BioMarin eyes first profits in 2022 as dwarfism drug launch kicks into gear, hemophilia gene therapy refiling nears

After nearly 25 years of operating at a loss, BioMarin hopes to finally turn a corner and post its first profits in 2022 as the commercial launch of dwarfism drug Voxzogo kicks into gear. But some safety concerns might ruin the party.

Voxzogo, which BioMarin believes could become its largest-ever brand, brought in $5.9 million in sales in its first quarter on the market. Based on the demand seen so far, BioMarin has slightly dialed up Voxzogo’s 2022 sales expectations to between $90 million and $115 million.

Voxzogo could drive a double-digit sales growth for the entire company as the impact from the 2020 market exclusivity loss of phenylketonuria (PKU) drug Kuvan starts to take a back seat, execs said. With that, BioMarin plans to be profitable for the first time in 2022.

As of last week, about 210 children with achondroplasia were being treated with Voxzogo across 10 markets, with an additional 54 children waiting for treatment in the U.S., BioMarin chief commercial officer Jeff Ajer told investors on a call Wednesday.

In Europe, BioMarin has already secured access in Germany and France. As for the U.S., Voxzogo has secured some early coverage policies from payers, which Ajer said are consistent with the firm’s expectations.

Voxzogo enjoys a broader label in the EU, reaching children as young as 2 years old; it’s currently only allowed for patients 5 years and above in the U.S. To reach even younger patients, Biomarin is running a separate phase 2 trial. One-year data from that study “trended in favor” of Voxzogo compared with placebo on annualized growth velocity, BioMarin’s R&D chief Hank Fuchs, M.D., said on the call.

One case of sudden infant death syndrome (SIDS) occurred in the Voxzogo arm, which Fuchs said was deemed by investigator as unrelated to treatment. There was also a higher rate of sleep apnea for Voxzogo. The sign might pose a problem as there has been a speculated link between apnea and SIDS in the dwarfism disorder, Evercore ISI analyst Josh Schimmer said in a Wednesday note.

BioMarin plans to talk to regulatory authorities to discuss next steps to potentially expand Voxzogo’s label for younger patients, Fuchs said.

RELATED: BioMarin gears up for two 'transformational' launches, Voxzogo and Roctavian: CEO

BioMarin had originally gunned for profitability in 2020, but COVID-19 and a surprising FDA complete response letter for hemophilia A gene therapy Roctavian dashed that hope.

The company now plans to resubmit Roctavian to the FDA in June, followed by an expected six-month review, Fuchs said. But Schimmer spotted one wrinkle in the drug’s upcoming resubmission.

The FDA recently slapped a clinical hold on BioMarin’s PKU gene therapy program after reports of cancer in mice and evidence that the viral vector used to deliver the gene therapy had integrated into the genome.

As the agency demands more preclinical experiments for that program, Schimmer said he’s concerned the safety signal could affect the Roctavian review.

“Since both PKU and hemophilia A have very good and safe current gold standards, the urgency to approve an entirely new platform such as gene therapy may (should?) be low,” the Evercore ISI analyst said.

RELATED: BioMarin's new launch Voxzogo could surprise investors and become a blockbuster brand: analyst

For 2022, BioMarin expects to see sales of $2.05 billion to $2.15 billion, which by midpoint would mean 14% growth over 2021's result.