With Ascendis Pharma entering the achondroplasia space last month and BridgeBio on deck, BioMarin faces competition. Adding to the pressure, the company suffered a setback Monday when it halted two studies of Voxzogo in other growth-related conditions following multiple cases of hip injuries in other trials of the drug.
The hits just keep on coming for BioMarin’s Voxzogo. After gaining a competitor in the achondroplasia space last month with the FDA approval of Ascendis Pharma’s Yuviwel, BioMarin is halting dosing and enrollment in trials for Turner syndrome, SHOX-deficiency and aggrecan (ACAN) deficiency.
BioMarin made the decision after several slipped capital femoral epiphysis (SCFE) events were reported in two ongoing investigator-sponsored trials of the drug, according to a Monday SEC filing. SCFE occurs when the rounded head of the femur slips off the neck of the thigh bone at the growth plate, causing pain, stiffness and difficulty walking.
SCFE events have not been detected in BioMarin’s Phase 2 trials of Voxzogo in the conditions studied in the investigator-sponsored trials, BioMarin said in the filing, nor in the “more than 5,000 infants and children” who have been treated with Voxzogo for achondroplasia, a genetic disorder at the root of the most common form of dwarfism.
Shares of BioMarin fell about 4.6% to $55.80 in premarket trading Monday, Seeking Alpha reported, but stabilized around $58.01 when the market opened.
The FDA approved Voxzogo in November 2021 as the first drug to improve growth in children with achondroplasia. The company is also developing the drug for several other growth-related conditions, including in Phase 2 trials for short stature associated with Turner syndrome and a basket study of Voxzogo in Turner syndrome, SHOX deficiency and Noonan syndrome (ACAN deficiency).
Two other Phase 2 trials of Voxzogo—in hypochondroplasia and Noonan syndrome, and in people with idiopathic short stature (ISS) without ACAN-deficiency—will continue as planned, BioMarin noted in the SEC filing. ISS without ACAN-deficiency represents approximately 95% of children enrolled in the ISS trial, the biotech added.
While Voxzogo was first to market in achondroplasia, the company has conceded that competition is threatening its position. During a third-quarter earnings call last year, BioMarin walked back a previous goal of hitting $4 billion in revenue by 2027, a decision that was heavily influenced by incoming competition, CEO Alexander Hardy said at the time.
Voxzogo requires a daily injection, compared to Ascendis’ Yuviwel, which is given on a weekly schedule and could provide a convenience advantage for patients.
Both companies are also facing potential competition from BridgeBio’s infigratinib, which in a Phase 3 trial last month “meaningfully exceeded even the most optimistic expectations” in terms of efficacy and safety, according to Truist Securities. BridgeBio expects to file a new drug application with the FDA in the second half of this year.
