Last reviewed 2026-04-22 · How we verify

RDD to Palynziq

BioMarin Pharmaceutical · FDA-approved active Small molecule

RDD to Palynziq is a Enzyme replacement therapy Small molecule drug developed by BioMarin Pharmaceutical. It is currently FDA-approved for Phenylketonuria (PKU) in patients with blood phenylalanine levels ≥600 μmol/L.

Palynziq is a recombinant phenylalanine ammonia-lyase (PAL) enzyme that breaks down phenylalanine, reducing blood phenylalanine levels in patients with phenylketonuria.

Palynziq is a recombinant phenylalanine ammonia-lyase (PAL) enzyme that breaks down phenylalanine, reducing blood phenylalanine levels in patients with phenylketonuria. Used for Phenylketonuria (PKU) in patients with blood phenylalanine levels ≥600 μmol/L.

At a glance

Generic name	RDD to Palynziq
Sponsor	BioMarin Pharmaceutical
Drug class	Enzyme replacement therapy
Target	Phenylalanine ammonia-lyase (PAL)
Modality	Small molecule
Therapeutic area	Metabolic/Genetic disorder
Phase	FDA-approved

Mechanism of action

Palynziq catalyzes the conversion of phenylalanine to trans-cinnamic acid and ammonia, thereby lowering circulating phenylalanine concentrations. This enzyme replacement therapy addresses the underlying metabolic defect in phenylketonuria (PKU), where patients lack functional phenylalanine hydroxylase, allowing them to tolerate a more normal diet while maintaining safe phenylalanine levels.

Approved indications

Phenylketonuria (PKU) in patients with blood phenylalanine levels ≥600 μmol/L

Common side effects

Injection site reactions
Anti-drug antibody formation
Arthralgia
Headache

Key clinical trials

Rapid Drug Desensitization Study in Adults Experiencing Hypersensitivity Reactions to Palynziq (PHASE4)

Primary sources

Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.

Source	Used for
ClinicalTrials.gov	Trial enrolment, design, endpoints, results

Competitive intelligence

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Frequently asked questions about RDD to Palynziq

What is RDD to Palynziq?

RDD to Palynziq is a Enzyme replacement therapy drug developed by BioMarin Pharmaceutical, indicated for Phenylketonuria (PKU) in patients with blood phenylalanine levels ≥600 μmol/L.

How does RDD to Palynziq work?

Palynziq is a recombinant phenylalanine ammonia-lyase (PAL) enzyme that breaks down phenylalanine, reducing blood phenylalanine levels in patients with phenylketonuria.

What is RDD to Palynziq used for?

RDD to Palynziq is indicated for Phenylketonuria (PKU) in patients with blood phenylalanine levels ≥600 μmol/L.

Who makes RDD to Palynziq?

RDD to Palynziq is developed and marketed by BioMarin Pharmaceutical (see full BioMarin Pharmaceutical pipeline at /company/biomarin-pharmaceutical).

What drug class is RDD to Palynziq in?

RDD to Palynziq belongs to the Enzyme replacement therapy class. See all Enzyme replacement therapy drugs at /class/enzyme-replacement-therapy.

What development phase is RDD to Palynziq in?

RDD to Palynziq is FDA-approved (marketed).

What are the side effects of RDD to Palynziq?

Common side effects of RDD to Palynziq include Injection site reactions, Anti-drug antibody formation, Arthralgia, Headache.

What does RDD to Palynziq target?

RDD to Palynziq targets Phenylalanine ammonia-lyase (PAL) and is a Enzyme replacement therapy.

Drug class: All Enzyme replacement therapy drugs
Target: All drugs targeting Phenylalanine ammonia-lyase (PAL)
Manufacturer: BioMarin Pharmaceutical — full pipeline
Therapeutic area: All drugs in Metabolic/Genetic disorder
Indication: Drugs for Phenylketonuria (PKU) in patients with blood phenylalanine levels ≥600 μmol/L
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