Summary study results show Ascendis drug for achondroplasia could be competitive to BioMarin’s Voxzogo, which is the foundation of the latter company’s growth plans.

BioMarin’s Voxzogo has been shown to increase growth rates in children with hypochondroplasia, a genetic cause of very short stature, in a phase 2 trial.

In his first presentation as the new CEO of BioMarin, Alexander Hardy left no doubt about his mission for the rare and genetic disease specialist.

The top priority for the former Genentech CEO is to accelerate and maximize the commercial potential of dwarfism drug Voxzogo, Hardy said Tuesday at the J.P. Morgan Healthcare Conference. His second objective is to ramp up the commercialization of Roctavian, a newly approved gene therapy for hemophilia A. Thirdly, it is up to Hardy to identify BioMarin’s most promising R&D projects and make tough decisions about what to cut.

The U.S. health regulator on Friday approved the expanded use of BioMarin Pharmaceutical's (BMRN.O) once-daily injection to treat children under the age of 5 with the most common form of short-limbed dwarfism.

This June, the FDA cleared Roctavian as a one-time gene therapy for adults with severe hemophilia A. For BioMarin, that was just the start, with plans to expand within the hemophilia population to adolescents, for instance, or into the Japanese market. The same goes for Voxzogo, which snagged FDA approval in achondroplasia—a genetic disorder that impairs bone growth and is the most common form of disproportionately short height—in late 2021.

Yesterday Biomarin confirmed what many had suspected: the company has yet to treat any patients with its haemophilia A gene therapy Roctavian. In the US, this is understandable, since the product only received an FDA nod in June.

The first half of 2023 saw the first approvals of therapies for a genetic subset of ALS and a pair of rare neurological disorders, Rett syndrome and Friedreich’s ataxia—and the regulator has several decisions for rare and orphan diseases on the docket in the second half of the year.

BioMarin refuses to give BridgeBio ground in their battle for the achondroplasia market. Tuesday, the rare disease biotech announced the FDA has accepted its supplemental New Drug Application (sNDA) to expand Voxzogo (vosoritide) to children younger than five years.

The Food and Drug Administration approved the first treatment for the most common cause of dwarfism Friday, a drug that has proved to increase children’s height but has been polarizing among adults with short stature.

After a period of "stagnant" growth at BioMarin—as characterized by one analyst team—the drugmaker could be on the brink of its next big launch. The company's dwarfism drug Voxzogo already has its European approval in hand, and it's due for an FDA decision later this week.