BioMarin Seeks Label Growth For Palynziq After Constructive Part 3 Outcomes In Adolescent Sufferers – Biomarin Pharmaceutical (NASDAQ:BMRN)

BioMarin Pharmaceutical Inc. BMRN launched topline information from the Part 3 PEGASUS trial evaluating Palynziq (pegvaliase-pqpz) in comparison with weight loss program alone in 55 adolescents aged 12-17 with phenylketonuria (PKU).

PKU is a uncommon, inherited metabolic dysfunction that forestalls the physique from breaking down the amino acid phenylalanine. Untreated phenylketonuria can result in mind injury, mental disabilities, behavioral signs or seizures.

The first endpoints are the modifications in blood Phe focus and characterization of the protection profile in adolescents. Secondary endpoints embody modifications in complete dietary protein consumption and pharmacokinetics. 

Additionally Learn: Why Is BioMarin Inventory Buying and selling Increased On Thursday?

The examine met its major efficacy endpoint, demonstrating a statistically vital decreasing in blood Phe ranges in adolescents aged 12-17 with phenylketonuria (PKU) in comparison with weight loss program alone.

Security outcomes have been per the identified profile of the drugs.

Palynziq is the primary and solely enzyme remedy authorised to deal with adults with PKU.

Detailed outcomes from the PEGASUS examine will likely be introduced at an upcoming medical assembly and submitted to world well being authorities later this 12 months to request a label growth for Palynziq to incorporate adolescents.

Palynziq substitutes the poor phenylalanine hydroxylase (PAH) enzyme in PKU with a PEGylated model of the enzyme phenylalanine ammonia lyase to interrupt down Phe. Palynziq is run utilizing a dosing routine designed to facilitate tolerability.

Palynziq is authorised to cut back blood Phe concentrations for adults within the U.S., for folks 16 and older within the EU, Canada, and Brazil, and for folks 15 and older in Japan with PKU who’ve uncontrolled blood Phe concentrations better than 600 micromol/L on current administration.

Worth Motion: BMRN inventory is up 0.29% at $68.14 on the final test on Wednesday.

Learn Subsequent: