformed Voxzogo's 1.57 cm/year; YUVIWEL showed 1.49 cm/year in its trial
1 in 21,000
Birth prevalence
Approximately 250,000 people worldwide live with achondroplasia
$735M
Voxzogo 2024 sales
BioMarin's injectable grew rapidly since 2021 approval, ~$900M projected for 2025 amid new competition
0%
Serious adverse events
No discontinuations or serious drug-related adverse events in BridgeBio's Phase 3 trial
Once-weekly
YUVIWEL dosing
FDA-approved injection for children 2+ years, first alternative to daily Voxzogo

Voices

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People Involved

Organizations Involved

Timeline

November 2021 February 2026

9 events Latest: February 27th, 2026 · 3 months ago
Tap a bar to jump to that date
  1. FDA Approves YUVIWEL, First Once-Weekly Achondroplasia Treatment

    Latest Regulatory

    FDA grants accelerated approval to Ascendis Pharma's YUVIWEL (navepegritide) to increase linear growth in children 2 years and older with achondroplasia. Commercial availability expected early Q2 2026; Rare Pediatric Disease Priority Review Voucher also issued.

  2. BridgeBio Reports Positive Phase 3 Results

    Clinical

    BridgeBio announces PROPEL 3 Phase 3 trial met primary endpoint with 2.10 cm/year height gain versus placebo and achieved first statistically significant improvement in body proportionality for achondroplasia.

  3. FDA Delays Ascendis Decision

    Regulatory

    FDA extends review period for Ascendis's TransCon CNP by three months to February 28, 2026, citing a major amendment to the application.

  4. Phase 2 Results Published in NEJM

    Publication

    New England Journal of Medicine publishes BridgeBio's Phase 2 PROPEL 2 study results for infigratinib in achondroplasia.

  5. FDA Grants Breakthrough Therapy Designation

    Regulatory

    FDA designates BridgeBio's oral infigratinib as a Breakthrough Therapy for achondroplasia, potentially expediting review.

  6. BridgeBio Reports Durable Phase 2 Results

    Clinical

    BridgeBio announces 12- and 18-month data from PROPEL 2 Phase 2 trial showing sustained 2.5 cm/year height gains with infigratinib.

  7. BridgeBio Signs $100M Japan Deal

    Business

    BridgeBio's QED Therapeutics licenses infigratinib rights in Japan to Kyowa Kirin for $100 million upfront plus potential milestones.

  8. Voxzogo Expanded to All Ages

    Regulatory

    FDA approves supplemental application expanding Voxzogo to children of all ages with achondroplasia and open growth plates.

  9. FDA Approves First Achondroplasia Treatment

    Regulatory

    BioMarin's Voxzogo (vosoritide) receives accelerated FDA approval for children aged 5 and older with open growth plates, becoming the first drug approved to treat the genetic condition.

Historical Context

3 moments from history that rhyme with this story — and how they unfolded.

1997

Growth Hormone for Turner Syndrome (1997)

The FDA approved growth hormone treatment for Turner syndrome, a chromosomal condition causing short stature in girls. The approval established a precedent for pharmacological intervention in genetic growth disorders, though treatment required daily injections for years.

Then

Treatment became standard of care for Turner syndrome, with most affected children receiving growth hormone therapy.

Now

Demonstrated that chronic injection therapy could be successfully adopted in pediatric populations, paving the way for Voxzogo's approach two decades later.

Why this matters now

Voxzogo's approval followed a similar regulatory path, and the Turner syndrome experience showed that families will accept daily injections for growth benefits—but also highlighted the opportunity for less burdensome alternatives.

December 2013 - October 2014

Hepatitis C Oral Therapy Revolution (2013-2014)

Gilead's Sovaldi and Harvoni replaced interferon injections with oral pills for hepatitis C treatment. The shift from injectable regimens with severe side effects to well-tolerated oral pills transformed treatment uptake and cure rates.

Then

Cure rates exceeded 95% as patients completed oral regimens they had previously abandoned due to injection burden.

Now

Hepatitis C became a curable disease for most patients. The example demonstrated how oral alternatives can dramatically expand treatment adoption.

Why this matters now

BridgeBio's oral infigratinib represents a similar shift from injection to pill. If approved, the convenience advantage could similarly expand treatment uptake among achondroplasia families who have declined or discontinued Voxzogo.

1980s-present

Limb Lengthening Surgery Era (1980s-Present)

Before Voxzogo, the only intervention for achondroplasia height was limb lengthening surgery using distraction osteogenesis—a painful process involving cutting bones and gradually separating them over months. Patients underwent an average of 3-4 procedures to gain 10-15 cm of height.

Then

Surgery provided meaningful height gains but with significant pain, complications, and years of recovery.

Now

Only about 19% of achondroplasia patients chose surgery, with nearly half uncertain whether to recommend it. The invasiveness created demand for pharmacological alternatives.

Why this matters now

Voxzogo's approval in 2021 marked the first non-surgical option. BridgeBio's oral therapy represents the next evolution—from surgery to injection to pill—potentially making treatment accessible to families who rejected both prior options.

Sources

(11)