BioMarin Ends Voxzogo Development for Noonan Syndrome, Citing “Changes in the Treatment Landscape”

BioMarin is ending development of Voxzogo for Noonan syndrome.

According to Fierce Pharma on the 16th (local time), BioMarin has decided to discontinue development of Voxzogo for children with Noonan syndrome, a genetic disorder that can affect height, facial features and the heart.

In a securities filing, BioMarin said the decision was not related to Voxzogo’s safety or efficacy, but to “the feasibility of the study and the current treatment landscape for children with Noonan syndrome.”

Voxzogo is approved by the U.S. Food and Drug Administration (FDA) to treat achondroplasia, a form of dwarfism. Earlier this month, BioMarin reported positive results from a phase 3 trial in children with hypochondroplasia, a milder form of dwarfism, and applied to the FDA to expand the indication.

Noonan syndrome is not the first indication to be dropped from Voxzogo’s pipeline this year. This spring, BioMarin stopped dosing and enrollment in phase 2 trials for three indications: Turner syndrome, SHOX deficiency and aggrecan (ACAN) deficiency.

At the time, the company cited reports of adverse events, including slipped capital femoral epiphysis (SCFE), as the reason for stopping the trials, while saying it would proceed as planned with the Noonan syndrome trial.

The pipeline adjustment comes amid changes in the competitive landscape for Voxzogo. In February, the FDA approved Ascendis’ treatment for achondroplasia, Yuviwel. Unlike Voxzogo, which is injected daily, Yuviwel has the advantage of requiring only once-weekly administration.

Analysts at Leerink Partners said the newly secured hypochondroplasia indication alone may not be enough for BioMarin to offset competitive losses from Yuviwel.

BioMarin recouped some losses last month through a patent dispute settlement. Under the terms of the agreement ending patent disputes in five countries, Ascendis will pay BioMarin royalties of 20% of U.S. net sales and 18% of net sales in the European Union, Brazil and South Korea.

In August, BioMarin raised its Voxzogo sales forecast for the year to $1 billion-$1.05 billion, citing strong patient demand and adherence. The company’s shares fell nearly 3% after the announcement that it was discontinuing the Noonan syndrome program.