Sponsor or institution

Clinical trials and research at BioMarin Pharmaceutical

• 10Clinical trials
• 14Countries

Newest trials

All recruiting trials (10)

• Immune Modulation During Palynziq Treatment in Adults

  This study tests whether adding low-dose methotrexate can help people with PKU tolerate Palynziq better and achieve better blood levels of phenylalanine.

  Recruiting at 10 sites Phase 4 BioMarin Pharmaceutical · Los Angeles, California and 11 other locations · NCT07477691

• BMN 333 versus daily vosoritide for growth in children with achondroplasia

  This trial tests whether weekly BMN 333 injections help children with achondroplasia grow as well as daily vosoritide.

  Recruiting at 25 sites Phase 2/3 BioMarin Pharmaceutical · Oakland, California and 27 other locations · NCT07441876

• Daily vosoritide versus placebo for infants and toddlers with hypochondroplasia

  This trial will test whether daily vosoritide injections for 52 weeks help infants and toddlers under 36 months with genetically confirmed hypochondroplasia grow better and stay healthier.

  Recruiting at 17 sites Phase 2 BioMarin Pharmaceutical · Phoenix, Arizona and 25 other locations · NCT07126262

• Evaluating Vosoritide for Children with Short Stature

  This study is testing a new treatment called vosoritide to see if it helps children with short stature grow better than the standard growth hormone treatment.

  Recruiting at 43 sites Phase 2 BioMarin Pharmaceutical · California City, California and 46 other locations · NCT06382155

• Assessing Growth in Children with Short Stature

  This study is trying to gather information on how children with unexplained short stature grow to help improve future treatments for them.

  Recruiting at 21 sites BioMarin Pharmaceutical · Phoenix, Arizona and 37 other locations · NCT06309979

• Long-term evaluation of immune response in PKU patients receiving Palynziq

  This study is testing how well Palynziq works over 10 years for people with PKU by looking at their immune responses and overall health.

  Recruiting at 11 sites BioMarin Pharmaceutical · Aurora, Colorado and 10 other locations · NCT06305234

• Observational study of children with Hypochondroplasia

  This study is tracking the growth and development of children with Hypochondroplasia to see how the condition affects them as they grow up.

  Recruiting at 32 sites BioMarin Pharmaceutical · Phoenix, Arizona and 42 other locations · NCT06212947

• Observational study of achondroplasia in the US

  This study looks at children in the U.S. with achondroplasia to see how they are doing with and without the treatment VOXZOGO™ over at least five years.

  Recruiting at 8 sites BioMarin Pharmaceutical · Oakland, California and 7 other locations · NCT06168201

• Long-term safety assessment of pegvaliase in patients with PKU

  This study is testing the long-term safety of pegvaliase in people with PKU to see how well it works and if there are any side effects over time.

  Recruiting at 26 sites BioMarin Pharmaceutical · Aurora, Colorado and 25 other locations · NCT05813678

• Assessing the effects of Palynziq during pregnancy and breastfeeding

  This study looks at how the medication Palynziq affects women with PKU and their babies during pregnancy and breastfeeding.

  Recruiting at 4 sites BioMarin Pharmaceutical · Wilmington, North Carolina and 3 other locations · NCT05579548

Quick answers

How many clinical trials run by BioMarin Pharmaceutical are recruiting right now?

10, as of November 12, 2026. We update this list each time we refresh our data from ClinicalTrials.gov.

Where are these trials running?

In 14 countries. The most sites are in United States (10 trials), Italy (6 trials), Germany (5 trials), Australia (4 trials), France (3 trials). Each study page lists its sites and which ones are recruiting.

What phases are they in?

Phase 2: 2, Phase 4: 1, Phase 2/3: 1. Trials with no phase listed are usually device, behavioural or observational studies.

How do I join one?

Open a study, check the "Is this trial a fit for you?" section, then contact the study team listed on the page. They will ask a few screening questions. Asking is free and does not commit you to anything. Talk it over with your doctor first.

Find the studies that fit you

A list is a starting point. Tell us your situation in your own words and we'll rank the studies that match and explain why. It's free and needs no account.

Describe your situation →