US FDA Approves BioMarin’s Palynziq To Help Treat Rare Metabolic Disorder

FDA approved BioMarin’s Palynziq to help treat adults with a rare metabolism disorder called phenylketonuria (PKU)

The United States Food and Drug Administration (FDA) approved BioMarin Pharmaceutical Inc’s Palynziq (pegvaliase-pqpz) for adults who have rare and severe genetic illness known as phenylketonuria (PKU). 

Palynziq injections are used to reduce blood phenylalanine (Phe) concentrations in grown-up patients with phenylketonuria (PKU). Patients with PKU have uncontrolled blood Phe concentrations greater than 600 micromol/L on existing management.

Phe is a naturally occurring amino acid needed for optimal growth in infants and nitrogen equilibrium in adults. BioMarin’s Palynziq is a PEGylated recombinant phenylalanine ammonia lyase enzyme, and it is the first approved enzyme substitution treatment that aims to help the body to break down Phe.

Phenylketonuria or PKU is an uncommon genetic disease that flourishes at birth and usually ends in a range of growing toxic effects on the brain. Each year, PKU affects 1 in 12,500 births in the United States.

In line with the severity of the illness, babies are screened at birth to guarantee that they are diagnosed early and treated to circumvent intellectual disability and other complications. Patients living with PKU require management for their entire lifetime.

Patients need to devote and discipline themselves with a challenging and ruthlessly limiting daily diet of medical food and formula to avoid the consumption of Phe which is present in most food.

BioMarin waited two years for FDA to approve Palynziq. Palynziq has an estimated cost of $192,000 which is a 28 percent premium to BioMarin’s previous yet similar drug called Kuvan.

The success of the approval of the Palynziq in the US serves as a significant milestone for adults living with PKU who will now have access to a useful treatment option for controlling their blood Phe.

Cary Harding, M.D., a professor at Oregon Health and Science University said that the goal in treating PKU is to retain blood Phe levels within the range set in the medical guidelines, as an elevation of Phe can be deadly and damaging to the brain. Palynziq delivers another essential tool for professionals to help adult patients control their Phe levels, which it did not achieve before.

The company will ignite the promotion of Palynziq immediately and will be available in the United States by the end of June. For the meantime, Palynziq is available only through a restricted program under a Risk Evaluation, and Mitigation Strategy (REMS) called the Palynziq REMS. However, patients who need the Palynziq should not worry because BioMarin is committed to bringing Palynziq to adult PKU patients outside of the United States.